Behind the Headlines: Sickle Cell Therapies; Mpox Vaccines; and ARCH Ventures’ Biotech Fund

This Behind the Headlines news roundup panel discussion on recent news items includes updates regarding sickle cell therapies (e.g,, Pfizer’s withdrawal of Oxbryta and Vertex’s choosing of Lonza as the commercial manufacturer of Casgevy); Emergent BioSolutions $400 million BARDA contract for an mpox vaccine; and the first in a generation novel schizophrenia drug, Cobenfy (xanomeline and trospium chloride). The panel also examines investment revival with ARCH Ventures $3 billion biotech fund raise and the $3.6 billion for Flagship Pioneering. Rounding things out is a discussion on COVID-19-associated childhood myopia and patent thickets artificially keep the price of drugs high.

Pfizer’s withdrawal of Oxbryta, one of a limited number of treatments that won accelerated FDA approval in 2019, surprised doctors, patients, investors, and analysts. CNBC News stated, “Oxbryta was one of the centerpieces of Pfizer’s $5.4 billion acquisition of Global Blood Therapeutics in 2022” (1). Vertex’s gene therapy, Casgevy (exagamglogene autotemcel), was given commercial-scale manufacturing capabilities when Vertex chose Lonza as its contract manufacturing and development organization for global release. This marks a major milestone in gene editing’s future deployment, and much will be learned about what is feasible, and what requires a re-think. Interestingly, Casgevy does not address the root cause of sickle cell but instead suppresses the suppressor of infant hemoglobin production, hence essentially diluting the problems of limited healthy hemoglobin by creating an alternate supply naturally only available to infants (2).

Emergent BioSolutions contract with the Biomedical Advanced Research and Development Authority (BARDA), worth $400 million, for mpox vaccine production comes at a time when we must ask ourselves, was this new strain outbreak controllable had the US government not destroyed its aging stockpiles of the older traditional? In an example of vaccine nationalism, “While waiting on a freeze-dried long shelf-life vaccine to be developed, the United States let its stockpile of liquid monkeypox vaccine languish and expire. By 2017, all 27,993,370 doses in the national Jynneos stockpile had expired, although the US still had a huge stockpile of its other smallpox vaccines. While waste per se is shameful, it’s akin to Greek tragedy when the rise in monkeypox cases internationally is associated with lost opportunities to improve millions of people’s lives in sub-Saharan Africa, where smallpox was eradicated in 1980, and vaccinations halted” (3).

After a generation of few new treatments for schizophrenia, Bristol Myers Squibb’s $14 billion deal for Karuna Therapeutics has borne fruit with the approval of a highly promising new drug with a novel mechanism of action (4).

References

1. Constantino, A. and Capoot, A. Healthy Returns: Pfizer Pulls Sickle Cell Disease Drug from Markets–Here’s Why it Matters. CNBC News. Oct. 1, 2024.
2. Spivey, C. New Medicines, Markets, and Manufacture: CRISPR for Sickle Cell Disease and β-thalassemia. PharmTech.com. Nov. 30, 2023.
3. Spivey, C. Vaccine Disconnect. Pharmaceutical Technology 2022 46 (9).
4. BMS. US Food and Drug Administration Approves Bristol Myers Squibb’s COBENFY (xanomeline and trospium chloride), a First-In-Class Muscarinic Agonist for the Treatment of Schizophrenia in Adults. Press Release. Sept. 26, 2024.

Panelists

Ed Silverman is a Senior Writer at The Boston Globe's Stat and Pharmalot.

Benjamin McCloud is co-founder of Stealth Co.

Moderator

Chris Spivey is director of Industry Relations and Strategic Partnership at MJH Life Sciences.