Roche boosts its gene ther­a­py game, ex­pand­ing part­ner­ship with 4D Mol­e­c­u­lar Ther­a­peu­tics

Join­ing the Big Phar­ma rush to pick up po­ten­tial­ly valu­able gene ther­a­py tech, Roche has ex­pand­ed its part­ner­ship with 4D Mol­e­c­u­lar Ther­a­peu­tics to work on a drug for night blind­ness, among oth­er oph­thal­mol­o­gy pro­grams.

David Kirn

Roche had first part­nered with the Emeryville, Cal­i­for­nia-based biotech back in 2016, when the duo was re­search­ing a small hand­ful of rare reti­nal dis­ease in­di­ca­tions. Now, it’s ex­pand­ed that com­mit­ment to take in oph­thal­mol­o­gy pro­grams that in­clude prospects for much larg­er mar­kets.

The progress of Spark and its gene ther­a­py hit Lux­tur­na — along with new part­ner­ships forged by its phar­ma brethren Pfiz­er and No­var­tis — like­ly laid the ground­work for Roche to re­con­sid­er its part­ner­ship with 4DMT.

Roche and 4DMT’s first col­lab­o­ra­tion pro­gram is for 4D-110, a clin­i­cal can­di­date to treat choroi­deremia (an in­her­it­ed form of night blind­ness that can show up in ear­ly child­hood). 4DMT is just now do­ing IND-en­abling stud­ies on the pro­gram. 4D-110 al­ready has some ad­vanced com­pe­ti­tion in the field, as Lon­don’s Night­star $NITE is al­so tack­ling this dis­ease with a Phase III gene ther­a­py drug.

4DMT’s edge would be its ade­no-as­so­ci­at­ed virus plat­form, which the com­pa­ny says is su­pe­ri­or to its ri­vals.

The part­ner­ship al­so in­cludes ad­di­tion­al clin­i­cal can­di­date pro­grams that are un­der­way to treat reti­nal dis­eases with high un­met need, 4DMT says. The com­pa­ny’s CEO David Kirn said this in a state­ment:

To­geth­er we elect­ed to ex­pand our part­ner­ship af­ter 4DMT com­plet­ed pro­pri­etary in­trav­it­re­al vec­tor dis­cov­ery and char­ac­ter­i­za­tion. We have cre­at­ed clear syn­er­gies be­tween 4D’s vec­tor dis­cov­ery, gene ther­a­py de­vel­op­ment and man­u­fac­tur­ing ca­pa­bil­i­ties and Roche’s ex­per­tise in late-stage clin­i­cal de­vel­op­ment and glob­al bi­o­log­ics com­mer­cial­iza­tion. The de­ci­sion to ex­pand our part­ner­ship rep­re­sents val­i­da­tion of our oph­thal­mol­o­gy plat­form, clin­i­cal can­di­dates and team.

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.”