Gilead bags Sarep­ta's pri­or­i­ty re­view vouch­er for a dis­count, pay­ing $125M

Sarep­ta CEO Ed Kaye

Back in the sum­mer of 2015, Ab­b­Vie agreed to pay $350 mil­lion for a pri­or­i­ty re­view vouch­er, which can be used to shave four months off the sched­ule for any FDA drug re­view. But now, less than two years lat­er, the price has come down con­sid­er­ably.

Ear­ly Mon­day Sarep­ta $SRPT said that it has auc­tioned off its PRV for $125 mil­lion, still a con­sid­er­able sum of cash. Sarep­ta CEO Ed Kaye start­ed the sale process right af­ter the ap­proval for Ex­ondys 51 land­ed, look­ing for some added cash as they launched their drug.

Sarep­ta didn’t say in its state­ment who is buy­ing the vouch­er or what drug it will be used for, but in its SEC fil­ing to­day the biotech iden­ti­fied the buy­er as Gilead, which helped get this mar­ket es­tab­lished in 2014 when it ac­quired its first PRV for $125 mil­lion.

The news should help en­cour­age Marathon, which al­so land­ed a PRV just days ago af­ter its con­tro­ver­sial ap­proval for its cheap, gener­ic steroid de­flaza­cort, to be sold in the US as a treat­ment for Duchenne MD. Marathon like­ly spent far less than that for its own de­vel­op­ment pro­gram, ac­cord­ing to a pair of ex­perts. And a deal for the PRV at $125 mil­lion could eas­i­ly make their drug prof­itable, be­fore it’s even sold.

These PRVs have had a con­tro­ver­sial his­to­ry. Reg­u­la­tors have made it clear that they don’t like to be forced to give the in­side track at the FDA to any com­pa­ny which can af­ford to pay the price to hur­ry along a ther­a­py that’s not so ur­gent­ly need­ed. But law­mak­ers like the added in­cen­tive, claim­ing that it en­cour­ages in­no­va­tion where it’s need­ed most.

As a re­sult, the num­ber of PRVs on the mar­ket has mul­ti­plied. Io­n­is won one, for ex­am­ple, and Alex­ion has won two by it­self. Gilead has been an ea­ger buy­er.

Leerink’s Joseph Schwartz was dis­ap­point­ed to hear what the PRV had fetched. He not­ed:

Up­on Ex­ondys 51 ap­proval and the re­ceipt of the PRV, we had ini­tial­ly es­ti­mat­ed a ~$350M PRV val­ue in our DCF. With the reau­tho­riza­tion of the PRV pro­gram, we de­creased our es­ti­mate to $200M to ac­count for a re­duc­tion in the scarci­ty val­ue of these in­stru­ments and to re­flect the min­i­mal ben­e­fit ac­cord­ed by the pre­vi­ous PRV pur­chased by REGN (OP) from BM­RN (OP). To­day’s an­nounce­ment of $125M is even low­er than our ad­just­ed es­ti­mates. And while this non-di­lu­tive amount will sure­ly add ad­di­tion­al run­way to Sarep­ta’s cash po­si­tion, we can­not help but won­der if this trans­ac­tion re­flects a 1) broad­er de­cline in PRV in­ter­est among bid­ders, or 2) an un­der­val­ued as­set sale.

Cred­it Su­isse helped mar­ket the PRV, ac­cord­ing to Sarep­ta’s state­ment, mak­ing the out­reach to “mul­ti­ple” bio­phar­ma com­pa­nies.

“Our mis­sion at Sarep­ta Ther­a­peu­tics is to treat more boys with Duchenne mus­cu­lar dy­s­tro­phy,” said Ed­ward Kaye, Sarep­ta’s chief ex­ec­u­tive of­fi­cer. “The sale of the PRV pro­vides an im­por­tant source of non-di­lu­tive cap­i­tal to sup­port the rapid ad­vance­ment of our fol­low on ex­on skip­ping can­di­dates and next gen­er­a­tion RNA tar­get­ed an­ti­sense plat­form.”

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.