In­hi­brx shares rise on PhI tri­al re­sults; Bragli­a's Helsinn en­ters part­ner­ship with Fo­s­un

Cal­i­for­nia biotech In­hi­brx re­leased in­ter­im re­sults to­day from a Phase I tri­al look­ing at a re­com­bi­nant fu­sion pro­tein pro­gram, IN­BRX-101. The study looked at pa­tients with al­pha-1 an­tit­rypsin de­fi­cien­cy, or AATD.

The study re­vealed in­ter­im phar­ma­co­ki­net­ic da­ta from 21 pa­tients with AATD, all with the ZZ mu­ta­tion of the SER­PINA1 gene, and in­ter­im safe­ty da­ta are from 24 pa­tients with AATD. There were no drug-re­lat­ed se­vere or se­ri­ous ad­verse events at dos­es up to and in­clud­ing 120 mg/kg sin­gle dose, and 80mg/kg mul­ti-dose, ac­cord­ing to a com­pa­ny re­lease. Ad­verse events were pre­dom­i­nant­ly mild with a few mod­er­ate events.

More re­sults re­gard­ing pa­tients’ func­tion­al AAT and bron­choalve­o­lar lavage da­ta are ex­pect­ed in the first half of next year, look­ing at three IN­BRX-101 dos­es of 80 or 120 mg/kg IV ad­min­is­tered every three weeks.

Share prices $IN­BX went on a high af­ter the an­nounce­ment, in­creas­ing over 25% be­fore the mar­ket open and hov­er­ing up 20% to 25% af­ter the open­ing bell.

“These ini­tial da­ta demon­strate the po­ten­tial of IN­BRX-101 to sig­nif­i­cant­ly im­prove treat­ment for pa­tients with AATD by main­tain­ing them in the nor­mal range of func­tion­al AAT and re­duc­ing in­fu­sions from 52 an­nu­al­ly to pos­si­bly as few as 12 an­nu­al­ly,” said In­hi­brx CEO Mark Lappe.

Helsinn and Fo­s­un part­ner up for an­ti-nau­sea and vom­it­ing drugs

Swiss biotech Helsinn an­nounced to­day a new li­cens­ing and dis­tri­b­u­tion part­ner­ship with Shang­hai-based Fo­s­un, ef­fec­tive Oc­to­ber 1.

This part­ner­ship with Fo­s­un grants the Chi­nese biotech li­cense to com­mer­cial­ize and sell three Helsinn drugs in Chi­na, Hong Kong and Macau — an­ti-nau­sea and an­ti-vom­it­ing drugs Aloxi and Akynzeo, along with anorex­ia and ma­lig­nan­cy-as­so­ci­at­ed weight loss treat­ment anamore­lin for pa­tients with NSCLC.

Ad­di­tion­al­ly, Helsinn’s Chi­nese sub­sidiary will pro­vide a Fo­s­un sub­sidiary with cer­tain med­ical, mar­ket­ing and com­mer­cial ser­vices, in­clud­ing co-pro­mo­tion ser­vices in Shang­hai.

Wu Yi­fang, Chair­man and CEO of Fo­s­un Phar­ma, com­ment­ed: “We are look­ing for­ward to work­ing with Helsinn and to mak­ing these ther­a­pies avail­able to pa­tients in these ter­ri­to­ries as soon as pos­si­ble.”

Han­soh and OliX get to­geth­er on siR­NA de­vel­op­ment deal

In an­oth­er Asia-fo­cused col­lab­o­ra­tion, Chi­na’s Han­soh Phar­ma and South Ko­rea RNA biotech OliX Phar­ma­ceu­ti­cals are team­ing up in a new li­cens­ing and col­lab­o­ra­tion agree­ment.

The deal will be to dis­cov­er, de­vel­op and com­mer­cial­ize siR­NA ther­a­peu­tics for cer­tain in­di­ca­tions in Chi­na, Tai­wan, Hong Kong and Macau, ac­cord­ing to a com­pa­ny state­ment.

While OliX will use its plat­form to dis­cov­er and de­vel­op can­di­dates for tar­gets in car­dio­vas­cu­lar, meta­bol­ic, and oth­er dis­eases as­so­ci­at­ed with the liv­er, Han­soh will have ex­clu­sive com­mer­cial rights to these can­di­dates in the four Asian mar­kets.

OliX will re­ceive an ini­tial up­front pay­ment of $6.5 mil­lion, with up to $450 mil­lion in mile­stones plus roy­al­ties, along with rights to the ther­a­peu­tic can­di­dates in the rest of the world.

“We are thrilled to part­ner with OliX Phar­ma­ceu­ti­cals, a leader in siR­NA de­vel­op­ment with an es­tab­lished clin­i­cal stage port­fo­lio, and look for­ward to uti­liz­ing their tech­nol­o­gy plat­form in our de­vel­op­ment process,” said Han­soh board ex­ec­u­tive di­rec­tor Eliza Sun.

Roche’s Spark to li­cense Com­bi­Gene’s epilep­sy gene ther­a­py treat­ment

In yet an­oth­er col­lab­o­ra­tion an­nounced to­day, Com­bi­Gene and Spark signed an agree­ment to ex­clu­sive­ly col­lab­o­rate on the for­mer’s fo­cal epilep­sy gene ther­a­py — with a $300M+ li­cens­ing deal to boot.

The agree­ment gives Roche-owned Spark the world­wide li­cense to de­vel­op, man­u­fac­ture and com­mer­cial­ize CG01 while Com­bi­Gene con­tin­ues parts of the pre­clin­i­cal pro­gram in tan­dem with the com­pa­ny.

Com­bi­Gene will get $8.5 mil­lion up­front from Spark as part of the arrange­ment — with an ad­di­tion­al $50 mil­lion in mile­stones and a po­ten­tial grand to­tal of $328.5 mil­lion. And that does not in­clude roy­al­ties, ei­ther.

Once the gene ther­a­py is com­mer­cial­ized, Com­bi­Gene is el­i­gi­ble for tiered roy­al­ties based on net sales.

“We look for­ward to ad­vanc­ing this po­ten­tial­ly trans­for­ma­tive ther­a­py to­geth­er with Spark for the ben­e­fit of a pa­tient group in need of bet­ter treat­ments,” said Com­bi­Gene CEO Jan Nils­son.

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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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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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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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.

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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