Trick, or treat? FDA plans Hal­loween ad­comm for Ipsen’s sec­ond chance at rare dis­ease drug

An oft-ma­ligned rare bone dis­ease pro­gram at Ipsen is go­ing to get a clos­er look from reg­u­la­tors on a day that’s proven to be mighty spooky.

The FDA has sched­uled an ad­vi­so­ry com­mit­tee to ex­am­ine Ipsen’s palo­varotene on Oct. 31, ac­cord­ing to a no­tice post­ed in the Fed­er­al Reg­is­ter, set­ting up a show­down for a drug that once failed a Phase III test. The hear­ing will come about two months ahead of palo­varotene’s de­ci­sion date, which is slat­ed for Dec. 29.

An Ipsen spokesper­son con­firmed the ad­comm date, telling End­points News that it is “com­mon” for the FDA to con­vene a pan­el of out­side ex­perts in ar­eas with no treat­ment op­tions, par­tic­u­lar­ly in rare dis­eases.

Re­searchers had been de­vel­op­ing the can­di­date to try to pre­vent new bone growth as­so­ci­at­ed with fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va (FOP) and mul­ti­ple os­teo­chon­dro­mas. Ipsen took on the drug fol­low­ing a $1 bil­lion-plus ac­qui­si­tion of Cana­di­an biotech Clemen­tia Phar­ma­ceu­ti­cals in Feb­ru­ary 2019, which had re­pur­posed it af­ter Roche punt­ed on em­phy­se­ma tri­als.

In De­cem­ber 2019, reg­u­la­tors slapped Ipsen with a par­tial hold on dos­ing pa­tients 14 and un­der in a Phase II and Phase III study af­ter re­ports of “ear­ly growth plate clo­sure,” a con­di­tion that can se­vere­ly stunt a child’s growth. The next month, the drug failed a Phase III fu­til­i­ty test and Ipsen de­cid­ed to pause dos­ing in all re­main­ing pe­di­atric pa­tients from that study.

Ipsen ap­peared to turn things around in short or­der, re­sum­ing dos­ing a few months lat­er af­ter amend­ing tri­al pro­to­cols. The com­pa­ny even won pri­or­i­ty re­view in May 2021. But things took a turn af­ter Ipsen with­drew its ap­pli­ca­tion in Au­gust 2021, cit­ing the FDA’s re­quest for ad­di­tion­al da­ta from its two piv­otal stud­ies.

The move came as a sur­prise to an­a­lysts but didn’t seem to dent their ex­pec­ta­tions. Jef­feries’ Pe­ter Welford said at the time that the with­draw­al wouldn’t change his pro­jec­tion of $180 mil­lion in peak sales, which had as­sumed a 2021 US ap­proval.

But Ipsen re­sub­mit­ted its ap­pli­ca­tion this June, once again win­ning pri­or­i­ty re­view. The com­pa­ny ap­plied for ap­proval in FOP in adults and chil­dren, in­clud­ing girls old­er than eight and boys old­er than 10. The drug has al­so been ap­proved in Cana­da, win­ning an OK this past Jan­u­ary.

Ed­i­tor’s note: This ar­ti­cle has been up­dat­ed with com­ment from an Ipsen spokesper­son.

Am­gen lays off about 300 work­ers, cit­ing 'in­dus­try head­wind­s'

Amgen has laid off about 300 employees, a company spokesperson confirmed to Endpoints News via email Sunday night.

Employees posted to LinkedIn in recent days about layoffs hitting Amgen last week. The Thousand Oaks, CA-based biopharma, which employs about 24,000 people, said the reduction “mainly” impacted US-based workers on its commercial team.

Drug developers of all sizes, including small upstarts and pharma giants, have let employees go in recent months as the biopharma market drags through a quarters-long winter doldrum.

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Bob Bradway, Amgen CEO (Stephen Lam/Reuters)

Am­gen launch­es the first US Hu­mi­ra biosim­i­lar at two dif­fer­ent list prices

The bizarre dynamics of the US prescription drug market were on full display once again this morning as Amgen announced that it would launch the first US biosimilar for Humira, the best-selling drug of all time, at two completely different list prices.

One price for Amgen’s Amjevita (adalimumab-atto) will be 55% below the current Humira list price, which is about $84,000 per year, and another at a list price 5% below the current Humira list price, but presumably (pharma companies don’t disclose rebates) with high rebates to attract PBMs and payers.

