Jan Møller Mikkelsen, Ascendis

As­cendis wins ap­proval for once-week­ly growth hor­mone drug as Pfiz­er, Op­ko Health keep up the pres­sure

Den­mark’s As­cendis Phar­ma won its first FDA ap­proval Wednes­day af­ter­noon, beat­ing out a ri­val pro­gram at Pfiz­er for pe­di­atric growth hor­mone de­fi­cien­cy.

As­cendis notched the OK for lon­apeg­so­ma­t­ropin, to be mar­ket­ed as Skytro­fa, as the first once-week­ly treat­ment for chil­dren aged one and old­er who don’t pro­duce enough growth hor­mone and weigh at least 11.5 kilo­grams, the biotech an­nounced. There’s no in­fo on the pric­ing yet and End­points News has reached out for com­ment, but CEO Jan Møller Mikkelsen said on a con­fer­ence call Wednes­day the cost will be “re­spon­si­ble.”

Pre­vi­ous GHD treat­ments re­quire dai­ly in­jec­tions of so­ma­t­ropin, a syn­thet­ic form of hu­man growth hor­mone, but As­cendis hopes the sus­tained re­lease over week­ly dos­ing will pro­vide pa­tients with a more con­ve­nient op­tion. The com­pa­ny said the ap­proval cov­ers not on­ly the drug, but car­tridges that can be stored at room tem­per­a­ture for up to six months.

“This once-week­ly treat­ment could re­duce treat­ment bur­den and po­ten­tial­ly re­place the dai­ly so­ma­t­ropin ther­a­pies, which have been the stan­dard of care for over 30 years,” As­cendis Phase III in­ves­ti­ga­tor Paul Thorn­ton said in a state­ment.

Wednes­day’s ap­proval was based on a Phase III tri­al that en­rolled 161 chil­dren who hadn’t tak­en any pri­or treat­ment. The study com­pared lon­apeg­so­ma­t­ropin to Genotropin dai­ly in­jec­tions, and demon­strat­ed As­cendis’ pro­gram was not just non-in­fe­ri­or to af­ter one year, but al­so su­pe­ri­or. Chil­dren tak­ing the As­cendis drug grew an av­er­age of 0.9 cm per year faster than those on Genotropin.

The FDA’s de­ci­sion al­so came ear­li­er than ex­pect­ed af­ter As­cendis’ PDU­FA date was pushed back in June. Orig­i­nal­ly slat­ed for a June de­ci­sion, reg­u­la­tors de­layed the PDU­FA to Sep­tem­ber 25 af­ter As­cendis sub­mit­ted ad­di­tion­al in­for­ma­tion as part of its ap­pli­ca­tion.

At least one an­a­lyst ap­peared con­fi­dent in Skytro­fa’s abil­i­ty to shake things up. SVB Leerink’s Joseph Schwartz wrote to in­vestors Wednes­day evening that, de­spite the lim­it­ed in­for­ma­tion re­vealed on the con­fer­ence call re­gard­ing pric­ing and oth­er mat­ters, As­cendis’ fo­cus on long-term gains rather than strict­ly sales num­bers is a pos­i­tive sign.

“Con­sid­er­ing the growth hor­mone mar­ket is frag­ment­ed with mul­ti­ple un­dif­fer­en­ti­at­ed prod­ucts that must be treat­ed dai­ly, [As­cendis] be­lieves that mar­ket is primed for dis­rup­tion,” Schwartz wrote. He added that he ex­pects the drug to be ap­proved in Eu­rope by the end of the year and that As­cendis is set­ting up ac­cess pro­grams to help pa­tients switch from dai­ly treat­ment.

As­cendis’ ap­proval beat out a can­di­date tout­ed by Pfiz­er and Op­ko Health, which re­vealed its lat­est Phase III da­ta in Oc­to­ber 2019 and Oc­to­ber 2020. In the first study, the pair’s so­ma­tro­gon pro­gram showed non-in­fe­ri­or­i­ty to Genotropin and that the drug arm saw pa­tients grow at an av­er­age of 0.33 cm per year faster than stan­dard of care.

The sec­ond tri­al, mean­while, ob­served im­prove­ment on the av­er­age score on a com­mon GHD test, known as the Life In­ter­fer­ence Ques­tion­naire, af­ter 12 weeks on each treat­ment.

Pfiz­er and Op­ko are ex­pect­ed to hear back from the FDA in Oc­to­ber and are al­so hop­ing their long-act­ing hor­mone treat­ment can shake up the mar­ket, par­tic­u­lar­ly af­ter a high-pro­file flop way back in 2016. But As­cendis beat them to the punch by a lit­tle over a month, giv­ing the biotech a po­ten­tial leg up on get­ting its drug to mar­ket.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Up­dat­ing the Covid-19 vac­cine: FDA of­fers a qual­i­fied thumbs-up ahead of ad­comm

The FDA’s adcomm of outside vaccine experts will meet tomorrow to discuss how to protect the US from a likely coming wave of Omicron cases in the fall and winter, and whether to deploy vaccines that specifically target the Omicron variant.

While the data so far are limited, the FDA sounded an upbeat tone in the briefing documents on Pfizer/BioNTech’s candidates, released this weekend ahead of the VRBPAC meeting.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.

Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.