Spark adds fresh ev­i­dence that pi­o­neer­ing gene ther­a­py could be a game chang­er for pa­tients

Spark Ther­a­peu­tics has added an­oth­er im­pres­sive round of late-stage da­ta demon­strat­ing that its gene ther­a­py for an in­her­it­ed eye dis­ease could be a game chang­er for pa­tients. This lat­est read­out in­clud­ed pos­i­tive out­comes for 9 crossover pa­tients who re­ceived voreti­gene nepar­vovec (SPK-RPE65) a year ago, along with promis­ing longer term re­sults on the orig­i­nal group of 20 pa­tients.

Nine pa­tients with in­her­it­ed reti­nal dis­ease caused by mu­ta­tions in the RPE65 gene were put in the con­trol arm of the study, and all vol­un­teered for the gene ther­a­py af­ter in­ves­ti­ga­tors con­clud­ed that the orig­i­nal treat­ment group had ben­e­fit­ed marked­ly with sig­nif­i­cant gains in vi­sion. A year af­ter treat­ment, Spark says that 8 of the 9 “demon­strat­ed the max­i­mum im­prove­ment mea­sur­able on the pri­ma­ry end­point at one year.” And the first 20 pa­tients who were treat­ed with their gene ther­a­py have demon­strat­ed a sus­tained ben­e­fit in vi­sion, speak­ing to the dura­bil­i­ty of the change.

But it wasn’t all up­beat for Spark to­day. An­a­lysts not­ed that the biotech ex­pects a sig­nif­i­cant de­lay in its reg­u­la­to­ry fil­ing, cit­ing CMC is­sues.

“ONCE al­so re­port­ed that the BLA sub­mis­sion will be de­layed un­til ear­ly 2017 due to in­com­plete CMC da­ta,” not­ed Wed­bush an­a­lyst David Nieren­garten. “(W)e have pushed back the ex­pect­ed launch from 4/1/17 to 10/1/17 in the US, which re­duces our PT by $1.” In­vestors didn’t over­look the un­ex­pect­ed set­back, send­ing Spark’s shares down slight­ly.

Voreti­gene nepar­vovec is a lead­ing gene ther­a­py in a di­verse field that has pro­duced re­mark­ably mixed re­sults. Wide­ly billed as the first gene ther­a­py like­ly to go to the FDA in search of mar­ket­ing ap­proval, it’s be­come a test case for dis­cus­sions on pric­ing a po­ten­tial one-time treat­ment. And it’s al­so helped un­der­score the sim­ple fact that not all gene ther­a­pies are equal, as de­vel­op­ers test new and bet­ter de­liv­ery ve­hi­cles and cor­rec­tive genes.

Spark wants to be the leader, and it’s been mak­ing its case with da­ta like this.

Look­ing for ev­i­dence of a func­tion­al im­prove­ment in vi­sion, Spark re­port­ed that the 8 ad­di­tion­al pa­tients hit the top rung in the tracked change in bi­lat­er­al mo­bil­i­ty test­ing. On light sen­si­tiv­i­ty test­ing, the eight al­so im­proved, “with an av­er­age im­prove­ment of near­ly 200-fold, com­pared to the more than 100-fold im­prove­ment av­er­age seen in the 301 study sub­jects. Sub­jects in the (crossover) study demon­strat­ed an av­er­age vi­su­al acu­ity im­prove­ment of 4.5 let­ters, av­er­aged across both eyes, com­pared to an av­er­age im­prove­ment of eight let­ters by the same analy­sis in the (orig­i­nal) 301 study.”

Spark al­so added some fresh ev­i­dence that its pi­o­neer­ing treat­ment could live up to its stock sym­bol {$ONCE), with ev­i­dence that the treat­ment was equal­ly ef­fec­tive two years af­ter treat­ment as one. Ac­cord­ing to Spark:

The mean dura­bil­i­ty of ben­e­fit was sus­tained at 1.9 lux lev­els af­ter two years, com­pared with the im­prove­ment of 1.9 lux lev­els af­ter one year. The more than 100-fold im­prove­ment av­er­age seen in the orig­i­nal in­ter­ven­tion group at one year sim­i­lar­ly was main­tained through at least two years.

Spark—a spin­out of Chil­dren’s Hos­pi­tal of Philadel­phia—has a mar­ket cap of $1.8 bil­lion.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Anthony Coyle (Repertoire)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

Embattled CDC director Robert Redfield (AP Images)

Covid-19 roundup: CDC ad­vi­so­ry com­mit­tee de­lays pri­or­i­ty dis­tri­b­u­tion vote; EU re­port­ed­ly in­dem­ni­fy­ing vac­cine mak­ers

A federal committee that advises the CDC was expected to hold a vote Tuesday on a plan regarding the distribution for initial doses of approved Covid-19 vaccines. But that vote has been scrapped.

The Advisory Committee on Immunization Practices, or ACIP, won’t be voting until the committee members learn more about which vaccines become available first, the Wall Street Journal reported. The vote could potentially wait until a specific vaccine is authorized before recommending how to dole out the first doses.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.