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

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.