With a po­ten­tial cure for lyso­so­mal stor­age dis­or­ders in hand, Avro­bio rais­es $60M to back gene ther­a­py pipeline

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.

Ge­off MacK­ay

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.

Bruce Booth

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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Steven James, Pionyr Immunotherapeutics CEO

Gilead pass­es on ful­ly ac­quir­ing Pi­o­nyr, as eyes now turn to Tizona, a fel­low sum­mer 2020 buy­out op­tion

Gilead and Pionyr Immunotherapeutics, a biotech trying to follow up on the first generation of checkpoint inhibitors, have “mutually agreed” on a rewrite to their 2020 terms, with Gilead deciding not to buy out the company.

The California biopharma waived its option to acquire the remaining 50.1% of Pionyr, which would have triggered a $315 million upfront payment and up to $1.15 billion down the road. Had Gilead waited to decide, the drugmaker would have had a potential payment to make in the near term under their agreement, a spokesperson said in an email to Endpoints News.

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Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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