Dato data lack upside for Gilead; Padcev 'must-win' does win

Dato data lack upside for Gilead; Padcev 'must-win' does win
Today's news in brief
BioWorld briefs for Sept. 22, 2023.
UK tribunal upholds ruling against Actavis, Allergan, for ‘unfair’ hiked hydrocortisone prices
The U.K.’s Competition Appeal Tribunal has rejected appeals by drugmakers, including Actavis plc and Allergan Inc., against a ruling by regulators that they hiked the price of a life-saving adrenal insufficiency drug excessively for almost a decade, saddling the companies with fines amounting to nearly £130 million (US$159 million).
Wuhan YZY raises $15 million in Hong Kong IPO to advance BsAbs
Wuhan YZY Biopharma Co. Ltd. made its debut on the Hong Kong Stock Exchange, raising HK$121 million (US$15.48 million) in its IPO on Sept. 22, with shares (HKEX:02496) opening at the lower end of its range at HK$16 per share and ending the day at HK$16.60, a rise of 3.75%.
US FDA approvals up 16% over last year
Approvals from the U.S. FDA are up more than 16% from last year, with 113 drugs approved in the first eight months of 2023. New approvals include two different treatments for relapsed or refractory multiple myeloma, two treatments for ultra-rare diseases and a biosimilar to Biogen Inc.’s Tysabri. In August the FDA greenlighted 13 drugs, approving three NDAs, six BLAs, two supplemental NDAs and two supplemental BLAs.
Filspari trips in IgAN phase III; data good enough to Protect against FDA withdrawal?
Travere Therapeutics Inc.’s narrow phase III miss in the study called Protect with the approved endothelin and angiotensin II receptor antagonist Filspari (sparsentan) in IgA nephropathy (IgAN) had Wall Street speculating about the fate of the compound, which is available for the indication by way of accelerated approval in the U.S., having been given the nod in February.

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Analysis and data insight

Lilly closes two $1B+ acquisitions in August as M&As pace similar to last year
Deals and M&A
Biopharma deal values through August are down 13.05% year-over-year, while the number of deals has fallen by 18.51% compared to 2022. M&A value, meanwhile, is down just 3.6% from last year. Through August, 2023 saw 885 biopharma deals worth $111.87 billion, compared to 1,086 deals totaling $128.66...
Biopharma financings up from last year with $42.8B raised through August 2023
Financings
In August 2023 biopharma companies raised a collective $4.02 billion through 86 transactions, down from the previous seven months’ average of $5.53 billion.
Biopharmas receive $1.43B in BARDA grants in 2023
Grant
Biopharma grants are up nearly 150% compared to the same time period last year, largely due to a number of $100 million-plus grants from the Biomedical Advanced Research and Development Authority (BARDA). Meanwhile, nonprofit deal value has declined year-over-year, with the number of transactions...
Clinical trial data down from 2021 but up 18% from 2019
Clinical
The first seven months of 2023 saw clinical trial data up 1.43% compared to the same time period last year. From January through July this year, BioWorld reported on 2,064 drugs in phase I-III, compared to 2,035 in the same time period in 2022. The number of trial updates is down 14.07% from the...

Financings

Chinabio 2023: Cansino, Gracell, Immuneonco’s C-suites offer IPO insights in China, US
Conferences
Raising capital has always been a challenge for small to medium biotech firms worldwide, but the economic whiplash and the wider downturn across international markets post-pandemic have pushed Chinese biotechs to make-it-or-break-it scenarios for crossing the IPO threshold, speakers at the Chinabio...

Science

Machine learning tool Alphamissense analyzes human mutations to predict diseases
Drug design, drug delivery and technologies
Proteome analysis with artificial intelligence has made it possible to create a catalog of all possible missense mutations in the human genome to predict diseases. The new Alphamissense tool from the technology company Google Deepmind, available online, will allow scientists to refine diagnoses and...

Study identifies cause of death for Alzheimer’s neurons
Neurology/Psychiatric
By creating a new mouse model of Alzheimer’s disease that better recapitulated how the disease plays out in humans, investigators at KU Leuven have gained new insights into how amyloid plaques, tau tangles and neuronal death are related at the molecular level.
Cloning pioneer Ian Wilmut dies at 79
Scientist Ian Wilmut, who led a team from Scotland’s Roslin Institute and biotech company PPL Therapeutics plc to clone Dolly the Sheep in 1996, died on Sept. 10 at age 79. Dolly was the first mammal cloned from an adult cell taken from the mammary gland of a 6-year-old Finn Dorset sheep and an egg...

