Blackstone Life Sciences, Merck, Bluebird Bio

March 27, 2024

51 3 minutes read

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Good morning, everyone. Damian here with the details on a potentially major drug approval, an idea to make CAR-T more equitable, and the slow launch of a gene therapy.

The need-to-know this morning

Blackstone Life Sciences said it will invest up to $750 million to support Moderna’s mRNA-based flu vaccine programs.
The European academic “TUDCA” study in ALS failed to its primary goal of slowing disease progression. The study was designed to investigate one of the two drug ingredients in Amylyx Pharma’s Relyvrio, which also recently failed its own clinical trial.

Merck’s potential blockbuster gets a clean label

The FDA approved Merck’s new treatment for a rare cardiovascular disease yesterday, clearing a drug that could bring in nearly $5 billion a year at its peak.

The approval of Merck’s injectable medicine for pulmonary arterial hypertension was widely expected, but the drug’s final label presents an upside for Merck. The FDA decided not to add a boxed warning on its side effects, which included an increased number of bleeding events compared to placebo. Merck’s drug will carry the brand name Winrevair and a list price of about $245,000 a year (each vial costs $14,000, and it’s injected every three weeks).

Analysts have described Winrevair as a promising potential growth driver for Merck. Keytruda, the company’s blockbuster cancer treatment, is expected to lose patent protection in 2028, and eventual biosimilar competition could substantially cut into Merck’s revenue.

Bluebird’s off to a slow start

More than three months after Bluebird Bio’s gene therapy for sickle cell disease won FDA approval, not one patient has received treatment, the company said yesterday.

Bluebird said it has “multiple patients” in the queue to receive its medicine, called Lyfgenia, and the first dosing is “imminent.” It takes about four months to prepare patients for the one-time infusion of Lyfgenia, and Bluebird only gets paid at the time of administration. The company said it expects to record its first revenue from the treatment in the third quarter of this year.

Bluebird’s stock price fell about 12% on the news, reflecting Wall Street’s concerns about patient demand for the company’s sickle cell gene therapy. Bluebird will be competing for patient attention with Vertex Pharmaceuticals and CRISPR Therapeutics, whose genome-editing treatment for sickle cell won approval the same day as Lyfgenia.

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Brazil’s plan for low-cost CAR-Ts

CAR-T therapies have proved curative for some blood cancer patients, but their high prices — they retail between around $350,000 and $475,000 in the U.S., much of that driven by manufacturing costs — have limited access in much of the world.

The Brazilian government will now test whether they can bring those costs down — dramatically. On Tuesday, Fiocruz, a foundation from the government’s ministry of health, signed an agreement with a 3-year-old U.S. nonprofit called Caring Cross. Under the deal, Caring Cross will provide equipment, materials, and expertise to develop CAR-T treatments at one-tenth of the current cost.

It’s part of a new movement to produce CAR-Ts cheaply in hospitals or other care centers themselves, rather than at centralized, pharma-owned factories. Caring Cross will set up mobile manufacturing units at multiple sites and help Fiocruz, which made Covid vaccines for much of Brazil, produce viruses and transform cells. If it works, it could set up a model for making CAR-Ts available across Latin America and much of the world, including for lower costs in the U.S.