COVID-19 is a worthy opponent for the world's healthcare community: medical professionals and the pharmaceutical, diagnostic, medical device and technology companies that support them

There is an array of organizations deploying substantial clinical, financial and technical resources to develop a solution to the COVID-19 pandemic. Different approaches are being explored as COVID-19 is a new and complex threat. Although it may share similarities to other viral pathogens, its unique characteristics, unknown biological attributes, contagious mobility and varying impact on individuals makes it an unwieldy foe to weaponize bioscience against.

A shared challenge to mitigate or eliminate the threat of COVID-19

Medical professionals, hospitals, health systems, universities, research institutions, government laboratories, biotech and pharmaceutical companies are hotly pursuing a victory against COVID-19. Their playbooks include conventional vaccine development, use of biological therapies including monoclonal antibodies (mAbs) as well as basic and advanced antiviral medications. 

Experience and education fueling the effort 

Those working on an answer to COVID-19 are applying their experience and knowledge based on past and ongoing threats including Cholera, Dengue, Ebola, H1N1, H5N1, H7N9 Hendra, HIV, Herpes, Marburg, Measles, MERS, Mumps, Nipah, Polio, Rubella, SARS, Typhoid Fever and Zika. The clinical researchers and healthcare data scientists plus medical professionals working with patients and healthcare authorities are sharing their knowledge and learning on-the-go.

$50 Million From The Bill & Melinda Gates Foundation 

The lineup of clinical intellect is impressive; the amount of support from governments and even private citizens and charitable groups is admirable. A leading example of enormous generosity: The Bill & Melinda Gates Foundation donated $50 million to 12 pharmaceutical companies to advance COVID-19 research.

Immunizations: the ideal long term solution to COVID-19

A vaccine to prevent COVID-19 or assertively mitigate its ability to cause significant illness is a perfect solution but challenging to come by. It has to be evaluated for efficacy as well as suitability for mass production. A pathogen like COVID-19 will likely require millions of doses of a vaccine to protect those at risk. 

Once a vaccine candidate clears those hurdles a manufacturing facility has to be identified that can produce it and additional test batches produced to assure quality standards are met. The creation of a new vaccine can take considerable time, funding and other resources.

Exploring a COVID-19 solution based on mRNA characteristics

CureVac, BionTech, Moderna are three companies with an even more exacting strategy to developing an effective COVID-19 immunization. Their development experience and focus centers on messenger RNA (mRNA) therapeutic dynamcis that align well with the characteristics of COVID-19. 

An added plus to this approach, depending on the final formulation if found to be effective, is that the approved candidate could possibly be ramped up for mass production in a short amount of time.

This is a list of leading firms engaged in the development of a COVID-19 immunization:

• BioNTech (NASDAQ: BNTX)
• Clover BioPharmaceuticals (Privately Held)
• CureVac (Privately Held)
• Fosun Pharma (OTCMKTS: SFOSF)
• Gilead (NASDAQ: GILD)
• GlaxoSmithKline (NYSE: GSK)
• Inovio Pharmaceutical (NASDAQ: INV)
• Johnson & Johnson (NYSE: JNJ)
• MIGAL (Research Institute)
• Moderna (NASDAQ: MRNA)
• Pfizer (NYSE: PFZ) – partnering with BioNTech and Fosun
• Regeneron (NASDAQ: RGN)
• Sanofi (NASDAQ: SNY) – partnering with Regeneron
• Takeda (NYSE: TAK)
• VIR Biotechnology (NASDAQ: VIR)

Monoclonal Antibody versus COVID-19

Another approach is the potential of Regeneron’s Kevzara (sarilumab), a monoclonal antibody, against COVID-19. Kevzara is an interleukin-6 (IL-6) receptor antagonist. Kevzara is presently indicated for severe rheumatoid arthritis (RA). IL-6 could be triggering the overactive inflammatory response in the lungs of patients who are critically ill with COVID-19. 

Regeneron and Sanofi are partnering to manage clinical trials of Kevzara and closely study the ability of an interleukin-6 (IL-6) receptor antagonist to combat COVID-19’s effects. The use of mAbs is also being explored in the treatment of Ebola virus and Malaria.

Regeneron’s and Sanofi’s exploration is based on the initial signs of success another IL-6 receptor antibody, tocilizumab, had in China against COVID-19. While tocilizumab is still being assessed, China added it to their formal COVID-19 treatment guidelines.

Tocilizumab, developed by Chugai, is marketed as Actemra by Genentech/Roche in the United States. Actemra has four FDA approved indications: moderate to severe rheumatoid arthritis, giant cell arthritis, Polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis.

A monoclonal antibody agent effective against COVID-19 would be a welcome answer. They are complex therapies to develop and produce but if highly targeted could bring about the desired results no matter the cost.

Antiviral Medications versus COVID-19

Flu, herpes, HIV and other viral pathogens have large patient populations. They have fostered the development of numerous brand and generic formulations of antiviral medications plus more in pharma pipelines or castoffs from earlier clinical failures against other viruses. 

New, old or on the clinical trial scrap heap, there is great potential to align them with COVID-19 to find selections most likely to demonstrate efficacy at a therapeutically acceptable level against it. By inhibiting a viral pathogen's ability to replicate, they slow or stop its progress and enable a patient to better withstand and outdistance its effects.

An antiviral product or a combination of therapies including an antiviral agent, could be a solution to COVID-19. Presently, a combination of a protease inhibitor, Prezista (darunavir) and cobicistat (a pharmacokinetic enhancer), are being evaluated in tandem against COVID-19. Remdesivir, an investigational nucleotide by Gilead, is an example of another antiviral treatment pathway being explored.

Johnson & Johnson has issued a warning regarding use of Prezista for COVID-19. The product was used for treating SARS patients; while SARS and COVID-19 have some similarities, the differences between the two mean Prezista may not be effective against COVID-19. 

Data that may indicate its effectiveness against SARS does not convert to efficacy against COVID-19. This is an instance where close clinical study of an agent like Prezista and any adjunctive therapies used to treat COVID-19 is required to confirm level of effectiveness.

Just as with vaccines and mAbs, an antiviral therapy solution must demonstrate efficacy and have the right attributes to be mass-produced then facilities and production processes come next that are rigorously fortified with quality reinforcements.

