Rin­voq nabs new ap­proval in sec­ond-line UC; Sen­so­ri­on says its failed drug now works in sub­set analy­sis

Ab­b­Vie’s Rin­voq racked up an­oth­er ap­proval late Wednes­day af­ter­noon.

The drug is now OK’d in adults with mod­er­ate­ly to se­vere­ly ac­tive ul­cer­a­tive col­i­tis who have had an in­ad­e­quate re­sponse or in­tol­er­ance to one or more TNF block­ers, Ab­b­Vie said in a press re­lease. It’s the first ap­proval for the drug in gas­troen­terol­o­gy, the com­pa­ny added, with da­ta stem­ming from three Phase III stud­ies.

“There re­mains an un­met need for pa­tients with mod­er­ate­ly to se­vere­ly ac­tive UC, who suf­fer from de­bil­i­tat­ing symp­toms that are of­ten un­pre­dictable and bur­den­some,” said Thomas Hud­son, Ab­b­Vie CSO. “With the ap­proval of Rin­voq as a new treat­ment op­tion, Ab­b­Vie con­tin­ues our lead­er­ship in ad­vanc­ing re­search that can help im­pact the lives of peo­ple liv­ing with ul­cer­a­tive col­i­tis.”

Wednes­day’s ap­proval comes a few months af­ter the agency slapped a new warn­ing on the drug af­ter Pfiz­er’s Xel­janz saw in­creased side ef­fects, rel­e­gat­ing the drug to sec­ond-line use in rheuma­toid arthri­tis. The warn­ing came amid the back­drop of a larg­er safe­ty saga play­ing out for the en­tire JAK class of drugs. — Max Gel­man

Sen­so­ri­on claims vic­to­ry on ex­plorato­ry end­points af­ter PhII fail­ure

French biotech Sen­so­ri­on got ham­mered back in Jan­u­ary af­ter its on­ly clin­i­cal stage drug failed to meet the pri­ma­ry end­point in a Phase II study — los­ing 40% of its share val­ue af­ter the news was an­nounced about its lead drug SENS-401, a treat­ment of sud­den sen­sorineur­al hear­ing loss (SSNHL).

To­day, the biotech says it has some more da­ta. And this time, it says that they found some sta­tis­ti­cal­ly sig­nif­i­cant treat­ment ef­fects in spe­cif­ic pop­u­la­tions af­ter they re­viewed ex­plorato­ry end­points. Those in­clude pa­tients suf­fer­ing from pro­found hear­ing loss treat­ed with cor­ti­cos­teroids see­ing an im­prove­ment of 19-26 dB from base­line com­pared to place­bo.

Ex­plorato­ry end­points, how­ev­er, are gen­er­al­ly con­sid­ered far less con­clu­sive than end­points that were set ahead of time, and are gen­er­al­ly used on­ly to de­sign fu­ture tri­als, rather than to of­fer de­fin­i­tive an­swers on ef­fi­ca­cy.

In­vestors re­spond­ed fa­vor­ably to the new de­vel­op­ment, ini­tial­ly sky­rock­et­ing the stock up over 43% be­fore sta­bi­liz­ing at around a 20% in­crease.

The drug is still in play in a col­lab­o­ra­tion on hear­ing preser­va­tion fol­low­ing cochlear im­plants. Sen­so­ri­on and part­ner Cochlear Lim­it­ed are mov­ing for­ward with a tri­al and have sub­mit­ted tri­al de­sign plans to reg­u­la­tors in Aus­tralia and France. — Paul Schloess­er

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

Kirk Myers is shown in a still image from a new film series showcasing the efforts of HIV advocates funded by Gilead.

Gilead spot­lights HIV projects and the com­mu­ni­ty lead­ers dri­ving them in new mi­ni-doc­u­men­tary films

Gilead is going behind the scenes of some of the HIV initiatives it funds through grants in a new film series narrated by the people helming the projects.

The first four films and leaders come from across the US — Arianna Lint in Florida and Puerto Rico, Cleve Jones in San Francisco, June Gipson in Mississippi and Kirk Myers in Texas. Their HIV-focused efforts range from addressing unmet needs of the transgender community to delivering social services and high-quality health care in underserved communities.

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EMA pulls an opi­oid from the 1950s used to treat dry cough

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

John Evans, Beam Therapeutics CEO

Beam's base-edit­ed al­lo­gene­ic CAR-T gets FDA go-ahead af­ter four-month wait

The FDA wanted more information on four key areas before it would let Beam Therapeutics proceed with human testing for a cell therapy in a certain type of leukemia. It appears the biotech has answered the agency’s queries.

The US regulator cleared the base-edited, off-the-shelf CAR-T, Beam said Friday morning, lifting a hold from this summer. More details on specific next steps for the Phase I will come out next year, the Boston-area biotech said.

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