Phar­varis lands an­oth­er $80M as it looks to tack­le Take­da and CSL with an oral ap­proach for HAE

A year af­ter their $66 mil­lion launch, the Swiss up­start Phar­varis has man­aged to con­vince an­oth­er fleet of new and re­turn­ing in­vestors to back their up­start ap­proach for a rare ge­net­ic con­di­tion called hered­i­tary an­gioede­ma, or HAE.

The com­pa­ny said Wednes­day that they’ve raised an $80 mil­lion Se­ries C led by Viking Glob­al In­vestors and Gen­er­al At­lantic. They’ll plow that cash di­rect­ly in­to a pair of Phase II tri­als to test whether their lead mol­e­cule can be­come the first oral drug to suc­cess­ful­ly treat and pre­vent the painful episodes HAE trig­gers.

A hand­ful of ef­fec­tive ther­a­pies al­ready ex­ist for HAE, a dis­ease char­ac­ter­ized by painful swelling in hands, feet and oc­ca­sion­al­ly in the air­ways or in­testi­nal walls. But the drugs, in­clud­ing CSL’s Hae­gar­da and Take­da’s Cin­ryze, Takhzy­ro and Fi­razyr, are all in­jectable.

The dis­ease is marked by a de­fi­cien­cy in the blood plas­ma pro­tein C1 es­terase in­hibitor and both Hae­gar­da and Cin­ryze try to boost the lev­el of that pro­tein in the blood. Fi­razyr, though, blocks bradykinin, a blood pro­tein that is over­pro­duced in HAE pa­tients and di­rect­ly caus­es blood cells to leak and tis­sues to swell.

Berndt Modig

Found­ed by Berndt Modig, their CEO, and oth­er vet­er­ans from Jeri­ni, the biotech that orig­i­nal­ly de­vel­oped Fi­razyr, Phar­varis is try­ing to de­vel­op a mol­e­cule that blocks the same bradykinin re­cep­tor but with a pill. Last week, they re­leased Phase I da­ta from 16 healthy vol­un­teers that they say sug­gest their mol­e­cule is 24 times as po­tent as Fi­razyr.

The com­pa­ny will now launch two Phase II tri­als, one for pro­phy­lax­is and one for treat­ing acute pain. If those are suc­cess­ful, they’ll fol­low on in with piv­otal Phase III stud­ies.

They are al­so de­vel­op­ing a new for­mu­la­tion that they plan to even­tu­al­ly use in pro­phy­lax­is tri­als in­stead of the cur­rent one.

The chal­lenges in get­ting there will be sig­nif­i­cant, though. Oth­er com­pa­nies have strug­gled to de­vel­op oral HAE treat­ments in the past. Most no­tably, BioCryst saw their shares cut in half last year af­ter Phase III re­sults fell short of in­vestor ex­pec­ta­tions.

Mean­while, al­though the com­pa­ny is con­fi­dent that pa­tients want an oral op­tion, the in­jecta­bles are al­ready high­ly ef­fec­tive and en­trenched. Takhzy­ro, the newest one on the mar­ket, is ex­pect­ed to be a block­buster.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

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.

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.

Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.