This is a list of leading companies that develop, manufacture and commercialize antiviral products:

• AbbVie (NYSE: ABV)
• Arbutus Biopharma (NASDAQ: ABUS)
• Aurobindo Pharma (NSE: AUROPHARMA)
• Bristol-Meyers Squibb (NYSE: BMY)
• Gilead (NASDAQ: GILD)
• GlaxoSmithKline (NYSE: GSK)
• Johnson & Johnson (NYSE: JNJ)
• Merck (NYSE: MRK)
• Roche (OTCMKTS: RHHBY)
• Sino Biopharmaceutical (HKG:1177)

Summary

Vaccines, monoclonal antibodies (mAb) and antiviral therapies are just 3 of the options being explored to find an effective agent to deploy against COVID-19. Upfront experience and analytics in qualifying and identifying options is part of the process, critically assessing their activity and outcomes is the next step. It's still too early to tell which of these 3 or other routes to a solution will emerge as the first primary contender.

These approaches, along with many others, are making progress towards an answer to this global threat.The discoveries and advancements made along the way are the building blocks for future healthcare breakthroughs.

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A large scale digital health initiative

Apple and Johnson & Johnson have launched a new study to detect atrial fibrillation among seniors while promoting wellness and pro-active heart health.

The clinical study focuses on Atrial Fibrillation

Atrial fibrillation (referred to as "A-fib") is a cardiovascular issue which may increase the risk of strokes and other serious heart-related problems. 

Patients over age 65 can pick up a discounted Apple Watch ($49 plus applicable sales tax) at a Best Buy store if they are assigned one for the study or are issued an iPhone app called Heartline which also connects to Medicare’s Blue Button API that provides the patients access to their Medicare billing and claims data. 

This unique study is one of the largest randomized digital health trials in history, at a minimum, the goal is to recruit 150,000 people.

Collaborating Technology, Clinical and Retail Partners

Its working model is unique in that it represents a partnership between a technology company (Apple, NASDAQ: AAPL), a global healthcare conglomerate (Johnnson & Johnson, NYSE: JNJ) and a retailer (Best Buy, NYSE: BBY) to facilitate the study program.

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Vyepti: Lundbeck new product launch expected in April 2020 for Lundbeck

Lundbeck (OTCMKTS: HLUYY) has received FDA approval for its migraine therapy product Vyepti (eptinezumab-jjmr). Vyepti is indicated for the preventive treatment of migraine in adults. Vyepti is an infused pharmaceutical therapy. Lundbeck anticipates the product being available to prescribers and patients in April 2020.

Vyepti is an early result of Lundbeck’s purchase of Alder

The product originated from the product pipeline of Alder Pharmaceuticals. Lundbeck acquired Alder for about $2 billion in 2019.

Vyepti is an intravenous (IV) mAb therapy

The recommended dose of Vyepti is 100 mg every 3 months and it is noted some patients may benefit from a dose of 300 mg. Vyepti is a humanized monoclonal antibody (mAb) that binds to calcitonin gene-related peptide (CGRP) ligand and blocks its binding to the receptor.

Vyepti will be up against strong competition from Aimovig (Amgen and Novartis), Ajovy (Teva), Emgality (Eli Lilly) and other migraine products.

Lundbeck is a global pharmaceutical company

Headquartered in Copenhagen, Denmark with a U.S. base located in Deerfield, Illinois (a Chicago suburb) Lundbeck is centered on brain and psychiatric pharmaceutical therapies.

Some of Lundbeck's best-known products include:

·         Abilify (aripiprazole)

·         Northera (droxidopa)_

·         Onfi (clobazam)

·         Rexulti (brexpiprazole)

·         Sabril (vigabatrin)

·         Trintellix (vortioxetine)

·         Xenazine (tetrabenazine)

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Three FDA Rx to OTC Switches Approved

The U.S. Food and Drug Administration (FDA) has approved these 3 products presently available only by prescription to be purchased without one:

• Pataday Twice Daily Relief (olopatadine HCl ophthalmic solution/drops, 0.1%)
• Approved for itching, redness of eyes triggered by allergies
• Manufactured by Alcon (NYSE: ALC)

• Pataday Once Daily Relief (olopatadine HCl ophthalmic solution/drops, 0.2%)
• Approved for itching, redness of eyes triggered by allergies
• Manufactured by Alcon (NYSE: ALC)

• Voltaren Arthritis Pain (diclofenac sodium topical gel, 1%)
• Approved for arthritis pain
• Manufactured by GlaxoSmithKline (NYSE: GSK)

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The Super Bowl is a global event; marketers and advertisers invest millions in audience research and testing plus media ad spending to air the best television commercials money can buy

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A first for Thyroid Eye Disease

Horizon's recently FDA approved Tepezza is indicated for the treatment of Thyroid Eye Disease (TED). TED is a healthcare condition associated with the inflammation of the eyes, eye muscles, eyelids, tear glands and fatty tissues behind the eye.

Tepezza (teprotumumab-trbw) can be described as a biologic or biotherapy agent as it is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor 1 receptor (IGF-1R).

Medical issues associated with TED

TED is also known as Graves’ Orbitopathy or Ophthalmopathy and is an autoimmune condition. TED’s symptoms include outward bulging of the eye which may cause a series of problems such as double vision, eye pain, light sensitivity or issues with closing the eye. Tepezza may help some patients prevent more serious challenges with their eyes as a result of TED including the potential need for surgical procedures. Tepezza is an injectable product administered via infusion; once every 3 weeks for a total of 8 infusions.

FDA Director of The Division of Transplant and Ophthalmology Weighs In On Tepezza:

Wiley Chambers, M.D., deputy director of the Division of Transplant and Ophthalmology Products in the FDA’s Center for Drug Evaluation and Research shares these comments about Tepezza:

“Currently, there are very limited treatment options for this potentially debilitating disease. This treatment has the potential to alter the course of the disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, non surgical treatment option. Additionally, thyroid eye disease is a rare disease that impacts a small percentage of the population, and for a variety of reasons, treatments for rare diseases are often unavailable. This approval represents important progress in the approval of effective treatments for rare diseases, such as thyroid eye disease.”

FDA requires Horizon to conduct a post-approval study for Tepezza

As part of the FDA's approval of Tepezza, Horizon will continue monitoring Tepezza’s performance in a post marketing study. The study will assess the product's safety profile in a larger patient population. 

About Horizon Therapeutics 

Horizon Therapeutics (the recently renamed Horizon Pharma) is based in Dublin, Ireland with a U.S.  headquarters located in the Chicago suburb of Lake Forest, Illinois. Founded in 2005, Horizon's annual revenues are about $1.25 billion.

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Organ donation saves lives

National Donor Day is dedicated to organ donors plus the patients and medical professionals needing their help. Advancements in medicine have made incredible progress in organ transplant success but this record of achievement cannot continue without more donors. While more transplant procedures are being conducted each year, many never happen because there were not enough donors.

