Pharmaceutical Market Europe • July/August 2024 • 6-7
NEWS
Roche and Ascidian agree to $1.8bn neurological disease partnership
Roche and Ascidian Therapeutics have partnered to develop RNA exon editing therapies for neurological diseases, with the deal worth over $1.8bn.
The research collaboration and licensing agreement gives Roche exclusive, target-specific rights to Ascidian’s RNA exon editing technology for undisclosed neurological targets.
Ascidian will conduct discovery and certain preclinical activities in collaboration with Roche, which will be responsible for further preclinical work as well as clinical development, manufacturing and commercialisation.
In exchange, Ascidian will receive an initial payment of $42m and will be eligible to receive up to $1.8bn in research, clinical and commercial milestone payments, plus royalties on global commercial sales.
More than three billion people worldwide were living with a neurological condition in 2021, according to a new study released earlier this year by The Lancet Neurology.
Ascidian said its technology allows for the therapeutic targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels. The approach is designed to provide the durability of gene therapy and reduce the risks associated with direct DNA editing and gene replacement.
AbbVie gains rights to FutureGen’s IBD candidate worth up to $1.7bn
AbbVie and FutureGen Biopharmaceutical have announced a licence agreement worth $1.7bn to develop a preclinical therapy for inflammatory bowel disease (IBD).
The deal gives AbbVie an exclusive global licence to develop, manufacture and commercialise the candidate, FG-M701.
In exchange, FutureGen will receive $150m in upfront and near-term milestone payments and will be eligible to receive up to $1.56bn in clinical development, regulatory and commercial milestones, as well as tiered royalties.
An estimated 3.1 million adults in the US have been diagnosed with IBD, a term for conditions characterised by chronic inflammation of the gastrointestinal tract, including Crohn’s disease and ulcerative colitis.
The exact cause of IBD is unknown and symptoms include persistent diarrhoea, abdominal pain, rectal bleeding, weight loss and fatigue.
FutureGen’s FG-M701 is a fully human monoclonal antibody targeting TL1A, a clinically validated target in IBD, that is hoped to offer greater efficacy and less frequent dosing for patients.
The deal came less than two months after AbbVie and Parvus Therapeutics entered into an exclusive global licence collaboration and option agreement to develop and commercialise novel therapies for IBD.
Sanofi and Belharra agree on $700m immunological disease deal
Sanofi and Belharra Therapeutics have entered into a $700m strategic collaboration aimed at advancing the discovery of small molecule therapeutics for immunological diseases.
The partnership will utilise Belharra’s proprietary non-covalent chemoproteomics platform to screen and validate small molecules against immunology targets chosen by Sanofi, which will then lead further preclinical, clinical and commercial activities for selected candidates.
In exchange, Belharra will be eligible to receive up to $40m in upfront and near-term milestone payments, with a potential total deal value of almost $700m over the course of the collaboration in research, development and commercial milestone payments, as well as tiered royalties on net sales.
Sanofi’s immunology portfolio already includes treatments for atopic dermatitis, severe asthma and rheumatoid arthritis.
Belharra outlined that its platform leverages a “computationally designed library of non-covalent drug-like molecules that use photoaffinity chemistry to identify which proteins bind the molecules as well as the precise binding location on the target proteins”.
The company added that it has already used the platform to build an internal pipeline focused on oncology and immunology.
Eli Lilly’s Alzheimer’s drug Kisunla granted FDA approval
Eli Lilly’s amyloid plaque-targeting Alzheimer’s disease (AD) drug has been approved by the US Food and Drug Administration (FDA) to treat adults with early-symptomatic cases.
Kisunla (donanemab-azbt), administered as a once-monthly intravenous infusion, has been authorised to treat patients with mild cognitive impairment or the mild dementia stage of AD with confirmed amyloid pathology.
Eisai/Biogen’s Leqembi (lecanemab), another amyloid-targeting treatment, is already approved in the US for patients with mild cognitive impairment or early-stage AD.
Lilly outlined that Kisunla is the first and only amyloid plaque-targeting therapy “with evidence to support stopping therapy when amyloid plaques are removed”, potentially resulting in lower treatment costs and fewer infusions.
The FDA’s decision on Kisunla comes after its Peripheral and Central Nervous System Drug Advisory Committee voted 11 to zero that the benefits of the drug outweighed its risks and that trial data submitted by Lilly showed it was effective.
Results from the late-stage TRAILBLAZER-ALZ 2 study showed that Kisunla slowed cognitive and functional decline by up to 35% compared to placebo at 18 months in AD patients who were less advanced in their disease.
AstraZeneca’s Truqap/Faslodex granted EC approval for breast cancer
AstraZeneca’s (AZ) AKT inhibitor Truqap (capivasertib) and its endocrine therapy Faslodex (fulvestrant) have been approved by the European Commission (EC) as a combination treatment for a subset of adults with advanced breast cancer.
Patients eligible for the combination will have oestrogen receptor (ER)-positive, HER2-negative locally advanced or metastatic breast cancer with at least one PIK3CA, AKT1 or PTEN alteration and will have experienced disease recurrence or progression on or after an endocrine-based regimen.
The EC’s decision follows a recent recommendation from the European Medicines Agency’s human medicines committee and is supported by positive results from the late-stage CAPItello-291 trial, in which Truqap plus Faslodex reduced the risk of disease progression or death by 50% compared with Faslodex alone in patients with tumours harbouring PI3K, AKT pathway or PTEN alterations.
The growth of HR-positive breast cancer progression is often driven by ERs, and endocrine therapies that target ER-driven disease are widely used as a first-line treatment in the advanced setting and often paired with CDK4/6 inhibitors. However, resistance to these therapies develops in many patients with advanced disease, underscoring the need for new treatment options.
Merck’s adult-specific pneumococcal vaccine approved by FDA
Merck & Co – known as MSD outside the US and Canada – has received approval from the US Food and Drug Administration (FDA) for its adult-specific 21-valent pneumococcal conjugate vaccine.
Capvaxive is now approved to prevent invasive pneumococcal disease and pneumococcal pneumonia in individuals aged 18 years and older.
The authorisation for Capvaxive for the prevention of pneumococcal pneumonia was granted under the FDA’s accelerated approval pathway.
Merck’s Capvaxive is specifically designed to help protect against the types of pneumococcal bacteria that are responsible for the majority of adult invasive pneumococcal disease.
The clinical data supporting the FDA’s decision included results from the late-stage STRIDE-3 trial, which compared Merck’s vaccine to Pfizer’s PCV20 in individuals aged 18 years and older who had not previously received a pneumococcal vaccine.
The approval was also supported by results from the phase 3 STRIDE-5 study, which evaluated Capvaxive co-administered with a quadrivalent influenza vaccine in adults aged 50 years and older, and the phase 3 STRIDE-6 study of Capvaxive in adults aged 50 years and older who had received a pneumococcal vaccine at least one year before enrolment.
Pharmaceutical Market Europe (PME)
Pharmaceutical industry news, articles, jobs, reports, advice and services