Terrie Curran, Phathom CEO (Credit: Arcutis)

Phath­om's old Take­da drug bests Pre­vacid in a PhI­II GI tri­al. Next stop? The FDA

There’s no time for rest in bio­phar­ma — at least not at Phath­om Phar­ma­ceu­ti­cals. Just over a month af­ter sub­mit­ting two NDAs for its lead acid-fight­er vono­prazan, the biotech is al­ready lin­ing up a third, and col­lect­ing an ex­tra $50 mil­lion to push things along.

Vono­prazan met its pri­ma­ry non-in­fe­ri­or­i­ty end­points in a Phase III study com­par­ing it to stan­dard-of-care Pre­vacid in a type of gas­troe­sophageal re­flux dis­ease (GERD) called ero­sive esophagi­tis (EE). It al­so proved su­pe­ri­or to the pop­u­lar heart­burn drug by mul­ti­ple mea­sures, in­clud­ing heal­ing rate and main­te­nance of heal­ing.

“The re­sults fur­ther so­lid­i­fy vono­prazan’s po­ten­tial to be the first ma­jor in­no­va­tion in the U.S. and Eu­ro­pean GERD mar­ket in more than 30 years,” CEO Ter­rie Cur­ran said in a state­ment.

Phath­om Phar­ma­ceu­ti­cals has come a long way since it was spun out of Take­da, with the help of Fra­zier, two years ago to han­dle the ex-Japan de­vel­op­ment of vono­prazan. Vono­prazan, dis­cov­ered by Take­da, is a P-CAB: a class of drugs that blocks the potas­si­um-bind­ing site of gas­tric hy­dro­gen potas­si­um AT­Pase (al­so known as the pro­ton pump), which is the en­zyme large­ly re­spon­si­ble for acid­i­fi­ca­tion of the stom­ach. It’s al­ready ap­proved in Japan to treat a va­ri­ety of acid-re­lat­ed con­di­tions, in­clud­ing EE.

The Phase III Phal­con-EE tri­al en­rolled just over 1,000 EE pa­tients in the US and Eu­rope, who were treat­ed with a 20 mg dose of vono­prazan in a heal­ing phase and one of two dos­es (10 mg or 20 mg) in a main­te­nance phase. In the heal­ing phase, vono­prazan proved non-in­fe­ri­or to Pre­vacid in terms of pa­tients who achieved com­plete heal­ing by Week 8, with a heal­ing rate of 93% in the vono­prazan arm com­pared to 85% in the Pre­vacid arm (p<0.0001). It al­so passed an ex­plorato­ry su­pe­ri­or­i­ty test, with a p-val­ue of 0.0001.

Pa­tients on vono­prazan saw sig­nif­i­cant­ly faster heal­ing in this phase. How­ev­er, a su­pe­ri­or­i­ty test com­par­ing a 20 mg dose of vono­prazan to a 30 mg dose of Pre­vacid for sus­tained res­o­lu­tion of heart­burn by Day 3 did not turn up sta­tis­ti­cal­ly sig­nif­i­cant (p=0.2196).

Vono­prazan met the pri­ma­ry and all sec­ondary end­points in the main­te­nance phase, though, demon­strat­ing su­pe­ri­or­i­ty in main­te­nance of heal­ing (79% for vono­prazan 10 mg, 81% for vono­prazan 20 mg, and 72% for Pre­vacid). The p-val­ues there were less than 0.0001 for both non-in­fe­ri­or­i­ty com­par­isons, 0.0218 for the 10 mg su­pe­ri­or­i­ty test and 0.0068 for the 20 mg su­pe­ri­or­i­ty test.

Both vono­prazan dos­es al­so achieved su­pe­ri­or­i­ty in the per­cent­age of pa­tients with mod­er­ate-to-se­vere dis­ease who main­tained heal­ing of EE through Week 24 (75% for vono­prazan 10 mg, 77% for vono­prazan 20 mg, and 61% for Pre­vacid).

The fre­quen­cy of se­ri­ous ad­verse events were rough­ly the same in the 20 mg vono­prazan and Pre­vacid arms of the heal­ing phase, but about twice as high in the 20 mg vono­prazan arm of the main­te­nance phase, ac­cord­ing to Phath­om. How­ev­er, that does in­clude five pa­tients in the 20 mg arm who got Covid-19, which was un­re­lat­ed to the treat­ment.

The new da­ta will al­low Phath­om to sub­mit an NDA in the first half of next year, and trig­gers an ex­tra $50 mil­lion from its term loan fa­cil­i­ty with Her­cules Cap­i­tal.

It’s al­so good news for Catal­ent, which inked a deal to man­u­fac­ture the drug just a few weeks ago.

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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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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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.

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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