Bio­Marin lays out its PhI­II strat­e­gy for its close­ly-watched gene ther­a­py for he­mo­phil­ia A

Hen­ry Fuchs

When it comes to gene ther­a­py stud­ies, any­thing oth­er than a per­fect score al­ways rais­es a red flag about their fu­ture in late-stage test­ing. And no­body gets a per­fect score.

Bio­Marin was re­mind­ed of that les­son again to­day as it care­ful­ly laid out the lat­est promis­ing ear­ly-stage da­ta from a hand­ful of he­mo­phil­ia A pa­tients who re­ceived their gene ther­a­py BMN 270.

Their key da­ta were good to great. Ex­clud­ing one of the pa­tients in the high dose group, leav­ing 6 for analy­sis, the mean an­nu­al­ized bleed­ing rate was re­duced by 97% — from 16.3 to 0.5. “The me­di­an ABR for those same pa­tients was re­duced from 16.5 to ze­ro. The mean an­nu­al­ized Fac­tor VI­II in­fu­sions were re­duced by 94% from 136.7 to 8.5. The me­di­an an­nu­al­ized Fac­tor VI­II in­fu­sions were re­duced from 138.5 to ze­ro.” And the low­er, mid-range dose al­so demon­strat­ed an ex­cel­lent score on bleed­ing and in­fu­sion rates, with a drop to ze­ro.

So far, so good. Bio­Marin ex­ecs – which saw its shares $BM­RN climb about 2% — say they are ready to take the high dose in­to a Phase III study dur­ing the 4th quar­ter, plan­ning on a piv­otal pro­gram with few­er than 100 pa­tients.

But a few fac­tors caught an­a­lysts’ eyes in ex­am­in­ing the de­tailed break­down on the da­ta. One of the key is­sues was a wide vari­abil­i­ty in Fac­tor VI­II ex­pres­sion need­ed to keep he­mo­phil­ia in check. Joseph Schwartz at Leerink notes this morn­ing that in­vestors will look close­ly to see if reg­u­la­tors are con­cerned by the much-high­er-than-nor­mal lev­els of Fac­tor VI­II in some pa­tients be­fore ap­prov­ing the Phase III de­sign.

In­ter­est­ing­ly, there were al­so sev­er­al cas­es of el­e­vat­ed ala­nine amino­trans­ferase (ALT) re­quir­ing a ta­per­ing treat­ment of cor­ti­cos­teroids. At one point not long ago, that sort of re­ac­tion would rat­tle a con­sid­er­able num­ber of in­vestors. Now, it seems a com­mon and eas­i­ly man­aged side ef­fect with no ev­i­dence of de­rail­ing ther­a­py.

Just as Spark $ONCE is push­ing ahead with its he­mo­phil­ia B gene ther­a­py, Bio­Marin is look­ing to rev­o­lu­tion­ize ther­a­py in he­mo­phil­ia. A suc­cess in the loom­ing Phase II­Is for once-and-done treat­ments could put to rest a full slate of block­buster ther­a­pies com­pet­ing in this mar­ket. So look for plen­ty of care­ful da­ta crunch­ing in the months to come.

In the mean­time, Bio­Marin con­tin­ues to in­vest heav­i­ly in the work, com­plet­ing a man­u­fac­tur­ing fa­cil­i­ty for BMN 270.

Bio­Marin R&D chief Hank Fuchs laid out the stakes for pa­tients:

“We are striv­ing to make a big dif­fer­ence for pa­tients with se­vere he­mo­phil­ia A by de­vel­op­ing a treat­ment that not on­ly has the po­ten­tial to elim­i­nate bleeds, but al­so has the po­ten­tial to elim­i­nate the re­quire­ment for re­com­bi­nant Fac­tor VI­II in­fu­sions for he­mo­phil­ia A pa­tients re­lat­ed to trau­ma, surgery and day-to-day ac­tiv­i­ties.”

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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As­traZeneca trum­pets the good 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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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

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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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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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: 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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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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