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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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Judge al­lows ex­pert tes­ti­mo­ny in GSK tri­al al­leg­ing Zan­tac link to can­cer

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.