PHILADELPHIA – With a major gap remaining between the demographics of people enrolled in clinical trials and those most likely to use the drugs the FDA approves, the agency is pushing forward with new steps to tackle the complex problem, issuing a draft guidance on enhancing trial population diversity on Thursday. If successful, the move could complement the efforts of industry and its partners as they pursue everything from community outreach to simple logistical supports.
PHILADELPHIA – Against a backdrop of President Donald Trump's controversial comments about U.S.-Britain trade Wednesday, panelists at the Biotechnology Innovation Organization's (BIO) international convention painted a mixed picture of Brexit's potential impacts. Preparations for likely disruptions started at day one following the referendum, an EMA representative said. Despite that, "if there's one thing certain about Brexit, it's that's there's lots of uncertainties," said Tineke Van hooland, chairwoman of Bio.be, a trade organization representing Belgian biotechs.
Excited by early data on an investigational IL-4/IL-13 receptor antibody it in-licensed in 2014, Aslan Pharmaceuticals Pte. Ltd. has agreed to pay its source, CSL Ltd., as much as $780 million for full global rights to the asset, ASLAN-004, setting aside earlier plans to collar a global partner. Instead Aslan's team now plans to pay CSL $30 million upon starting a phase III study of the drug itself, after first tackling a multiple ascending-dose study in patients with moderate to severe atopic dermatitis (AD).
Excited by early data on an investigational IL-4/IL-13 receptor antibody it in-licensed in 2014, Aslan Pharmaceuticals Pte. Ltd. has agreed to pay its source, CSL Ltd., as much as $780 million for full global rights to the asset, ASLAN-004, setting aside earlier plans to collar a global partner. Instead Aslan’s team now plans to pay CSL $30 million upon starting a phase III study of the drug itself, after first tackling a multiple ascending-dose study in patients with moderate to severe atopic dermatitis (AD).
A medicine that may bring people with bipolar depression and acute suicidal ideation a safer, non-hallucinogenic alternative to repeated ketamine use for relapse prevention has succeeded in a small midstage study, supporting pursuit of a pivotal trial by its developer, Neurorx Inc. The Philadelphia-based company said based on its phase II data, the FDA has granted the fixed-dose cycloserine/lurasidone combination, NRX-101, a breakthrough therapy designation and a special protocol assessment for the pivotal study, expected to begin in the second half of this year.
Ayala Pharmaceuticals Inc., a company developing small-molecule therapies for genetically defined cancers, has completed a $30 million series B financing. The Rehovot, Israel-based startup said the funds will fuel plans to advance its lead candidate, a Notch inhibitor called AL-101, through a phase II study in adenoid cystic carcinoma (ACC) and into a second midstage study in triple-negative breast cancer (TNBC). Novartis AG, Ayala's partner on a second candidate for the potential treatment of multiple myeloma, AL-102, led the financing with a $10 million equity investment. Other participants included SBI JI Innovation Fund, a partnership between the Japanese-based financial services group SBI Holdings and Vertex Venture Capital, and all Ayala's previous investors.
New data on Biomarin Pharmaceutical Inc.'s investigational treatment for severe hemophilia A, valoctocogene roxaparvovec (valrox), sparked mild debate among analysts who expressed appreciation for the gene therapy's apparent durability but uncertainty regarding interim data from a phase III test of the medicine. But overall, it "at least met – and in some cases exceeded – the majority of investors' expectations," J.P. Morgan analyst Cory Kasimov said. Though the market rendered its own verdict, driving company shares (NASDAQ:BMRN) 5.1% lower to $84.50 by Tuesday's close, the program seemed poised to retain significant interest going forward.
A new gene therapy developed by Novartis AG's Avexis Inc. unit, Zolgensma (onasemnogene abeparvovec), has won FDA approval for the treatment of all types of spinal muscular atrophy (SMA) in children under 2 with biallelic mutations in the survival motor neuron 1 (SMN1) gene, news that was rapidly followed Friday by approval of Piqray (alpelisib), the first PI3K inhibitor to be marketed for the treatment of breast cancer.
Showing clear confidence that its investigational mu-opioid agonist, NKTR-181, will gain FDA approval this summer, Nektar Therapeutics Inc. is talking up a recently established subsidiary poised to launch and market the chronic back pain drug. The new entity, Inheris Biopharma Inc., "will lead all of the preparations for the potential commercialization of NKTR-181, as well as development of other CNS programs, enabling Nektar to remain focused on advancing our immuno-oncology and immunology development pipeline," said Howard Robin, Nektar's president and CEO.
Little more than a week after corralling $150 million to create and build a portfolio of cell and gene therapy companies, Elevatebio Inc. is revealing its first play, backing a Houston-based startup making allogeneic cell therapies to battle viral pathogens. The Baylor College of Medicine spinout, Allovir, will use a new $120 million series B financing to initiate multiple pivotal studies for its lead product, Viralym-M, in the next 12 months, while also advancing a second candidate to the clinic.