Organ transplants are more common but still challenging 

Since each patient and donor is different, no two transplants are the same. With each transplant, medical professionals, clinical researchers and other transplant science stakeholders learn more. For each transplant, medical professionals conduct an intense and time sensitive assessment of the organ recipient and the organ donor to determine the best match that will help increase the chances of success and survival. 

Organ transplant patients require considerable care after the procedures are completed

Following the transplant procedure, transplant patients are still at risk for complications as a result of the transplant including organ rejection. Even with highly effective immunosuppressive drugs and other therapies plus the care and support of healthcare professionals, organ rejection can occur years after a transplant is performed. In some cases the person is added to the transplant waiting list again. Based on new patients and transplant patients needing a replacement for their transplanted organ, there is an ongoing demand for more organ donors.

Ten facts about organ donation and organ transplantation:

Over 120,000 persons in the United States are in need of an organ transplant; for many of them their very life depends on it

Almost 40,000 organ transplants were conducted in 2019

One organ donor can save the lives of or tremendously improve the quality of life for 8 people or even more

In the United States more than 700,000 organ transplants have been conducted since 1988

These are the most common organ transplants:

• Heart
• Intestine
• Kidney
• Liver
• Lung
• Pancreas

• Blood Vessels
• Bones
• Corneas
• Heart Valves
• Ligaments
• Skin Tendons

Survival rates vary depending upon the patient and post-transplant recovery challenges including organ rejection

A new person is added to the organ transplant list in the United States every ten minutes

In the United States it is estimated 20 persons die each day that were waiting for transplants

Organ donation is free and there are no costs to register as an organ donor

Sign up to be an organ donor or share this information with someone else so they can be an organ donor, too 

You can help over 120,000 persons currently waiting for an organ transplant!

Visit this resource to learn how to sign up; if you are already registered, please share it with those who would like to join yourself and others as life saving organ donors:

 OrganDonor.Gov - U.S. Government Information on Organ Donation and Transplantation

Spread the word: Friday February 14th is National Donor Day!

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Published on 1/22/2020; updated 2/23/2020

Amazon hires Dr. Vin Gupta

Amazon continues to fortify itself with healthcare talent and technology assets. It has ramped up its online pharmacy unit Amazon Pharmacy / PillPack, acquired healthcare software and technology startup Health Navigator plus launched Amazon Comprehend Medical and Amazon Transcribe Medical. Amazon is also involved in a collaboration with the Pittsburgh Health Data Alliance.

Its latest addition is the reported hiring of Dr. Vin Gupta for its Amazon Care unit. Amazon Care (formed in 2019) is dedicated to serving the headquarters staff of Amazon and provides an array of healthcare services to employees and their dependents. Its programs include conventional patient and clinician care visits, telemedicine access plus other medical support and pharmacy services.

Dr. Gupta has a distinguished clinical, commercial and government background

Vin's experience spans these and other organizations in full time and advisory roles:

• Institute of Health Metrics and Evaluation
• Council on Foreign Relations
• U.S. Air Force
• U.S. State Department
• MIT
• NBC News
• Apple
• World Bank Group
• Brigham & Women’s Hospital

He has extensive academic credentials including:

• MPA; Public Administration, Harvard University
• USAF United States Air Command and Staff College
• MSc; International Relations and Affairs, University of Cambridge
• MD; Columbia University College of Physicians and Surgeons
• BA; Chemistry, Princeton University

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Initially published 1/06/20, updated 2/23/20

The 2019-2020 Flu season is well underway across the United States

The Centers for Disease Control ( CDC ) plus other government healthcare agencies and medical professionals are closely monitoring what is shaping up to be a formidable Flu season. 

Who is most at risk for Flu?

Individuals spanning infant to elderly are susceptible to the Flu virus. Expectant mothers, seniors and persons with chronic conditions such as asthma, an airway abnormality or other respiratory issues, diabetes, hypertension, heart disease, neurological or neurodevelopmental disease, kidney, liver or blood disease could be more vulnerable to Flu and severe Flu symptoms.  

Who are some of the leading healthcare companies developing and marketing vaccine products to help protect against the Flu?

The largest Flu vaccine manufacturers include:

• AstraZeneca ( NYSE: AZN ) 
• GlaxoSmithKline ( NYSE: GSK )
• Sanofi ( NASDAQ: SNY )
• Seqirus / CSL ( ASX: CSL )

Most Flu vaccine formulations are administered via a single injection although an inhaled nasal mist Flu vaccine is available ( FluMist Quadrivalent manufactured by AstraZeneca ). Healthcare professionals determine and prescribe which Flu immunization option, injection or inhaled formulation, is most appropriate for each patient.  

What products are available to treat the Flu for those diagnosed with it?

Depending on the consumer or patient there is an array of over-the-counter medications available which treat Flu symptoms that do not require a prescription.

There are several companies manufacturing Flu antiviral drugs which can be administered to patients with confirmed Flu diagnosis that require a prescription including:

• Rapivab ( peramivir; injection ) - manufactured by CSL 
• Relenza ( zanamivir; dry powder for inhalation ) -  manufactured by GSK  
• Tamiflu ( oseltamivir phosphate; oral ) - manufactured by Roche but also available as as a generic 
• Xofluza (baloxavir marboxil; oral) - manufactured by Genentech / Roche ( OTCMKTS: RHHBY ) 

How is the Flu season unfolding so far?

• The predominant Flu virus strain varies by region
• Age groups are another variable in profiling specific Flu virus strains
• Nationwide, influenza B/Victoria virus has been the most frequently reported Flu strain thus far; the second most frequently occurring is A(H1N1)
• Influenza B/Victoria viruses are most commonly reported in children age 0-4 years ( 46% of reported viruses) and persons age 5-24 years ( 57% of reported viruses)
• A(H1N1) pdm09 viruses are most commonly identified in persons age 25-64 years (41% of reported viruses)
• For adults 65 years of age and older almost equal proportions of influenza A(H1N1) pdm09 ( 38% ) and A(H3N2) viruses ( 37% ) have been reported
• Current hospitalization rate has been 6.6 cases per 100,000 ( similar to previous Flu season trends at this calendar interval but on the increase )
• Overall the CDC estimates there have been 4.6 million Flu cases  so far this year resulting in about 39,000 hospitalizations and over 2,100 fatalities

What are some ways I can protect myself and others from the Flu?

