BioMarin taps AI upstart Deep Genomics for discovery pact; Praxis says it has a path to lifting clinical hold
Years after double FDA/EMA rejections left BioMarin’s oligonucleotide for Duchenne muscular dystrophy languishing on the shelf, the company is going back to the drawing board for new oligonucleotide drug candidates — with a healthy dose of artificial intelligence.
Its partner of choice is Deep Genomics, a Toronto-based shop led by star researcher Brendan Frey. Paid an undisclosed upfront, their mandate is to identify and validate new target mechanisms in rare diseases as well as lead candidates. BioMarin will then take over the preclinical work.
Founded in 2014 ahead of a new crop of startups that promise to boost the probability of success in pharma, Deep Genomics’ claim to fame is going from target identification to declaring a candidate in 18 months with a platform that “does the whole thing.”
“I expect that we’ll identify lead candidates for over half of the programs within 12 months,” Frey told Endpoints News. “That’s fast and it means that we can see paths to success and growth in a matter of a year or two, instead of five years like you see with other deals.”
As Deep Genomics positions itself as the go-to AI therapeutics company for novel targets and oligonucleotides, Frey added that he and chief business officer Amanda Kay are looking to do another deal in the first quarter of 2021.
With the discovery deal, BioMarin is taking a crack at an area where it was severely bruised.
The rare disease drug developer once bet $680 million on Prosensa and drisapersen, its exon-skipping antisense oligonucleotide, which ultimately flopped pivotal studies and failed to gain approvals. In recent years it’s turned its focus to gene therapy, with the hemophilia A program Roctavian leading a pack that also features potential once-and-done treatments for phenylketonuria and hereditary angioedema. — Amber Tong
Praxis says it has a path to lifting clinical hold
One week after the FDA slapped Praxis with a stock-denting full clinical hold, the neuroscience upstart says it has a path to get its trial up and running.
Praxis said Tuesday that the agency had asked the company to conduct more animal toxicology studies, particularly for how the drug affects fertility, reproduction and embryo development. Praxis said they believe their ongoing fertility and reproductive tests will answer the agency’s questions.
Those studies are set to be completed in the first quarter of 2021. The FDA also asked for changes to the investigator’s brochure, which summarizes all data on a drug for the agency, and to the contraception requirements in their study.
If they can manage to convince the FDA, they plan to start the Phase II/III study in major depressive disorder in the first half of 2021. Investors had previously expressed high hopes for the drug, PRAX-114, in the hard-to-treat condition, seeding the company with a $100 million launch round and a $190 million IPO.
For now they’ll wait and see. The stock $PRAX is down 9% pre-market, from $31.48 to $28.75. — Jason Mast
Sketching plans for accelerated OK in Fabry, Avrobio touts promising first looks for its other gene therapies
Avrobio’s second — and third — gene therapy programs have shined in their clinical debuts, setting a rosy backdrop for the expansion of the pipeline.
In addition to data on the first patients to be treated with their lentiviral ex vivo gene therapy for Gaucher disease and cystinosis, the biotech said that the Fabry disease data continue to “reflect sustained and durable results” 3.5 years after initial dosing. That paves the way for potential accelerated approval.
“Three months post-gene therapy infusion, the first Gaucher disease patient’s levels of the toxic metabolite plasma lyso-Gb1, as well as plasma chitotriosidase, were lower than the baseline levels when the patient was still on enzyme replacement therapy (ERT),” CEO Geoff MacKay summarized in a statement. “Additionally, the first patient in the investigator-sponsored trial for cystinosis, now out one year, remains off both oral and eye drop cysteamine and we are thrilled to announce that a third patient has been dosed.”
With Gaucher disease type 1, the lyso-Gb1 reduction registered at 22% and plasma chitotriosidase, a biomarker of the “Gaucher cells” that lead to inflammation and severe organ damage, dropped by 17% compared to the patient’s ERT baseline. She remains off ERT, having discontinued one month before the gene therapy infusion.
As for cystinosis — a condition characterized by buildup of the amino acid cystine — a 56% decrease in the number of crystals in the patient’s skin suggests Avrobio’s drug helped him produce his own functional cystinosin protein, in turn preventing the toxic accumulation.
Having added a new program for Gaucher type 3, Avrobio now has six pipeline assets that they say “share tremendous synergies in clinical development, manufacturing, regulatory processes and commercialization.” — Amber Tong
Evox partners with its old parent
The Oxford spinout announced Tuesday that it signed a strategic collaboration with Oxford University. The biotech will team with the Oxford-Harrington Rare Disease Centre to devise ways of applying exosomes in the treatments of rare disease. The partnership will last three years.
An increasingly hot space for drug development, exosome therapeutics involve mimicking the cellular postal services tissues use to send messages throughout the body. By copying these discrete envelopes, companies hope to hit new targets and shuttle drugs into spaces they wouldn’t ordinarily reach. — Jason Mast