| | | |   | Hello, all. Damian here with news on the importance of vials, the definition of "productive," and what the CRISPR patent ruling actually means. | | | Gilead’s next blockbuster was tripped up by a vial Gilead Sciences believes lenacapavir, a long-acting HIV drug, could become the backbone of combination treatments for years to come, making it a multibillion-dollar asset in waiting. But the wait got a bit longer yesterday, as the FDA rejected the treatment on a curious technicality.
The issue, according to Gilead, relates to vials. The company packages lenacapavir in borosilicate glass, the same material used in Pyrex measuring cups. In its rejection, the FDA cited concerns that Gilead’s drug is incompatible with those vials, the company said. In December, the agency put lenacapavir under clinical hold over the risk that using borosilicate vials could lead to “the formation of sub-visible glass particles in the solution of lenacapavir,” Gilead said at the time.
The FDA is not requesting any new clinical trials, but Gilead did not disclose how long it might take to switch vials and convince the agency to approve lenacapavir. Baird analyst Brian Skorney wrote in a note to clients that the rejection will likely delay lenacapavir’s approval by a year. | What does that CRISPR patent ruling mean for the field? Monday’s federal ruling on just who invented the process for turning CRISPR into human medicine appeared to end a seven-year legal battle. But the exact implications for the rapidly expanding industry of genome editing remain more than a little murky.
As STAT’s Megan Molteni reports, the ruling could spell a multimillion-dollar windfall for the Broad Institute, which came out on the winning end. And that money would likely come out of the pockets of Intellia Therapeutics, CRISPR Therapeutics, and any company that signed a license agreement with the University of California, Berkeley.
But what remains up for debate is whether the decision will impact newer versions of the genome editing technology, what it means for companies aligned with the Broad, and whether the ruling is indeed as final as it seems.
Read more. | Pharma at the intersection of private enterprise and public interest The pharmaceutical industry is global, highly competitive, and tightly regulated. As its challenges and opportunities grow, so does its need for professionals who fully understand the legal, business, and scientific considerations involved in the drug development process. Tomorrow’s pharma leaders can gain that expertise with a Master of Science in Law: Life Sciences. | 2022’s hottest biotech stock has returned to Earth Last month, Karyopharm Therapeutics became 2022’s best-performing biotech stock on the news that its approved blood cancer treatment had succeeded in a Phase 3 trial and was on the way to an expanded label. As it turns out, the FDA did not agree with the company’s take on the data, and Karyopharm has now given back nearly all of its 2022 gains.
The news, disclosed yesterday after market close, is that Karyopharm is going to conduct another randomized trial of its drug, Selinexor, after “a productive meeting with the FDA” in which the two parties “had differing views on the study significance and overall clinical benefit” demonstrated in its Phase 3 trial in endometrial cancer. Karyopharm’s share price fell about 30% after hours.
The company’s volatile month started with the Feb. 8 announcement that Selinexor had met its primary endpoint in the endometrial cancer study. Over the next week, Karyopharm’s share price rose about 75%, reaching a 52-week high after the company pulled out of a scheduled appearance at a banking conference, which investors read as a sign the company was negotiating a buyout deal. Then, on Feb. 23 came news that Karyopharm’s chief medical officer had left the company, sending shares down 26%. Now, with Selinexor’s label expansion indefinitely delayed, Karyopharm’s valuation is back to where it started the year. | Not every Pfizer vaccine is a winner Pfizer, which is making world-historic revenue on its Covid-19 vaccine, missed the mark in its effort to develop a vaccine for C. diff, putting a little more pressure on the company to find new sources of growth.
The three-dose vaccine missed its primary endpoint of preventing C. diff infection at a significantly higher rate than placebo, the company said yesterday. The vaccine succeeded on its secondary endpoints of reducing the severity and duration of symptoms, which, according to Mizuho analyst Vamil Divan, might be enough to eventually win approval for high-risk patients. But even in the best-case scenario, Pfizer’s C. diff vaccine is now unlikely to reach the $1 billion peak annual sales management had previously hoped.
The setback amplifies Pfizer’s precarious situation. Comirnaty, the company’s Covid-19 vaccine, and Paxlovid, its oral treatment for the disease, are expected to bring in about $60 billion in revenue this year alone, which would make 2022 Pfizer’s best year ever. But no one is certain how long those medicines will be in such demand, and it’s unclear whether Pfizer’s pipeline can compensate for their eventual decline. | More reads - Tennessee judge sanctions Endo for withholding opioid documents, refuses to recuse himself from case. STAT+
- NIH Foundation taps former CDC head, Merck exec Julie Gerberding. Bloomberg
- Rediscovering a language of healing that doesn’t require ‘cure’. STAT
- Inovio says lab tests show Covid vaccine weaker against Omicron. Reuters
| Thanks for reading! Until tomorrow,  | | | |
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