| | | |   | Good morning, all. Damian here with the latest from a biotech rainmaker, a look at the next big news in Alzheimer's disease, and an FDA hearing with sweeping implications. | | | After an $8 billion deal, ex-Loxo CEO has a well-funded new startup Three years removed from selling Loxo Oncology to Eli Lilly for $8 billion, Josh Bilenker has raised at least $473 million from private investors for a new company that is yet to go public with just what it’s working on.
As STAT’s Matthew Herper and Allison DeAngelis report, it’s called Treeline Biosciences, and Bilenker, who served as Loxo’s CEO, is running it with Jeffrey Engelman, formerly Novartis’s top cancer researcher. The company has hired more than 130 people across three research sites in little more than a year, according to a company blog post, regulatory filings, and STAT reporting.
Treeline’s focus remains a mystery, though it’s fair to assume, based on the prior work of its founders, that cancer will be involved.
Read more. | What to know ahead of the next big Alzheimer’s news After the surprising Phase 3 success of Biogen and Eisai’s latest treatment for Alzheimer’s disease, pivotal data from rival Roche could again upend the field.
Any day now, Roche will disclose top-line results from a pair of Phase 3 studies on gantenerumab, yet another Alzheimer’s treatment meant to slow the disease’s progression by clearing out toxic plaques from the brain. Unlike the intravenous lecanemab, Biogen and Eisai’s treatment, Roche’s drug is injected just under the skin. And its pair of two-year studies are each six months longer than lecanemab’s pivotal trial.
Whether those differences make gantenerumab a better medicine remains to be seen. But lecanemab’s ostensible success sets a bar for Roche clear. And with both treatments scheduled for detailed dissection at a medical conference next month, the stakes are high.
Read more. | Protect patients’ healthcare privacy and genetic data The U.S. Department of Defense recently listed BGI as a Chinese military company operating in the United States. American health information collected by BGI has no legal protection against disclosure to the Chinese government. The healthcare community must ensure patients’ health records and genetic data are protected and not used for nefarious purposes, including genetically targeted bioweapons and bio-surveillance. Learn more at protectmypatientdata.com. | A massive test of the FDA’s authority The FDA is going to trial today, kicking off a three-day hearing that will both decide the future of a controversial drug and shed light on just how much power the agency has to enforce its own regulations.
At issue is Makena, a drug that received accelerated approval in 2011 after the FDA determined it was “reasonably likely” to reduce deaths associated with premature birth. Eight years later, when the manufacturer completed a confirmatory study, Makena showed no benefit. The FDA called for its approval to be revoked, and the manufacturer, Covis Pharma, protested, leading to this week’s slate of hearings.
Beyond the implications for Makena and any patients who might take it, the dispute could have sweeping consequences for the FDA, whose accelerated approval program has come under intense scrutiny in recent years. The pathway was designed to provide early access to potentially transformational medicines with the promise that drugs would be pulled from the market if they don’t ultimately prove beneficial. If the FDA can’t successfully revoke Makena’s clearance, accelerated approval will only face more criticism. | Milestone’s nasal spray hit the mark in Phase 3 An investigational nasal spray from Milestone Pharmaceuticals met its primary goal in a pivotal study, the company said today, restoring normal heart rhythm for patients dealing with rapid palpitations.
As STAT’s Adam Feuerstein reports, Milestone’s drug is meant to treat paroxysmal supraventricular tachycardia, or PSVT, a condition characterized by episodes of rapid heartbeat that start and stop suddenly. In a Phase 3 study of about 700 people, 64% of patients who self-administered Milestone’s drug at the start of a PSVT episode returned to normal heart rhythm within 30 minutes, compared to 31% for participants who treated themselves with a placebo. The difference was statistically significant.
Milestone expects to file for FDA approval by the middle of next year, and its drug would be the first at-home treatment for PSVT, which affects about 1 million people in the U.S. A majority of health care investors estimated peak annual sales of the drug in the range of $300 million to $500 million, according to a survey conducted by Cowen biotech analyst Ritu Baral.
Read more. | More reads - Lawmakers push NIH to disclose steps being taken to ensure clinical trial results are reported, STAT
- CRISPR pioneer Feng Zhang is building a secretive new startup trying to solve gene editing's biggest problem, Business Insider
- Biden takes aim at Big Pharma, Republicans in California, Reuters
| Thanks for reading! Until tomorrow,  | | | |
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