LONDON – Evox Therapeutics Ltd. has validated its exosome drug delivery technologies in a $882 million deal with Takeda Pharmaceutical Co. Ltd., in which the partners will develop protein replacement and messengerRNA (mRNA) therapies in five rare disease indications. As an indicator of the potential therapeutic power of exosome delivery, the lead program in the collaboration rests on the ability of exosomes to cross the blood-brain barrier and deliver a correct copy of the NPC1 gene, to treat the inherited neurodegenerative disorder, Niemann-Pick type C.
Barely a day after its PDUFA date, despite the unfolding COVID-19 pandemic, the FDA has approved Bristol Myers Squibb Co.'s immunomodulator, ozanimod, an oral treatment for adults with relapsing-remitting multiple sclerosis (MS) and active secondary progressive disease branded as Zeposia. The win, a much-anticipated milestone precipitated by the company’s multibillion-dollar acquisition of ozanimod developer Celgene Corp. in November 2019, gives patients a new treatment option amid a growing field of therapies for MS.
The stimulus bill passed by the U.S. Senate last night on a vote of 96-0 does more than throw $2 trillion into the war against COVID-19. “This is not … a stimulus package. It is emergency relief,” Senate Majority Leader Mitch McConnell (R-Ky.) said on the Senate floor before the vote. Some of that relief translates into new authorities federal health agencies can use in the future. For instance, the bill, which is scheduled for a House vote tomorrow, gives the FDA more tools to mitigate potential drug and device shortages, requires the National Academies to report on the security of the U.S. drug and device supply chain and allows the Biomedical Advanced Research and Development Authority to more easily partner with the private sector on R&D during public health emergencies.
Oncopeptides AB remains on track for an NDA filing in the second quarter for its peptide-drug conjugate melflufen in relapsed refractory multiple myeloma (RRMM). The Stockholm-based firm reported today that the drug attained an overall response rate (ORR) of 30% – as assessed by the independent review committee (IRC) – in the intent-to-treat analysis of the pivotal open-label phase II Horizon study (n=157). According to the IRC assessment, it attained an ORR of 26% in a subgroup of patients who were refractory to three drug classes (n=119) and an ORR of 27% in patients with extramedullary (metastatic) disease (n=57). “They are notoriously hard to treat,” Oncopeptides CEO Jakob Lindberg told an analyst call audience.
Shares of Tel Aviv, Israel-based VBL Therapeutics Ltd. (NASDAQ:VBLT) rose 20 cents, or 17%, to $1.34 midday as Wall Street learned of upbeat findings from the planned interim analysis in the phase III study with gene therapy VB-111 (ofranergene obadenovec) called Oval, a double-blind, controlled, potential registration experiment in patients with platinum-resistant ovarian cancer. An independent data safety monitoring committee reviewed unblinded data and assessed CA125 biomarker response, measured according to Gynecological Cancer Intergroup criteria, in the first 60 enrolled subjects evaluable. The panel confirmed that the study met the interim prespecified efficacy criterion of an absolute percentage advantage of 10% or higher CA125 response rate for the VB-111 treatment arm, and recommended the study go on. The overall CA125 response rate turned up at 53%. Assuming a balanced randomization, the response rate in the treatment arm – testing VB-111 plus weekly paclitaxel – would be 58% or higher. In patients who had post-dosing fever, which is a marker for VB-111 treatment, the response rate totaled 69%.
Privately held Recode Therapeutics Inc., of Menlo Park, Calif., and Dallas, brought in $80 million through an oversubscribed series A financing with plans to continue its preclinical work in primary ciliary dyskinesia and cystic fibrosis. INDs for both programs are set to be filed next year. The company platform will also benefit from the financing, as Recode plans to advance its nonviral lipid nanoparticle delivery technology for organ-specific delivery of RNA therapies and gene editing component. Orbimed Advisors LLC and Colt Ventures co-led the round, with participation from MPM Capital, Vida Ventures LLC, Hunt Technology Ventures and Osage University Partners.
BEIJING – Suzhou-based Innovent Biologics Inc. has inked a deal with Alector Inc. to obtain the rights of the U.S. biotech’s AL-008, a dual function anti-SIRP-alpha antibody, to develop and commercialize it for oncology indications in China. AL-008 has yet to enter clinical trials anywhere. Under the terms, Innovent will be responsible for developing and commercializing the drug in China and will also manufacture the molecule. Alector will continue to develop AL-008 globally, except China. Both companies remained tight-lipped on the financial terms.
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Admare, Agios, Allovir, Arvelle, Astrazeneca, Bellus, Biopharmx, Bluebird, Bolt, Bristol Myers Squibb, Celgene, Cerecor, Citius, Decibel, Delmar, Dynavax, Evofem, Grifols, Healios, Imcheck, Inflazome, Inovio, Ipsen, Ironwood, Jazz, Lift, Lipocine, Medimetriks, Mylan, Novartis, Oncopeptides, Pear, Pfizer, Qurient, Relief, Revive, Saniona, Scholar Rock, Secarna, Sutrovax, Tetra, Tolero, Twist, Uniqure, VBL, Vir, Xellia, Xencor