• It is not too late to get the Flu shot ( or FluMist if appropriate ); there are ample supplies presently available
• Take precautions to avoid contracting and spreading Flu virus:

• Avoid persons who are sick
• Avoid directly touching eyes, nose, mouth; cover nose and mouth with a tissue when coughing or sneezing; properly dispose of the tissue quickly
• Wash hands with soap and water frequently and clean / disinfect surfaces where hand contact is frequent ( desks, counters, table tops, cell phones, keyboards, tablets, door handles, remote controls, appliance handles, etc. )
• If diagnosed with the Flu and prescribed Flu antivirus medication, patients should be certain to complete the entire course of therapy and limit close contact with others

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Tokyo-based Astellas buys second California biotech company in less than a month

Astellas Pharma ( OTCMKTS: ALPMY ) is acquiring Xyphos Biosciences in a deal worth up to $665 million. Astellas is seeking to build out its  immuno-oncology business capabilities. This is the second acquisition Astellas has made in a month. Historically the company has made few buyouts since it was formed in 2005 from the merger of Yamanouchi Pharmaceutical and Fujisawa Pharmaceutical.

Xyphos is centered on advanced oncology treatment science

Astellas covets Xyphos’ proprietary molecules which can be delivered to natural immune cells or to engineered Chimeric Antigen Receptor (CAR) cells to generate immunotherapies for oncology treatment. The deal provides Astellas with additional clinical talent plus proprietary processes and technology focused in novel cancer treatment research and development. Xyphos’ first CAR cell product investigational agent is in preclinical development and scheduled to be tested in a first-in-human clinical study in 2021.

Astellas announced a multi-billion dollar deal earlier this month 

Earlier in December, 2019 Astellas bought Audentes Therapeutics for $3 billion. Audentes is a biotech firm focused on gene therapy. Xyphos and Audentes are each based in South San Francisco.

Astellas is a worldwide pharmaceutical leader

Based in Tokyo, Japan with a U.S. headquarters in Northbrook, Illinois, Astellas is Japan’s second largest pharmaceutical company ( Takeda is ranked number one). Astellas has over 17,000 employees and generally speaking is aligned with 5 disease categories:

• Immuno-Oncology
• Immunotherapy
• Neuro-Muscular
• Regenerative
• Gene Therapy

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Gene therapy continues to draw multi-billion dollar investments from biotech and pharmaceutical companies worldwide

Roche ( OTCMKTS: RHHBY ) is committing over $1 billion dollars up front to Sarepta Therapeutics ( NASDAQ: SRPT )  in a licensing deal with substantially more to follow depending upon the progress of SRP-9001, an investigational gene therapy targeting Duchenne muscular dystrophy.

Roche and Sarepta develop a product development and licensing deal

Based on its $1.15 billion deal with Sarepta, Roche will have global rights to launch and market SRP-9001 except for the United States:

• Sarepta received $750 million in cash from Roche
• Roche purchased about $400 million of Sarepta stock at $158. 59 per share

For now Sarepta, based in Cambridge, Massachusetts, has decided to retain the U.S. market sector for its own commercial ventures.  Sarepta will continue to manage clinical development and production manufacturing of SRP-9001; Roche has agreed to cover half of the global clinical development costs. If the drug meets unspecified regulatory and sales milestones, Sarepta could receive up to $1.7 billion more in funds from Roche, as well as royalties on any net sales, if SRP-9001 achieves certain regulatory approval requirements as well as sales revenue targets.

The patient care science of SRP-9001

SRP-9001 has the potential to be a breakthrough therapy in a challenging patient care sector. SRP-9001 is presently in Phase II clinical development. The agent is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein.

Sarepta's strategy to fund clinical trials for SRP-9001

With Roche’s investment, Sarepta can continue to invest in the development of the product and conduct clinical trials. They are selecting patients for the two-part Phase II SRP-9001-102; a 40-patient study focused on safety and efficacy of SRP-9001 in a 48-week randomized, double-blinded, placebo-controlled timeframe plus a 96-week, double-blinded extension period. This clinical study program has an anticipated wrap up targeting the 4^th quarter of 2022.

Based on positive results, Sarepta and Roche will pursue marketing opportunities worldwide

If the product is approved, Sarepta will have funds to orchestrate commercialization initiatives to launch the product in the United States. Roche, headquartered in Basel, Switzerland, has the global expertise and commercial resources that will enable it to move forward in other markets around the world. Roche, which just completed a $4.3 billion acquisition of gene therapy company Spark Therapeutics, will be a lucrative partner for Sarepta to market other gene therapy pipeline products with in the future or perhaps be wholly-acquired by Roche.

Partnerships and licensing agreements are popular options for large and mid-sized biotech and pharmaceutical companies to explore gene therapy commercial opportunities while avoiding risk

Gene therapy and other highly-focused clinical research or product development firms do not always have the financial resources or business structure to orchestrate a complete commercial lifecycle of their work. Established, publicly-held drug companies frequently collaborate with gene therapy and other smaller, highly advanced biotherapeutic concerns. As pipeline candidates move through the clinical trials process, it is easier to gauge the likelihood of their chances for approval by regulators. They provide financial support to these activities and when it appears their success is almost imminent, larger investments and commitments are made to assure trial completion, approval and subsequent clinical / commercial launch traction. 

The high cost and high risk involved with gene therapy research is a barrier to entry; less firms and highly focused therapies reduce competition in the sector

As an advanced area of life sciences with potential to cure diseases by replacing missing or mutated versions of a gene found in a patient’s cells with healthy copies gene therapy is not a sector many companies can participate in based on time, clinical and financial commitments. Depending on the patient and the genetic-based issue involved, gene therapy in has been proven to significantly reduce or eliminate complex, life threatening conditions. Success in the gene therapy sector also enriches the clinical insights and manufacturing attributes of their organizations which can be applied in other product development ventures outside of the gene therapy realm.

