As­cendis Phar­ma of­fers pos­i­tive sur­prise as its tech not just equals, but im­proves out­comes in key study

Tech­nol­o­gy de­vel­oped by Den­mark’s As­cendis Phar­ma to re­lease a par­tic­u­lar drug with­in the body in a pre­dictable man­ner at reg­u­lar in­ter­vals with­out the need for mul­ti­ple sep­a­rate ad­min­is­tra­tions has been shown to work well in a late-stage tri­al in pe­di­atric pa­tients with growth hor­mone de­fi­cien­cy — val­i­dat­ing the com­pa­ny’s plat­form that is used across its port­fo­lio.

The tech­nol­o­gy, called TransCon or tran­sient con­ju­ga­tion, com­pris­es an un­mod­i­fied ap­proved drug, an in­ert car­ri­er that pro­tects it, and a link­er that tem­porar­i­ly binds the two. When bound, the car­ri­er in­ac­ti­vates and shields the par­ent drug from be­ing re­leased, but once the prod­uct is in­ject­ed in­to the body, phys­i­o­log­ic pH and tem­per­a­ture con­di­tions al­low for the pre­dictable dis­sem­i­na­tion of the ac­tive drug — this can be done dai­ly or even half year­ly. The tech can be used for sys­temic ad­min­is­tra­tion or lo­cal­ly in ar­eas that are dif­fi­cult to treat with con­ven­tion­al ther­a­py.

The 161 treat­ment-naïve-pa­tient-tri­al, called heiGHt, com­pared the once-week­ly TransCon growth hor­mone (hGH) against a dai­ly growth hor­mone — Pfiz­er’s Genotropin — in chil­dren with growth hor­mone de­fi­cien­cy, an or­phan dis­ease caused by the in­suf­fi­cient pro­duc­tion of growth hor­mone by the pi­tu­itary gland that is char­ac­ter­ized by short stature, meta­bol­ic ab­nor­mal­i­ties, psy­choso­cial chal­lenges and cog­ni­tive gaps.

TransCon hGH is de­signed to de­liv­er un­mod­i­fied hGH — the same growth hor­mone used in dai­ly ther­a­pies — at a pre­dictable rate over one week. Cur­rent­ly in the Unit­ed States and Eu­rope, pa­tients must con­tend with dai­ly hGH in­jec­tions re­sult­ing in thou­sands of in­jec­tions over time, which of­ten leads to poor pa­tient com­pli­ance, the com­pa­ny said.

Tri­al da­ta showed that As­cendis’ prod­uct not on­ly met the main goal of non-in­fe­ri­or­i­ty in an­nu­al­ized height ve­loc­i­ty (a mea­sure of speed of growth) at 52 weeks, but ac­tu­al­ly proved su­pe­ri­or to dai­ly hGH. These re­sults could con­sti­tute a home run, con­sid­er­ing var­i­ous an­a­lysts were ex­pect­ing just the non-in­fe­ri­or­i­ty bench­mark to be met.

Joseph Schwartz

In the pri­ma­ry analy­sis of the in­tent-to-treat pop­u­la­tion, TransCon hGH demon­strat­ed a sig­nif­i­cant­ly greater an­nu­al­ized height ve­loc­i­ty of 11.2 cm/year com­pared to 10.3 cm/year for the dai­ly hGH — the treat­ment dif­fer­ence amount­ed to 0.86 cm/year, hit­ting a p-val­ue of 0.0088. The ex­per­i­men­tal treat­ment be­gan to sur­pass the com­para­tor at a sta­tis­ti­cal­ly sig­nif­i­cant rate from week 26 on­ward.

As­cendis’ shares $AS­ND sky­rock­et­ed more than 64.6% in morn­ing trad­ing to $114.10.

“The stock is not cheap, but we do see room for sig­nif­i­cant up­side up­on pos­i­tive piv­otal da­ta that may be used for reg­is­tra­tion of the first long-act­ing growth hor­mone treat­ment, which has been a prized goal for many com­pa­nies over sev­er­al decades,” Leerink’s Joseph Schwartz wrote in a note in late Jan­u­ary.

The com­pa­ny, which has part­nered with Roche’s Genen­tech and Sanofi to eval­u­ate its tech­nol­o­gy for oph­thal­mol­o­gy and di­a­betes drugs re­spec­tive­ly, plans to sub­mit a mar­ket­ing ap­pli­ca­tion for its pe­di­atric GHD prod­uct in the first half of 2020.

“As­cendis’ man­age­ment con­tin­ues to de­liv­er with this best-case out­come for TransCon hGH in a Ph3 tri­al, fur­ther val­i­dat­ing the tech­nol­o­gy plat­form and with pos­i­tive read-through to oth­er clin­i­cal pro­grams and pipeline po­ten­tial. We al­so see the pos­i­tive Ph3 rais­ing the com­pa­ny’s pro­file as a po­ten­tial ac­qui­si­tion tar­get, as As­cendis of­fers bal­anced clin­i­cal pipeline with fair­ly de-risked pro­grams, po­ten­tial best-in-class as­sets, an emerg­ing new ther­a­peu­tics ver­ti­cal and plen­ty of op­tion­al­i­ty with­in the TransCon tech­nol­o­gy plat­form,” Cred­it Su­isse an­a­lysts wrote in a note.

Top-line da­ta for the fliGHt Tri­al, eval­u­at­ing TransCon hGH in sub­jects who switch from dai­ly hGH, are ex­pect­ed in the sec­ond quar­ter. The com­pa­ny is al­so eval­u­at­ing its tech­nol­o­gy for use in hy­poparathy­roidism and achon­dropla­sia.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.