With new buy­out, J&J jumps back in on a sto­ried but trou­bled Holy Grail tar­get

Over a decade ago, some phar­ma ex­ec­u­tives fell in love with a mol­e­cule called ra­pamycin and its ge­net­ic tar­get, called mTOR. (Lit­er­al­ly: “mol­e­c­u­lar tar­get of ra­pamycin.”)

It was easy to see why. Orig­i­nal­ly iden­ti­fied in 1972 by an In­di­an-Cana­di­an chemist, who hid it in ice cream tubs in his freez­er and then smug­gled it in­to the US af­ter the com­pa­ny he worked for asked him to aban­don the pro­gram, it was fi­nal­ly ap­proved in 1999 as an im­muno­sup­pres­sant for or­gan trans­plant re­cip­i­ents.

But it showed a wide range of strange ef­fects. Broad­ly speak­ing, it seemed to stop cells from di­vid­ing — a fea­ture seen most no­tably in ex­per­i­ments show­ing mice, giv­en the drug, lived about 12% longer.

It could al­so, it seemed, slow the growth of heart tis­sue on stents — an in­di­ca­tion where it’s now used — and slow the growth of can­cer cells. This was seen in rig­or­ous an­i­mal mod­els and, anec­do­tal­ly, in the In­di­an-Cana­di­an chemist him­self, Suren­dra Nath Se­h­gal, who start­ed tak­ing ra­pamycin af­ter be­ing di­ag­nosed with what doc­tors said was ter­mi­nal colon can­cer. He lived five years, on­ly dy­ing af­ter he stopped.

So Mer­ck, No­var­tis and oth­ers start­ed de­vel­op­ing new com­pounds to go af­ter the same tar­get. In the clin­ic, though, the ef­fects were less pro­nounced. And the drugs could some­times bring fa­tal side ef­fects. In 2012, an FDA pan­el round­ly re­ject­ed a Mer­ck can­di­date in a rare sar­co­ma, say­ing the ben­e­fits didn’t out­weigh the risks.

Al­though one No­var­tis mTOR-tar­get­ing drug, everolimus, was ap­proved for sev­er­al can­cers, most drug com­pa­nies moved on. No­var­tis it­self of­floaded its mTOR pipeline to PureTech in 2017.

Now, though, J&J is get­ting back in the game. The phar­ma an­nounced on Wednes­day it bought, for an undis­closed sum, a tiny biotech known as Anakuria Ther­a­peu­tics.

Spun out of Nav­i­tor Phar­ma­ceu­ti­cals, an mTOR-fo­cused biotech that’s hung around qui­et­ly since its launch in 2009, Anakuria has a port­fo­lio of ra­pamycin-like mol­e­cules. One, known as AT-20494, is ready for first-in-hu­man tri­als, Nav­i­tor said in a state­ment.

Tom Hugh­es

J&J will test it in dom­i­nant poly­cys­tic kid­ney dis­ease, a pro­gres­sive ge­net­ic dis­or­der where cysts grow to cov­er the kid­neys, caus­ing a range of symp­toms.

Al­though re­searchers have ad­vised against us­ing ra­pamycin di­rect­ly in such pa­tients be­cause of its ex­treme side ef­fects, they’ve the­o­rized some type of mTOR in­hibitor could help slow cyst growth. Pre­sum­ably, J&J sees the new mol­e­cule as safer and more se­lec­tive.

“We are thrilled that the po­ten­tial val­ue and sub­stan­tial­ly dif­fer­en­ti­at­ed pro­file of Anakuria’s mTORC1 in­hibitor pro­gram can be ex­plored with Janssen,” Nav­i­tor CEO Tom Hugh­es said in a state­ment.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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Ab­bott pumps $450M+ in­to new Ire­land-based man­u­fac­tur­ing site project and hir­ing spree

As Ireland continues to see more investments and building projects from pharma companies, another contender is looking to place more investment in the Emerald Isle.

According to a report from The Irish Times on Friday, Abbott Laboratories is investing €440 million, or about $451 million, to build a new manufacturing plant in Kilkenny, located in the country’s southeast, to make more of its glucose monitors.

Tony Coles, Cerevel CEO

Cerev­el takes the pub­lic of­fer­ing route, with a twist — rais­ing big mon­ey thanks to ri­val da­ta

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

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Benjamin Oakes, Scribe Therapeutics CEO

CEO of Doud­na spin­out: With­in five years, genome ed­i­tors will have a 're­al­ly big im­pact' on pa­tients' lives

“CRISPR-by-design” is the idea behind Scribe Therapeutics, a company spun out from Jennifer Doudna’s Nobel-winning lab that’s competing in a closely-tracked field of genome editor companies just starting to make their way to the clinic.

After nabbing $100 million last March for its Series B funding round, Scribe is taking a different tack from some of its competitors, crafting a new enzyme isolated from bacteria called CasX, which has now been tweaked extensively and may be targeted to a range of genome-related diseases, offering a plethora of therapeutic options.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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