In­ozyme brings $67M to the bank to build up its cal­ci­fi­ca­tion-fo­cused rare dis­ease pipeline

In­ozyme founder Ax­el Bolte is in­ten­si­fy­ing his faith in his rare-dis­ease up­start’s lead ex­per­i­men­tal drug by test­ing it for a sec­ond in­di­ca­tion fo­cused on dis­or­ders char­ac­ter­ized by ab­nor­mal cal­ci­fi­ca­tion. An ex­pand­ed clin­i­cal pro­gram for the drug — which is be­ing read­ied for hu­man test­ing — needs more cap­i­tal, and the biotech has raised some in a fresh $67 mil­lion round, led by Piv­otal bioVen­ture Part­ners and Sofinno­va In­vest­ments.

Bolte launched the com­pa­ny with a $49 mil­lion round and armed with slate of no­table bio­phar­ma ex­ec­u­tives, af­ter run­ning in­to Yale sci­en­tist Demetrios Brad­dock and his work on cal­ci­fi­ca­tion in his ca­pac­i­ty as an in­vest­ment ad­vi­sor for HBM Part­ners. He spent most of 2016 rais­ing funds, and in 2017 In­ozyme was of­fi­cial­ly born.

Sarah Bha­gat

The Boston, Mass­a­chu­setts-based com­pa­ny —  which has raised $116 mil­lion in ven­ture cap­i­tal to date — has bet on the role of py­rophos­phate, or PPi, in rare, life-threat­en­ing dis­or­ders that are man­i­fest­ed by the over-cal­ci­fi­ca­tion of soft tis­sues and un­der-min­er­al­iza­tion of bone. Armed with this sci­en­tif­ic ba­sis In­ozyme is de­vel­op­ing en­zyme re­place­ment ther­a­pies us­ing in­or­gan­ic PPi to treat cal­ci­fi­ca­tion dis­or­ders.

This new in­fu­sion will help shep­herd its lead en­zyme re­place­ment ther­a­py — INZ-701 — in­to hu­man stud­ies for use in for se­vere dis­or­ders of cal­ci­fi­ca­tion as­so­ci­at­ed with de­fi­cien­cies in the en­zyme ENPP1, in­clud­ing Gen­er­al­ized Ar­te­r­i­al Cal­ci­fi­ca­tion of In­fan­cy and Au­to­so­mal Re­ces­sive Hy­pophos­phatemic Rick­ets Type 2. In­ozyme is cur­rent­ly com­plet­ing IND‑en­abling stud­ies for the drug and ex­pects to ini­ti­ate clin­i­cal tri­als in 2020.

The funds will al­so be used to test the drug in pa­tients with mu­ta­tions in the ABCC6 gene, which can lead to a con­di­tion known as pseu­dox­an­thoma elas­ticum.

In­ozyme has al­so added to its pipeline by adding a sec­ond re­search pro­gram tar­get­ing an undis­closed rare bone dis­ease.

The Se­ries A2 fi­nanc­ing al­so in­clud­ed the par­tic­i­pa­tion of RA Cap­i­tal Man­age­ment, Cowen Health­care In­vest­ments, Rock Springs Cap­i­tal, Lon­gi­tude Cap­i­tal, NEA, No­vo Hold­ings, and Sanofi Ven­tures.

Rob Hopfn­er, man­ag­ing part­ner of Piv­otal bioVen­ture Part­ners, and Sarah Bha­gat, prin­ci­pal at Sofinno­va In­vest­ments, al­so joined In­ozyme’s board.

Im­age: Ax­el Bolte. TUR­OS CAP­I­TAL

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.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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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.

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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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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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, 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.