LinkedIn: John G. Baresky

Twitter: Healthcare & Content Marketing Guy

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Published on 12/19/19, updated 2/23/20

Danaher achieves critical European regulatory approval milestone in acquisition of GE biopharma business unit

Danaher (  NYSE: DHW.WD ) is closing out 2019 at full speed with its attention squarely centered on 2020. The European Union has approved their acquisition of GE Biopharma which completes a pivotal stage of their quest to buy one of GE's most prestigious and profitable corporate divisions. To satisfy the European Commission and other government regulatory requirements, Danaher will sell off these operating groups:

• MolDev FortéBio molecular characterization business located in Fremont, California and Shanghai, China
• Pall Biotech chromatography hardware unit operating from Portsmouth, United Kingdom and Westborough, Massachusetts
• Pall Biotech chromatography resins company operating out of Cergy, France
• Pall Biotech Single-Use Tangential Flow Filtration ("SUT TFF") systems located in Portsmouth, United Kingdom and Westborough, Massachusetts plus its stainless-steel Hollow-Fibre TFF (“SS HF TFF”) systems operations in Shanghai, China
• Pall Biotech SoloHill microcarriers and particle validation standards unit based in Farmington Hills, Michigan

With addition of GE biopharma assets, Danaher's business will engage multiple bioprocessing industry sectors

As a combined organization, Danaher and the acquired assets of GE Biopharma Business will be a worldwide leader in products and services aligned with bioprocessing including consumable single-use technology products such as bioreactors, mixers or connectors plus cell culture media and sera, microcarriers, bioprocessing filtration, molecular characterization, microscopy, high-content screening and laboratory filtration. Its enormous portfolio will enable it to embrace the needs of multiple customer stakeholders. 

These include academic institutions and commercial research organizations plus an array of companies involved with research, development, manufacturing, and commercialization of products prepared from or used by biological systems such as biopharmaceutical developers and manufacturers, cosmetics, food, fuel, livestock feed and other biochemical product and service providers.

Danaher corporate profile 

Headquartered in Washington, D.C., the Danaher organization is generally defined through three business groups: 

• Environmental & applied solutions
• Diagnostics
• Life sciences

With the addition of the GE biopharma assets, Danaher’s abilities in each of the 3 will be significantly multiplied and likely reach into new business sectors as well. Excluding the addition of the GE biopharma assets and their revenue potential, Danaher already generates just under $20 billion in annual sales.

Danaher will be disrupting the competitive balance in their business sectors

These top 10 Danaher rivals plus other firms will be sizing up the new competitive threats Danaher is formulating:

• Agilent Technologies ( NYSE: A ) 
• Bruker Corporation ( NASDAQ: BRKR )
• Harvard Bioscience ( NASDAQ: HBIO ) 
• Illumina ( NASDAQ: ILMN )
• Lonza Group ( OTCMKTS: LZAGF )
• PerkinElmer ( NYSE: PKI )
• Roche ( OTCMKTS: RHHBY )
• Thermo Fisher Scientific ( NYSE: TMO )
• VWR / Avantor ( privately held ) 
• Waters Corporation ( NYSE: WAT )

Danaher completes its divestiture of Envista including stock ownership and voting rights

Another strategic corporate development milestone at Danaher is the completion of its dental product assets into a fully independent spinoff  known as Envista Holding Corporation which was launched in 2018.  Danaher recently accepted an aggregate of 22,921,984 shares of Danaher common stock in exchange for all of its 127,868,000 shares of Envista common stock. Danaher is now completely separated from Envista as it no longer possesses any voting rights or economic interest in the newly formed company.

Danaher spinoff formed a global professional dental product leader 

Overlooked in the broader sense of the investor and dental healthcare communities, through spinning off its dental assets to form Envista ( NYSE: NVST ) Danaher launched a professional dental products giant comprised of: 

• 3 primary business units: KaVo Kerr, Nobel Biocare, Ormco
• Over 12,000 employees
• More than 30 dental brands and product franchises
• $2.8 billion in annual sales generated from over 150 nations

Danaher's organizational and revenue goal plans for 2020

Moving forward, Danaher will be busy with the arrangements to integrate its new assets and aligning their revenue producing capabilities according to their annual business plan. At the same time, they will be shopping the 4 Pall business units and MolDev FortéBio to interested parties. 2020 will unveil a significantly larger and somewhat redefined Danaher that customers, competitors and investors will be paying much more attention to in the future.

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Commercial synergy, market access and profit in strategic partnerships

Walgreens Boots Alliance (NASDAQ: WBA ) ranks at number 17 on the Fortune 500 list of largest firms. Reportedly they are exploring options to take the company private which may also be a strong signal to other industry leaders that it is open to takeover offers. Regardless of what their ultimate goals are, they remain a global force in retail, pharmacy, wholesaler and other sectors. The company’s CEO, Stefano Pessina, has communicated Walgreens is actively pursuing more partnerships as a business strategy that enable it to generate revenue by asserting its corporate, financial, clinical and operations resources to build market access and revenue while disrupting competitors --without having to deploy excessive funding for complete acquisitions that contribute further to debt loads.

Walgreens Boots Alliance ( WBA ) financial profile based on the formation of its present corporate structure

For many, Walgreens is a widely recognized retail drugstore chain that competes against consumer and pharmacy players such as CVS Health, Target, Walmart, grocery store pharmacies and online juggernaut Amazon's consumer and pharmacy businesses. In 2014, Walgreens completed an acquisition exercise valued at $4.9 billion cash plus 144.3 million common shares valued at $10.7 billion to acquire UK and Swiss-based Alliance Boots ( Walgreens had earlier committed $4 billion cash and 83.4 million common shares to get the deal underway ). Based in Deerfield, Illinois, ( a Chicago suburb ), the new organization, which deems itself as a holding company, immediately transformed into a global, multi-sector competitor. 

Primary elements of Walgreens Boots Alliance financial position

• Market capitalization of $50 billion
• Annual sales: $136.86 billion ( 2019 figures which represented a 5.8% increase over 2018 )
• Earnings: $3.982 billion
• Debt: $15 billion
• Ownership stake of 16% held by CEO Stefano Pessina

• Business operations in more than 25 nations
• Over 415,000 employees
• More than 18,500 stores located in 11 countries
• Over 390 distribution centers servicing pharmacies ( including pharmacies not owned by WBA ), physician offices and healthcare provider organizations
• Ownership stake of 26% in AmerisourceBergen, a global leader in healthcare wholesaler operations ranked at number 12 on the Fortune 500 list; annual sales of $153 billion

Based on its success and expertise dating back to 1901, Walgreens is acknowledged as an industry leader in consumer, pharmacy, wholesale and other business sectors. While it has various collaborations in place with a number of entities, it has 4 partnerships with established industry leaders equal to its own scope and scale. They demonstrate how attractive Walgreens is as an ally and business opportunity. Their relationships with Walgreens underscores Walgreens' ability to innovate and operate outside of conventional business model structures to share knowledge and resources which benefits both partners while increasing sales.

U.S. grocery store giant Kroger Co.

Walgreens has been collaborating with grocery store giant Kroger ( NYSE: KR )  in several marketing initiatives and late in 2019 announced the formation of their own GPO, Retail Procurement Alliance. By combining their orders, Kroger and Walgreens seek to lower costs and boost operational efficiencies. The two companies are already selectively marketing each other’s private label brand products in test market stores across the nation.

