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

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.

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.