Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

In an­oth­er PhI­II, Al­bert Bourla con­tin­ues to build case for a once-flopped GHD drug

A growth hor­mone treat­ment that’s seen a tur­bu­lent path may fi­nal­ly be reach­ing the light at the end of the tun­nel.

Pfiz­er and Op­ko Health an­nounced Thurs­day morn­ing that their once-week­ly so­ma­tro­gon met its pri­ma­ry end­point in a Phase III study, beat­ing out Pfiz­er’s old dai­ly Genotropin (so­ma­t­ropin) ther­a­py in the per­cep­tion of treat­ment bur­den. The news fol­lows pos­i­tive Phase III da­ta from 2019 show­ing clin­i­cal ben­e­fits, as well as a pre­vi­ous flop near the end of 2016 mea­sur­ing the pro­gram against a sug­ar pill.

Aimed at treat­ing chil­dren with growth hor­mone de­fi­cien­cy old­er than 3 and younger than 18, so­ma­tro­gon im­proved the av­er­age score on a com­mon GHD test, known as the Life In­ter­fer­ence Ques­tion­naire, af­ter 12 weeks on each treat­ment. The 87 pa­tients were ran­dom­ized in­to two “se­quences” — one tak­ing Genotropin fol­lowed by so­ma­tro­gon and the oth­er tak­ing so­ma­tro­gon fol­lowed by Genotropin.

In the topline re­sults re­vealed Thurs­day, tak­ing so­ma­tro­gon re­sult­ed in an av­er­age score of 8.63 while Genotropin’s av­er­age was 24.13. The ques­tion­naire is a com­pos­ite score tak­ing in­to ac­count sev­er­al fac­tors such as ease of use, con­ve­nience and will­ing­ness to con­tin­ue treat­ment, among oth­ers. Like in golf, low­er scores are bet­ter.

The es­ti­mat­ed dif­fer­ence be­tween the two arms, neg­a­tive-15.49, reached a p-val­ue low­er than 0.0001. Sec­ondary end­points al­so showed that pa­tients on so­ma­tro­gon showed an over­all ben­e­fit in treat­ment ex­pe­ri­ence over Genotropin.

GHD ther­a­py has cen­tered around dai­ly in­jec­tions since their in­tro­duc­tion sev­er­al decades ago. Pfiz­er’s Genotropin it­self has been on the mar­ket for near­ly a quar­ter cen­tu­ry, and while it was once a fran­chise drug, biosim­i­lars have flood­ed the de­vel­op­ment space. The phar­ma com­pa­ny like­ly wants to stay ahead of the com­pe­ti­tion, hence the part­ner­ship with Op­ko.

As of late 2019, the leader in the space is Norditropin, a growth hor­mone from No­vo Nordisk that us­es the same ba­sic in­gre­di­ent as Genotropin. The Dan­ish com­pa­ny sells the treat­ment with a kid-friend­ly self-in­jectable pen.

It’s been a long road for so­ma­tro­gon, which in 2016 flopped a Phase III tri­al show­ing that the can­di­date did not show an im­prove­ment in change in trunk fat mass from base­line to 26 weeks. At the time, Op­ko in­sist­ed the re­sult was caused by “one or more out­liers” that af­fect­ed the out­come, and in 2017 fol­lowed up with an analy­sis say­ing they were the un­der­ly­ing rea­son for the fail.

Things be­gan look­ing up to­ward the end of 2019, when the com­pa­nies re­leased da­ta say­ing that so­ma­tro­gon showed a bet­ter in­crease in height ve­loc­i­ty than Genotropin. The tri­al arm showed a ve­loc­i­ty of 10.12 cm/year, edg­ing out the old­er drug’s 9.78 cm/year.

Pfiz­er orig­i­nal­ly part­nered with Op­ko in 2014 to the tune of $295 mil­lion up­front and up to $275 mil­lion more in reg­u­la­to­ry mile­stones.

Last month, Pfiz­er CEO Al­bert Bourla pinned peak sales es­ti­mates on each of the com­pa­ny’s late-stage pro­grams, a step most ex­ec­u­tive teams might be re­luc­tant to take. Though so­ma­tro­gon wasn’t list­ed among the top pro­ject­ed block­busters, Bourla pegged it for the $500 mil­lion to $1 bil­lion cat­e­go­ry.

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.

So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; 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.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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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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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 are 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 are 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 Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

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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