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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

Bags of shred­ded docs: In­di­an drug­mak­er Lupin hand­ed a Form 483 by FDA in­spec­tors

The generics manufacturer Lupin has been given another Form 483 from the FDA this year.

US regulators inspected Lupin’s pharmaceutical manufacturing site in the town of Mandideep, India from Nov. 14 through Nov. 23, with the 14-page report marking 16 observations.

The inspection report stated that the site did not have the appropriate controls over its computer systems to ensure that changes in “master production” or records are only done by authorized personnel, along with written procedures not being established to conduct annual reviews of records associated with drug batches.

Mark Cuban (Jed Jacobsohn/AP Images)

Mov­ing to the em­ploy­er side of health­care, Mark Cuban's Cost Plus Drugs part­ners with a PBM

From “Shark Tank” to direct-to-consumer generic drugs, Mark Cuban has made another inroad in the ongoing battle over prescription drug prices. His cost-plus-15% generic drug company, frequently undercutting many competitors, now has its sights set on the employer healthcare market.

Cost Plus Drugs, which originally pledged to cut out PBMs, has now partnered with the PBM EmsanaRx, majority owned by the Purchaser Business Group on Health, to launch a supplemental drug discount program designed specifically for self-funded employers, the company announced Thursday.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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FDA re­view­ers head back to White Oak in 2023, with lead­er­ship look­ing to ap­pease a new Con­gress

Republicans have taken a stand against the pandemic era habit of lax work-from-home schedules. Now that they’ve wrestled control of the House majority, the FDA’s leadership is playing ball, sending many of the agency’s more than 18,000 employees back to their desks early next year.

Whether this exodus back to White Oak in Silver Spring, MD (many staff will still be allowed to work from home for multiple days per week) will mean more defections to industry and elsewhere remains to be seen.