$49M round; ex­pe­ri­enced team; A-list VCs — In­ozyme launch­es with rare dis­ease strat­e­gy and a yen for big­ger things to come

Ax­el Bolte

It was Ax­el Bolte’s work as a ven­ture part­ner for HBM that orig­i­nal­ly took him to the cam­pus at Yale, with a chance to start meet­ing the sci­en­tists there. And that in­tro­duc­tion even­tu­al­ly led him to Demetrios Brad­dock, who had been do­ing lab work on cal­ci­fi­ca­tion that he’s de­vout­ly hop­ing could have an im­pact on a long line of dis­eases.

“Ul­ti­mate­ly I al­ways want­ed to be di­rect­ly in­volved as an en­tre­pre­neur and op­er­a­tional in com­pa­nies,” says Bolte, “which is what I did be­fore in large or­ga­ni­za­tions.”

It was Brad­dock’s work that made that hap­pen. And to­day Bolte is of­fi­cial­ly open­ing the shop at In­ozyme, the lat­est in a long line­up of star­tups to set out to make their mark in Cam­bridge, MA.

The re­al re­search work at In­ozyme got start­ed ear­ly this year, Bolte tells me. That was af­ter he spent much of 2016 rais­ing cash, which led to to­day’s an­nounced $49 mil­lion A round with some VCs he knows well. Lon­gi­tude Cap­i­tal took the lead for a round that in­cludes New En­ter­prise As­so­ci­ates, No­vo Ven­tures and Sanofi Ven­tures.

Demetrios Brad­dock

They’re back­ing a very fa­mil­iar strat­e­gy that starts with rare dis­eases, a clas­sic strat­e­gy that al­lows for piv­otal da­ta on small pa­tient groups.

Their lead en­zyme re­place­ment ther­a­py is for the treat­ment of Gen­er­al­ized Ar­te­r­i­al Cal­ci­fi­ca­tion of In­fan­cy — GACI, which hits new­borns — and Au­to­so­mal Re­ces­sive Hy­pophos­phatemic Rick­ets Type 2.  GACI is trig­gered by a ge­net­ic mu­ta­tion that caus­es low lev­els of py­rophos­phate and cal­ci­fi­ca­tion of the ar­ter­ies and heart. But af­ter get­ting es­tab­lished in rare dis­eases, Bolte sees a big­ger fu­ture ahead in ma­jor dis­eases like chron­ic kid­ney dis­ease.

Right now the com­pa­ny has 8 staffers, right in line for where it’s at at this stage. It in­cludes some high­ly ex­pe­ri­enced biotechies with re­sumes that lean heav­i­ly on rare dis­eases:

  • COO Hen­ric Bjarke: for­mer vice pres­i­dent and ther­a­peu­tic area head for the meta­bol­ic busi­ness unit at Alex­ion, where he was re­spon­si­ble for as­fo­tase-al­fa for the treat­ment hy­pophos­phata­sia and oth­er de­vel­op­ment pro­grams.
  • Chief tech­ni­cal op­er­a­tions of­fi­cer Steven Jun­gles, a vet from Ul­tragenyx and Bio­Marin.
  • Chief med­ical of­fi­cer Er­ic Yuen, with stints at Mer­ck, John­son & John­son, Ul­tragenyx and Bio­Clin.
  • Reg­u­la­to­ry chief Ruhi Ahmed from Ul­tragenyx and Bio­Marin.
  • Fi­nance VP Stephen Bas­so out of Alex­ion.

Now Bolte plans to dou­ble that num­ber as the com­pa­ny heads to the clin­ic.

“We have some ex­pe­ri­ence in CMC and these kinds of things,” says the CEO. “We ex­pect an IND in 2019 and then clin­i­cal tri­als should start at the end of ’19, with ini­tial tri­al re­sults in 2020.”

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

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Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.