| | | |   | Good morning, everyone. Damian here with a watershed moment for gene therapy, the dimming of a few bright ideas in oncology, and the successor to a particularly efficient biotech CFO. | | | Bluebird won FDA approval. Now what?  The FDA approved Bluebird Bio’s gene therapy for the blood disorder beta thalassemia yesterday, a long-in-the-making victory for a company in desperate need of a financial lifeline.
As STAT’s Adam Feuerstein reports, the one-time treatment, called Zynteglo, helped about 90% of patients in a clinical trial live without the need for the regular blood transfusions used to treat beta thalassemia. Bluebird, which tried and failed to market the same treatment in Europe, set a price of $2.8 million per patient, promising to return up to 80% of that cost if patients need to go back to regular transfusions within two years of therapy.
The question now is whether Bluebird, which recently laid off employees and cut spending to conserve cash, can build a sustainable business with its first FDA-approved medicine. The company’s stock price, which hit an all-time low in June, rose about 26% immediately after yesterday’s approval before closing about 10% above the prior day.
Read more. | Another bright idea in oncology is looking shaky Sanofi is discontinuing all research into a once-promising cancer treatment, the company said yesterday, raising doubts about a cadre of similar medicines aimed at the same biological target.
The company’s decision follows a second clinical failure for amcenestrant, a selective estrogen receptor degrader, or SERD, designed to treat certain forms of breast cancer. To Cowen analyst Steve Scala, the end of amcenestrant could bode poorly for the entire class of SERD medicines, which includes late-stage projects from AstraZeneca, Roche, and Eli Lilly.
The news only compounds the pressure on Sanofi, which, thanks to a spate of pipeline setbacks, has become increasingly reliant on the blockbuster anti-inflammatory treatment Dupixent. The company’s share price has fallen about 15% this month over worries about its future growth and concerns that it will suffer losses in ongoing litigation related to the heartburn drug Zantac. | When it comes to drug development, we need both conventional and cutting-edge therapies Two things the world learned from the pandemic: go quickly and be ready. The biopharma industry can take advantage of the momentum gained and continue pushing the boundaries of medicine. In this three-part article series, explore the era of ‘and’, where mRNA and other novel molecules will feature prominently. And learn how we can prepare for that reality. Read the first article here. | Another blow to the ‘don’t eat me’ hypothesis Speaking of once-promising ideas in oncology, the protein CD47, which tumors hijack to send a “don’t eat me” signal to certain types of immune cells, emerged in the last five years as a winning target for cancer therapy. But a metronomic series of setbacks has forced researchers to rethink whether impressive early data will ever translate into an actual medicine.
The latest setback comes from the Chinese-headquartered I-Mab, which said yesterday that partner AbbVie had discontinued a Phase 1 study of its CD47-targeting molecule, sending the company’s share price down about 14%. Last month, AbbVie terminated a separate study of the same treatment.
The news follows years of mounting concern that CD47, despite early promise, might be an imperfect target. While tumors can exploit it to escape immune detection, healthy red blood cells use CD47 to go about their business without meddling from the immune system. Blocking it risks exposing those cells to destruction, which could lead to dangerous bouts of anemia and make for an unsafe medicine. | Moderna has a permanent CFO once more Three months after Moderna Chief Financial Officer Jorge Gomez made $700,000 for about 48 hours of work, the company has found a permanent replacement.
James Mock, outgoing CFO at the scientific instruments company PerkinElmer, will take the Moderna job starting Sept. 6, the company said yesterday. He succeeds Gomez, who was fired days into his tenure after a prior employer revealed an internal investigation into its financial reporting, marking a brief corporate embarrassment for Moderna. (“I think it’s safe to say the company did a little bit more diligence this time around,” Jefferies analyst Michael Yee told the Wall Street Journal.)
Mock’s hiring ticks a box for Wall Street, which has spent months asking Moderna just how it intends to spend its roughly $18 billion in cash to offset a decline in profits from its blockbuster Covid-19 vaccine. Hiring a permanent CFO clears the way for the company to put its plans in motion. | More reads - Pharma’s likely to sue over Medicare negotiation. Here are the arguments they might use, STAT
- Endo International files for bankruptcy to weather opioid lawsuits, Wall Street Journal
- In an effort to address its missteps during Covid, CDC plans an ‘ambitious’ agency overhaul, STAT
| Thanks for reading! Until tomorrow,  | | | |
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