Af­ter an odyssey of set­backs, FDA fi­nal­ly green-lights Newron’s Parkin­son’s drug Xada­go

For Italy’s Newron, per­sis­tence has fi­nal­ly paid off. Af­ter fol­low­ing two rad­i­cal­ly dif­fer­ent paths to get its add-on ther­a­py sa­fi­namide on the mar­ket in Eu­rope and the US, the FDA has fi­nal­ly of­fered a green light for their Parkin­son’s drug sa­fi­namide af­ter sev­en years of tri­al and er­ror.

While the com­pa­ny sub­mit­ted an MAA in Eu­rope in 2013, set­ting them on a path to ap­proval on the con­ti­nent in ear­ly 2015, the FDA re­fused to even ac­cept their first ap­pli­ca­tion in 2014, fault­ing them for a se­ries of mis­takes in the NDA. A re­jec­tion fol­lowed their first ac­cept­ed fil­ing, with con­cerns about po­ten­tial abuse of the drug that need­ed to be ad­dressed first.

All of that oc­curred af­ter Mer­ck Serono dropped their part­ner­ship on the drug in 2011, af­ter de­cid­ing they weren’t bull­ish on its com­mer­cial prospects. And then Bi­oTie in Fin­land punt­ed a deal to ac­quire the com­pa­ny for $63 mil­lion in stock.

To­day, though, Newron and its new part­ners, Zam­bon and US WorldMeds, had cause for cel­e­bra­tion. Sa­fi­namide will now be sold as Xada­go by US WorldMeds, which did a deal with Zam­bon for the com­mer­cial rights.

The drug is a se­lec­tive MAO-B in­hibitor and has demon­strat­ed in two stud­ies its abil­i­ty to re­duce “off” time for pa­tients tak­ing lev­odopa, as well as a re­duced rate of dysk­i­ne­sia. The drug works by tar­get­ing an en­zyme that breaks down dopamine in the brain, al­low­ing for bet­ter re­sults from lev­odopa in con­trol­ling mo­tor func­tions.

Char­lotte Key­wood, glob­al head of R&D for Zam­bon, said:

We are de­light­ed with the ap­proval of Xada­go in the USA. This first new treat­ment for a decade rep­re­sents an im­por­tant ad­di­tion to the treat­ment choic­es for pa­tients with PD and their doc­tors. In or­der to more ful­ly char­ac­ter­ize the clin­i­cal ben­e­fits of Xada­go, Zam­bon will con­tin­ue to work with our part­ners on new clin­i­cal tri­als.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,800+ biopharma pros reading Endpoints daily — and it's free.

Credit: Shutterstock

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,800+ biopharma pros reading Endpoints daily — and it's free.

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.

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.”

Pfiz­er ax­es 6 ear­ly to late-stage can­cer stud­ies from the pipeline — with one oth­er cut for sick­le cell dis­ease

Pfizer trimmed a group of 3 R&D programs using their PD-L1 Bavencio — partnered with Merck KGaA — in their latest pipeline cull.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,800+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: In­cyte scores much need­ed PhI­II suc­cess — and of course it’s de­liv­ered by rux­oli­tinib

Incyte’s efforts to breathe a second life into ruxolitinib — its JAK inhibitor sold in pill form as Jakafi — has been greeted with clear, if preliminary and unsurprising, Phase III success.

Topline data from the TRuE-AD2 cements ruxolitinib’s foundational importance for Incyte, and gives analysts hope that there might yet be room for growth in a pipeline that’s suffered multiple R&D setbacks.

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.