Galecto shelves high dose of IPF drug after SAE imbalance; Longtime Aveo partner hands back HER3 antibody
Galecto $GLTO is yanking the top dose of its IPF drug GB0139 in a clinical study after the safety monitoring board flagged an imbalance of adverse events in the study pointing to the therapy.
Galecto expects to continue recruiting patients who are not taking nintedanib or pirfenidone at screening and who would be randomized to receive GB0139 3 mg or placebo. The DSMB recommended the patients randomized to the 10mg group and all those taking nintedanib or pirfenidone should be discontinued from the study. Based on these recommendations, the Company plans to work with both the study investigators and the appropriate regulatory authorities to implement these changes promptly.
“Based on our prior phase 1b/2a study of GB0139 in IPF patients, we believe the 3 mg dose has the potential to be an effective clinical dose for these patients,” said Hans Schambye, CEO of Galecto. As of now, the company adds, they don’t expect the board’s recommendations to affect other studies. — John Carroll
Aveo gets back a drug as longtime partner leaves the collaboration
Boston-based Aveo is getting back rights to a drug dubbed AV-203, an IgG1 monoclonal antibody that targets ErbB3 (also known as HER3). And the biotech says it’s glad to have control again.
CANbridge Life Sciences has terminated their 5-year relationship, handing back its rights for ex-North American sales. The move comes after Aveo completed a small study of the drug, seeing a dose-limiting adverse event and one of two neuregulin positive (NRG1+) patients experiencing a partial response.
“AV-203 has demonstrated early signs of activity in an NRG1+ patient that suggest it could have meaningful application in several areas of high unmet need in cancer,” says CEO Michael Bailey. “We look forward to advancing AV-203 in the clinic as part of our strategy for delivering long-term value from our pipeline programs.” — John Carroll
Japanese investors back a small A round for vaccine player VLP
A low-profile vaccine effort has raised a $16 million A round to back their development work.
The Gaithersburg, MD-based VLP Therapeutics has a lead program for a cancer vaccine, with other programs in place for malaria, dengue as well as Covid-19. The money c0mes from Miyako Capital Co., Sojitz Corporation, and Konishiyasu Co. in Japan along with three existing investors in the US: Robert G. Hisaoka, SK Impact Fund and RJ Fund.
Masayoshi Fujimoto, president and CEO of Sojitz Corporation, noted: “We, Sojitz Corporation, are very pleased to work with VLPT CEO Wataru Akahata, an ambitious scientist with considerable experience in vaccine R&D, as well as with the members of the research team and the company founders who are well-versed in pharmaceutical development. We will do whatever we can to help VLPT grow going forward.” — John Carroll
Novartis pushes early SMA screening with more data on quick intervention with Zolgensma
Novartis wants parents to know that their children have a lot more to gain if they’re diagnosed on a pre-symptomatic basis with SMA.
Pointing to long-term follow-up data from existing studies the pharma giant underscored continued gains for children who received the groundbreaking gene therapy before they started to exhibit symptoms.
“When treated with Zolgensma prior to the onset of symptoms, children in the SPR1NT trial achieved milestones like sitting, standing and walking at an appropriate age, grew as expected without nutritional assistance, and remained free of all forms of mechanical ventilatory support,” said Kevin Strauss, medical director for the Clinic for Special Children in Pennsylvania. “This stands in sharp contrast to the natural progression of SMA Type 1, which would otherwise render them helpless within the first year of life and unable to swallow, breathe, or survive without mechanical support.” — John Carroll
Ocuphire reveals positive PhIII for eye exam dilating agent
Ocuphire is potentially one step closer to seeing a new product approval following a positive Phase III readout Monday.
The Farmington Hills, MI-based biotech released data Monday showing its Nyxol product for pupil dilation in eye exams met its primary endpoint. In 185 individuals, mostly adults but which included 14 children over age 12, 49% of patients returned to less than 0.2 mm of their baseline pupil diameter at 90 minutes compared to 7% of subjects treated with placebo.
That resulted in a sparkling p-value of p<0.0001. Patients received either Nyxol or placebo, in a 1-to-1 randomization, one hour after getting one of three dilating agents in their study eye.
“In addition, Nyxol demonstrated significant benefit through 6 hours across the range of commonly used mydriatic agents, light and dark iris colors, and age cohorts,” said Ocpuhire board member Jay Pepose in a statement.
With Monday’s data in hand, Ocuphire is looking at an early 2023 NDA application. — Max Gelman
BioXcel wins BTD for dementia-related agitation program
A little over two months after putting out top-line data in an early stage trial for agitation in dementia and Alzheimer’s disease, BioXcel has picked up backing from the FDA.
Known as BXCL501, the candidate received breakthrough therapy designation Monday for agitation in dementia, including Alzheimer’s, the company said Monday. The designation was based on January’s Phase Ib/II trial, which showed a statistically significant calming effect compared to placebo without serious side effects.
“Managing dementia related agitation, specifically in elderly patients, represents a significant challenge for physicians and caregivers, as there are currently no FDA-approved therapies and off-label drugs come with black box warnings,” CEO Vimal Mehta said in a statement.
BXCL501 is a reformulation of Pfizer’s 21-year-old sedation drug Precedex. BioXcel created a dissolving film-based, sublingual version of the drug — like Listerine strips, but placed under the tongue — that patients administered themselves. The candidate produces a calming effect without knocking a patient out. — Max Gelman