No­var­tis' PI3K drug, al­ready ap­proved in breast can­cer, nabs a speedy OK to treat ul­tra-rare con­di­tion

Half a year af­ter No­var­tis show­cased the po­ten­tial of its PI3K in­hibitor alpelis­ib among a group of young pa­tients with an ul­tra-rare con­di­tion, the FDA has green­light­ed the drug’s use based sole­ly on re­al-world ev­i­dence.

Brand­ed Vi­joice, it is the first and on­ly treat­ment aimed at the root cause of PIK3CA-re­lat­ed over­growth spec­trum (PROS), ac­cord­ing to No­var­tis, a dis­ease es­ti­mat­ed to af­flict 14 peo­ple per mil­lion.

The un­der­ly­ing mu­ta­tions in the PIK3CA are just what alpelis­ib tar­gets. The same drug is al­ready mar­ket­ed as Piqray to treat a sub­set of ad­vanced or metasta­t­ic breast can­cer that’s HR-pos­i­tive, HER2-neg­a­tive and PIK3CA-mu­tat­ed. No­var­tis re­port­ed $390 mil­lion in sales for 2021.

With PROS, pa­tients’ body parts can grow too large or have an un­usu­al shape, and they may have blood ves­sel ab­nor­mal­i­ties.

“PROS con­di­tions can be de­bil­i­tat­ing and dis­abling and can re­sult in dis­rup­tions to every­day ac­tiv­i­ties,” said Kris­ten Davis, ex­ec­u­tive di­rec­tor of CLOVES Syn­drome Com­mu­ni­ty, in a pre­pared state­ment. “Un­til to­day, of­ten the on­ly treat­ment op­tions for pa­tients were sur­gi­cal or in­ter­ven­tion­al ra­di­ol­o­gy pro­ce­dures.”

Through a com­pas­sion­ate use pro­gram, a num­ber of PROS pa­tients re­ceived the drug. No­var­tis then ran a re­al-world study dubbed EPIK-P1 re­view­ing the med­ical charts of 57 pa­tients, in­clud­ing 39 pe­di­atric and 18 adult pa­tients, across five coun­tries. It found that those treat­ed with Vi­joice saw a re­duc­tion in the size of PROS le­sions as well as im­prove­ments in re­lat­ed signs and symp­toms.

Since Vi­joice was OK’d un­der the ac­cel­er­at­ed ap­proval path­way, No­var­tis will be re­quired to run a con­fir­ma­to­ry tri­al.

The phar­ma gi­ant said it will con­duct two ad­di­tion­al Phase II stud­ies: EPIK-P2 to as­sess safe­ty, ef­fi­ca­cy and phar­ma­co­ki­net­ics, and EPIK-P3 to look in­to long-term safe­ty and ef­fi­ca­cy for pa­tients who were in EPIK-P1.

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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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.

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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Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

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.

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.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.