| |   | Hello, everyone. Damian here with news on the return of a biotech stalwart, the debate over a new treatment for ALS, and unanswered questions about Pfizer's Covid-19 pill. | | | With biotech in a swoon, Third Rock returns with its biggest fund Stalwart biotech investor Third Rock Ventures has raised $1.1 billion to seed the next generation of startups, the firm said today, marking its largest-ever fund and arriving in the middle of a lengthy downturn for the sector.
As STAT’s Allison DeAngelis reports, the lion’s share of those dollars will go to companies in their first rounds of venture fundraising. Each individual investment will likely come in at between $80 million and $100 million, partner Reid Huber said, which will help get companies “to mature their pipelines a little bit more than in the past, so these companies can have a much clearer line of sight to their first product and the value proposition for investors.”
That’s in part a reaction to the market, Third Rock partner Abbie Celniker said. For years, early-stage biotech firms have gone public without having a clear path to running actual clinical trials, and the many resulting setbacks have dragged down the broader biotech market. Third Rock is looking at companies with “a reasonable out-of-the-gate asset that has a line of sight to the clinic,” Celniker said.
Read more. | Here’s why Amylyx might win FDA approval after all Amylyx’s first date with the FDA didn’t exactly go well, with agency staff plainly skeptical of the evidence supporting its treatment for ALS and independent advisers voting narrowly against recommending the drug’s approval. But that doesn’t mean Amylyx is doomed to rejection.
As STAT’s Adam Feuerstein points out, there’s plenty of precedent for the FDA granting accelerated approval to drugs with incomplete supporting evidence that might make a difference for patients with devastating diseases. It did so for Biogen’s Aduhelm last year and Sarepta Therapeutics’ first treatment for Duchenne muscular dystrophy in 2016.
And Amylyx arguably has a stronger case for leniency. Unlike Biogen and Sarepta, the company has already begun a larger study that would confirm its drug’s benefits, and data are expected in 2024. That makes a potential approval seem more palatable to the FDA: Give Amylyx a two-year grace period; if the trial succeeds, the agency will look wise, and if it fails, regulators can rescind approval.
Read more. | How Tech Can — and Can't — Eliminate Barriers to Good Health Care Telemedicine and big data are just a few of the technological developments that may help doctors reach patients who currently don’t get high-quality care. On June 29, Join STAT for a look at what these efforts can accomplish and also what the new technology’s limitations are likely to be. | And here’s why people are already fighting about its price Amylyx’s ALS drug would only be cost-effective if the company charged about $13,700, according to the nonprofit Institute for Clinical and Economic Review, a conclusion that has disturbed patient advocates and could set the stage for arguments to come.
As STAT’s Ed Silverman reports, ICER reached its conclusion by using a metric called quality-adjusted life year, or QALY, which measures a drug’s value based on patients’ life expectancy and health for their remaining years when using a given treatment. Patient groups, including the ALS Association, have argued that QALY is a discriminatory metric for diseases like ALS, contending that people without disabilities should not be making judgments about what constitutes “quality of life.” Amylyx has not committed to a price, saying in a statement that it “will work closely with payers on coverage, reimbursement, and access” if its drug is approved.
For now, the debate is largely academic. But come September, when Amylyx’s drug is up for a final FDA decision, the company’s chosen price could seed a backlash that loops in patients, payers, and even lawmakers.
Read more. | Pfizer’s latest Paxlovid study presents more questions than answers Pfizer said yesterday that a closely watched study of Paxlovid failed to speed up recovery for Covid-19 patients who don’t have risk factors for severe disease.
As STAT’s Matthew Herper and Jason Mast report, the data in no way invalidate earlier results showing that Paxlovid prevents hospitalizations and saves lives in patients at high risk of severe disease, but they do complicate the question of just when doctors should reach for Paxlovid.
For instance, because of the small number of hospitalizations overall in the study, Pfizer’s latest trial failed to produce a statistically significant finding on whether patients already vaccinated against Covid-19 were hospitalized less often if they received Paxlovid.
Read more. | More reads - Biotech edges toward gender parity, but C-suites stay male, white. FierceBiotech
- The Senate health committee finally marks up its user fee bill. STAT
- Alnylam wins FDA approval of rare disease drug in step toward profitability. BioPharma Dive
- Newest Omicron Covid-19 lineages gaining ground in United States. STAT
| Thanks for reading! Until tomorrow,  | | | |
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