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.

Spe­cial re­port: Meet 20 ex­tra­or­di­nary women who are su­per­charg­ing bio­phar­ma R&D

Even though many biopharma leaders have come together in recent years to address its gender gap, the consensus is clear: We still have a long way to go.

Companies this year were 2.5 times more likely than last year to have a diversity and inclusion program in place, according to a recent BIO survey, but women are still largely absent from executive roles. Getting women to enter the industry isn’t the problem — studies show that they represent just under half of all biotech employees around the world. But climbing through the ranks can be challenging, as women still report facing stereotypes, and, unfortunately, harassment.

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Op­ti­miz­ing Oral Drug De­liv­ery us­ing Zy­dis® Oral­ly Dis­in­te­grat­ing Tablet Tech­nol­o­gy to Ad­dress Pa­tient Chal­lenges

KEY POINTS

Patients prefer oral dosing, but swallowing tablets can be a challenge for many patients.
The Zydis® orally disintegrating tablet (ODT) platform addresses challenges associated with oral dosing, expanding benefits for patients and options for healthcare providers.
A strong growth trajectory is expected for ODTs given therapeutic innovation and continued technology development.

Many patients prefer conventional tablets for the administration of medications, but some geriatric and pediatric patients and those with altered mental status and physical impairments find swallowing tablets to be difficult. Orally disintegrating tablets (ODTs), which dissolve completely without chewing or sucking, offer a patient-friendly dosage form for the administration of small-molecule drugs, peptides and proteins. With the potential for multiple sites of drug absorption, often faster onset action for the active pharmaceutical ingredient (API), and potentially greater bioavailability, ODTs are an attractive option for drug developers considering first-to-market formulations or product line extensions of existing drugs with compatible API. In this report, we look at how innovation in the industry-leading Zydis ODT platform is expanding oral formulation options and bringing benefits to patients.

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Geoffrey Porges (SVB Leerink)

The 2022 wave com­ing? Top an­a­lyst says Big Phar­ma will have more than $1T avail­able to sat­is­fy its grow­ing ap­petite for biotech M&A

All through this year you could practically feel the frustration of the biotech investor class as M&A activity continued to drag behind expectations — or desires. Buyouts of public companies provide the essential juice for keeping stocks lively, and there’s been a notable lack of juice in 2021.

So is all that about to change, big time?

SVB Leerink’s Geoffrey Porges, a longtime student of biotech M&A, thinks so. In a lengthy analysis he put out last week, Porges totted up the cash flow of the major pharmas and determined that there was a good long list of industry buyers who would have around a half trillion dollars of cash to play with in 2022. Leverage that up with added debt and you could get that deal cache to $1.6 trillion.

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Mer­ck pumps the brakes on two more PhI­II tri­als for its lead an­ti-HIV drug

After trial investigators flagged a drop in immune cell counts that an external committee determined was related to treatment last month, Merck has been pausing HIV-related Phase II and III trials ever since.

On Monday, the biopharma company announced it’s pausing enrollment in two of its Phase III trials evaluating its leading anti-HIV drug candidate, which is the once-monthly, oral islatravir.

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Bolt Bio CEO Randy Schatzman

Bolt Bio goes bust as in­vestors boo sin­gle re­sponse in ear­ly test against HER2-ex­press­ing tu­mors

Bolt Bio’s BDC-1001, an antibody conjugate drug designed to amp up the body’s innate immune system response to tumors, posted a single partial response in a Phase I/II study in patients with HER2-expressing solid tumors after a year of dosing. Just 13 of 40 evaluable patients showed any signs of “clinical activity,” the biotech said Monday.

BDC-1001 links a HER2-targeting biosimilar of Herceptin with a TLR7/8 agonist, which is designed to activate myeloid cells in the innate immune system and drive tumor cytotoxicity, Bolt said. But the early results paint the picture of a drug with little effect on HER2 tumors, one of the most highly validated tumor targets in drug development.

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Biohaven CEO Vlad Coric (Photo Credit_ Andrew Venditti)

Bio­haven shakes up lead­er­ship team as it feels the heat from mi­graine com­peti­tors

As Biohaven Pharma comes off a rollercoaster pipeline year, its CEO will take on more responsibility in a full C-suite makeover.

Vlad Coric was unanimously elected to the role of chairman of the board of directors, after Declan Doogan’s retirement. Matthew Buten will take over the role of CFO after James Engelhart’s retirement, and director Michael Heffernan has been appointed lead independent director. All of the appointments are effective immediately, a company press release said.

Chen Schor, Adicet CEO

Adicet un­veils ear­ly re­spons­es for off-the-shelf drug lever­ag­ing rare T cells. Will dura­bil­i­ty hold up?

On the hunt for the next generation of “off-the-shelf” cell therapies, biotech players like Adicet Bio have looked to leverage some of the less-obvious members of the immune system as potent cancer fighters. In Adicet’s case, scarce gamma delta T cells are on the menu, and an early cut of data is showing some promise.

Adicet’s AD-001, an off-the-shelf cell therapy developed by engineering a CD20-targeting chimeric antigen receptor (CAR) onto a donor’s gamma delta T cells, posted two complete responses across four patients in an early Phase I study testing the drug in patients with heavily pretreated B cell non-Hodgkin’s lymphoma, the biotech said Monday.

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Mar­ket­ingRx Matchup: How Ab­b­Vie and Bio­haven ads rank in head-to-head mi­graine chal­lenge

Are you ready to rumble? DTC brands that is. MarketingRx is launching a new monthly feature today called MarketingRx Matchup. We’re pitting two pharma brands’ DTC advertising in the same therapeutic category against each other to find out what consumers and patients really think.

Market research company Leger is handling the polling and analysis each month, and I’ll be writing up the results — along with my own take — inside MRx on the first Tuesday of the month.

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Wendy Lund, Organon chief communications officer

Q&A: Organon chief com­mu­ni­ca­tions of­fi­cer Wendy Lund talks about the Mer­ck spin­off, women’s health and why it mat­ters

One of Wendy Lund’s earliest jobs was head of marketing at Planned Parenthood. As the youngest person on its management team, she introduced them to emerging new technologies, and in return, she learned the importance of fighting for what you believe in.

Now as chief communications officer at Organon, the women’s health company recently spun off by Merck, Lund is keeping that point top of mind. That’s in part because women’s health hasn’t been a spotlight therapy area for Big Pharma in years. Several companies have spun off, sold or at least considered selling women’s health assets to focus on “core” products.

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