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.

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Habib Dable, Acceleron CEO

Days of heat­ed ru­mors cul­mi­nate in a re­port that Ac­celeron is in ad­vanced buy­out talks

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

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Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Merck CEO Rob Davis

Mer­ck emerges as lead bid­der in po­ten­tial Ac­celeron buy­out with deal pos­si­ble this week — re­port

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Alexander Lefterov/Endpoints News

The coro­n­avirus vac­cine that the world for­got could still help save it

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

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Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi calls it quits on mR­NA Covid-19 shots, scrap­ping vac­cine from $3.2B Trans­late Bio buy­out

Sanofi is throwing in the towel on mRNA-based Covid-19 vaccines.

The French drugmaker will halt development on its unmodified mRNA Covid-19 shot despite what it said were positive Phase I/II results, a spokesperson told Endpoints News on Tuesday morning. Sanofi said the reason it’s stopping the Covid-19 mRNA program, developed in partnership with its new $3.2 billion acquisition Translate Bio, is because the market is too crowded.

Brian Hubbard, Anji Pharmacetuticals CEO

Look­ing to rewrite the rules of drug li­cens­ing, start­up An­ji is on the hunt for 'dy­nam­ic eq­ui­ty' joint ven­tures

Licensing is one of the most common ways big drugmakers leverage biotech innovation to drive gains across their pipelines — and the structure of those deals is pretty well established. But one biotech with home bases in China and the US thinks it may have a better way.

On Tuesday, Cambridge-based biotech Anji Pharma closed a $70 million Series B with two late-stage molecules in the fold and a mission to rewrite the rules of drug licensing through what it calls “dynamic equity” deals and a joint venture-heavy game plan. The round was funded in whole by Chinese hedge fund CR Capital.

Con­tract re­search is hav­ing a mo­ment right now. Will M&A splash­es dri­ve the in­dus­try to even greater heights?

Contract research organizations are a fairly mysterious bunch. They’re typically considered the skilled laborers behind big drug development — the stage crews who run the trials behind some of the most (and least) successful data reveals in biopharma history.

But all that is changing.

This year, a couple of huge, out-of-the-blue M&A deals sounded the alarm on just how much money is flying around in this corner of the industry.

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Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

Covid-19 roundup: Pfiz­er/BioN­Tech sub­mit vac­cine da­ta to FDA for younger chil­dren; Doc­tors kept pre­scrib­ing hy­drox­y­chloro­quine

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.