biotech
Athira's Alzheimer's drug falls short
Athira Pharma, which has already been marred by a data manipulation scandal, said yesterday that Alzheimer's patients who received its experimental injection fosgonimeton did not decline at a significantly slower rate than patients on placebo.
Athira's approach is different from the conventional mechanism that's been tested by many companies, which is clearing amyloid plaques in the brain. Instead, the company hopes to protect neurons and strengthen the synapses between them, by targeting a network of genes known as the HGF/MET pathway.
But in 2021, allegations arose that founder and CEO Leen Kawas altered images in several papers that helped form the basis for the company's formation and biological thesis.
Read more from STAT's Jason Mast.
biotech
BioMarin's big day today
From my colleague Adam Feuerstein: Later this morning, BioMarin will unveil its long-term growth and R&D strategy to an audience of investors and analysts gathering inside a New York City hotel ballroom. The event has been billed as a potentially transformative moment for the rare-disease drugmaker — a chance for new-ish CEO Alexander Hardy and his handpicked team to show Wall Street that BioMarin can not only invent new drugs, but generate sustainable profits from them.
The BioMarin event is expected to focus on four big topics: 1) improving operating margins, mostly by reining in expenses, 2) growing sales of Voxzogo, its treatment for achondroplasia, the most common, hereditary form of dwarfism, and also BioMarin's most important source of immediate revenue growth, 3) revamping its research pipeline, which includes a treatment for Duchenne muscular dystrophy, and 4) looking at potential business development deals under newly appointed Chief Business Officer James Sabry, who joined the company after a long career at Roche.
BioMarin is one of the industry's most successful developers of drugs for rare diseases, but shareholders have long been frustrated by its inability to deliver sustainable profits — or better stock returns. But hopes are high that today's event delivers: BioMarin shares are already up 25% from its recent May low.
venture capital
George Church's transplant startup raises $191 million
From my colleague Allison DeAngelis: eGenesis, the George Church-cofounded startup using gene editing to alter porcine organs for human transplantation, has new funding on hand. The company announced today that it raised $191 million for a Series D round from ARCH Venture Partners, Khosla Ventures, Farallon Capital Management, Alta Partners, and others.
In March, eGenesis became the first company to successfully transplant a kidney from a CRISPR gene-edited pig into a living patient. The Series D funds will be used to test the company's lead candidate, EGEN-2784, in more living patients through a clinical trial. It hasn't disclosed exactly when it plans to launch that trial.
eGenesis aims to tackle the U.S. shortage of transplantable organs by genetically editing pigs to knock out genes that trip up the human immune system, and add in human genes that make the organs more compatible with the human circulatory system. The small breed of Yucatan minipigs that received EGEN-2784 before the March transplant carried a 69 DNA edits.
market check
Vaxcyte challenges Pfizer in David vs. Goliath story
Shares of Vaxcyte soared 36% yesterday, after the biotech reported that its pneumococcal vaccine outperformed Pfizer's Prevnar in a small early trial. (Pfizer's stock dropped 2.5%.) With this new data, Vaxcyte is emerging as one of biotech's hottest David-vs.-Goliath stories, my colleague Matt Herper writes.
Vaxcyte's vaccine includes coverage of 31 different strains of the pneumococcus bacteria, compared with 20 for the Pfizer vaccine. Investors had expected that on some strains that two vaccine had in common, Prevnar would do better, but Vaxcyte said its vaccine did better on every one. This raises hopes that Vaxcyte's candidate will perform well in a larger study set to begin next year.
Read more.
And late yesterday, after Vaxcyte's stock rose, the company said that it would seek to raise $1 billion by selling shares and pre-funded warrants.
regulation
Novartis probed by Greece's antitrust regulator
Greece's antitrust regulator is investigating allegations that Novartis paid doctors and institutions to prescribe its pricey eye drug, Lucentis, and also disparaged rival medicines.
The agency is probing claims that the company paid for doctors' travel and disseminated "defamatory claims and news" in hopes of influencing physicians and consumers to favor its own treatment for age-related macular degeneration, a common disease among the elderly that can lead to blindness.
There's been ongoing concern among European antitrust regulators about the lucrative market for drugs to treat macular degeneration, and Novartis has featured heavily in such probes.
Read more from STAT's Ed Silverman.
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