Drug de­vel­op­ers face in­creas­ing­ly risky tightrope walk be­tween top-line re­sults and hard da­ta

Even the biggest phar­ma com­pa­nies can hype the re­sults of a big drug study, giv­en the cu­ri­ous dance-of-the-sev­en-veils chore­og­ra­phy pro­vid­ed to late-stage da­ta. And the Wall Street Jour­nal is of­fer­ing up an ex­am­ple to un­der­score just how per­ilous this dance can be for all play­ers.

Case in point: Roche and Per­je­ta. The Swiss phar­ma gi­ant first of­fered a top-line glimpse of its pos­i­tive re­sults for the APHIN­I­TY study in­volv­ing Per­je­ta, but had to deal with some bit­ter re­ac­tions when an­a­lysts got a chance to ex­am­ine the mar­gin­al ben­e­fit that has been pro­vid­ed.

Some of this is the sim­ple re­sult of a sys­tem where drug de­vel­op­ers toss out top-line out­comes but re­serve the re­sults for a sci­en­tif­ic con­fer­ence.

The Jour­nal could have picked from any num­ber of cas­es, in­clud­ing the big Am­gen car­dio out­comes study on its PC­SK9 drug Repatha. The FOURI­ER study was built up as a big suc­cess, with Am­gen ex­ec­u­tives al­most in­stinc­tive­ly in­sist­ing that sta­tis­ti­cal sig­nif­i­cance would trans­late in­to mar­ket ac­cep­tance. But with no mor­tal­i­ty ben­e­fit in the mix, the whole per­for­mance came in look­ing weak and un­con­vinc­ing.

For the WSJ writ­ers, the push to tout re­sults is tied to some par­tic­u­lar risks for ex­ecs who sell shares be­tween the hype and the re­al­i­ty. But a big­ger les­son may be that in­creas­ing­ly we’re see­ing that suc­cess in a clin­i­cal tri­al is no guar­an­tee of a win on mar­ket gains. The bar on suc­cess can be much high­er than clin­i­cal­ly sta­tis­ti­cal suc­cess for pay­ers ex­pect­ed to cov­er these drugs.

One oth­er ob­ser­va­tion: If you’re go­ing to pre­dict that your drug can clear a spe­cif­ic hur­dle, that be­comes the bar you have to jump to win ap­plause. Iron­wood found that out the hard way re­cent­ly when its Phase IIb da­ta for a would-be block­buster failed to hit its own mark – even though the da­ta were sta­tis­ti­cal­ly sig­nif­i­cant.

That trend to con­tin­u­al­ly raise the bar – and risk the con­se­quences of falling short of ex­pec­ta­tions – is on­ly go­ing to get tougher for drug de­vel­op­ers.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Mer­ck makes a triple play on Covid-19: buy­ing out a vac­cine biotech, part­ner­ing on an­oth­er pro­gram and adding an an­tivi­ral to the mix

Merck is making a triple play in a sudden leap into the R&D campaign against Covid-19.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

The deal with IAVI covers recombinant vesicular stomatitis virus (rVSV) technology that is the basis for Merck’s successful Ebola Zaire virus vaccine. That’s going into the clinic later this year.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Piv­otal myas­the­nia gravis da­ta from ar­genx au­gur well for FcRn in­hibitors in de­vel­op­ment

Leading the pack of biotechs vying for a piece of the generalized myasthenia gravis (gMG) market with an FcRn inhibitor, argenx on Tuesday unveiled keenly anticipated positive late-stage data on its lead asset, bringing it one step closer to regulatory approval.

Despite steroids, immunosuppressants, acetylcholinesterase inhibitors, and Alexion’s Soliris, patients with the rare, chronic neuromuscular disorder (more than 100,000 in the United States and Europe) don’t necessarily benefit from these existing options, leaving room for the crop of FcRn inhibitors in development.

Af­ter de­cou­pling from Re­gen­eron, Sanofi says it’s time to sell the $13B stake picked up in the mar­riage

With Regeneron shares going for a peak price — after doubling from last fall — Sanofi is putting a $13 billion stake in their longtime partner on the auction block. And Regeneron is taking $5 billion of that action for themselves.

Sanofi — which has been decoupling from Regeneron for more than a year now — bought in big in early 2013, back when Regeneron’s stock was going for around $165 a share. Small investors flocked to the deal, buzzing about an imminent takeover. The buyout chatter wound down long ago.

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Andrew Hopkins, Exscientia founder and CEO (Exscientia)

Af­ter years of part­ner­ships, AI biotech Ex­sci­en­tia lands first ma­jor fi­nanc­ing round at $60M

After years racking up partnerships with biotechs and Big Pharma, the AI drug developer Exscientia has landed its first large financing round.

The UK-based company raised $60 million in a Series C round led by Novo Holdings — more than double the $26 million it garnered in a Series B 18 months ago. The round will help further the company’s expansion into the US and further what it calls, borrowing a term from the software world, its “full-stack capabilities,” i.e. its ability to develop drugs from the earliest stage to the market.

Covid-19 roundup: Janet Wood­cock steps aside — for now — as FDA drug czar; WHO hits the brakes on hy­droxy study af­ter lat­est safe­ty alarm

The biopharma industry will soon get a look at what the FDA will look like once CDER’s powerful chief Janet Woodcock retires from her post.

Long considered one of the most influential regulators in the agency, if not its single most powerful official when it counts, Woodcock is being detached to devote herself full-time to the White House’s special project to fast-forward new drugs and vaccines for the pandemic. The move comes a week after some quick reshuffling as Woodcock and CBER chief Peter Marks joined Operation Warp Speed. Initially they opted to recuse themselves from any FDA decisions on pandemic treatments and vaccines, after consumer advocates criticized the move as a clear conflict of interest in how the agency exercises oversight on new approvals.

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Janet Woodcock, director of the Center for Drug Evaluation and Research (AP Images)

Covid-19 roundup: Hit with new con­flict ac­cu­sa­tions, Janet Wood­cock steps out of the agen­cy's Covid-19 chain of com­mand

Two weeks ago, FDA drug chieftain Janet Woodcock was assuring a top Wall Street analyst that any vaccine approved for combating Covid-19 would have to meet high agency standards on safety and efficacy before it’s approved. But over the weekend, after she and Peter Marks took top positions with the public-private operation meant to speed a new vaccine to lightning-fast approvals — they both recused themselves from the review process after an advocacy group argued their roles close to the White House could pose a conflict of interest.

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An­oth­er NASH de­lay for In­ter­cept frus­trates in­vestors, shares wilt

A previous FDA advisory committee delay for Intercept’s NASH drug may have dampened spirits, but investors perked up after French rival Genfit recently failed to best a placebo with its offering in a keenly anticipated pivotal study. In yet another twist on Friday, the New York drugmaker said the FDA is postponing its adcom again to accommodate the review of additional data it has asked the company to furnish.

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Eric Edwards, Phlow president and CEO (PR Newswire)

BAR­DA of­fers a tiny start­up up to $812M to cre­ate a US-based drug man­u­fac­tur­er — and the CEO comes with a price goug­ing con­tro­ver­sy on his ré­sumé

BARDA has tapped a largely unknown startup to ramp up production of a list of drugs that may be at risk of running short in the US. And the deal, which comes with up to $812 million in federal funds, was inked by a CEO who found himself in the middle of an ugly price gouging controversy a few years ago.

The feds’ new partner — called Phlow — won a 4-year “base” contract of $354 million, with another $458 million that’s on the table in potential options to sustain the outfit. That would make it one of the largest awards in BARDA’s history.

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