Pour one out for Rubius

Rubius Therapeutics first rose to attention with a 2018 press release claiming its red blood cell platform, still in preclinical development, would lead to "life-changing" treatments, fueling a then-record $2 billion IPO. Five years and a 99% valuation decline later, the company is shutting down and liquidating its remaining assets.

As STAT's Adam Feuerstein writes, the demise of Rubius marks one of biotech's highest-profile flameouts, and it closes the book on the yesteryear industry trend of venture capitalists pouring money into risky, early-stage startups and then foisting them onto the public markets at questionable valuations.

Biotech's protracted bear market is, at least in part, a product of the spendthrift unicorn era that put Rubius on the map. When things eventually turn around for the sector, investors would be wise to remember Rubius' journey from riches to rags before buying into the next hot biotech startup.

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Biotech

Speaking of flameouts

Graphite Bio has issued two press releases in 2023. The first, on Jan. 5, disclosed that the company had paused a Phase 1 study of its genome-editing treatment for sickle cell disease after the first patient dosed developed a serious adverse event. The second, posted yesterday, says Graphite is abandoning that treatment, cutting 50% of its staff, and exploring "strategic alternatives."

The decision, according to CEO Josh Lehrer, came after the company realized the daunting time and resources needed to restart its study and the progress of other potentially curative treatments for sickle cell disease.

It's a rapid unraveling for a company that raised nearly $240 million in a 2021 IPO. Graphite is now "exploring the potential" to sell its lead therapy to another company, and the company is still spending money on research in hopes of finding one or more development candidates.

Research

The monkey-smuggling scandal has implications for pharma

An international smuggling scandal has dramatically constrained the supply of research primates that are integral to preclinical drug development, and it's about to start delaying studies.

As STAT's Ed Silverman reports, Charles River Laboratories, one of the largest contract-research organizations in the world, is suspending shipments of research primates amid a federal investigation into a smuggling ring selling poached macaques instead of purpose-bred ones. The company is waiting on federal authorities to develop new procedures for ensuring imported primates aren't smuggled, a process that will take an unknown amount of time and thus lead to study delays for the company's many pharmaceutical clients.

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R&D

What's going on at Cerevel?

Cerevel Therapeutics, one of biotech's few SPAC success stories, lost about 14% of its value yesterday after the company disclosed yet another delay to one of its pipeline treatments for neurological diseases.

The news is that a mid-stage study testing a drug called darigabat in focal epilepsy is not going to read out this year, as Cerevel had previously expected. That follows last quarter's disclosure that two other studies, in Parkinson's disease and dementia, would be delayed by a year or more, meaning Cerevel will have no data to offer in all of 2023.

The company's most promising treatment, a drug called emraclidine, is still on track to have Phase 2 data in schizophrenia next year. But as Mizuho analyst Graig Suvannavejh wrote in a note to clients yesterday, after three straight pipeline delays, it wouldn't be a surprise "if investor confidence in Cerevel's ability to execute may begin to be called into question."

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