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.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Covid-19 roundup: WSJ of­fers in­side look at Ox­ford-As­traZeneca deal, in­clud­ing fi­nan­cial terms; Lil­ly plant un­der scruti­ny again

Oxford scientists developing a Covid-19 vaccine are working with British drugmaker AstraZeneca, and together the pair have become one of the frontrunners in the race to end the pandemic. But a new Wall Street Journal report out Wednesday offered a behind-the-scenes look at how that deal came together in the wake of a scholar-led revolt over a potential collaboration with Merck, and included previously unreported financial terms of the AstraZeneca deal.

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Jean-Pierre Sommadossi, Atea president and CEO (file photo)

Roche wades deep­er in­to Covid-19 fight, ink­ing an­tivi­ral pact with $350M cash fol­low­ing Re­gen­eron deal

Roche is making its first bet on an antiviral against Covid-19 in style, shelling out $350 million in cash to grab ex-US rights.

The drug comes from Atea Pharmaceuticals, the 7-year-old biotech created by Pharmasset co-founder Jean-Pierre Sommadossi, which essentially rebranded itself as a Covid-19 fighter in May when it closed a whopping $215 million venture round. Over a dozen investors bought in, including marquee names like Bain Capital and RA Capital.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Peter Kolchinsky and Raj Shah (file photo)

Pe­ter Kolchin­sky and Raj Shah's RA Cap­i­tal has $461M more to play with, af­ter 'rapid' in­vest­ment in the last 15 months

Just over 15 months after launching its first venture fund, RA Capital Management is ready for more. And this time the firm is bringing an even bigger load of cash to the table.

Announcing the close of its Nexus II fund on Wednesday, RA said it raised $461 million for investments in private companies across the biotech industry. The first venture fund, which raised $300 million, has churned through roughly 80% of its capital already, a pace that managing partner Raj Shah called unusually quick.

RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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