Sarep­ta read­ies FDA pitch af­ter a small study spot­lights 1% dy­s­trophin add for golodirsen in Duchenne MD

Af­ter win­ning an FDA ap­proval for Ex­ondys51 as a new ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy with­out hav­ing to ac­tu­al­ly pro­duce da­ta to prove it worked, Sarep­ta $SRPT fol­lowed up this new morn­ing with a fresh set of da­ta from a small study un­der­scor­ing the abil­i­ty of its ex­per­i­men­tal fol­lowup ex­on-skip­ping drug golodirsen to trig­ger added pro­duc­tion of dy­s­trophin.

Doug In­gram, Sarep­ta CEO

The num­bers it cites, though, are tiny. De­spite that, Sarep­ta is telling an­a­lysts to­day that it plans to see if the FDA could be will­ing to give it an ac­cel­er­at­ed green light.

Prin­ci­pal in­ves­ti­ga­tor Francesco Muntoni not­ed:

All (25) treat­ed boys showed the an­tic­i­pat­ed ex­on skip­ping af­ter treat­ment and this re­sult­ed in a mean in­crease of dy­s­trophin pro­tein, as mea­sured by West­ern blot, from 0.095 per­cent at base­line to 1.019 per­cent of nor­mal af­ter at least one-year of treat­ment with golodirsen.

To un­der­score, that’s a claimed win with slight­ly more than 1% of nor­mal dy­s­trophin. And it will ar­rive at the FDA af­ter an in­ter­nal war was fought over Ex­ondys51, which Janet Wood­cock even­tu­al­ly won by shov­ing aside a line­up of reg­u­la­tors that tried to get in her way.

Some of the an­a­lysts found the out­come to be more than a lit­tle lean. Not­ed RBC’s Matthew Eck­ler:

To­day’s re­lease con­firms the abil­i­ty of Sar­pe­ta’s ex­on-skip­ping ap­proach to in­crease dy­s­trophin in the mus­cles of DMD pa­tients, in our view. How­ev­er, re­call that dur­ing FDA re­view of Ex­ondys 51’s NDA, the agency raised con­cerns around the clin­i­cal ben­e­fit of pro­duc­ing small amounts of dy­s­trophin (cit­ing 10% as the thresh­old for clin­i­cal ben­e­fit). In­deed, one of the bear cas­es we hear for SRPT is that the small amount of dy­s­trophin pro­duc­tion and lack of a con­firmed clin­i­cal ben­e­fit leave the door open to pay­or push­backs and de­nials. We’d still ex­pect some push­back around the head­line “1% dyst. How­ev­er re­gard­less of the ul­ti­mate clin­i­cal ben­e­fit, we think that with the cur­rent da­ta re­lease (and pos­si­bil­i­ty of ac­cel­er­at­ed ap­proval), in­vestors will start as­crib­ing val­ue to Sarep­ta’s pipeline, and that shares will bet­ter re­flect the in­trin­sic val­ue of the com­pa­ny be­yond Ex­ondys 51.

Bri­an Sko­r­ney at Baird likes it. His rea­sons in­clude:

Re­call Sarep­ta sub­mit­ted base­line and 48-week treat­ment dy­s­trophin analy­ses to the FDA last year from the PRO­MOVI study, which showed eteplirsen in­creased dy­s­trophin from 0.16% of nor­mal to 0.44% at week 48, a 0.28% ab­solute in­crease. To­day’s SRP-4053 re­sults are clear­ly more im­pres­sive, with an in­crease from 0.095% to 1.019% (p<0.001), al­most 3.5x the dy­s­trophin pro­duc­tion seen in the PRO­MOVI pa­tients. Get­ting across the 1% of nor­mal thresh­old ap­pears to be an im­por­tant psy­cho­log­i­cal thresh­old, as crit­ics have of­ten cit­ed the “frac­tion of a per­cent” is­sue for eteplirsen.

The com­pa­ny is out to prove that its ex­on-skip­ping tech does work ef­fec­tive­ly, but the biotech still has a very long way to go on that score. Nev­er­the­less, with some help­ful cheer­lead­ing on the side­lines, its stock jumped 15% on the re­lease.

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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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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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.

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.