Brainstorm Cell Therapeutics Inc. said it’s exploring all its options in the wake of yesterday’s U.S. FDA advisory committee vote, in which the committee overwhelmingly disagreed with the company that the data it presented supported the effectiveness of Nurown (debamestrocel) for the treatment of mild to moderate amyotrophic lateral sclerosis. While trading was halted during the committee meeting, the Street reacted strongly to the vote today, with shares of Brainstorm (NASDAQ:BCLI) dropping as low as 15 cents in premarket trading, down more than 61% from a close of 39 cents Sept. 26. Brainstorm, which had been trading as high as $1.08 last week, had been in a bit of a free fall since the FDA released its briefing document for the adcom.

Bionomics stock charges ahead with positive PTSD data

Positive top-line data from the phase IIb study of BNC-210 to treat post-traumatic stress disorder (PTSD) has super charged Bionomics Ltd.’s stock. At midday, shares (NASDAQ:BNOX) were trading 255% higher at $2.50 each. The oral, selective negative allosteric modulator of the alpha7 nicotinic acetylcholine receptor demonstrated stumbled last December in a phase II trial for treating social anxiety disorder but has regained momentum. The new data show the monotherapy met the primary endpoint of change in Clinician-Administered PTSD Scale for DSM-5 total symptom severity score from baseline to week 12 (p=0.048). There was also a statistically significant change in the score at weeks four (p=0.015) and eight (p=0.014). The symptoms include nightmares, flashbacks and emotional distress.

Immunovant to raise $450M on ‘potent’ falls in IgG levels with MAb in phase I

Immunovant Inc. is seeking a $450 million windfall on the back of the “best-in-class potential” of its early stage monoclonal antibody for IgG-mediated autoimmune diseases, IMVT-1402, which a phase I readout demonstrated could supersede the firm’s lead, batoclimab. In total, the company selling 7.4 million shares of common stock at a price of $38 per share and has also granted underwriters a 30-day option to buy up to an additional 1.1 million shares. Separately, it has also agreed to sell 4.5 million of its common stock to Roivant Sciences, Ltd. at $38 per share, in a private placement.

Samsung, Celltrion set record targets as S. Korea banks on Bio Economy 2.0

As South Korea increases its stakes on the “bioeconomy” as its next growth engine and as its “second semiconductor industry,” leading domestic biologic and biosimilar drug producers like Samsung Biologics Co. Ltd. and Celltrion Inc. are setting record production targets to become forerunners in the global playing field.

Genetic editing of individual cells points to late targets for developmental disorders

A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus to target, edit and analyze single-cell genetic perturbations.

Fabre-Kramer’s NME Exxua approved in US for MDD 

It has been a long time coming, but Fabre-Kramer Pharmaceuticals Inc. finally received U.S. FDA approval for its major depressive disorder candidate, Exxua (gepirone hydrochloride extended-release tablets). The approval comes three months after the June 23 PDUFA date (as a result of amendments filed by the company in April and May), but 24 years after the original NDA was filed in 1999. The company resubmitted the NDA in December 2022, which was considered a complete response to the November 2007 not approvable letter. Fabre-Kramer received a favorable ruling for the product, then called Travivo, in 2016 by the FDA’s Office of New Drugs. Exxua, a new molecular entity, selectively targets the serotonin 1A receptor, a key regulator of mood and emotion, and has been shown to relieve depressive symptoms without the sexual side effects seen with other treatments.

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