In gene therapy, responses from just one or two patients can have a huge influence on analysts’ opinions. Just ask the executive team at bluebird bio, which trumpets each patient’s progress with delight — or calibrates a new development strategy when smiles turn to frowns.
So it’s no wonder that Avrobio is so proud of its very first patient.
At a scientific conference in Japan last fall, researchers reviewed the case of a patient with Fabry disease whose blood levels of the enzyme a-Gal A were near zero at the start of treatment. Within 45 days of receiving their gene therapy AVR-RD-01, the patient’s plasma a-Gal A activity increased into the normal range, they said. And 6 months later that hadn’t changed.
In other words, they got the response they wanted and Patient 1 cleared the first hurdle on durability. All systems go for a young company that is out to create a once-and-done fix.
Atlas seeded the company — Bruce Booth is the proud chairman — and then launched it in the spring of 2016 with a $25 million A round that backed the work of 15 staffers. Over the last few months, its staff has doubled. And more hires are being planned for later this year.
That upbeat start has helped inspire a hefty $60 million round to back the mid-stage work that will soon begin for a company that has laid out a broad plan to follow some of the leaders in lysosomal storage disorders with the promise of a cure that could one day relegate the current standard of care for these ailments to an historic footnote.
The Series B round was co-led by Cormorant Asset Management and Surveyor Capital (a Citadel company), and included Aisling, Brace Pharma Capital, Eventide, Morningside and Leerink Partners, along with existing investors Atlas Venture, SV Health Investors and Clarus Ventures.
Getting an investment group like Leerink in your round — not an everyday event in biotech — might make you think that there’s an IPO in the offing, especially with 2018 looking like an especially good year to take a biotech public. Gene therapy pioneers have also earned a lot of respect on Nasdaq, even with some turbulence for every setback.
But Avrobio CEO Geoff MacKay is circumspect about any near-term offerings.
The company’s focus this year is on kicking off a critical Phase II trial for Fabry disease, he says, to run parallel to the Phase I.
There are no timelines for the trials on public display, though.
In gene therapy, even after the first US approval, all companies in the field tend to get striped with the same brush. MacKay’s desire now is to make sure that everyone knows that Avrobio is in the lentiviral camp, where you’ll find leaders like bluebird bio.
“AAV is a different vector,” he says. “It’s not fair to extrapolate to lentiviral.”
And at that point I hadn’t even mentioned the James Wilson study sounding the alarm around high-dose gene therapies using AAV tech earlier in the week.
A breakthrough with a gene therapy would undoubtedly have a huge impact on the patients who depend on steady infusions of some high-priced therapies.
“We’re very respectful of the contribution Genzyme and Shire’s made,” says MacKay. “Twenty years ago these patients had no options, then the patients had an option. When we look at it with fresh eyes, we see a significant opportunity to improve on the standard of care.”
“Patients now rely on biweekly infusions of enzymes, with severe challenges to cost, compliance and quality of life,” adds the CEO. “When ERT succeeds it slows the progression of disease but doesn’t halt it. The central premise is to deliver via a one-time infusion for 24-7 protection, which we hope will halt the progression rather than just slow it down.”
Avrobio has a long ways to go, but it has big plans for when it arrives.
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