Adamas wins its first FDA OK, set­ting up mar­ket launch for a re­for­mu­lat­ed Parkin­son’s drug

More than a year af­ter Adamas $ADMS post­ed pos­i­tive re­sults from a piv­otal Phase III study of ADS-5102 — an ex­tend­ed-re­lease ver­sion of the gener­ic Parkin­son’s treat­ment aman­ta­dine — the com­pa­ny got a green light from the FDA to sell the ther­a­py.

The drug will be sold as Go­cov­ri.

Gre­go­ry Went, CEO

Prob­a­bly not un­ex­pect­ed­ly, the drug was able to beat out a place­bo in sig­nif­i­cant­ly re­duc­ing the side ef­fects of Parkin­son’s. And the Emeryville, CA-based biotech boasts that this is the first and on­ly drug ap­proved for “dysk­i­ne­sia in pa­tients with Parkin­son’s dis­ease re­ceiv­ing lev­odopa-based ther­a­py” — an or­phan in­di­ca­tion. The ther­a­py is de­signed to help pa­tients who suf­fer from the sharp, in­vol­un­tary move­ment dis­or­der brought on by lev­odopa, com­mon­ly used to con­trol the dis­ease.

In­vestors em­braced the news, send­ing shares rock­et­ing up 57%.

The gener­ic drug, though, is al­ready used off la­bel for dysk­i­ne­sia, which may well in­ter­fere with the com­pa­ny’s sales ex­pec­ta­tions. Walid Gel­lad, an as­so­ciate pro­fes­sor of med­i­cine at the Uni­ver­si­ty of Pitts­burgh, high­light­ed the off la­bel use in a Tweet.

The ap­proval marks a ma­jor shift for Adamas, which has been lin­ing up job of­fers for 59 sales ex­ecs they be­lieve can reach the vast ma­jor­i­ty of the physi­cians and spe­cial­ists who han­dle the bulk of the cas­es. And the team there has been lay­ing the ground­work with pay­ers to help roll out the drug.

What Adamas doesn’t have right now is a price. “We an­tic­i­pate a list price for Go­cov­ri around the range of the pre­vi­ous dis­clo­sure: $10K – $30K,” the com­pa­ny told me in an email. The fi­nal price, they say, will be heav­i­ly in­flu­enced by pa­tient ac­cess. In the US, whole­sale prices are typ­i­cal­ly heav­i­ly dis­count­ed for ma­jor pay­ers.

The gener­ic drug is sold by on­line phar­ma­cies, of­ten for the flu or Parkin­son’s, for a lit­tle more than $2 per 100 mg cap­sule.

Adamas ran two Phase III stud­ies for the drug, demon­strat­ing that it trig­gered a 37% re­duc­tion in Uni­fied Dysk­i­ne­sia Rat­ing Scale (UDysRS) to­tal score vs. 12 per­cent for place­bo at week 12. The re­sults were con­firmed in the sec­ond study, with Go­cov­ri achiev­ing a 46% re­duc­tion in UDysRS vs. 16% for place­bo.

The drug launch starts in Jan­u­ary, ac­cord­ing to Adamas, which can now wrap up its pre-launch ef­forts.

“To­day’s ap­proval is a tremen­dous mile­stone for Adamas and for the Parkin­son’s dis­ease com­mu­ni­ty,” said Adamas CEO Gre­go­ry T. Went. “Go­cov­ri has the po­ten­tial to help peo­ple with Parkin­son’s dis­ease suf­fer­ing from dysk­i­ne­sia by fi­nal­ly pro­vid­ing physi­cians with an ef­fec­tive tool to ad­dress this long-stand­ing un­met med­ical need.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

Finally freed from the restraints of a partial FDA clinical hold on its lead HER2-positive solid tumor candidate, Pieris Pharmaceuticals is now racing toward Phase II.

The FDA slapped a partial hold on Pieris’ PRS-343 back in July, restricting the biotech from enrolling new patients in a Phase I trial. While Pieris was allowed to continue dosing patients who were already enrolled, the agency requested they conduct an additional “in-use and compatibility study” before recruiting any more.

News brief­ing: Ve­rastem CMO ex­its two weeks af­ter join­ing com­pa­ny; Ther­mo Fish­er inks $550M M&A deal

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

FDA hits the brakes on His­to­gen's knee car­ti­lage ther­a­py, ask­ing for more in­fo on man­u­fac­tur­ing process

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Artist rendering of the Assembly Square site in Somerville, MA (BioMed Realty)

Bio­Med Re­al­ty snaps up in­no­va­tion cam­pus site with­in earshot of pricey and bustling Boston biotech hub

On the short list of the premier biotech hubs in the world, the Boston area has transformed into a home for innovation — and ridiculously high rent. Now, a real estate firm is seeking tenants for a major site in neighboring Somerville with more than enough elbow room.

Snapped up by BioMed Realty, the land — which consists of an existing 162,000 square-foot office building and a 7.5 acre site — will serve as an “innovation space” for a variety of research, technology and life science tenants, the real estate company said in a press release. Financial terms weren’t disclosed.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Andrew Allen, Gritstone CEO (Gritstone via website)

Grit­stone con­tin­ues Covid-19 push with deal to de­vel­op 'self-am­pli­fy­ing RNA' vac­cines, as shares con­tin­ue bal­loon­ing

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.