The best news in biotech, September 2 - 8

Last week saw a plethora of positive news in biotech! The private markets continue to gain momentum with a mix of NewCo launches and big raises at Series C/D for more mature companies. This included a >$300M raise from a cell therapy biotech, proving that there is still investor interest in differentiated programs for the otherwise out of favor modality. The public markets saw two IPOs priced up and a third announced, as well as a >$1B raise by a biotech that is poised to dominate a vaccine space after presenting some outstanding data. Collaborations continue to be a key route to cash for early biotech as pharma companies keep seeking to strengthen their pipelines and remain at the forefront of innovative technology spaces. Keep reading to our cool science spotlight demonstrating a new and simple approach to achieving tissue transparency in live animals!

?? New Biotech Launches & Private raises…

• Seattle, Boston and Tel Aviv based ZipBio , founded by Roee Farber , Ariel Ben-Sasson Ori Hassin and Ori Zelichov , launched with $4M seed funding to develop biologics and gene therapies using generative AI to compress complex biological structures into smaller de novo-designed proteins that retain functionality. Link
• San Francisco based OrsoBio , led by Founder and CEO G. Mani Subramanian , announced an oversubscribed $67M series B financing to advance its clinical pipeline of mitochondrial protonophores that increase energy expenditure and may be an ideal combination drug for obesity GLP-1 inhibitors. 礼来 notably joined the funding round. Link
• Following a world-first gene engineered pig kidney transplant earlier this year, Boston based eGenesis, Inc. , led by President and CEO Michael Curtis , has raised a $191M series D financing to advance its lead product candidate to a first-in-human study for kidney transplant. Link
• San Francisco based Arsenal Biosciences, Inc. , led by CEO Ken Drazan , raised $325M in an oversubscribed series C to advance its programable CAR-T cell therapy treatments into clinic for the treatment of solid tumors. Link
• 美国杜克大学 spinout Adaptin Bio, Inc. led by CEO Michael Roberts , emerged from stealth with news of FDA clearance of its IND for its lead Brain Bispecific T-cell Engager therapy for the treatment of glioblastoma. Link
• California based Circle Pharma, Inc. , led by David J. Earp, JD, PhD , raised a $90M series D round to fund development of its cell-permeable macrocycle pipeline, including clinical development of a first-and-only-in-class cyclin A/B RxL inhibitor. Link
• Toronto based HDAX Therapeutics , led by Co-Founder and CEO Nabanita Nawar, PhD , raised $3.2M in an oversubscribed seed financing to advance its HDAC6 targeting therapeutics towards preclinical development in cardiometabolic diseases. Link
• Quebec City and Toronto based Feldan Therapeutics raised a $21M series B extension to complete a Phase 1/2a study for FLD-103 – a first-in-class non-invasive intralesional treatment for basal cell carcinoma. Link ?

?? IPO News & Public raises…

• Bicara Therapeutics , a phase 1 oncology biotech based in Boston, priced up its IPO and is hoping to raise $182M to fund a pivotal phase 2/3 trial of a bi-functional antibody targeting EGFR and TGF-β to treat head and neck cancer. Link
• Zenas BioPharma , also Boston based, priced up its IPO at a similar amount, $180M, to advance obexelimab in multiple indications, including an ongoing phase 3 trial treating IgG4-RD and phase 2 trials in MS, Lupus and warm autoimmune hemolytic anemia. Link
• California based BioAge Labs announced plans to file for an IPO to fund two mid stage obesity trials. The 礼来 partnered metabolic disease and obesity biotech's apelin receptor agonist is designed to combine with GLP-1 drugs to boost weight loss and preserve muscle mass. Link
• What a week for Vaxcyte ! The California based biotech reported incredibly positive results from a phase 1/2 trial of its 31-valent pneumococcal conjugate vaccine candidate and subsequently raised an oversubscribed $1.5B public offering as it goes for pneumococcal market dominance. Link

?? Deals deals deals…

• Boston based Voyager Therapeutics, Inc. announced another licensing deal, expanding its partnership with 诺华 to license its capsids against a rare neurological disease target. This brings Voyager's portfolio of partnered gene therapy programs to 14! The new deal is worth $15M upfront and $305M milestone payments. Link
• NY based start-up Genetic Leap , led by Founder and CEO Bertrand Adanve, Ph.D. , is partnering with 礼来 in a deal worth up to $409M? to leverage Genetic Leap's RNA-targeted AI platform to generate genetic medicines against selected targets. Link
• Switzerland based Basilea Pharmaceutica received a $25M milestone payment from 辉瑞 for its sales of a licensed antifungal drug in Europe. https://www.basilea.com/news#news_1512
• Stealth biotech Vignette Bio , incubated by Foresite Labs , has entered a strategic collaboration agreement with EpimAb Biotherapeutics, Inc. to develop a BCMA/CD3 targeting bispecific antibody in a deal worth $60M upfront and up to $575M in milestone payments. Link
• Switzerland and San Diego based HAYA Therapeutics , led by Founding Scientist and CEO Samir Ounzain , announced a collaboration with 礼来 to discover novel targets and develop RNA-guided regulatory genome targeting drugs for obesity and related metabolic conditions using its proprietary RNA platform. Haya will receive an upfront payment as well as up to $1B in milestone and royalty payments. Link
• Global research organization Evotec are to partner with DNA Encoded Library specialist X-Chem, Inc. in a collaboration focused on accelerating early-stage discovery. Link