Cincinnati, Ohio based Kroger operates over 2,800 stores under 17 different names in 35 states with annual sales exceeding $122 billion. By working together, Kroger and Walgreens are developing wider market access for each of their product portfolios while developing synergies to more effectively operate and compete against Amazon, Walmart, Target, CVS Health, Albertsons, Costco plus other grocery and pharmacy rivals.

The global leader in healthcare wholesale operations McKesson Corporation

Late in 2019, Walgreens entered into a collaboration with McKesson, the world’s largest wholesaler. The two companies formed a joint venture to combine each of their drug wholesale units ( Alliance Healthcare Deutschland and GEHE Pharma Handel ) in Germany. McKesson ( NYSE: MCK ) has a 30% stake int the venture and WBA owns the remaining 70%. The organization will optimize efficiencies and strengthen a competitive position in Germany. The joint venture ( yet to be named and still requiring approval by regulators) is exclusively deployed within Germany's borders and does not extend into other nations. 

This arrangement is particularly interesting relative to Walgreens 26% ownership in AmerisourceBergen, one of McKesson’s primary competitors. McKesson, headquartered in San Francisco, ranks at number 7 on the Fortune 500 list by producing over $208 billion in yearly sales.

Technology champion and innovator Microsoft Corporation

Early in 2019 Walgreens Boots Alliance entered into a deal with Microsoft to enroll over 380,000 employees in Microsoft 365 cloud apps which encompass mobile, Office 365, security features and other applications. Walgreens will also transition the majority of its IT workload to Azure and Microsoft’s cloud platform. The two organizations are further working on solutions to optimize data workflow throughout WBA including administrative, pharmacy, operations and other areas through artificial intelligence ( AI ) and other technology. Microsoft has deep AI capabilities spanning, voice, advanced machine learning, data science, Internet Of Things ( IoT ) / Interoperability applications and robotics.

This arrangement benefits Walgreens as it gives them access to Microsoft’s technology resources and IT savvy while Microsoft gains a wider presence in the consumer / patient data management, retail, pharmacy, operations and other sectors ripe for advanced technology development. Through the partnership, both are staking a competitive position against a mutual consumer, healthcare and commercial technology rival, Amazon. Ranked at number 24 in the Fortune 500, Seattle based Microsoft churns out more than $110 billion in annual sales.

Blue Cross Blue Shield Pharmacy Benefit Manager Leader Prime Therapeutics

In 2017 Walgreens entered into a partnership arrangement with Prime Therapeutics, one of the largest pharmacy benefit management companies in the United States. The venture, called AllianceRx Walgreens Prime, is a ten year commitment for the 2 partners. Walgreens is the central ( but not exclusive ) retail pharmacy provider for Prime Therapeutics and the two jointly operate a specialty pharmacy plus mail order pharmacy services. AllianceRx Walgreens Prime benefits WBA with enhanced market access to the membership of 14 BCBS plans plus other customer groups. 

Prime Therapeutics realizes the benefit of being aligned with a leader in retail, mail and specialty pharmacy services and operations. Prime Therapeutics, based in Eagan, Minnesota, was founded in 1998. It services more than 28 million members and patients. Their cooperative ownership structure is comprised of 14 Blue Cross Blue Shield plans across the United States.

Walgreens building its future through clinical, operations, and technical transformation plus strategic alliances

Through the company it keeps in strategic partnerships and willingness to reinvent itself; Walgreens fortifies its ability to profitably grow and compete. Maximizing its assets and attributes with equally adept and equipped partners, Walgreens cultivates and improves its organizational effectiveness. Moving forward, Walgreens has numerous options to satisfy the demands of consumers, patients, payers and shareholders while continuing to be a preferred strategic ally for existing and future partners.  

LinkedIn: John G. Baresky

Twitter: Healthcare & Content Marketing Guy

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Medical school applicants and medical school enrollees transforming based on gender, ethnicity, race

The Association of American Medical Colleges ( AAMC ) reports that based on its data, more females than males are enrolled as medical students throughout the nation. The margin is narrow but reflects an ongoing trend that has been building over the last several years. This is a breakthrough for women, the medical community, academia and the nation.

AAMC is a champion for better patient care and healthcare academics

Based in Washington, D.C., AAMC was founded in 1876. AAMC is a non-profit organization. Its members comprise all 154 accredited United States and 17 accredited Canadian medical schools encompassing almost 400 teaching hospitals and health systems, 51 Department of Veterans Affairs ( VA ) medical centers plus over 80 academic medical societies. The AAMC is a healthcare leader dedicated to improving medication education and patient care while driving breakthrough medical research and healthcare innovation. 

A positive and growing trend that cleared a medical school enrollment milestone in 2017

Presently, 50.5% of all enrolled medical students are women. This number is built from crossing an earlier landmark threshold from 2017 when for the first time females outnumbered males as first-year medical students.

Kaiser Family Foundation Data

According to the Kaiser Family Foundation ( KFF ) there were 25,955 medical school graduates in 2018. This number includes allopathic medical school graduates and osteopathic medical school graduates. The top 10 states of combined allopathic and osteopathic graduates were:

• California: 1,477
• Florida: 1,316
• Illinois: 1,315
• Michigan: 1,143
• Missouri: 922
• New York: 2,402
• Ohio: 1,122
• Pennsylvania: 1,910
• Texas: 1,735
• Virginia: 858

Based in San Francisco and founded in 1948, the Kaiser Family Foundation is a commercially and clinically recognized and respected non-profit organization dedicated to the major healthcare issues of the United States and the role of the nation as an influential global healthcare policy leader.

As an industry, healthcare is becoming more diverse

The medical school academic sector is changing in many other ways. A greater number of minorities are applying and the percentage of them being accepted is on the rise as well.

Data from AAMC reveals a small and steady transformation in healthcare academics and the future healthcare workforce:

• Native American or Alaska Native applicants increased by 4.8% and their acceptance levels increased by 5.5%
• Hispanic or Latino applicants grew by 5.1% and those accepted grew 6.3%
• African American or black applicants rose by 0.6% and those accepted increased by 3.2%
• For African American or black males, their applicant count grew by .5% and those accepted increased by 3.7%

If current trends continue, the physician workforce of the future will be significantly changed

While women and minorities are presently outnumbered in the physician ranks, the current trends mark an important change from the past norms which is favorable for patients and clinicians moving forward. For persons early in their education and forming ideas about their career aspirations or for those people seeking a change in their existing careers, the prospects of gaining entry into the healthcare industry is greater than ever --including the opportunities for medical doctor roles as long as they can meet the academic rigor ( average GPA of undergraduates admitted to medical school is above 3.7 ) and take on the economic burden involved.

