Den­mark's Gubra to col­lab­o­rate with Bay­er on pep­tides; Sam­sung and Bio­gen re­ceive FDA ap­proval for Lu­cen­tis biosim­i­lar

Dan­ish biotech Gubra an­nounced a re­search col­lab­o­ra­tion and li­cense agree­ment with Bay­er to de­vel­op pep­tide ther­a­peu­tics to treat car­diore­nal dis­eases. The col­lab­o­ra­tion will uti­lize Gubra’s pep­tide drug dis­cov­ery plat­form to iden­ti­fy po­ten­tial can­di­dates.

This is not the first time Gubra has part­nered with a com­pa­ny on pep­tide ther­a­peu­tics — they part­nered with Boehringer In­gel­heim back in 2017 to cre­ate pep­tide ther­a­peu­tics to treat obe­si­ty.

“We are very pleased to en­ter this re­search col­lab­o­ra­tion and li­cense agree­ment with Bay­er,” said Gubra CEO Hen­rik Blou in a state­ment. “We are pleased that the po­ten­tial of our tech­nol­o­gy is be­ing rec­og­nized by such a qual­i­fied part­ner ca­pa­ble of ad­vanc­ing in­no­v­a­tive med­i­cines in­to the clin­ic.”

As part of the agree­ment, Gubra will re­ceive an undis­closed pay­ment up­front, as well as up to $253 mil­lion for reach­ing de­vel­op­ment and com­mer­cial­iza­tion mile­stones.

Sam­sung and Bio­gen re­ceive FDA ap­proval for Lu­cen­tis biosim­i­lar

Sam­sung Bioepis and Bio­gen an­nounced to­day that the FDA has ap­proved Lu­cen­tis biosim­i­lar By­ooviz for three in­di­ca­tions:

  • Neo­vas­cu­lar (wet) age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD)
  • Mac­u­lar ede­ma fol­low­ing reti­nal vein oc­clu­sion (RVO)
  • My­opic choroidal neo­vas­cu­lar­iza­tion (mC­NV)

While By­ooviz is the first oph­thal­mol­o­gy biosim­i­lar ap­proved in the Unit­ed States, this ap­proval was like­ly a mat­ter of time as CHMP had rec­om­mend­ed the drug’s ap­proval back in June.

By­ooviz was ap­proved in Eu­rope on Au­gust 18 and in the UK on Au­gust 31.

“We are very ex­cit­ed to be able to open a new chap­ter with the ap­proval of By­ooviz in the US,” said Bio­gen SVP and glob­al head of biosim­i­lars Ian Hen­shaw in a state­ment.

How much will BY­OOVIZ cost? Bio­gen spokesper­son Al­li­son Parks de­clined to say.

“Be­cause we’re not launch­ing un­til June, it’s too pre­lim­i­nary to an­nounce pric­ing,” Parks told End­points News. “We will work in part­ner­ship with ex­ter­nal stake­hold­ers, in­clud­ing pay­ers and providers to en­sure a pre­dictable and sus­tain­able ac­cess po­si­tion is de­vel­oped for BY­OOVIZ.”

Syn­log­ic un­veils da­ta from mul­ti­ple clin­i­cal stud­ies on PKU

Mass­a­chu­setts biotech Syn­log­ic an­nounced pos­i­tive da­ta from clin­i­cal stud­ies look­ing at two en­gi­neered mi­crobes to treat phenylke­tonuria, or PKU — SYNB1618 and SYNB1934.

SYNB1618 showed re­duc­tions of pheny­lala­nine (Phe) at dif­fer­ent dose lev­els, across mul­ti­ple time points, in an in­ter­im analy­sis of a Phase II Syn­Phe­ny-1 study. SYNB1934, an op­ti­mized strain evolved from SYNB1618, showed two-fold high­er ac­tiv­i­ty than SYNB1618 in a head-to-head Phase I study in healthy vol­un­teers, as mea­sured by bio­mark­ers of Phe me­tab­o­lism.

Based on these clin­i­cal da­ta, Syn­log­ic will start plan­ning a piv­otal Phase III study.

“These events mark a ma­jor mile­stone for Syn­log­ic’s Syn­thet­ic Bi­ot­ic plat­form. We look for­ward to com­plet­ing our Phase 2 Syn­Phe­ny-1 study and ad­vanc­ing the PKU pro­gram in­to a piv­otal study,” said Syn­log­ic pres­i­dent and CEO Aoife Bren­nan in a state­ment.

Glax­o­SmithK­line to em­bark on clean en­er­gy in­vest­ment

As GSK CEO Em­ma Walm­s­ley par­tic­i­pat­ed at the open­ing event for New York Cli­mate Week, GSK an­nounced ma­jor up­dates on ef­forts to achieve cer­tain en­vi­ron­men­tal goals, in­clud­ing ma­jor new in­vest­ment in re­new­able elec­tric­i­ty at man­u­fac­tur­ing sites in the UK and US.

There is al­so a new ini­tia­tive to re­duce green­house gas emis­sions from its me­tered dose asth­ma in­halers, which ac­count for al­most half of the com­pa­ny’s car­bon emis­sions.

“For GSK, our sus­tain­abil­i­ty com­mit­ments are an in­te­gral part of our strat­e­gy, mak­ing our busi­ness more re­silient, pro­tect­ing our op­er­a­tions and ul­ti­mate­ly help­ing us to achieve health im­pact on a glob­al scale while de­liv­er­ing re­turns for our share­hold­ers,” said Walm­s­ley.

GSK said these in­vest­ments al­so co­in­cid­ed with con­fir­ma­tion of the glob­al phar­ma­ceu­ti­cal and med­ical tech­nol­o­gy sec­tor reach­ing the Race to Ze­ro ‘break­through’ tar­get of 20% of ma­jor com­pa­nies (by rev­enue) com­mit­ting to net ze­ro car­bon emis­sions by 2050.

FDA ac­cepts Mar­i­nus’s NDA on epilep­sy seizure treat­ment

Mar­i­nus Phar­ma­ceu­ti­cals an­nounced to­day that the FDA ac­cept­ed Mar­i­nus’ NDA for the use of ganax­olone in the treat­ment of seizures as­so­ci­at­ed with rare ge­net­ic epilep­sy CD­KL5 de­fi­cien­cy dis­or­der.

The NDA was grant­ed pri­or­i­ty re­view and the FDA as­signed a PDU­FA ac­tion date of March 20, 2022. In its ac­cep­tance let­ter, the FDA in­di­cat­ed that it is not cur­rent­ly plan­ning to hold an ad­vi­so­ry com­mit­tee meet­ing to dis­cuss the ap­pli­ca­tion.

“We be­lieve that ganax­olone has the po­ten­tial to pro­vide mean­ing­ful clin­i­cal ben­e­fit for pa­tients and we look for­ward to work­ing close­ly with the FDA dur­ing the re­view process,” said Mar­i­nus CEO Scott Braun­stein in a state­ment.

Ganax­olone re­ceived or­phan drug des­ig­na­tion and Rare Pe­di­atric Dis­ease (RPD) des­ig­na­tion for CDD in June 2017 and Ju­ly 2020, re­spec­tive­ly.

The ac­cep­tance of the NDA for fil­ing en­ables the biotech to draw $30 mil­lion un­der its May 2021 fi­nanc­ing agree­ment with in­vest­ment firm Oak­tree Cap­i­tal Man­age­ment, based on cer­tain con­di­tions de­scribed in the cred­it agree­ment. If the NDA is ap­proved by the end of next year, an ad­di­tion­al $30 mil­lion is avail­able un­der the agree­ment.

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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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.

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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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.