| |   | Good morning, all. Damian here with an update on a scandal in Alzheimer's disease, news on the latest rare disease gold rush, and tips on how to spend $5 billion. | | | The race to treat a not-so-rare rare disease gets a little more complicated Partners AstraZeneca and Ionis Pharmaceuticals said yesterday that their investigational medicine for transthyretin amyloidosis succeeded in Phase 3, further crowding a competitive space ahead of this year’s most important data readout.
The news is that the duo’s eplontersen met its two primary goals in a study: improving nerve pain caused by transthyretin amyloidosis, or ATTR, and reducing patients’ levels of the protein transthyretin, whose malformation causes the disease. The former point means eplontersen might be approvable to treat polyneuropathy related to ATTR, which is a small market, but the latter suggests the treatment might eventually prove itself in the much larger and rapidly growing indication of ATTR-caused cardiomyopathy.
That’s where 2022’s biggest binary comes in. Alnylam Pharmaceuticals, whose treatment is already approved for ATTR polyneuropathy, will soon have Phase 3 data in cardiomyopathy. Analysts expect that study to set the bar for other therapies, which include eplontersen and a potentially curative ATTR treatment from Intellia Therapeutics. But success is hardly guaranteed, as BridgeBio learned last year when its promising treatment for ATTR cardiomyopathy failed in a trial similar to Alnylam’s. | PTC’s years-long confirmation process might yet work out PTC Therapeutics, which has spent the last decade insisting that its drug can improve the lives of children with Duchenne muscular dystrophy, said yesterday that the treatment met its goals in a pivotal study.
As STAT’s Adam Feuerstein reports, patients who got PTC’s treatment were able to walk an average of 14.4 meters farther in six minutes compared to 176 patients offered a placebo, a statistically significant difference.
That result seems likely to keep PTC’s therapy on the market in Europe, where it won a conditional approval in 2014, but it might not resolve the company’s lengthy quest to persuade the FDA. PTC conducted a separate analysis of the study using the statistical method preferred by the FDA, and while it showed a relative improvement favoring Translarna, the result was not statistically significant.
Read more. | Nominate a talented young scientist to be named a STAT Wunderkind For the sixth year in a row, STAT is on the lookout for the most impressive young minds in science to give them the recognition they deserve. Nominees must be done with their degree, and on the next leg of their career, but not yet running their own research program independently. Winners will be profiled on STAT and invited to the flagship STAT Summit. Nominate your Wunderkind by July 29. | Meanwhile, in Theranos Five months after a jury found Elizabeth Holmes guilty of fraud, her former second-in-command is heading toward a verdict on identical charges.
Yesterday the court heard closing arguments in the trial of Ramesh “Sunny” Balwani, who served as chief operating officer at Theranos during its head-turning rise and catastrophic downfall. According to prosecutors, Balwani defrauded investors and patients alike by repeatedly lying about what Theranos’ blood-testing machines could do and how much clients were willing to pay for them. To Balwani’s attorneys, he was a true believer in the technology who was misled by Holmes, his boss and former girlfriend.
Theranos’ lies are indisputable, but the jury, which could begin its deliberations as early as this week, will have to decide just how culpable Balwani was for the company’s deception. In January, a different jury found Holmes guilty of lying to investors but innocent of defrauding patients. Balwani, whose job was more directly tied to Theranos’ labs, might not be so fortunate. | A downtrodden biotech is ready to go shopping Things have not gone well for Galapagos. The company has lost about 80% of its valuation since early 2020, and its enterprise value — which is market cap plus debt and minus cash — is a biotech-worst negative $1.4 billion.
But despite all those red numbers, Galapagos is in arguably the best position of any under-performing drug developer. The Belgian firm ended the first quarter with nearly $5 billion in cash, and, as new CEO Paul Stoffels has told analysts since taking over the company in April, it has been fielding enquiries from financially troubled biotech companies looking to out-license their ideas to stay alive.
Yesterday brought the first evidence of how a cash-rich Galapagos might seek to rebuild its pipeline. The company is buying into CAR-T with a roughly $130 million acquisition of the privately held CellPoint, promising another $100 million more tied to future milestones. Separately, it's paying $14 million for AboundBio, a company that makes therapeutic antibodies.
Each is a sign of deals to come, Stoffels told the Belgian newspaper De Tijd, as “it is currently difficult for young biotech companies to raise money on the stock exchange,” and Galapagos has a big enough war chest to bail them out and expand its pipeline in the process. | More reads - Novartis loses in patent appeal over multiple sclerosis drug. Reuters
- Biden picks former DARPA director Prabhakar as next science adviser. STAT
- Startup will target cystic fibrosis with a new twist on gene therapy. Boston Globe
- To protect people with addiction from discrimination, the Justice Dept. turns to a long-overlooked tool: the ADA. STAT
| Thanks for reading! Until tomorrow,  | | | |
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