Em­bat­tled AM­AG braces for Oc­to­ber FDA Mak­e­na ad­com, but it need not wor­ry — an­a­lyst

When the FDA ap­proved AM­AG Phar­ma­ceu­ti­cals’ fe­male li­bido drug, Vyleesi, months ago sans an ad­vi­so­ry com­mit­tee de­spite weak da­ta — con­tro­ver­sy en­sued. But the Mass­a­chu­setts-based drug­mak­er must now con­tend with the reg­u­la­to­ry fall­out of an­oth­er one of its women’s health drugs: Mak­e­na.

Mak­e­na — which has been on the mar­ket for years as a ther­a­py de­signed to cut the risk of preterm birth in preg­nant women — ear­li­er this year failed a post-con­fir­ma­to­ry study. Now, the FDA has set up a meet­ing of in­de­pen­dent ad­vi­sors in Oc­to­ber to fig­ure out whether or not to take it off the shelves.

For long the ther­a­py, a man-made ver­sion of the hor­mone prog­es­terone, was used off-la­bel sourced from un­reg­u­lat­ed con­coc­tions pre­pared by com­pound­ing phar­ma­cies. Even­tu­al­ly, the FDA grant­ed the prod­uct ac­cel­er­at­ed ap­proval in 2011, de­spite lim­it­ed clin­i­cal da­ta. The man­u­fac­tur­er was ac­quired by AM­AG in 2014. Last year, a pre­filled au­to-in­jec­tor ver­sion with a thin­ner, al­most in­vis­i­ble, nee­dle (ver­sus the orig­i­nal bulky in­tra­mus­cu­lar Mak­e­na in­jec­tion) was en­dorsed by the US reg­u­la­tor.

In March, AM­AG $AM­AG re­vealed the ther­a­py had fiz­zled in the 1,700-pa­tient post-ap­proval PRO­LONG study — blam­ing the fail­ure on the fact that 75% of the pa­tients en­rolled were not from the Unit­ed States, sug­gest­ing that the dif­fer­ence in de­mo­graph­ics had played a role in the set­back.

Back when the FDA orig­i­nal­ly ap­proved Mak­e­na, the agency was “pre­sent­ed with a very dif­fi­cult choice: ac­cept fair­ly lim­it­ed clin­i­cal da­ta (al­beit via spon­sor­ship from the NIH) or con­tin­ue to al­low preg­nant women to re­ceive an in­jec­tion from un­reg­u­lat­ed sources. The Agency clear­ly ac­cept­ed the lim­it­ed clin­i­cal da­ta and grant­ed ap­proval for Mak­e­na as we be­lieve (and spec­u­late) that the true goal was to bring the man­u­fac­tur­ing un­der FDA com­pli­ance,” Cowen’s Ken Cac­cia­tore wrote in a note.

“For these rea­sons, we do not be­lieve that the Agency would now rel­e­gate clin­i­cians and pa­tients to once again sourc­ing this crit­i­cal care prod­uct from un­ap­proved man­u­fac­tur­ing sources.”

As part of its sec­ond-quar­ter re­sults un­veiled in Au­gust, AM­AG said that gener­ic com­pe­ti­tion for Mak­e­na was eat­ing in­to sales, that it had faced sup­ply con­straints re­lat­ing to the prod­uct, that it had de­cid­ed to ex­it the in­tra­mus­cu­lar Mak­e­na mar­ket, and that it had ter­mi­nat­ed an arrange­ment with Pras­co, its au­tho­rized gener­ic part­ner.

Mean­while, AM­AG is bat­tling on oth­er fronts. On Mon­day, the com­pa­ny put out a state­ment ad­mon­ish­ing the ac­tivist hedge fund Cali­gan Part­ners, which has been ac­cu­mu­lat­ing its shares.

“(L)ast month Cali­gan ini­ti­at­ed what ap­pears to be a rushed, ag­gres­sive and mis­lead­ing scheme to seize near con­trol of your AM­AG Board. Now you are es­sen­tial­ly be­ing asked by Cali­gan — as part of a rarely used cor­po­rate ac­tion called a con­sent so­lic­i­ta­tion — to re­move and re­place four mem­bers of the re­cent­ly-re­elect­ed Board as a pre­cur­sor to risky and ill-in­formed changes that Cali­gan wants to make at AM­AG,” the com­pa­ny wrote in a let­ter to share­hold­ers, urg­ing them to dis­re­gard Cali­gan’s cam­paign.

Last month, the com­pa­ny low­ered its full-year fi­nan­cial guid­ance af­ter ex­it­ing the Mak­e­na in­tra­mus­cu­lar mar­ket and re­vis­ing its ex­pec­ta­tions of cira­paran­tag de­vel­op­ment mile­stone rev­enue. Now, the drug­mak­er ex­pects $325 – $355 mil­lion in rev­enue, ver­sus its pre­vi­ous range of $365 – $415 mil­lion.

So­cial im­age: Wes­t­i­form

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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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.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

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.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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