Bill Hinshaw, Axcella CEO

Ax­cel­la touts in­ter­im NASH da­ta but needs cash to stay afloat for 2024 topline read­out

Ax­cel­la Ther­a­peu­tics, with an emp­ty bank ac­count loom­ing in the first quar­ter of next year and a re­cent set­back in a mid-stage long Covid study, is out with some in­ter­im da­ta on its NASH drug.

Mar­garet Koziel

The biotech isn’t yet say­ing how the drug per­formed on the pri­ma­ry end­point of im­prove­ment in the non­al­co­holic fat­ty liv­er dis­ease (NAFLD) Ac­tiv­i­ty Score (NAS), but the first batch of re­sults has sent shares $AXLA soar­ing near­ly 15% be­fore Thurs­day’s open­ing bell.

At 24 weeks, Ax­cel­la said all pa­tients tak­ing the high dose of its drug, AXA1125, showed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in liv­er stiff­ness mea­sure­ment com­pared to place­bo, com­ing in at a p-val­ue of 0.0096 with an ab­solute change in LSM at -4.07 kilo­pas­cals, or kPa. The low-dose arm did not reach sta­tis­ti­cal sig­nif­i­cance with -2.01 kPa and a p-val­ue of 0.0992. Pos­i­tive changes in liv­er stiff­ness are cor­re­lat­ed with im­prove­ment in fi­bro­sis, CMO Mar­garet Koziel said in a state­ment.

An Ax­cel­la spokesper­son told End­points News via email that the com­pa­ny is “eval­u­at­ing var­i­ous op­tions in­clud­ing fi­nanc­ing and busi­ness de­vel­op­ment op­por­tu­ni­ties to fund the or­ga­ni­za­tion.”

Ax­cel­la is try­ing to po­si­tion the oral drug — a com­bi­na­tion of six amino acids and de­riv­a­tives — as a treat­ment for NASH and long Covid, with the lat­ter not meet­ing the bar in a Phase II pri­ma­ry end­point, but the biotech nonethe­less is at­tempt­ing to move for­ward on fa­tigue mea­sures.

“[The re­sults] in­di­cate that ad­min­is­tra­tion of AXA1125 over 24-weeks leads to sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments com­pared to place­bo in bio­mark­ers for me­tab­o­lism, in­flam­ma­tion and fi­bro­sis, un­der­scor­ing its mul­ti-tar­get­ed ef­fi­ca­cy,” CEO Bill Hin­shaw said in a state­ment Thurs­day.

The pre­planned in­ter­im analy­sis, con­duct­ed once 30% of 270 ex­pect­ed pa­tients were en­rolled, looked at 82 pa­tients at week 12 and 58 who had thus far made it 24 weeks. The on­go­ing blind­ed study ran­dom­ized pa­tients 1:1:1 to ei­ther place­bo, 22.6 g or 33.9 g of AXA1125 twice dai­ly.

At both time points, all pa­tients an­a­lyzed were shown to have sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in ala­nine amino­trans­ferase (ALT), which is in­dica­tive of liv­er cell in­flam­ma­tion, Ax­cel­la said. The im­prove­ments came in at -28.61% (p=0.0183) for the low dose and -36.3% (p=0.0017) for high dose.

A field lit­tered with clin­i­cal tri­al fail­ures, NASH has long been a snag for drug de­vel­op­ers, but a re­cent crop of mid-stage da­ta out of Akero Ther­a­peu­tics sent the biotech straight to a $175 mil­lion pub­lic of­fer­ing af­ter the com­pa­ny said its drug led to at least one stage of im­prove­ment in fi­bro­sis with NASH wors­en­ing in 41% of pa­tients on 50 mg and 39% of those in the 28 mg arm.

Ax­cel­la will soon need to raise mon­ey if it wants to make it to the topline 48-week biop­sy re­sults for AXA1125. That Phase IIb da­ta read­out is on tap for the first half of 2024, Hin­shaw said, but the com­pa­ny on­ly has the cap­i­tal to keep the lights on in­to the first quar­ter of 2023, Ax­cel­la re­port­ed in earn­ings last month.

Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Robert Califf, FDA commissioner (Jose Luis Magana/AP Images)

Fourth ac­cel­er­at­ed ap­proval in Duchenne? Sarep­ta gets pri­or­i­ty re­view for gene ther­a­py amid FDA scruti­ny

Sarepta is once again on the accelerated approval path for a Duchenne drug, picking up a priority review Monday morning.

The FDA granted the accelerated review to SRP-9001, Sarepta announced Monday, which would become the biotech’s fourth Duchenne drug if approved. Much like SRP-9001 will do, each of the previous three therapies went through the accelerated approval pathway. But unlike the others, SRP-9001 is a gene therapy.

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Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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Merck targets vaccine-hesitant parents in its latest 'Why Vaccines' campaign. (Image: Shutterstock)

Mer­ck­'s lat­est 'Why Vac­ci­nes' cam­paign seeks to bet­ter in­form vac­cine-hes­i­tant moms

From Hollywood couple endorsements to targeted equity efforts, Merck has been pushing the value of vaccinations, especially since the Covid-19 pandemic disruption. Now the pharma is turning to a new target — vaccine-hesitant parents, and moms in particular.

Merck’s “Why Vaccines” latest social media and digital campaign spotlights real-life new moms who have questions about vaccinating their children.

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In­dus­try groups, CVS pick apart FDA's pro­posed path­way for gener­ics to carve out OTC in­di­ca­tions

Pharma industry groups like the Association for Accessible Medicines (AAM) and PhRMA are raising pointed questions about an FDA plan to create a new pathway for marketing prescription drugs with an additional condition for nonprescription use (ACNU), which would require more safeguards than the current OTC pathway but essentially carve out new OTC uses for some generic drugs.

Chief among the concerns were: Insurance companies dropping coverage for the Rx version, new ACNU patents to block competition, and industry essentially governing the pathway.

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Marc de Garidel, CinCor Pharma CEO (Eric Piermont/AFP via Getty Images)

Cin­Cor’s hy­per­ten­sion can­di­date flops a PhII test but plans to charge ahead with piv­otal study

Toward the end of summer, Massachusetts-based CinCor Pharma was riding high with a $225 million raise, touting a positive data readout. But a closer look under the hood has revealed a few hiccups in its Phase II.

CinCor on Monday unveiled topline results from its Phase II trial, dubbed HALO, evaluating baxdrostat in patients with uncontrolled hypertension who are taking up to two blood pressure medications. The trial whiffed on its primary endpoint of evaluating change from the “baseline in mean seated” systolic blood pressure (SBP) in the intent-to-treat population in all dosage levels.