Medical school expense is a daunting challenge but interest in the healthcare profession and physician careers is robust

Based on data from the American Medical Student Association ( AMSA ), the annual cost of attending  a public medical school ( complete with tuition, fees, and health insurance included ) is approximately $34,592 for in-state students and $58,668 for out-of-state students. This cost rises considerably for those attending private institutions. Private medical school tuition, fees and health insurance average over $50,000 annually for in-state or out-of-state enrollees. These figures are based on statistics from 2016-2017; for public and private universities these costs continue to increase. Founded in 1950, AMSA is a student governed non-profit organization with over 30,000 members that is based in Washington, D.C. which cultivates healthcare academic and student advocacy while providing a framework for educational and clinical communication and networking. 

The sustained and growing interest in healthcare as a career and physician-centered roles is encouraging. 20 new medical schools have opened in the last ten years and class sizes have been expanded which has resulted in a 33% growth trend that dates back to 2002. 

Americans are living longer; will there be enough physicians to care for us?

Amazingly, the expansion may not be sufficient to meet the healthcare needs of the nation. Thanks to rapidly evolving healthcare knowledge, better techniques and advanced healthcare products as well as a decline in tobacco use, U.S. citizens are living longer. To meet the needs of today and those of future generations, the number of physicians needs to increase as present data indicates a drastic shortfall by up to 122,000 doctors is approaching by the year 2032.

LinkedIn: John G. Baresky

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Synthorx: a fourth multi-billion dollar acquisition for Sanofi in less than 10 years

Sanofi is expanding its oncology therapeutics business through acquiring Synthorx for approximately $2.5 billion. Based in La Jolla, California, Synthorx has a primary focus in immuno-oncology. Synthorx's most current primary development project is THOR-707, a form of interleukin-2 (IL-2) which, if approved and launched, can be deployed to treat multiple solid tumor types as a monotherapy and potentially in combination with checkpoint inhibitors. The agent is intended to increase effector T-cells and natural killer cells that eliminate cancerous cells in patients.

Synthorx has value-added biotech research & development attributes for Sanofi to build on

An added asset for Sanofi in the deal is Synthorx’s Expanded Genetic Alphabet platform. It can be integrated with Sanofi’s existing initiatives in nanobody research and development. This will support Sanofi’s ventures in advanced patient care and healthcare product sectors such as:

• Biologics
• Conjugates
• Protein Fusions

$2.5 billion deal a mega win for Synthorx investors

Synthorx had gone public in 2018. These three initial backers will realize an enormous windfall from their investments in it:

• Avalon Ventures
• OrbiMed
• RA Capital

Sanofi expanding its ability to compete in advanced pharmaceutical product sectors while stacking on debt

Sanofi, which has been reported to be shopping its consumer healthcare products unit as a spinoff or joint venture that could be worth up to $30 billion, has made three sizable acquisitions since 2011 that, combined with the Synthorx deal, will push up its $28 billion debt load:

• Synthorx: bought for $2.5 billion in 2019
• Bioverativ: acquired for $11.5 billion in 2018 ( a spinoff from Biogen)
• Ablynx purchased for $2.4 billion in 2018
• Genzyme bought for $20 billion in 2011 

 Based in Paris, France, Sanofi ( NASDAQ: SNY ) is a global contender ranked within the top 10 of pharmaceutical manufacturers. It generates over $42 billion in annual sales and has over 100,000 employees.

Sanofi transforming its corporate organization and clinical focus

The Synthorx deal is part of a newly emerging change in strategy at Sanofi. They are not only seeking to further monetize their consumer healthcare products unit through a spinoff, partnership or outright sale but also to channel more focus into global healthcare opportunities based on clinically complex medicines. Their senior leadership team has communicated they are going to be significantly reducing their clinical and financial investments in the cardiovascular / hypertension and diabetes / endocrinology patient care sectors in favor of more challenging life science commercial opportunities.

LinkedIn: John G. Baresky

Twitter: Healthcare & Content Marketing Guy

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Could 3M be seeking to boost revenue or pay down long term debt?

3M ( NYSE: MMM ) is reportedly exploring it options to sell or spinoff its drug delivery systems unit. Based in Maplewood, Minnesota ( a suburb of St. Paul ), 3M ( formerly known as the Minnesota Mining and Manufacturing Company ) is a global conglomerate with long established business enterprises in consumer goods, healthcare, industrial products and worker safety.

3M has a robust presence in the healthcare sector

While 3M generates about $33 billion in sales annually, the drug delivery portfolio of products is a smaller part of a substantially large healthcare products unit. It is estimated drug delivery product revenue accounts for less than 2% of 3M’s total annual sales. Medical device manufacturers, brand and generic pharmaceutical manufacturers as well private equity investors can be considered as likely buyers if 3M does move forward with the sale of the business unit.

 3M drug delivery has 5 primary product groups:

• Inhalation
• Microneedle
• Nasal
• Packaging
• Transdermal

Possible plans for proceeds from sale of the drug delivery business unit

If 3M were to spinoff or sell the drug delivery unit, it could be marketed for around $1 billion. 3M may have plans to drop the proceeds of the drug delivery unit into the bottom line of its annual revenue totals or could apply it to pay down some of its long term debt which is approximately $19.4 billion.

LinkedIn: John G. Baresky

Twitter: Healthcare Marketing Guy

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Astellas Expands Therapeutic Portfolio With Gene Therapy Deal

Astellas ( OTCMKTS: ALPMY ) has announced it is acquiring Audentes Therapeutics ( NASDAQ: BOLD ) for $3 billion and plunge itself into the gene therapy market. Based in San Francisco, California, Audentes is a clinical stage research company centering on the development of  AAV-based genetic medicines for people with significant and rare neuromuscular diseases.

Astellas is a global pharmaceutical leader 

Astellas, based in Tokyo, Japan with a U.S. headquarters in Northbrook, Illinois, is Japan’s second largest pharmaceutical company ( Takeda is ranked at number 1). The current product portfolio of Astellas is defined by 4 categories and the addition of Audentes will establish a 5^th with the specialty of genetic therapy:

• Immuno-Oncology
• Immunotherapy
• Neuro-Muscular
• Regenerative
• Gene Therapy

Astellas employs approximately 17,500 worldwide; Audentes has less than 1,000 staff members. 

