News briefing: Axovant faces months of delay on lead Parkinson's gene therapy; Chinese CAR-T biotech nabs $100M
One of Axovant’s top gene therapy prospects for its second act is hitting a roadblock that could push its clinical timelines back by almost a year.
In an update, the biotech said it was informed about delays in CMC data and third-part fill-finish issues around mid-October by its manufacturing partner, Oxford Biomedica. Axovant has been developing a suspension-based process for the Parkinson’s drug; with that taking longer than expected, it now believes “it is unlikely that its planned randomized, sham-controlled trial of AXO-Lenti-PD will enroll patients by the end of calendar year 2021.”
It had previously expected the manufacturing to be complete by the end of the year and to enroll the first patient by early 2021.
Axovant had licensed the therapy, AXO-Lenti-PD, from Oxford Biomedica for $30 million upfront. Comprising a lentiviral vector, It’s designed to deliver three genes — for tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase — to restore dopamine levels.
In Part A of the SUNRISE-PD trial, Axovant had reported that two patients who were given the lowest dose saw an improvement in motor function after being washed out of oral levodopa therapy.
The company said new follow-up data on the second cohort continue to support its hope for a one-shot Parkinson’s treatment.
Faster, cheaper CAR-T promises spur $100M financing for Chinese player
Chinese CAR-T player Gracell Biotechnologies has secured $100 million — and the backing of marquee investors — in a Series C.
Wellington Management, OrbiMed and Morningside (freshly rebranded 5Y Capital) co-led the round. Vivo Capital pitched in for the first time, joined by old-timers Temasek, Lilly Asia Ventures and King Star Capital.
Based out of Shanghai with a manufacturing site in Suzhou, Gracell set out to dismantle some of the constraints limiting the first generation of CAR-T therapies: high production costs, lengthy manufacturing process, lack of off-the-shelf products and a short duration of effects.
Its tech platforms can generate both autologous and allogeneic cell therapy candidates, promising a manufacturing process under 24 hours, a way to target two antigens at once and genetic tweaks to donor T cells.
6 Dimensions helped founder and CEO William Cao get it off the ground with $10 million Series A three years ago, and a year later Gracell scored $85 million more to push its programs toward the clinic.
Cao had previously founded and led Cellular Biomedicine Group, which has a partnership with Novartis to develop and commercialize Kymriah in China as well as other research pacts.
Gracell said the new cash will go toward both its Phase I product for relapsed or refractory T cell acute lymphoblastic leukemia and other preclinical candidates.
J&J funnels two more IL-23 oral peptide drugs from Protagonist into pipeline
Three years after first buying into Protagonist’s lead oral peptide, J&J is grabbing two more IL-23 receptor antagonists for its portfolio.
“The IL-23 pathway is a validated therapeutic mechanism, but orally delivered therapeutics for this class have not been available,” Dinesh Patel, Protagonist president and CEO, said in a statement. “The advancement of three different oral co-development candidates provides us with several strategic options for development in multiple indications.”
PTG-200 (JNJ-67864238), the initial drug J&J licensed, is in a Phase II proof-of-concept study in moderate to severe Crohn’s disease — one of its top inflammatory bowel disease targets.
PN-235 (JNJ-77242113) and PN-232 (JNJ-75105186) are both preclinical at the moment, although PN-235 is expected to enter the clinic later this year.
AbCellera hipsters Kodiak Sciences re-up opthomalogy discovery pact
Kodiak Sciences got in on the once below-the-radar Canadian biotech AbCellera before it was cool, and evidently, they were pleased with the results.
Four years after their first partnership, Kodiak and AbCellera have signed a new deal under which AbCellera will use its microfluidics technology to develop antibodies against targets that Kodiak designates. As with the previous partnership, those targets are undisclosed, but Kodiak is squarely focused on opthalmology, namely preventing and treating leading causes of blindness.
AbCellera spent the last decade quietly with some of the industry’s top drug developers, including Lyell, Gilead, Denali, Autolos, and Novartis. Fame came earlier this year when Eli Lilly tapped them to develop a neutralizing antibody against Covid-19, which is now in late-stage trials and which, despite mixed results, the US government just signed a blockbuster contract to acquire in bulk.