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.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Martin Landray, Protas CEO (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Those big bil­lion-dol­lar PhI­II stud­ies? Mar­tin Lan­dray says they can be done for a tiny frac­tion of the cost

Martin Landray knows what controversy in clinical drug development feels like, from first-hand experience.

Landray was the chief architect of RECOVERY, a study that pitted a variety of drugs against Covid-19. And he offered some landmark data that would help push dexamethasone out into broader use as a cheap treatment, while helping ice hydroxy’s reputation as a clear misfire.

“Lots of people told us we shouldn’t use it,” Landray says about dexamethasone and Covid-19. “It was dangerous. We shouldn’t even do a trial. They also cared about hydroxychloroquine and lots of people said we shouldn’t do a trial because it must be used. I’ve got the letters from both sets of people.”

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FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs-up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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Geoffrey Porges, new Schrödinger CFO

Long­time an­a­lyst Ge­of­frey Porges de­parts SVB to lead fi­nances at a drug dis­cov­ery shop

Geoffrey Porges has ended his two-decade run as a biotech analyst, as the former SVB Securities vice chair began as CFO of Schrödinger on Thursday.

The long-running analyst, who previously headed up vaccines marketing at Merck before the turn of the millennium, will lead the financial operations of the 700-employee company as Schrödinger broadens its focus from a drug discovery partner to also building out an in-house pipeline, with clinical trial No. 1 set to begin next quarter.

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James Sabry, Roche global head of pharma partnering

Roche, Genen­tech plunk down $60M up­front to part­ner with Chi­nese phar­ma on PRO­TAC-based prostate can­cer drug

Roche and Genentech are always on the hunt for deals, and on Thursday they found their newest partner.

The pair will team up with the Chinese pharma company Jemincare to push forward a new program for prostate cancer, the companies announced. Roche is ponying up $60 million upfront to get its hands on the candidate and promising up to $590 million in biobucks, plus royalties, down the line.

In return, Genentech will get a worldwide license to develop the program, known as JMKX002992, and bring it to market.

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Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia ter­mi­nates Bay­er pact half a year ear­ly, col­lect­ing small por­tion of €240M promised

Bayer and Exscientia are winding down their three-year collaboration, leaving the big German pharma to take the AI-designed compounds born out of the pact further.

London-based Exscientia revealed in its Q2 update that the partners have “mutually agreed to end” their collaboration, which kicked off in early 2020, after recently achieving a drug discovery milestone. In an SEC filing, Exscientia said it terminated the pact on May 30, about six months early.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”