Yvonne Greenstreet, Alnylam CEO (via Alnylam)

Al­ny­lam boasts PhII win in kid­ney dis­ease cor­ner­stone of Re­gen­eron R&D block­buster deal

A drug at the heart of the $1 bil­lion-plus Al­ny­lam and Re­gen­eron col­lab­o­ra­tion ce­ment­ed three years ago has some new ef­fi­ca­cy da­ta to show off.

The RNAi biotech once helmed by John Maraganore re­port­ed pos­i­tive topline da­ta Thurs­day for its in­ves­ti­ga­tion­al cem­disir­an, an RNAi drug tar­get­ing the C5 com­po­nent of the path­way that both Al­ny­lam and Re­gen­eron are work­ing on in IgAN, or im­munoglob­u­lin A nephropa­thy. The da­ta come out of a Phase II de­scrip­tive tri­al, with no sta­tis­ti­cal hy­poth­e­sis test­ing.

Im­munoglob­u­lin A nephropa­thy is a kid­ney dis­ease, marked by when im­munoglob­u­lin A, an an­ti­body, builds up in the kid­neys. This can re­sult in in­flam­ma­tion and ul­ti­mate­ly kid­ney fail­ure.

Af­ter 32 weeks, treat­ment in 31 adult pa­tients with sub­cu­ta­neous­ly ad­min­is­tered cem­disir­an re­sult­ed in a 37% mean re­duc­tion from base­line in the 24-hour urine pro­tein to cre­a­ti­nine ra­tio (UP­CR) rel­a­tive to place­bo. As such, the study reached its pri­ma­ry end­point of pre­cent change in the UP­CR af­ter 24 hours.

The re­sults of sec­ondary end­points were al­so “con­sis­tent with a ther­a­peu­tic ben­e­fit of cem­disir­an in IgAN,” ac­cord­ing to Al­ny­lam. Those sec­ondary end­points:

  • Per­cent of pa­tients with par­tial clin­i­cal re­mis­sion (urine pro­tein <1g/24-hours)
  • Per­cent of pa­tients with >50% re­duc­tion in 24-hour pro­tein­uria
  • Per­cent change from base­line in 24-hour pro­tein­uria (g/24-hours)
  • Change from base­line in UP­CR as mea­sured in spot urine
  • Change from base­line in hema­turia
  • Fre­quen­cy of ad­verse events (AEs)

Al­ny­lam said there were no “sig­nif­i­cant drug-re­lat­ed safe­ty sig­nals,” but not­ed that two of nine pa­tients on place­bo and 12 of 22 pa­tients on cem­disir­an ex­pe­ri­enced treat­ment-emer­gent ad­verse events.

Shares of $AL­NY dipped slight­ly af­ter the re­lease, go­ing down less than 1% af­ter the an­nounce­ment.

Sonalee Agar­w­al

“We are en­cour­aged by these topline re­sults with cem­disir­an demon­strat­ing what we be­lieve to be clin­i­cal­ly mean­ing­ful re­duc­tions in pro­tein­uria – an im­por­tant prog­nos­tic fac­tor in IgA nephropa­thy,” said Al­ny­lam VP and Cem­disir­an pro­gram leader Sonalee Agar­w­al in a state­ment.

Agar­w­al added that Al­ny­lam and Re­gen­eron are work­ing on putting to­geth­er plans for Phase III clin­i­cal de­vel­op­ment. Full re­sults will be pre­sent­ed at a fu­ture med­ical con­gress.

When the deal was first inked in 2019, Re­gen­eron had agreed to pay Al­ny­lam $400 mil­lion up­front, and added an­oth­er $400 mil­lion eq­ui­ty — equat­ing to $90.01 a share in 2019 — with $200 mil­lion on the ta­ble in ear­ly clin­i­cal mile­stones. With up to 30 tar­gets in mind, Al­ny­lam was of­fered to get a $2.5 mil­lion bonus every time it ini­ti­at­ed a pro­gram, an­oth­er $2.5 mil­lion for lead iden­ti­fi­ca­tion and about $30 mil­lion in an­nu­al dis­cov­ery funds avail­able to boot.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,600+ biopharma pros reading Endpoints daily — and it's free.

Andy Kidd, Aptinyx CEO

‘The place­bo caught up’: Months af­ter di­a­bet­ic nerve pain fail, Aptinyx sees an­oth­er pain tri­al fall through

In 2019, Aptinyx’s stock cratered after it reported that its lead candidate failed a diabetic nerve pain trial. After some ‘further analysis,’ the biotech re-upped with that same non-opioid pain drug in two more mid-stage studies — another diabetic nerve pain trial and later a fibromyalgia trial.

In April, Aptinyx reported that its second diabetic nerve pain trial also fell through. However, the Illinois-based penny stock biotech had one more shot for its NMDA-modulating drug, dubbed NYX-2925, in fibromyalgia.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,600+ biopharma pros reading Endpoints daily — and it's free.

Randall Schatzman, Bolt CEO

Bolt throws a wrench in the pipeline, look­ing to con­serve cash

A meltdown in the biotech market is making most execs cautious, including Bolt Biotherapeutics’ CEO, as the company hits the brakes on one preclinical asset and pauses other early-stage work to extend cash reserves by two years.

The pipeline re-org will keep Bolt’s lights on through 2025 so the biotech can focus on its clinical-stage HER2 solid tumor drug candidate, which the company should have early-stage data on and a recommended Phase II dose by year’s end. The biotech also wants to focus on its preclinical asset BDC-3042, an immune-stimulating antibody conjugate (ISAC), for KRAS and TP53 mutated tumors.