???Other investments and expansions…

• Astellas Pharma officially opened its Cambridge Crossing facility, which it plans to house 400 researchers, medical and business development staff, as well as incubator labs. Link
• 诺华 began construction of 2 new radioligand manufacturing facilities in the US, a new slits in California and expansion of an existing facility in Indianapolis. Link

??? Positive clinical development updates…

• Summit Therapeutics, Inc. and Akeso took the limelight at WCLC last week presenting phase 3 (China-only) data reducing risk of lung cancer disease progression vs Keytruda by a whopping 49% in a similar patient group. Could we see a new standard in oncology? Link
• Montreal and North Carolina based Milestone Pharmaceuticals, Inc. announced positive topline results from a phase 3 study in China for its arrhythmia drug in people with an abnormal heart rhythm disorder, teeing up a potential China filing. Link
• 荷商葛蘭素史克藥廠 announced positive results from a phase 3 study of its IL-5 targeting antibody Nucala for the treatment of COPD. Link
• 勃林格殷格翰 announced plans to advance its antibody fragment treatment for Geographic Atrophy to phase 2 following positive phase 1 results. Link
• 礼来 presented data from a phase 3 trial demonstrating that its once weekly insulin drug has similar therapeutic benefit to daily basal injections. Link
• Biogen presented positive topline results from a phase 2/3 study of its ASO SPINRAZA, showing benefits at higher doses when treating spinal muscular atrophy, and plans to seek approval of this dose regimen. Link
• 罗氏公司 released positive phase 2 data for fenebrutinib in patients with relapsing multiple sclerosis. Link
• 辉瑞 and Valneva announced positive phase 2 booster results from its Lyme Disease vaccine candidate. Link
• Praxis Precision Medicines, Inc. announced positive topline results from a phase 2 pediatric epilepsy study, reducing seizures by 46%. Link
• Denali Therapeutics announced a successful meeting with the FDA as well as plans to file for accelerated approval for its Enzyme Transport Vehicle delivered drug Tividenofusp alfa, which is engineered to cross the blood-brain-barrier, for the treatment of Hunter Syndrome. Link
• Arrowhead Pharmaceuticals presented pivotal phase 3 data for its RNAi drug Plozasiran for the treatment of Familial Chylomicronemia Syndrome and plans to file an NDA by the end of 2024. Link
• Boston based Vor Bio announced positive results from a phase 1/2 study its Shielded Transplants approach to using cell and genome editing therapeutics to treat blood cancers. Link
• Colorado based Crestone Pharma announced positive results from a phase 2 trial for its antibiotic to treat C. Difficile infection, triggering NIAID to exercise a $4.5M option to find ongoing phase 2 studies. Link
• San Diego based Poseida Therapeutics, Inc. presented positive data from a case study of a patient with relapsed multiple myeloma treated in a clinical trial of P-BCMA-101, the Company's original investigational T stem cell memory (TSCM)-rich BCMA targeting autologous CAR-T cell therapy. Link

? Regulatory Approvals…

• San Diego based Travere Therapeutics received full FDA approval of FILSPARI?, the first and only non-immunosuppressive treatment that significantly slows kidney function decline in IgA nephrophy. Link

?? Cool science spotlight…

Our cool science of the week this week sits at the interface between biology and physics and is truly a marvel!?

A research team at Stamford University, led by Zihao Ou , Mark Brongersma , Guosong Hong and Nick Rommelfanger published the following paper in Science on 6 September:

Achieving optical transparency in live animals with absorbing molecules .

Using a food dye commonly found in Doritos, the team were able to turn the skin of live mice from opaque to transparent.

The technique seeks to reverse the light scattering that normally occurs in live tissue due to differing refractive indexes (RI) between aqueous and non-aqueous (lipids, fats, proteins) tissues by altering the low RI of the aqueous medium to match that of the high index components.?

Remarkably, by rubbing the harmless food-dye based solution onto the skin of mice, tissues were observed to become transparent to a depth of 3mm. The tissues then regained their opaque appearance when the solution was wiped off.?

Considering the simplicity of the approach, the results are impressive! -?

I am excited to see the application of this technology taken further!

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That's all for this week, thanks for tuning in. Subscribe for alerts to the latest positive news in the biotech industry delivered to your inbox every Monday ????

About the author: Max Robinson is an ex-scientist turned talent leader in the biotechnology industry, and currently Global Head of Discovery & Preclinical Research at Proclinical .