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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Spe­cial re­port 2022: Meet 20 women blaz­ing trails in bio­phar­ma R&D

When you run a special report for a fourth year, it can start feeling a little bit like a ritual. You go through the motions — in our case opening up nominations for top women in biopharma R&D and reviewing more than 500 entries — you make your choices of inclusion and exclusion. You host a ceremony.

But then things happen that remind you why you do it in the first place. Perhaps a Supreme Court rules to overturn the constitutional right to abortion and a group of women biotech leaders makes it clear they strongly dissent; perhaps new data on gender diversity in the industry come out that look all too similar to the old ones, suggesting women are still dramatically underrepresented at the top; perhaps protests and conflicts around the world put in stark terms the struggles that many women still face in earning the most basic recognition.

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Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Prometheus moves to raise cash hours af­ter PhII da­ta leads to stock surge

After releasing better-than-anticipated data on two mid-stage studies Wednesday morning, Prometheus Biosciences’ CEO said the company would “take some time to assess” its next financing options.

It only needed about seven hours. Wednesday afternoon after the market closed, the biotech announced it would seek $250 million through an equity offering as the company looks to edge out anti-TL1A competitor Pfizer and its new partner Roivant.

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Piper Trelstad, head of CMC, Bill & Melinda Gates Medical Research Institute

Q&A with Gates leader: Women tak­ing on more roles in phar­ma man­u­fac­tur­ing, but still work to do

More and more women are driving innovation and taking leadership roles in biotech – as evidenced today in the release of Endpoints News’ list of the top 20 women in the R&D world – but those gains are beginning to extend across pharma sectors.

In pharma manufacturing in the US today, around 46% of all roles are occupied by women, according to the US Bureau of Labor Statistics for 2021. And according to a Bloomberg report, women’s roles across manufacturing roles had a massive boost after the start of the pandemic.

Phar­ma rep­u­ta­tion re­tains 'halo' even as pan­dem­ic me­dia cov­er­age re­cedes — sur­vey

The Covid-19 halo effect on the pharma industry is continuing, according to a new global study from Ipsos. The annual survey for the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) finds considerable goodwill from consumers across measures of trust, cooperation with governments, and advancing research and drug development.

“Despite the pandemic in many countries no longer being the top of mind concern generally – although it does remain the top concern as a health issue – the industry’s reputation has remained positive,” said Ipsos research director Thomas Fife-Schaw.

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FDA commissioner Robert Califf (Jose Luis Magana/AP Images)

FDA pulls On­copep­tides' Pepax­to in­di­ca­tion, open­ing the door for dan­gling ac­cel­er­at­ed ap­proval en­force­ment

In a move all but ensured after an overwhelmingly negative adcomm vote this September, the FDA is yanking Oncopeptides’ dangling accelerated approval. And there may be more to come.

In recent months, US regulators have honed in on reforming the accelerated approval pathway and preventing drugmakers from continuing to sell their medicines in the event of a confirmatory study flop. The moves come after commissioner Rob Califf has called for companies to do more to produce post-marketing evidence quickly earlier this year.

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Mark McKenna, Prometheus Biosciences chairman & CEO

With clear PhII win in IBD, Prometheus thwarts Pfiz­er com­par­isons as it fol­lows Hu­mi­ra 'play­book'

Prometheus Biosciences reported a clear Phase II win in two inflammatory bowel disease conditions in a clinical development race with Pfizer, planting the biotech’s flag in a field of antibodies attempting to go against black box-cornered JAK inhibitors and AbbVie’s Humira.

Shares $RXDX have soared since the summer — a small dip last week notwithstanding when rival Pfizer teamed up with Roivant on a new company for their competing anti-TL1A monoclonal antibody. And they skyrocketed once again Wednesday morning, climbing from $36 apiece to more than $100 on the back of two Phase II studies: one placebo-controlled in ulcerative colitis and the other an open-label trial in patients with Crohn’s disease.

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