Ernest Loumaye, ObsEva CEO (ObsEva)

Ob­sE­va’s sec­ond uter­ine fi­broids PhI­II comes through, send­ing some in­vestors to the hills

In the three-com­pa­ny race to de­vel­op a new uter­ine fi­broid treat­ment, Ob­sE­va long lagged be­hind Ab­b­Vie and My­ovant. Still, they hoped that bet­ter ef­fi­ca­cy — in­clud­ing a 93.9% re­sponse rate in one Phase III tri­al — could ul­ti­mate­ly de­liv­er bet­ter sales.

Now, though, the sec­ond of those Phase III stud­ies is out, and it has brought the Swedish biotech back to earth.

In the sec­ond of their Phase III tri­als, 75.5% of pa­tients who took Ob­sE­va’s ex­per­i­men­tal tablet lin­zagolix plus a hor­mone saw their bleed­ing re­duced by at least 50% and at least 80 ml af­ter 24 weeks. Pool­ing those re­sults with new da­ta from their first Phase III tri­al — which showed a 93.9% re­sponse rate at 24 weeks and a 91.6% re­sponse rate at 52 weeks — Ob­sE­va cal­cu­lat­ed a col­lec­tive 84.7% re­spon­der rate for pa­tients tak­ing their ther­a­py and said the da­ta “con­firm po­ten­tial best-in-class” sta­tus.

“These ex­cel­lent da­ta move us clos­er to the po­ten­tial com­mer­cial­iza­tion of Ysel­ty and our im­me­di­ate pri­or­i­ty is to progress our reg­u­la­to­ry fil­ings,” Ernest Loumaye, Ob­sE­va CEO and co-founder said in a state­ment, us­ing the brand­ed name for his drug.

Yet some in­vestors and an­a­lysts were dis­ap­point­ed, not­ing that these new re­sults erase the edge that the first tri­al seemed to give Ob­sE­va. 75.5% falls right in line with the re­sults from Ab­b­Vie and My­ovant. Ab­b­vie had a 68.5% re­sponse rate in one Phase III tri­al and a 76.2% re­sponse rate in the sec­ond. My­ovant saw re­sponse rates of 71.2% and 73.4%.

In fact, SVB Leerink’s Ami Fa­dia wrote in a note to in­vestors that be­cause Ob­sE­va had high place­bo re­sponse in both tri­als. the com­pa­ny now has the worst place­bo-ad­just­ed re­sponse of any of the three.

“We be­lieve the place­bo-ad­just­ed re­sponse rate will be a key met­ric that in­vestors pay at­ten­tion to, and the PRIM­ROSE1 re­sults on that mea­sure like­ly fell short of ex­pec­ta­tions,” Fa­dia wrote, not­ing their own Ob­sE­va mod­el is now un­der re­view.

The mar­ket agreed, cut­ting Ob­sE­va’s stock $OB­SV by 40%, or $2.75, Mon­day morn­ing.

Even be­fore the new tri­al, an­a­lysts had ex­pressed reser­va­tions about Ob­sE­va’s ap­par­ent ef­fi­ca­cy. In De­cem­ber, Baird’s Bri­an Sko­r­ney chalked up their high score to a 29.4% place­bo re­sponse rate, far high­er than those seen in their two com­peti­tors’ tri­als. “We think the re­sound­ing take­away here is that the ef­fi­ca­cy pro­files of these 3 … in­hibitors are es­sen­tial­ly the same,” he wrote in a note to in­vestors.

If these drugs are ef­fec­tive­ly the same, Sko­r­ney bet that the first ones out of the gate will es­tab­lish a beach-head and gar­ner the most sales. That would spell bad news for Ob­sE­va. Tim­ing-wise, they now sit in third place. Ab­b­Vie’s drug has al­ready been ap­proved and My­ovant sub­mit­ted an NDA in June, while Ob­sE­va has yet to file an ap­pli­ca­tion. Known as GnRH in­hibitors, these drugs block a hor­mone re­cep­tor in the body.

Some an­a­lysts, though, bet that de­spite some ini­tial con­cerns, safe­ty could ul­ti­mate­ly be Ob­sE­va’s ad­van­tage. When the FDA ap­proved Ab­b­Vie’s drug, Ori­ahnn, they al­so put strict lim­its on its use af­ter mul­ti­ple pa­tients in their tri­al had throm­bot­ic events, in­clud­ing a pul­monary em­bolism. These like­ly came from ex­tra hor­mone treat­ments, known as add-back ther­a­py, pa­tients take to stop the menopause-like symp­toms GnRH in­hibitors can cause.

That’s part­ly why Ob­sE­va in­clud­ed an arm on both tri­als where pa­tients took a small­er dose of the drug and didn’t re­ceive the add-back ther­a­py. In the com­pa­ny’s first tri­al, a lit­tle over half of those pa­tients saw their bleed­ing cut in half. That could make it a good can­di­date for pa­tients with oth­er co-mor­bidi­ties, Fa­dia and oth­er an­a­lysts wrote. The new study showed a 56.6% rate of pa­tients on the no-add-back treat­ment arm, but it al­so had a 35% rate in place­bo, cut­ting the place­bo-ad­just­ed rate to just 21.4%. With new 52-week da­ta out, there were al­so ques­tions about how long the ther­a­py would last.

“We still view the pro­file of both the low dose and the high dose as ap­prov­able,” Fa­dia wrote, “al­though we see po­ten­tial ques­tions com­ing up re­gard­ing the treat­ment du­ra­tion on the la­bel and the com­mer­cial vi­a­bil­i­ty for the low dose.”

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Pascal Soriot, AstraZeneca CEO (via Getty images)

UP­DAT­ED: FDA slaps As­traZeneca's MCL-1 can­cer drug with a hold af­ter safe­ty is­sue — 2 years af­ter Am­gen axed a trou­bled ri­val

There are new questions being posed about a class of cancer drugs in the wake of the second FDA-enforced clinical hold in the field.

Two years after the FDA hit Amgen with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity, AstraZeneca says that regulators hit them with a hold on their rival therapy of the same class.

The pharma giant noted on clinicaltrials.gov that its Phase I/II study for the MCL-1 drug AZD5991 “has been put on hold to allow further evaluation of safety related information.”

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Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.