Strug­gling Sarep­ta bounces back as an­a­lysts cheer the first trick­le of rev­enue for DMD drug

Sarep­ta CEO Ed Kaye

Shares of Sarep­ta shot up 21% on Tues­day, buoyed by some en­thu­si­as­tic re­marks for the biotech’s first round of sales of its Duchenne mus­cu­lar dy­s­tro­phy drug Ex­ondys 51, which were talked up by CEO Ed Kaye at the JP Mor­gan con­fer­ence. And a deal to kick off a gene ther­a­py re­search pact may have helped.

The sales fig­ure for Ex­ondys 51 didn’t amount to much. Sales hit $5.4 mil­lion for the first three months the drug was on the mar­ket, which fell be­hind Leerink’s es­ti­mate and gen­er­al con­sen­sus — pegged by Joseph Schwartz at $7.9 mil­lion and $6 mil­lion. But with more than 250 DMD pa­tients amenable to an ex­on-skip­ping ther­a­py seek­ing cov­er­age, and a pos­i­tive as­sess­ment that Sarep­ta could man­age the back­lash that’s grown among pay­ers, the biotech earned a 21% spike in its stock price.

This is one of the most con­tro­ver­sial drugs to ever hit the mar­ket. The FDA’s la­bel spec­i­fies that there’s no proof of clin­i­cal ben­e­fit, and the in­ter­nal war at the FDA over this drug en­cour­aged a slate of in­sur­ers to ei­ther refuse to cov­er it or lim­it it, caus­ing the big share price spike that greet­ed an OK to melt away.

But Sarep­ta’s stock has been on a roller coast­er ride for years now, lurch­ing from big gains to big loss­es with each turn of the card.

Bri­an Sko­r­ney, Baird

Baird’s Bri­an Sko­r­ney was and is an op­ti­mist.

“While we ex­pect hur­dles to get­ting in­sur­ance au­tho­riza­tion and putting a plan in place for each pa­tient to have a week­ly in­fu­sion, we be­lieve the vast ma­jor­i­ty of these pa­tients will con­vert to rev­enue in 1H17,” he wrote. “We al­so be­lieve this is a strong in­di­ca­tor of ini­tial de­mand and ex­pect new start forms to con­tin­ue to in­crease over 2017.”

Gena Wang at Jef­feries, who nev­er ex­pect­ed to see this drug on the mar­ket, still ex­pects plen­ty of stub­born push­back from pay­ers. She writes:

Mgmt guid­ance on Ex­ondys 51 ac­cess and re­im­burse­ment con­sis­tent with our ex­pec­ta­tion of strict pay­er push­back. On­ly 13% of cov­ered lives have a fa­vor­able pol­i­cy, while 8% of cov­ered lives are deny­ing cov­er­age, per mgmt. Rest (79%) are ei­ther pend­ing pol­i­cy de­ci­sions, cov­er­ing with re­stric­tions, and/or re­view­ing pts case by case, in line with our ex­pec­ta­tions of strict pay­er push­back.

The drug is slat­ed to cost more than $300,000 a year.

The Cam­bridge, MA-based Sarep­ta, mean­while, signed up to sup­port a mi­crody­s­trophin gene ther­a­py pro­gram at Na­tion­wide Chil­dren’s Hos­pi­tal run by Jer­ry Mendell and Louise Rodi­no-Kla­pac. Sarep­ta gets an op­tion to ac­quire the pro­gram, with a Phase I/IIa tri­al start­ing lat­er this year.

Sarep­ta al­so struck a deal to part­ner with Ox­ford, UK-based Sum­mit in Oc­to­ber on a ther­a­py that fol­lows a com­ple­men­tary path­way to treat DMD.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

Top Har­vard chemist caught up in FBI’s 'T­hou­sand Tal­ents' drag­net, ac­cused of ly­ing about Chi­nese con­nec­tions, pay

The FBI’s probe into the alleged theft of R&D secrets by Chinese authorities has drawn Harvard’s top chemist into its net.

The agency accused Charles M. Lieber, who chairs the university’s chemistry and chemical biology department, with lying about his involvement in China’s Thousand Talents campaign, which was established as a way of drawing in innovators from around the world. And the scientist, 60, was charged with making false statements about his ties to China.

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Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Passage Bio — Jim Wilson’s self-described “legacy company” — has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.

UP­DAT­ED: Ac­celeron res­ur­rects block­buster hopes for so­tater­cept with pos­i­tive PhII — and shares rock­et up

Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

“We’re thrilled to report such positive topline results from the PULSAR trial,” Acceleron CEO Habib Dable said in a statement. The company said in a conference call they plan on discussing a Phase III trial design with regulators.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”