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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Cy­to­ki­net­ics’ ALS drug fails PhI­II, leav­ing the biotech with a sin­gle late-stage prospect

Cytokinetics’ candidate for the muscle disease amyotrophic lateral sclerosis, or ALS, failed a Phase III trial, the Bay Area biotech announced Friday morning.

At a second interim analysis of the trial, an independent review committee recommended that Cytokinetics discontinue its COURAGE-ALS trial for reldesemtiv, as it “found no evidence of effect” compared to placebo on the primary or key secondary endpoints.

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CHMP gives thumbs-up for We­govy use in ado­les­cents, along with nine new drug rec­om­men­da­tions

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended nine drugs for approval this week while also giving thumbs up for six expanded indications, including Novo Nordisk’s approved obesity medication Wegovy for younger people. Wegovy is already approved as an obesity treatment in the EU for adults, and the new indication would allow prescriptions for adolescents aged 12 and older.

Austin biotech Mol­e­c­u­lar Tem­plates lays off more than 100 staffers as pipeline nar­rows

Molecular Templates is ridding itself of a Phase I HER2 asset and fine-tuning its pipeline to focus on three programs and a preclinical Bristol Myers Squibb collaboration. With the narrowed scope on its so-called engineered toxin bodies, the Austin, TX biotech is laying off about half of its staff.

That’s a little more than 100 employees, per an SEC filing. Molecular’s layoffs, approved by its board Wednesday, add to the dozens of pullbacks in the industry in the first three months of 2023.

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Green­Light re­ceives buy­out of­fer; Apol­lomics com­pletes SPAC merg­er

RNA biotech GreenLight Biosciences has been handed an offer for potential acquisition.

GreenLight said in a release that it has received a non-binding “indication of interest” from Fall Line Endurance Fund to acquire GreenLight’s capital stock for $0.60 per share in cash. The release said any potential agreement between the two parties would depend on certain conditions.

Through a special committee, the biotech will evaluate the offer but added there’s no certainty a deal will go forward. GreenLight will also not make any more announcements until a deal comes through or “otherwise determines” a statement is necessary.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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TScan Therapeutics' departing CEO David Southwell and CSO/COO Gavin MacBeath

TCR up­start an­nounces CEO ex­it, with CSO now act­ing re­place­ment

A public T cell biotech’s chief executive has decided to leave the company.

TScan Therapeutics said Friday morning that CEO David Southwell stepped down earlier this week, leaving both his chief executive and board member roles. Filling in is Gavin MacBeath, the company’s CSO and COO. He became the acting CEO on Tuesday, and will continue to remain CSO and COO, TScan’s announcement read.

Aptinyx eval­u­ates fu­ture of the com­pa­ny fol­low­ing two failed tri­als, 60% lay­offs

This year has been tough for Aptinyx — two failed trials, a 60% cut in its workforce, and now the company has brought on a firm to help evaluate the future of the company.

The press release noted it’s working with the firm Ladenburg Thalmann as its financial advisor to assist in exploring and evaluating “strategic alternatives” — a process that a growing group of struggling biotechs has embarked on, sometimes ending in a merger, asset sale or wind-down.

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Sar­to­rius to ac­quire French man­u­fac­tur­er for $2.6B+ in cell and gene ther­a­py play

The German life science group Sartorius will be picking up French contract manufacturer Polyplus for the price of €2.4 billion, or $2.6 billion.

On Friday, Sartorius announced the acquisition through its French subgroup, Sartorius Stedim Biotech, which will be acquiring Polyplus from private investors ARCHIMED and WP GG Holdings IV. Polyplus has 270 employees and produces materials and components that go into making viral vectors that are used in cell and gene therapies. This includes DNA/RNA reagents as well as plasmid DNA. Polyplus has locations in France, Belgium, China and the US.