An­oth­er blow to NASH, dis­ap­point­ing tri­al re­sults spell end for dark­horse play­er Al­bireo

An­oth­er NASH pro­gram is head­ed to the scrap heap.

Al­bireo $AL­BO, a Boston biotech that took an un­con­ven­tion­al route to treat­ing the fat­ty liv­er dis­ease, an­nounced yes­ter­day that they were end­ing NASH de­vel­op­ment af­ter they re­ceived dis­ap­point­ing re­sults from a Phase II study. Just last week CEO Ron Coop­er, on their Q2 call, talked up their off­beat ap­proach and the po­ten­tial for their lead mol­e­cule to be used in com­bi­na­tion with agents now in de­vel­op­ment at oth­er coun­tries.

Ron Coop­er

On Tues­day, he em­pha­sized that Al­bireo’s in­vest­ment in the pro­gram was min­i­mal.

“We want­ed to in­ves­ti­gate the po­ten­tial of elo­bix­i­bat in NASH and al­lo­cat­ed min­i­mal re­sources to an ex­plorato­ry Phase 2 study,” Coop­er said in a state­ment. “Based on the re­sults of this study, we have made the de­ci­sion not to pur­sue fur­ther de­vel­op­ment of elo­bix­i­bat in NASH.”

Com­pared to the stock-melt­ing hits com­pa­nies such as Gen­Fit have tak­en af­ter their tri­al fail­ures, Al­bireo emerges from their NASH ex­plo­ration rel­a­tive­ly un­scathed. Their lead pro­grams are in oth­er liv­er con­di­tions, bile atre­sia and PF­IC, and the com­pa­ny took on­ly a 4.85% hit on the news, drop­ping from $27.01 to $25.70.

Still, the failed pro­gram is an­oth­er blow to a field that has seen lit­tle but fail­ure since JP Mor­gan 2019, when mul­ti­ple com­pa­nies seemed poised to break in­to a mar­ket es­ti­mat­ed to have mil­lions of pa­tients and room for mul­ti­ple mega-block­busters. Since then Gilead, Gen­Fit and a se­ries of small­er biotechs have seen ma­jor tri­al fail­ures. And, af­ter the FDA sur­pris­ing­ly re­ject­ed an In­ter­cept drug that showed mixed re­sults in Phase III, de­vel­op­ers have been left in lim­bo about what the bar for ef­fi­ca­cy will be.

Odd­ly enough, Al­bireo ac­tu­al­ly hit the pri­ma­ry end­point on their study. Coop­er in­sist­ed that he saw the dis­ease, large­ly dri­ven by obe­si­ty and sim­i­lar health fac­tors, as fun­da­men­tal­ly a car­dio­vas­cu­lar ail­ment.

So un­like In­ter­cept, Gen­fit and the rest of the biotechs that fo­cused on fi­bro­sis and NASH res­o­lu­tion, Al­bireo set up a tri­al de­signed to prove their lead drug, elo­bix­i­bat, could im­prove pa­tients’ cho­les­terol and show “proof-of-con­cept” on liv­er-spe­cif­ic end­points, such as liv­er fat and func­tion. If so, it could be used in tan­dem with oth­er fu­ture NASH drugs, as re­searchers in­creas­ing­ly turn to com­bi­na­tion ther­a­pies for the dis­ease.

Ul­ti­mate­ly, though, elo­bix­i­bat could do on­ly the for­mer. In the 47-per­son study, pa­tients on the treat­ment arm saw their cho­les­terol fall al­most twice as much as pa­tients on the place­bo arm (-20.5 mg/dL vs -11.1 mg/dL), but there was lit­tle to no change in liv­er end­points. Liv­er fat fell by 2.6% for the treat­ment arm, as mea­sured by MRI, and there was “no mean­ing­ful change” in lev­els of ala­nine amino­trans­ferase, a liv­er en­zyme com­mon­ly used to mea­sure func­tion.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

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It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Volker Wagner (L) and Jeff Legos

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Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Dave Lennon, former president of Novartis Gene Therapies

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Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.