Spark Ther­a­peu­tics of­fers a glimpse of ef­fi­ca­cy in first two he­mo­phil­ia A gene ther­a­py pa­tients

With its lead gene ther­a­py un­der FDA re­view, Spark Ther­a­peu­tics $ONCE is now un­veil­ing the first en­cour­ag­ing re­spons­es on the he­mo­phil­ia A front.

Jef­frey Mar­raz­zo

With an­a­lysts look­ing for a 12%-plus Fac­tor VI­II re­sponse, the com­pa­ny says that in­ves­ti­ga­tors tracked an 11% and 14% sta­bi­lized rise in Fac­tor VI­II ac­tiv­i­ty in the first two pa­tients who were giv­en the ther­a­py in the dose-es­ca­la­tion study.

Key to this part of the process, re­searchers say they have seen no Fac­tor VI­II in­hibitors ap­pear, no throm­bot­ic events, no spon­ta­neous bleeds and no need to use cor­ti­cos­teroids on the pa­tients, who have been tracked for 23 and 12 weeks.

As a re­sult, the biotech says that it has now dou­bled the dose and treat­ed the third pa­tient in the proof-of-con­cept study. States the com­pa­ny: “While the re­sults for this third par­tic­i­pant are ear­ly, his fac­tor ac­tiv­i­ty lev­el is track­ing pro­por­tion­al­ly high­er, con­sis­tent with the dose es­ca­la­tion.”

Spark shares surged 15% this morn­ing as the biotech up­dat­ed the da­ta as well as its Q2 re­sults.

Not­ed Jef­feries’ Michael Yee re­cent­ly: “First da­ta from Phase I/II of He­mo­phil­ia A gene ther­a­py SPK-8011 in Ju­ly/Aug could es­tab­lish ear­ly proof-of-con­cept and be a (+) cat­a­lyst. Giv­en small no. of pts to start, key is demon­strat­ing po­ten­tial to get to 12%+ Fac­tor VI­II.”

Spark Ther­a­peu­tics is the most ad­vanced biotech in the US gene ther­a­py field, with a good chance to win the first ever FDA ap­proval for a once-and-done treat­ment. As a re­sult, an­a­lysts are watch­ing every step CEO Jeff Mar­raz­zo makes. The biotech has been mak­ing steady progress with a gene ther­a­py for he­mo­phil­ia B af­ter get­ting over some ear­ly safe­ty jit­ters. And just days ago Spark was hand­ed a pri­or­i­ty re­view of its lead pro­gram for RPE65-me­di­at­ed in­her­it­ed reti­nal dis­ease, putting it first in line for a US ap­proval.

To be sure, Spark isn’t alone in he­mo­phil­ia A. Sang­amo has a pro­gram un­der­way. But the leader in this field is Bio­Marin $BM­RN, which has pro­duced some stel­lar – though al­so puz­zling — re­sults. Now ready to go in­to Phase III, ear­ly-stage stud­ies demon­strat­ed a wide vari­abil­i­ty in Fac­tor VI­II ex­pres­sion need­ed to keep he­mo­phil­ia in check. Joseph Schwartz at Leerink has not­ed 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. In the mean­time, look for some care­ful ex­am­i­na­tion of sta­bil­i­ty ver­sus high but vari­able im­pact.

Once over the fin­ish line, gene ther­a­pies will present pay­ers with a thorny is­sue. How do you cov­er ther­a­pies that have the promise of be­ing used just once, with­out any guar­an­tees that they can last a life­time? Prices are ex­pect­ed to be sky high, which has held back the two gene ther­a­pies that have been ap­proved in Eu­rope, though on­ly rarely used.

Kather­ine High

“The en­cour­ag­ing start of our SPK-8011 clin­i­cal tri­al re­in­forces the strength of our gene ther­a­py plat­form, de­liv­ers hu­man proof-of-con­cept in a sec­ond liv­er-me­di­at­ed dis­ease — a sig­nif­i­cant achieve­ment in the gene ther­a­py field — and po­si­tions us well to po­ten­tial­ly trans­form the cur­rent treat­ment ap­proach for this life-al­ter­ing dis­ease with a one-time in­ter­ven­tion,” said Kather­ine High, pres­i­dent and chief sci­en­tif­ic of­fi­cer of Spark Ther­a­peu­tics. “We are ex­cit­ed about the progress we are mak­ing to achieve our goals of our in­ves­ti­ga­tion­al he­mo­phil­ia A and B pro­grams: to safe­ly achieve pre­dictable, con­sis­tent and sus­tained ac­tiv­i­ty lev­els that pre­vent spon­ta­neous bleed­ing.”

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

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De­sert­ed by Astel­las and Mer­ck, lit­tle Cor­re­vio still can't win over FDA pan­el con­cerned with its AFib drug's safe­ty

When the FDA spurned Astellas’ pitch for atrial fibrillation drug vernakalant in 2008, regulators made it abundantly clear that it wasn’t the efficacy they had a problem with — two Phase III trials had shown the drug successfully restored 52% of patients’ heartbeat from irregular to normal — but the cardio safety issues for a drug that was to compete with well established, low-risk options. One licensing deal, one clinical hold and several studies later, the chances of approval aren’t looking any better.

New trade deal knocks out long-sought patent pro­tec­tions for drug­mak­ers

House Democrats negotiating with the Trump Administration on a new North American trade deal settled early on four issues: enforcement, labor and environmental standards and drug pricing.

On drug pricing, Politico reports, Trump crumbled within weeks of heightened negotiations.

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With loom­ing su­per­bug cri­sis, Iterum miss­es in a key an­tibi­ot­ic tri­al — and its shares tum­ble

A raft of antibiotic makers have crashed and burned recently despite getting their drugs across the finish line. Iterum Therapeutics hopes that its lead drug sulopenem, which has an outpatient focus in addition to hospitals and a plan to target areas with the highest levels of drug-resistant infections, will avert some of those reimbursement challenges. However, the company has now stumbled in a late-stage study, diminishing its shot at FDA approval it first requires.

UP­DAT­ED: Lit­tle Leo Phar­ma en­ters the prize-fight ring with pos­i­tive PhI­II atopic der­mati­tis da­ta. Now they just have to beat Dupix­ent

A day after new Sanofi CEO Paul Hudson staked his reputation and the future of the pharma giant on making Dupixent a megasuccess story, little Leo Pharma is throwing down the gauntlet on atopic dermatitis.

Three years ago Leo paid AstraZeneca $115 million to buy up rights to use tralokinumab against atopic dermatitis — with $1 billion more on the table in milestones — the Danish company says their drug has swept up positive results for all primary and secondary endpoints in three Phase III trials. Now they plan to start the final push for regulatory approvals so they can challenge the heavyweight champions in this slugfest.

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Otello Stampacchia. Omega Funds

Af­ter sev­er­al high pro­file start­up launch­es, om­niv­o­rous Omega Funds clos­es $438M fund to pur­sue more deals

Omega Funds likes to work backwards. It invests with the end game — denoted by the Greek letter in its name — in mind, and it keeps tabs on the number of marketed medical products that culminate from its ventures: 37 in 16 years.

So when founder and managing director Otello Stampacchia declares it’s the most exciting time to be investing in life sciences in a generation, it’s perhaps only natural that Omega has closed its largest fund to date. With $438 million in total commitments for Fund VI, the firm will continue injecting capital into a broad swath of companies across the US and Europe.

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