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New York City in­vests $20M in­to biotech 'in­no­va­tion space' at the Brook­lyn Navy Yard

New York City is investing $20 million in biotech this year in the form of a 50,000-square-foot “innovation space” at the Brooklyn Navy Yard, complete with offices, research laboratories and events and programming space to grow biotech startups and companies.

Mayor Eric Adams said during his State of The City Address last Thursday that there will be an “emphasis” on making more opportunities for women and people of color to further diversify the industry. The City first reported the news.

Dirk Thye, Quince Therapeutics CEO

Af­ter piv­ot­ing from Alzheimer's to bone con­di­tions, biotech piv­ots again — and halves its head­count

When troubled public biotech Cortexyme bought a private startup named Novosteo and handed the keys to its executive team, the company — which changed its name to Quince Therapeutics — said it would shift its focus from an unorthodox Alzheimer’s approach to Novosteo’s bone-targeting drug platform.

Less than a year later, Quince is pivoting again.

The biotech has decided to out-license its bone-targeting drug platform and its lead drug, NOV004, and instead look for clinical-stage programs to in-license or acquire, according to a press release.

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Boehringer In­gel­heim touts pre­ven­tion re­sults in rarest form of pso­ri­a­sis

Boehringer Ingelheim uncorked some positive results suggesting that Spevigo can help prevent flare-ups in patients with a severe form of psoriasis, months after the drug was approved to treat existing flares.

Spevigo, an IL-36R antibody also known as spesolimab, met its primary and a key secondary endpoint in the Phase IIb EFFISAYIL 2 trial in patients with generalized pustular psoriasis (GPP), Boehringer announced on Monday. While the company is keeping the hard numbers under wraps until later this year, it said in a news release that it anticipates sharing the results with regulators.

As­traZeneca, No­vo Nordisk and Sanofi score 340B-re­lat­ed ap­peals court win over HHS

AstraZeneca, Novo Nordisk, and Sanofi won an appeals court win on Monday, as the US Court of Appeals for the Third Circuit found that the companies cannot be forced to provide 340B-discounted drugs purchased by hospitals from an unlimited number of community and specialty pharmacies.

“Legal duties do not spring from silence,” the decision says as the court makes clear that the federal government’s interpretation of the “supposed requirement” that the 340B program compels drugmakers to supply their discounted drugs to an unlimited number of contract pharmacies is not correct, noting:

Ap­peals court toss­es J&J's con­tro­ver­sial 'Texas two-step' bank­rupt­cy case

A US appeals court has ruled against Johnson & Johnson’s use of bankruptcy to deal with mounting talc lawsuits, deciding that doing so would “create a legal blind spot.”

The Third Circuit Court of Appeals reversed a previous bankruptcy court decision on Monday, calling for the dismissal of a Chapter 11 filing by J&J’s subsidiary LTL Management.

Faced with more than 38,000 lawsuits alleging its talc-based products caused cancer, J&J spun its talc liabilities into a separate company called LTL Management back in October 2021 and filed for bankruptcy, a controversial move colloquially referred to as a “Texas two-step” bankruptcy. Claimants argued that the strategy is a misuse of the US bankruptcy code — and on Monday, a panel of judges agreed.

Troy Tazbaz, FDA's newly-named director of the Digital Health Center of Excellence (Oracle via YouTube)

Or­a­cle ex­ec­u­tive Troy Tazbaz named new FDA di­rec­tor of dig­i­tal health

The FDA has found a brand new director of the Digital Health Center of Excellence in Troy Tazbaz, a former senior vice president at Oracle.

According to Tazbaz’s LinkedIn, he took a five-month break after leaving an 11-year career at Oracle before joining the FDA in January. Stat News first reported the hire. Tazbaz also said on his LinkedIn that he biked all the way from Chesapeake Bay to the San Francisco Bay over 58 days during his career break.

Chad Mirkin, Flashpoint co-founder

‘The field is at a flash­point’: New Chad Mirkin-found­ed biotech hopes to make more ef­fec­tive can­cer vac­cines

Following the success of the mRNA Covid vaccines, cancer vaccines are seeing renewed interest after years of middling results. But a group of researchers suggests that more attention needs to be paid not to what goes into those vaccines, but how the parts are put together.

In a recent paper published in Nature Biomedical Engineering, researchers led by Northwestern University’s Chad Mirkin describe how the placement of different antigens in a cancer vaccine impacts its efficacy. The paper builds on past work done by Mirkin’s lab that suggests the structure, or how the parts of a vaccine are arranged, impact a vaccine’s efficacy, not just its components.

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