$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.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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