Al­ny­lam, Sanofi flag an­oth­er promis­ing step for­ward for PhI­II he­mo­phil­ia drug

Al­ny­lam $AL­NY and its part­ners at Sanofi $SNY say their he­mo­phil­ia drug fi­tusir­an has tak­en a promis­ing step for­ward in the clin­ic, clear­ing an­oth­er leg of a Phase II study with con­tin­ued ev­i­dence of safe­ty and ef­fi­ca­cy that backs its re­cent move in­to a piv­otal tri­al.

Fi­tusir­an was one of the key rea­sons why Sanofi de­cid­ed to buy in­to Al­ny­lam and its RNAi ap­proach with a $700 mil­lion eq­ui­ty deal three years ago. And it’s al­so cen­tral now to Al­ny­lam’s case to in­vestors that its Phase III pipeline is grow­ing with promis­ing new meds.

Akin Ak­inc

Ap­pear­ing at the In­ter­na­tion­al So­ci­ety of Throm­bo­sis and Haemosta­sis Con­gress in Berlin to­day, in­ves­ti­ga­tors not­ed that the 33 pa­tients tak­ing fi­tusir­an passed the 11-month me­di­an mark on dos­ing with an an­nu­al­ized bleed­ing rate of 1 in the full group and 0 among the 11 pa­tients who had de­vel­oped in­hibitors to stan­dard ther­a­pies. Most of the pa­tients suf­fered from he­mo­phil­ia A, though there were al­so 6 he­mo­phil­ia B pa­tients in the study.

Sig­nif­i­cant­ly, the me­di­an an­nu­al­ized bleed rate was 12 in all pa­tients pre-study, and 38 in in­hibitor pa­tients.

“We are quite en­cour­aged,” Al­ny­lam’s Akin Ak­inc – the gen­er­al man­ag­er of the pro­gram – told me in a pre­view of to­day’s da­ta up­date.

There was a de­cline in the num­ber of pa­tients who re­mained bleed-free in the study, down to 48%. But Ak­inc notes that isn’t un­ex­pect­ed.

“We are pret­ty pleased with that num­ber,” he says. You should ex­pect to see that fig­ure trick­le down, he adds, “that num­ber will nev­er go up.”

As in­ves­ti­ga­tors have al­ready not­ed, there was an is­sue with el­e­vat­ed liv­er en­zymes in the study, though Ak­inc says the one dis­con­tin­u­a­tion that prompt­ed was in a pa­tient with hep C, which could have been an is­sue. An­oth­er pa­tient on the drug dis­con­tin­ued due to seizure and con­fu­sion in a pa­tient with seizure dis­or­der.

I asked him about emi­cizum­ab be­fore Shire is­sued its broad­side against the Roche drug. But Ak­inc wasn’t in a crit­i­cal frame of mind. He of­fered a “tip of the hat” to Roche on the da­ta so far and not­ed that it is hard to com­pare drugs with­out a head-to-head study. That said, he added, the he­mo­phil­ia com­mu­ni­ty is now see­ing a va­ri­ety of dif­fer­ent ther­a­pies com­ing through late-stage de­vel­op­ment, in­clud­ing among gene ther­a­py play­ers. And that will ben­e­fit every­one.

Al­ny­lam and Sanofi an­nounced on Fri­day that the fi­tusir­an Phase III was get­ting un­der­way. Its im­por­tance was mag­ni­fied con­sid­er­ably af­ter the Cam­bridge, MA-based biotech had to scrap its num­ber 2 drug due to safe­ty con­cerns.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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