A ground­break­ing can­cer drug nabs fast OK, giv­ing Eli Lil­ly boast­ing rights as a ri­val speeds close be­hind

Eli Lil­ly $LLY has scored the fast FDA ap­proval it was gun­ning for when the phar­ma gi­ant plunked down $8 bil­lion to ac­quire Loxo On­col­o­gy back at the be­gin­ning of 2019. Reg­u­la­tors stamped their OK on selper­ca­tinib, which can now be mar­ket­ed as Retev­mo to pa­tients suf­fer­ing from 3 dif­fer­ent types of can­cers all dri­ven by the same gene al­ter­ation.

The ap­proval marks an­oth­er win for ad­vo­cates of rapid de­vel­op­ment, with the Loxo team jump­ing from their first treat­ment to an ap­proval on Phase I/II da­ta in 3 short years. And it comes just 3 months af­ter the FDA’s can­cer group hand­ed Lil­ly a pri­or­i­ty re­view, in­di­cat­ing that the agency still finds it easy to re­ward ad­vances in the field with speedy an­swers — de­spite the pan­dem­ic.

For Lil­ly, the drug OK al­so marks a rare first place fin­ish, but Blue­print Med­i­cines $BPMC is ex­pect­ed to quick­ly counter with new da­ta at AS­CO on a ri­val that is one step be­hind the phar­ma gi­ant.

The phar­ma play­er got the mar­ket­ing ball rolling with a $20,600 per month price on the ther­a­py.

But they don’t have a lock on best-in-class sta­tus. As Steve Scala at Cowen not­ed Mon­day morn­ing:

The la­bel con­tains pos­i­tives (line ag­nos­tic, in­creased ef­fi­ca­cy in thy­roid can­cer) and neg­a­tives (safe­ty warn­ings in­clud­ing liv­er mon­i­tor­ing, in­cre­men­tal downtick in lung ef­fi­ca­cy). All told, the la­bel is in­cre­men­tal­ly worse than hoped. Long-term dura­bil­i­ty da­ta is now more im­por­tant.

The FDA ac­tion cov­ers RET fu­sion-pos­i­tive non-small cell lung can­cer and thy­roid can­cer, along with RET-mu­tant medullary thy­roid can­cer. In the ab­sence of an ap­proved test to spot the al­ter­ation, Lil­ly sug­gests us­ing bio­mark­ers to iden­ti­fy pa­tients.

That is no easy propo­si­tion, as on­ly 2% of NSCLC pa­tients will be in­clud­ed. But it’s a large enough group to push the field to­ward more test­ing. It al­so helps that No­var­tis just got an OK for cap­ma­tinib a few days ago, adding an­oth­er drug used on the ba­sis of a gene al­ter­ation that dri­ves cell pro­lif­er­a­tion. As more of these drugs are ap­proved, test­ing and se­quenc­ing will grow more com­mon, al­low­ing for bet­ter out­comes for all.

Blue­print is al­so like­ly to help that process if their next round of da­ta for pralse­tinib — now un­der FDA re­view — in RET fu­sion pos­i­tive NSCLC high­lights pos­i­tive re­sults. An ap­pli­ca­tion for RET-mu­tant medullary thy­roid can­cer is ex­pect­ed any week now.

An­drew Berens at SVB Leerink has been track­ing the com­pe­ti­tion from Blue­print’s per­spec­tive, and re­cent­ly not­ed how the matchup will be scored based on AS­CO da­ta:

We ex­pect in­vestor fo­cus (on pralse­tinib) to be on DOR rel­a­tive to selper­ca­tinib, which has shown me­di­an DOR of 20.3 months. How­ev­er, some in­vestors are fo­cused on the me­di­an PFS as the rel­a­tive bench­mark of ef­fi­ca­cy, with LOXO’s selper­ca­tinib show­ing 18.4 months at WCLC 2019.

The FDA ac­tion on Fri­day serves as fur­ther val­i­da­tion for Lil­ly’s de­ci­sion to turn over the on­col­o­gy group to the Loxo team, led by Josh Bilenker. Lil­ly’s can­cer team has had a lack­lus­ter per­for­mance record over the years, which Bilenker’s group was brought on to rem­e­dy. Since then, they’ve been clean­ing up the pipeline and prep­ping new deals as Lil­ly looks to join big play­ers like GSK and Sanofi, which are look­ing to turn things around with some fast-ac­tion can­cer plays.

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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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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Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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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.

David Hung, Nuvation Bio president and CEO (Nuvation Bio)

FDA places par­tial clin­i­cal hold on David Hung biotech af­ter cer­tain can­cer pa­tients ex­pe­ri­ence eye in­flam­ma­tion

Two and a half years after setting out on another foray into oncology R&D, a biotech headed by David Hung — of Medivation fame — has run into its first setback.

San Francisco-based Nuvation Bio announced early Monday the FDA placed a partial clinical hold on a Phase I dose-escalation study of NUV-422, its CDK inhibitor program for certain types of solid tumors. The trial began enrolling patients in December 2020, and, according to Nuvation, researchers were in the middle of exploring dose escalation and defining the maximum dose tolerable in patients.

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.