Audentes has developed a robust pipeline of advanced gene therapies

Audentes' adeno associated virus ( AAV ) gene therapy technology platform and proprietary production process expertise is applied  in programs spanning 3 modalities:

• Gene Replacement
• Vectorized Exon Skipping
• Vectorized RNA Knockdown

Audentes has committed significant resources to develop potential approval candidates although risk remains high in terms of chances of approval by the Food and Drug Administration ( FDA ) in the United States and the government regulatory agencies of other nations:

• AT132 X-linked Myotubular Myopathy MTM1 ( Gene Replacement )
• AT845 Pompe GAA ( Gene Replacement )
• AT702 Duchenne Muscular Dystrophy Exons 2, 1-5 ( Vectorized Exon Skipping )
• AT751 Duchenne Muscular Dystrophy Exon 51 ( Vectorized Exon Skipping )
• AT753 Duchenne Muscular Dystrophy Exon 53 ( Vectorized Exon Skipping )
• AT466 Myotonic Dystrophy DMPK ( Vectorized Exon Skipping / Vectorized RNA Knockdown )

Presently, the leading candidate for approval in the Auduentes portfolio is AT312 which could happen as early as mid-2020. Its clinical trial performance has generated good results in the treatment of X-linked myotubular myopathy (XLMTM) occurring primarily in male infants.

Astellas began exploring gene therapy marketplace in 2018

Astellas began exploring gene therapy marketplace in 2018

Astellas has already licensed a gene therapy candidate. In 2018 Astellas entered into a licensing agreement with Juventas Therapeutics for JVS-100. JVS-100 is a non-viral gene therapy expressed from stromal cell-derived factor -1 ( SDF-1 ) which is a naturally occurring signaling protein activating the endogenous tissue repair pathways. Founded in 2007 and based in Cleveland, Ohio, Juventas is a private, clinical stage biotechnology company developing novel non-viral gene therapies which activate natural processes. Their clinical indication specialties encompass dermal scar prevention, heart failure, peripheral artery disease and other areas of investigational research and development.

Why is gene therapy such a hot marketplace for biotech and pharmaceutical companies to invest in?

Gene therapy is an advanced area of life sciences with potential to cure diseases by replacing missing or mutated versions of a gene found in a patient’s cells with healthy copies. Depending on the patient and the genetic challenge involved, gene therapy in some instances can resolve significant illnesses with one dose. Because of the advanced clinical technologies involved with developing and manufacturing gene therapies, they are quite costly and the processes to develop them are complex and highly coveted by pharmaceutical manufacturers.  

Large and mid-sized pharmaceutical companies are always seeking to build their clinical and commercial capabilities. Many product categories are crowded with competition including:

• Allergy
• Antibiotics
• Cholesterol
• Dermatology
• Diabetes
• Gastraoesophogeal Reflux Disease ( GERD )
• Hypertension
• Oral Contraceptives 
• Rheumatology

There are numerous brand and generic drugs in each of these categories. While the patient population prescribed these products is in the millions and the medications involved taken on a daily basis for months if not years, pharmaceutical companies devote large sums to develop these products and promote them. Then conversely, they frequently discount the products through market access strategy measures to contract for favorable positions on managed care organization ( MCO ), prescription benefit manager ( PBM ) and health system formularies or group purchasing organization ( GPO ) listings. 

While gene therapy and other advanced medications are costly and risky to produce, the investment to promote them following approval is less. The payout is larger as there are less competitors due to their high research & development and production costs plus the narrow indications they are approved for and reduced number of patients they are prescribed to results in significantly higher prices at time of launch. 

Astellas is highly selective in its acquisition strategy

It has been almost 10 years since Astellas last made such a sizable acquisition. In 2010, Astellas acquire OSI Pharmaceuticals for $3.8 billion. With annual sales of approximately $12 billion and a market capitalization estimated at $33 billion, Astellas is solidly established in global healthcare markets and has the clinical and financial resources to cultivate a successful gene therapy portfolio of products.

Astellas versus the competition in the gene therapy marketplace

By adding  Audentes’ research & development attributes and manufacturing capabilities plus its connections  with patient groups, academic institutions and the biotech research community, Astellas assertively positions itself in the expanding gene therapy marketplace.

Roche ( OTCMKTS: RHHBY ) Novartis ( NYSE: NVS ) and Pfizer ( NYSE: PFE  ) are presently the blue chip pharmaceutical manufacturer leaders in gene therapy. Roche acquired gene therapy company Spark Therapeutics for $4.8 billion in 2019, Novartis bought up AveXis, another gene therapy leader for $8.7 billion. Among Pfizer's many investments, they acquired gene therapy producer Bamboo Therapeutics for $645 million in 2016. 

Reportedly Pfizer and Novartis have been hard at work building out their gene therapy development and manufacturing capabilities. Pfizer has allocated about $600 million to gene therapy production and Novartis  slightly less at $500 million.

Potential gene therapy giants or acquisition targets

Upcoming gene therapy companies, which could substantially grow on their own if their pipeline candidates are approved, may also be attractive buyout targets. Some of the leading gene therapy companies of this caliber include:

• MeiraGTx Holdings PLC (NASDAQ: MGTX )
• Regenxbio Inc. ( NASDAQ: RGNX )
• Sarepta Therapeutics Inc. ( NASDAQ: SRPT )
• Solid Biosciences Inc. ( NASDAQ: SLDB )
• uniQure NV ( NASDAQ: QURE )
• Voyager Therapeutics Inc. ( NASDAQ: VYGR )

While it appears there are numerous contenders in the gene therapy market, their products are highly niche-focused so they have less or no competition within each of the therapeutic areas they are exploring to develop new medicines. The high rate of clinical trial failure also results in an ongoing reduction of competing agents throughout the product development cycle.

Drug manufacturers seek to avoid large financial outlays and risk involved with gene therapy and other biotherapy product development

Many large and mid-sized companies owned by the investment community prefer to have smaller, highly-focused companies develop products like gene therapies first. As they progress through the approval process, the pharma companies either license or buy outright the product or the firm when approval by regulators seems imminent. In advance, these companies may partially fund the research necessary to develop the products. Audentes provides Astellas with a solid candidate to enter the gene therapy market with and hopefully generate other products in the future.

Astellas and Audentes deal approval and product approval timelines

Even before the Astellas and Audentes deal is approved by regulators, the approval of AT132 X-linked Myotubular Myopathy MTM1 ( Gene Replacement ) will be highly anticipated by investors, company staff members and the medical community. Depending when the deal is approved and AT132 is approved, a combined torrent of activity will follow. Astellas and Audentes could be working to integrate their organizational structures and launching a strategic product venture at the same time. As 2020 rapidly approaches, each company has much to accomplish and look forward to.

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