Most biotechs start life with little money, one or several early-stage assets, a handful of employees and big dreams but face an uphill struggle to stay alive, let alone get to market with a new med.
Biogen’s new spinoff Bioverativ, however, will be trading on the Nasdaq next from the start of month with two products, $325 million in the bank, hundreds of staffers and a new preclinical pipeline of meds for non-cancer blood disorders and cash to boost its R&D efforts.
The biotech, led by Biogen’s former EVP of pharma ops and technology John Cox, will sell Biogen and Sobi’s hemophilia A and B meds Eloctate and Alprolix, but also has its own pipeline of drugs.
Speaking from the J.P. Morgan event in San Francisco, Cox tells FierceBiotech: “We’re not your normal upstart biotech, given what we start with. But we’re not just focused on selling the drugs we have, but on being an independent company with our own drug development strategy.
Cox says the capitalization of the company is “very healthy,” and the money side is also very good. “We start with $325 million in cash, we have essentially no debt, and Biogen will not own any of the company, so we’re fully independent. With the drugs we have, we’re in the line of sight to be a billion-dollar biotech company, so this is not your normal startup. Normal startups dream of having revenue someday, but we start out the gate with that.”
He says the biotech will also be looking for “really smart business opportunities” to expand its pipeline, such as in sickle cell.
On the R&D side, Cox says much of that cash will go into its current portfolio of early-stage assets: “We want to build a great, rare disease and drug development company around blood disorders, which will include R&D for new therapies in haemophilia and sickle cell disease. If you look at our pipeline, the first product that we intend to move into the clinic is BIVV-001, which relates to our Nasdaq symbol [$BIVV] and it’s our first one, and this is a molecule that is a Factor VIII drug, like Eloctate.”
Eloctate is a long-acting therapy, but Cox hopes that BIVV-001 will be even longer-acting. “It works by, what we believe, will overcome a natural half-life cap on Factor VIII. We’ve re-engineered it so it has a VWF component, a polypeptide sequence to increase half-life. Our animal studies are suggesting that its half-life will roughly be around 2x the half-life of Eloctate, which as I say is already long-acting.”
He says the reason they are excited by this is because, even though investors and even doctors talk a lot about gene therapy in the haemophilia space, if you talk to haemophilia A patients about what they really want, “being able to dose, once a week, which is our target with this drug, or even just less frequently, is what they want. I think what we’re doing can really help fill a near-term need for these patients,” Cox says. It could enter the clinic this year.
The second molecule the company is working on, again at an early-stage is BIVV-002, which also uses the so-called XTEN half-life extension tech, which originally comes from Amunix and was bought by Biogen for Bioverativ, with a Factor IX molecule.
“I think this could give us the potential to offer a subcutaneous dose, with the potential to dose less frequently as well. Of course, IV is a more challenging activity for people, and so much of the gene therapy work is being done in haemophilia B, and seems to be the more advanced, with a lot of people working in that space.
“But, if you have a choice of injecting yourself subcutaneously once a week with the Factor that a patient is missing, I think that results in good compliance, convenience and performance, and we hope really good efficacy.” This would mean that people could dose themselves “in accordance with their lifestyle,” which could give the biotech an edge in the increasingly crowded haemophilia market, and not have “the perceived risk profile that a gene therapy would have.”
The biotech is however also working on gene therapy approaches to haemophilia, where Cox says he hopes to test across the whole hemophilia community, of all ages and types.
I asked whether, as some have suggested, that gene therapies could be a cure for the disease. “We’re all hoping for a cure, and of course we’re doing work on gene therapy now, but I don’t think people are looking at these now as a permanent cure; the questions are over durability, rather than cure.”
He said they have a very early-stage product which is poised to integrate into the genome which could, Cox says, be potentially much longer acting than the typical approaches of today, but adds that the curative properties of the medicines today simply haven’t been determined.
“There are of course question marks over gene therapy: The obvious one is safety, because of the history here, and this is a risk-averse population, for good reason, and the other question is naturally over efficacy, and how long does it last, as well as manufacturing, scale and so on. But saying all that, it’s a cool biotechnology, and it is an exciting area to be involved in.