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.

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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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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The Avance 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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