Roberto Bellini, Bellus Health CEO

UP­DAT­ED: Bel­lus says its Mer­ck cough ri­val passed a PhI­Ib test af­ter ear­li­er flop, but in­vestors can't make up their minds

A lit­tle over a year af­ter flop­ping out of the chron­ic cough game and leav­ing Mer­ck as the sole play­er, Bel­lus Health is say­ing it’s back in con­tention.

The Cana­di­an biotech tout­ed a Phase IIb in­ter­im analy­sis Mon­day morn­ing it de­scribed as pos­i­tive, say­ing that “at least one dose” of its ex­per­i­men­tal chron­ic cough drug post­ed a clin­i­cal­ly mean­ing­ful re­duc­tion in place­bo-ad­just­ed 24-hour cough fre­quen­cy af­ter four weeks. It’s a re­sult that comes rough­ly 15 months af­ter an­oth­er Phase II tri­al re­port­ed a miss on all four dos­es, send­ing shares $BLU down more than 75% at the time.

Bel­lus, how­ev­er, re­port­ed ex­act­ly ze­ro da­ta, and the press re­lease an­nounc­ing the re­sults was vague. The biotech said on­ly that an in­de­pen­dent team re­port­ed the re­sults met a “pre­de­fined strin­gent prob­a­bil­i­ty thresh­old for clin­i­cal ef­fi­ca­cy.”

“We be­lieve the en­cour­ag­ing SOOTHE Phase IIb tri­al in­ter­im analy­sis will en­able us to ac­cel­er­ate the plan­ning for our Phase III pro­gram while await­ing SOOTHE fi­nal re­sults,” CEO Rober­to Belli­ni said in a state­ment. “With tri­al en­roll­ment pro­gress­ing as planned, we an­tic­i­pate an­nounc­ing topline da­ta in the fourth quar­ter of 2021.”

It wasn’t im­me­di­ate­ly clear when Bel­lus would launch the Phase III, or which of the dos­ing lev­els passed the Phase IIb test. In an emailed state­ment to End­points News, the com­pa­ny said it has not put out guid­ance for the Phase III study and will not be dis­clos­ing the dos­ing lev­el that hit the pri­ma­ry un­til the topline da­ta are re­leased.

“All we can dis­close at this time is the fact that at least one dose (and up to 3 dos­es) has met” the ap­pro­pri­ate re­duc­tion lev­els, a spokesper­son said in an email.

In­vestors, though, ap­peared to cheer the news — at least ear­ly on. In the im­me­di­ate wake of the an­nounce­ment Bel­lus stock moved up more than 35% but lev­eled off to flat af­ter Mon­day’s open­ing bell.

Bel­lus’ new da­ta come from a study that looked at a dif­fer­ent pa­tient pop­u­la­tion than its pre­vi­ous fail­ure. The tri­al looked at pa­tients with at least 25 coughs per hour, a cri­te­ri­on that was not in­clud­ed in the old­er study but was an area where some da­ta had sug­gest­ed the pro­gram might be more ef­fec­tive.

Re­searchers re­cruit­ed 300 pa­tients and ran­dom­ized them in­to three treat­ment arms — Bel­lus dropped the third-high­est dose, out of the orig­i­nal four — and a place­bo group, mak­ing this study about five times larg­er than the last. The com­pa­ny al­so made sure to spread out the num­ber of se­vere cough pa­tients, or those de­fined as ex­pe­ri­enc­ing at least 45 coughs per hour, even­ly across the four groups.

In­vestors will have a bet­ter idea of just how good the new da­ta are once Bel­lus reads out topline da­ta lat­er this year, but re­sults from a sub­group in the old­er tri­al may give some in­sight. Look­ing at 31 pa­tients with a cough fre­quen­cy at or above the me­di­an of 32.4 coughs per hour, the Bel­lus pro­gram re­duced awake cough fre­quen­cy by 28% to 32% across all four dos­es.

Though the end­points were slight­ly dif­fer­ent, fig­ures any­where close to that in the new study would come in high­er than Mer­ck’s gefapix­ant pro­gram, which is cur­rent­ly un­der FDA re­view. Cross-tri­al com­par­isons are al­ways a risky busi­ness, but Mer­ck reg­is­tered on­ly an 18.5% es­ti­mat­ed rel­a­tive risk re­duc­tion in 24-hour cough fre­quen­cy with a p-val­ue com­ing in at p=0.041.

This wouldn’t prove such a high bar for Bel­lus to cross. It’s al­so worth not­ing that the two pro­grams are the same class of drug — P2X3 re­cep­tor an­tag­o­nists — and Mer­ck’s re­sults came from a 45-mg dose. The class has been dinged for af­fect­ing a pa­tient’s abil­i­ty to taste, with more than half of Mer­ck’s high-dose pa­tients re­port­ing taste-re­lat­ed ad­verse events.

Bel­lus, mean­while, test­ed dos­es of 12.5 mg, 50 mg and 200 mg in the tri­al. An­a­lysts will like­ly be watch­ing close­ly for the taste-re­lat­ed side ef­fects here, de­pend­ing on which dose or dos­es passed the new Phase IIb pri­ma­ry. The biotech said on­ly that such events were “lim­it­ed” and “con­sis­tent” with what it’s seen in pre­vi­ous stud­ies.

Bel­lus shares have yet to re­cov­er from the 2020 flop as the biotech has hov­ered in pen­ny stock ter­ri­to­ry since peak­ing around $12 last year.

This ar­ti­cle has been up­dat­ed with com­ment from Bel­lus.

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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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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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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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