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.

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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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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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.

Iron­wood kicks de­layed-re­lease Linzess for­mu­la­tion to the curb af­ter tri­al fail­ure

The delayed-release formulation of Ironwood and Allergan’s bowel drug Linzess will not see the light of day.

The experimental drug, MD-7246, failed to help patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) in a mid-stage study, prompting the partners to abandon the therapy.

First approved in 2012, Linzess (known chemically as linaclotide) enhances the activity of the intestinal enzyme guanylate cyclase-C to increase the secretion of intestinal fluid and then transit through the intestinal tract, as well as reduce visceral pain, to relieve pain and constipation associated with IBS.

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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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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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:”.

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.