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.”

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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UP­DAT­ED: Stay tuned: Bio­gen’s num­bers are great — it’s their wor­ri­some fu­ture that leaves an­a­lysts skit­tish

Biogen came out with an upbeat assessment of their Q2 numbers today, discounting the arrival of a key rival for its blockbuster Spinraza franchise. But the top execs remain grimly determined to not say much anything new about the sore points that have dragged down its stock, including the future of its big investment in Alzheimer’s or how it plans to invest the considerable cash that the big biotech continues to reap.

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Why wait? Cel­gene re­struc­tures a big Jounce pact — ze­ro­ing in on new I/O path­way with $530M deal and bump­ing ICOS

Celgene’s business team isn’t waiting for the big merger with Bristol-Myers Squibb to go through before syncing its strategy with the new mother ship.

Tuesday evening the big biotech unveiled a $530 million deal — $50 million in upfront cash — to amend their alliance with Jounce Therapeutics $JNCE to gain worldwide rights to JTX-8064, an antibody that targets the LILRB2 receptor on macrophages. Their old, $2.6 billion deal is being scrapped, leaving Jounce with a pipeline that includes the lead drug, the ICOS-targeting vopratelimab.

PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Pharma, the lofty goal to unleash a “tsunami” of T cells personalized for each patient has hinged on the ability to correctly identify the neoantigens that form something of a fingerprint for each tumor, and extract the small group of T cells primed to attack the cancer. It still has a long way to go testing a treatment in humans, but the biotech says it has nailed that highly technical piece of the process.

UP­DAT­ED: My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ramaswamy’s Myovant $MYOV has closely matched its positive first round of Phase III data for their uterine fibroid drug relugolix, setting up a head-to-head rivalry with pharma giant AbbVie as the little biotech steers to the market with a planned filing in Q4.

Here’s how Myovant plans to prevail over the AbbVie $ABBV empire.

In the study, 71.2% of women receiving once-daily relugolix combination therapy achieved the clinical response they were looking for, compared to only 14.7% in the control arm. The data comfortably reflected the same outcomes in the first Phase III — 73.4% of women receiving once-daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo — which will reassure regulators that they are getting the carefully randomized data that qualifies for the FDA’s gold standard for success.

Lit­tle Mar­i­nus sees its shares eclipsed as the Sage ri­val fails to com­pare on PPD in PhII

The executive team at Sage $SAGE have skirted another potential pitfall on its way to racking up a big future for its depression drug Zulresso.

Little Marinus Pharmaceuticals $MRNS had sought to challenge the Sage drug with an IV formulation — followed by an oral version — of ganaxolone for postpartum depression. But researchers say their Phase II study failed to positively differentiate itself from a placebo at 28 days — leaving them to hold up “clinically meaningful” data within the first day of administration compared to the control arm.

Roche cuts loose Tam­i­flu OTC rights, hand­ing Sanofi the keys as the phar­ma gi­ant dou­bles down on Xofluza

Roche set out to make a better flu medicine than Tamiflu as that franchise was headed to a generic showdown. Now they’ll see just how well Xofluza stacks up against the mainstay drug after handing off over-the-counter rights in the US to Sanofi.

Sanofi $SNY says it will now step in to negotiate a deal with the FDA to steer Tamiflu into the OTC market, a role that could well involve new studies to ease passage of the drug out of doctor’s hands and into the consumer end of the market. And the French pharma giant will have first dibs over “selected” OTC markets around the world as they push ahead.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.