Ac­celeron un­corks more mid-stage da­ta for PAH drug so­tater­cept as part of the drug's res­ur­rec­tion cam­paign

Once on life sup­port in pul­monary ar­te­r­i­al hy­per­ten­sion, Ac­clereron’s so­tater­cept came back from the brink with win­ning da­ta ear­ly last year. Now, with Phase III piv­otal stud­ies on the way, Ac­celeron is adding more da­ta to its scrap­book in prepa­ra­tion for an even­tu­al sit-down with reg­u­la­tors.

PAH pa­tients treat­ed with Ac­celeron’s so­tater­cept showed im­prove­ments across a broad range of out­comes, in­clud­ing peak oxy­gen up­take or VO2 max, as well as ven­ti­la­to­ry ef­fi­cien­cy, to­tal work­load and ar­te­ri­ove­nous oxy­gen con­tent, ac­cord­ing to ear­ly da­ta from the open-la­bel Phase II SPEC­TRA II pre­sent­ed at the Amer­i­can Tho­racic So­ci­ety meet­ing.

In ad­di­tion, pa­tients’ six-minute walk dis­tance in­creased by an av­er­age of 72.4 me­ters from base­line to week 24 in nine pa­tients for whom da­ta were avail­able, Ac­celeron said. Pa­tients al­so saw a 35.9% re­duc­tion in pul­monary vas­cu­lar re­sis­tance from base­line to week 24 and a 29.5% re­duc­tion in pul­monary ar­te­r­i­al pres­sure dur­ing the same pe­ri­od.

The drug post­ed a sim­i­lar safe­ty pro­file to past tests, with treat­ment-re­lat­ed side ef­fects re­port­ed in 76% of pa­tients as of the Feb. 25 cut­off date. Three were deemed se­ri­ous but not enough to stop treat­ment, Ac­celeron said.

With grow­ing da­ta in hand, Ac­celeron is busy at work kick­start­ing its Phase III clin­i­cal pro­gram, which starts with en­roll­ment for the STEL­LAR tri­al. Mean­while, Ac­celeron is plan­ning two ad­di­tion­al Phase III stud­ies in pa­tients with PAH: the HY­PE­R­I­ON tri­al in new­ly di­ag­nosed pa­tients and the ZENITH in­ter­ven­tion tri­al.

SPEC­TRA II con­tin­ues so­tater­cept’s long march back from the brink of fail­ure af­ter Ac­celeron re­port­ed pos­i­tive Phase II da­ta from the PUL­SAR study in Jan­u­ary of last year. In that 106-pa­tient study, so­tater­cept hit its pri­ma­ry end­point: a sig­nif­i­cant re­duc­tion in pul­monary vas­cu­lar re­sis­tance. The drug al­so met three dif­fer­ent sec­ondary end­points, in­clud­ing the 6-minute walk­ing test.

Those re­sults warmed an­a­lysts’ hearts, in­clud­ing SVB Leerink’s Ge­off Porges, who pre­dict­ed so­tater­cept could be­come a $3 bil­lion-per-year prospect in the long run. But in a June fol­low-up note, Porges high­light­ed a CV death in the drug arm of the study, po­ten­tial­ly cast­ing doubt on the drug’s fu­ture. Here’s how he put it:

There was one car­diac ar­rest in the high dose co­hort, and while the pa­tient had a his­to­ry of CV dis­ease, this does raise the ques­tion of whether there might be an as­so­ci­a­tion with such events, and these will bear watch­ing in the piv­otal tri­al.

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

Learn more about Avance ClinicReady here.
Contact us about your next study.
Download our Frost & Sullivan APAC CRO Report here. 

The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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