Technology of genomically recoded organisms borne out of Yale and Stanford university laboratories and housed at Khosla Ventures-backed Pearl Bio received validation on March 12 through a $1 billion deal signed with Merck & Co. Inc. Cambridge, Mass.-based Pearl is eligible for the funds through up-front, option and milestone payments, plus potential royalties on sales of deal-related products that gain approval. The company aims to create multi-functionalized therapeutics with tunable properties, bringing new biologics for cancer to Merck. It was founded by Farren Isaacs, a Yale University professor of molecular, cellular and developmental biology, as well as biomedical engineering, and Michael Jewett, a Stanford University professor of bioengineering. Both are science advisers for Pearl Bio. Another co-founder is Amy Cayne Schwartz, who serves as the company’s chief business officer and chief operating officer.

US FDA’s briefing doc sends Geron tumbling

Although Geron Corp.’s imetelstat met its primary and key secondary endpoints in a phase III study, the U.S. FDA is questioning the magnitude and durability of the effect of the first-in-class telomerase inhibitor as a second-line treatment of transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes. The agency’s concerns resulted in a nearly 15% stock tumble today after the FDA released its briefing document for Thursday’s Oncology Drugs Advisory Committee meeting, in which the panel will be asked to vote on whether imetelstat’s benefits outweigh its risks. After a high of $2.40 yesterday, Geron (NASDAQ:GERN) opened today at $1.93. Following a few brief mid-morning peaks, it slid below $1.85.

Acadia halts pimavanserin development after a phase III fail

It’s the end of the development line for Acadia Pharmaceuticals Inc.’s main asset, pimavanserin. The company said it won’t conduct any more clinical studies on the selective serotonin inverse agonist and antagonist targeting 5-HT2A receptors after a phase III fail in schizophrenia. Top-line results from the Advance-2 study show the treatment missed the primary endpoint, the change in baseline from week 26 on an assessment of symptoms compared to placebo. The drug will still be around in the form of Nuplazid, pimavanserin’s brand name, which was approved for treating Parkinson's disease psychosis by the U.S. FDA in April 2016. The miss caused the stock (NASDAQ:ACAD) to plunge 16% to $2.28 a share at midday.

Regulus jumps on phase Ib data in kidney disease

Phase Ib data pushed shares of San Diego-based Regulus Therapeutics Inc. (NASDAQ:RGLS) almost 60% higher to trade at $2.20, up 82 cents, as the firm disclosed positive top-line results from the second cohort of patients in its with RGLS-8429 for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The multiple ascending-dose study is testing the safety, tolerability, pharmacokinetics and pharmacodynamics of RGLS-8429 in adults with ADPKD. Among other upbeat findings is a mechanistic dose response that turned up at the 2-mg/kg level, based on urinary biomarker analyses.

Dimerix raises AU$20M on positive phase III kidney disease data

Dimerix Ltd. announced a AU$20 million ($US13.22 million) capital raise following the news that its lead candidate, DMX-200, was successful in a prespecified interim analysis of the efficacy endpoint in its pivotal phase III trial in focal segmental glomerulosclerosis (FSGS), a rare kidney disease. “DMX-200 successfully passed its first interim analysis in the ongoing phase III trial – there were no safety concerns, and it is performing better than placebo in reducing proteinuria in patients with FSGS in a much larger cohort than the prior phase II study,” Dimerix CEO Nina Webster said during a March 12 conference call. Having passed the first analysis, the capital raise means the trial is now fully funded and is being formally expanded, Webster said. 

CROI 2024: New options, and concerns, on HIV drug resistance

One topic at the 31st Conference on Retroviruses and Opportunistic Infections held in Denver this month was that resistance to antiretroviral therapy (ART) has become a public health problem for people living with HIV. Without a vaccine or a cure, these patients depend on treatments that suppress viremia by preventing the virus from replicating. They are lifelong treatments and, until new advances succeed in eradicating the virus from reservoirs, the only option available.

Cognition aims for $11.5M in public offering to progress AD asset

With early phase II trial data from Cognition Therapeutics Inc.’s lead candidate, CT-1812, for Alzheimer’s disease (AD) expected in mid-2024, the firm is hoping to raise $11.5 million from an underwritten public offering of common stock of 6.6 million shares, priced at $1.75 each.

US DOJ sets more aggressive course for qui tam activity

U.S. deputy attorney general Lisa Monaco recently outlined some new programs related to federal enforcement across the economy, including some novel elements related to artificial intelligence. However, the more important take-away, according to Preston Pugh of Crowell & Moring LLP, is that the Department of Justice continues to work to make the legal environment more friendly to would-be whistleblowers, thus increasing the risk for companies inside and outside the life sciences.

Also in the news

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