Shares of Annexon Inc. (NASDAQ:ANNX) tumbled 57%, or $2.97, to trade midday at $2.20 on word of a primary endpoint phase II miss in geographic atrophy (GA). Twelve-month data from the study called Archer with ANX-007, which inhibits C1q and its downstream activity, fell short in the mean rate of change, or slope, in the GA lesion area compared to sham. But patients treated monthly and every other month were protected against vision loss as measured by changes from baseline in the functional endpoint of best corrected visual acuity. The firm will meet with regulators to talk about next steps.
Bioxcel has mixed phase III data and a tough stock day
Mixed phase III study results have Bioxcel Therapeutics Inc. moving ahead to complete the clinical trial while withstanding a hammering from investors. BXCL-501 (dexmedetomidine), a sublingual film being developed to treat bipolar disorders- or schizophrenia-associated agitation, produced clinically meaningful efficacy results in part 1 of the study with half of the approved dose, but the primary efficacy endpoint was not statistically significant at two hours (p=0.077). BXCL-501 separated from placebo at four hours (p=0.049). BXCL-501 was approved under the brand name Igalmi in April 2022 for acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. The company’s stock (NASDAQ:BTAI) was down 30% at midday to about $18 a share.
Varmx adds €30M to series B, preps for pivotal testing of DOAC reversal agent
Varmx BV has raised €30 million (US$32 million) in an extension to its series B to fund preparations for the pivotal trial of VMX-C001, a modified recombinant factor X that acts as a universal antidote to reverse the effects of direct oral anticoagulants. The drug is intended for use in patients suffering severe spontaneous bleeding or needing emergency surgery. The new funding was announced a month before the company publishes the results of a first-in-human study. While not able to disclose any of the data, Jan Öhrström, CEO said he is satisfied the trial has gone very well and Varmx has a clear idea of the dose range that will be tested in the pivotal trial.
Mirati’s sitravatinib combo fails in phase III lung cancer trial
Providing no details, Mirati Therapeutics Inc. failed to meet its overall survival primary endpoint in the phase III Sapphire trial with sitravatinib. The receptor tyrosine kinase inhibitor was tested in combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) vs. docetaxel in patients with second- or third-line advanced nonsquamous non-small-cell lung cancer. This patient population consists of about 70,000 people in the U.S. and Europe, according to Mirati. While the company intends to disclose the Sapphire data “at a future date,” shareholders expressed their displeasure on May 25, as shares (NASDAQ:MRTX) plunged 13.4%, or $5.86, and were trading at $37.98.
Let there be walk: Onward Medical sees first use of movement-restoring lead
The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external computer to direct leg movements.
Bayer’s PI3K inhibitor available in China market for follicular lymphoma
Bayer AG’s copanlisib was approved in China for the treatment of adult patients with relapsed follicular lymphoma who have received at least two prior systemic therapies. It is the first indication approved in the country for copanlisib, entering a market with room to grow but marked by some domestic competition.
Oral COVID-19 antiviral Paxlovid wins full FDA approval
Pfizer Inc.’s Paxlovid (nirmatrelvir/ritonavir) became the first oral antiviral to win full U.S. FDA approval for treating COVID-19, cleared for use in adults who are at high risk for progression to severe disease, including hospitalization or death. The nod follows two months after the agency’s Antimicrobial Drugs Advisory Committee voted 16-1 in favor of Paxlovid’s overall benefit-risk assessment.