Newco news

Not just another cytokine? Simcha looks to rejuvenate IL-18 field with decoy resistance
Clinical
Although preclinical studies of genetically engineered interleukin-18 (IL-18) historically indicated its potential in cancer treatment, interest in the cytokine languished after GSK plc shelved its IL-18 therapeutic on lack of responses in a phase II melanoma trial. But in 2020, Yale...
Galimedix targeting misfolded amyloid in retinal disease
Clinical
Galimedix Therapeutics Inc. plans to move its lead compound, GAL-101, into a phase IIa proof-of-concept trial next year, in order to test an intriguing hypothesis associated with certain retinal degeneration conditions. GAL-101 selectively binds misfolded amyloid beta species, and the company...
Precision med startup Actio draws $55M series A investment
Financings
“From one to many” is how Actio Biosciences Inc. describes its approach to drug development. The firm emerged with a $55 million series A financing and an eye for biological targets found in both rare and common diseases, starting with TRPV4, a target associated with Charcot-Marie-Tooth disease...
Acuitas tackles mRNA drug delivery challenges with lipid nanoparticles
Vaccine
Messenger RNA, or mRNA, represents a relatively new class of therapeutics with the potential to prevent and treat a wide range of diseases. A well-known success story is of the mRNA vaccines that controlled the COVID-19 pandemic, which has fueled enthusiasm for the field. But biotechs are also...
Faster than light drug discovery: Superluminal launches with $33M seed round
Seed
As its name suggests, Superluminal Medicines Inc. is aiming for speed. The startup, which closed a $33 million seed round led by RA Capital Management, is combining a biology-focused approach with a generative AI platform it says has the potential to create candidate-ready compounds in a matter of...

U.S.

Bipartisan momentum gathering for reforms of US Medicare program
CMS
The U.S. Centers for Medicare & Medicaid Services is once again in the crosshairs thanks to issues related to pharmacy benefits managers and coverage of novel medical devices, with Congress mulling over two dozen pieces of Medicare-related legislation.

Europe

One trial, two regulators, two opinions on Oncopeptides drug
Regulatory
Supporting their conclusions with data from the same phase III study, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for extending the use of Oncopeptides AB’s Pepaxti (melflufen) to earlier lines of treating relapsed, refractory multiple myeloma even as the FDA...

Corteria closes $70.7M series A to advance heart failure candidates
Financings
As part of its quest to advance its three first-in-class heart failure drug candidates toward the clinic, Corteria Pharmaceuticals SAS has closed an oversubscribed €65 million (US$70.7 million) series A, co-led by investors Orbimed and Jeito Capital, companies based in the U.S. and Europe,...
UK Phosp study finds biomarkers predictive of long COVID cognitive defects
Clinical
Blood biomarkers have been found in patients hospitalized with acute COVID-19 that are predictive of the cognitive defects of long COVID. Post COVID-19 deficits in cognition, including brain fog, are common and debilitating. They are also clinically complex, with both objective and subjective...

Clinical

Starpharma’s dendrimer platform delivers impressive results in bowel, ovarian cancer
Cancer
Starpharma Holdings Ltd.'s nanoscale drug delivery technology shows enhanced safety and durable responses in phase II trials compared to standard of care therapies in patients with advanced colorectal and ovarian cancer.
Let there Belite: Alkeus, Kubota help in Stargardt orals genesis
Ocular
Iveric Bio Inc.’s regulatory win Aug. 4 drew attention to the already hot eye-disease space, where intriguing new developments include the possibility of an oral therapy for Stargardt disease. Belite Bio Inc. in late July finished enrollment of a...
Anthos’ phase II in afib hits primary endpoint and makes an early stop
Cardiovascular
Anthos Therapeutics Inc.’s phase II study of abelacimab in treating atrial fibrillation in patients at moderate to high risk of stroke has met its primary endpoint. The data monitoring committee stopped the study early as the fully human...
Hanmi unveils new obesity pipeline under new strategy leadership
Obesity
Hanmi Science Co. Ltd. is rolling out a new obesity pipeline with five candidates under its relatively new leadership with Lim Ju-hyun, the eldest daughter of Hanmi Pharmaceutical Co. Ltd. founder Lim Sung-ki, who serves as president of Hanmi’s...
The more the Mereo as AATD lung space keeping busy
Gene therapy
Though data won’t be available for a few years, the disclosure in mid-July that Grifols SA completed enrollment in the phase III study called Sparta caused some ears to perk up in the alpha-1-antitrypsin deficiency (AATD) space, where a number of...
Ignition: Rocket Pharma offering and phase II launch the stock
Financings
The combination of new U.S. FDA phase II study guidance and a $175 million underwritten public offering sent gene therapy developer Rocket Pharmaceuticals Inc.’s stock soaring on Sept 13. Shares (NASADQ:RCKT) closed 38.8% upward to $21.23 each on...

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