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.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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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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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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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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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

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.

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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.