Pharmaceutical Market Europe • April 2022 • 6-7
NEWS
Novartis has announced a licence option agreement worth up to $1.7bn with Voyager Therapeutics for potential gene therapies for neurological diseases.
Voyager is a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies.
Voyager will receive an initial $54m to license its new class of AAV capsids to Novartis for application on three undisclosed CNS targets, along with an option to include two more targets.
Novartis is involved in developing treatments for diseases of the central nervous system, including neurological diseases that manifest in deep brain regions where commonly used vectors have not been shown to effectively target and penetrate.
“The challenge of biodistribution of AAV gene therapies to target cell types in the brain is long-standing, but we refuse to believe it is insurmountable,” said Jay Bradner, president of the Novartis Institutes for BioMedical Research (NIBR).
“We see the potential of Voyager’s innovative platform to imagine new types of therapeutics for life-threatening CNS diseases,” he added.
Bob Baloh, Head of Neuroscience at NIBR, said: “We have a robust pipeline of gene therapy programmes to target diseases of the central nervous system – a number of them requiring enhanced delivery vectors that reach cells in multiple brain regions, which has not been possible with technologies used to date.”
In a deal worth up to $2bn, Takeda has formed a new partnership with Code Biotherapeutics, to gain opt-in rights for four rare diseases candidates.
Code Bio will receive upfront milestone and research funding payments in the range of ‘double-digit million dollars’ from Takeda, while Takeda will make development and commercial milestone payments – apart from tiered royalties – at a future date. The potential value of the collaboration – once the milestones for all four programmes have been met – could total up to $2bn.
Takeda will gain access to Code Bio’s 3DNA non-viral genetic medicine delivery platform, with a view to investigating the technology for use in disorders surrounding the central nervous system.
At the beginning of the collaboration, the companies will work together on research until the selection of the candidates and, following the subsequent option exercise, Takeda will assume responsibility for overseeing the development and marketing of the programmes.
In October 2021, Takeda signed a $3.6bn deal with Poseida Therapeutics for gene therapies for six to eight programmes. These included liver and haematologic diseases, while another deal with Selecta Biosciences targeted lysosomal storage disorders and was valued at around $1.1bn.
AbbVie has announced its acquisition of Syndesi Therapeutics, further expanding AbbVie’s neuroscience portfolio. As part of the $1bn agreement, Abbvie will pay Syndesi an upfront payment of $130m, followed by milestone payments of up to $870m.
The deal will allow AbbVie to access Syndesi’s research into treatment for cognitive impairment and other symptoms associated with neurodegenerative and neuropsychiatric disorders, including major depressive disorder and Alzheimer’s disease.
AbbVie has been closely involved in various Alzheimer’s projects, including an amyloid-beta drug the company believes could work against the degenerative disease. However, AbbVie’s R&D team has also been hindered by setbacks, including a decision to scrap a phase 2 tau-focused drug, ABBV-8E12, after failing to record efficacy.
The acquisition will strengthen AbbVie’s neurology pipeline, as Syndesi bought the rights for a programme aiming to achieve synaptic efficiency from UCB four years ago. Communication between neurons in the brain typically deteriorates as degenerative illnesses progress, particularly in Alzheimer’s.
Tom Hudson, senior vice president, R&D, chief scientific officer, AbbVie, said: “There is a major unmet need for new therapies that can help improve cognitive function in patients suffering from difficult-to-treat neurologic diseases.
“With AbbVie’s acquisition of Syndesi, we aim to advance the research of a novel, first-in-class asset for the potential treatment of cognitive impairment associated with neuropsychiatric and neurodegenerative disorders.”
The US Food and Drug Administration (FDA) has approved Lynparza (olaparib) for breast cancer patients. The drug, jointly developed by AstraZeneca (AZ) and MSD – known as Merck & Co in the US and Canada – is the first and only approved medicine targeting BRCA mutations in early breast cancer.
The FDA approval is specifically for the treatment of patients with BRCA-mutated HER2-negative high-risk early breast cancer who have already received chemotherapy treatment either before or after surgery.
The FDA based its decision on results from the OlympiA phase 3 trial, which showed that Lynparza reduces the risk of invasive breast cancer recurrences, second cancers or death by 42% versus placebo, a significant improvement in survival rates.
As the most diagnosed cancer worldwide, an estimated 2.3 million patients were diagnosed with breast cancer in 2020. In the US, almost 91% of all breast cancer patients are diagnosed at an early stage of disease, with BRCA mutations found in approximately 5-10% of patients.
Dave Fredrickson, executive vice president, Oncology Business Unit, AZ, said: “This new data confirms Lynparza significantly extends patients’ lives versus placebo and underlines the importance of germline BRCA testing as soon as possible after diagnosis to identify patients that may be eligible for Lynparza.”
Sanofi and Regeneron Pharmaceuticals have announced positive results from a second phase 3 trial, showing Dupixent (dupilumab) significantly improved the signs and symptoms of eosinophilic oesophagitis when compared to placebo in patients aged 12 years and over.
The data was presented at the 2022 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting and showed that patients treated with 300mg of Dupixent on a weekly basis experienced a 64% reduction of in disease symptoms by week 24, compared to 41% for those who received placebo.
Additionally, the trial found that significantly more patients treated with Dupixent every two weeks reduced their oesophageal eosinophilic count to the normal range compared to placebo. Despite this, there was not a significant improvement in dysphagia symptoms.
In the US, there are approximately 160,000 people living with eosinophilic oesophagitis. It is a chronic, progressive type 2 inflammatory disease that damages the oesophagus and stops it from functioning normally. Swallowing the smallest amount of food or taking a sip of water can be painful, increasing the risk of choking.
Results from the second phase 3 trial, enrolling 80 patients in the Dupixent group and 79 patients in the placebo group, were announced in October 2021 and confirmed results from the first phase 3 trial.
The US Food and Drug Administration (FDA) has approved Janssen and Legend Biotech’s jointly developed drug, Carvykti, for adult patients with relapsed or refractory multiple myeloma (RRMM).
The FDA based its approval on data from the CARTITUDE-1 trial, involving patients who had received a median of six prior treatments and had previously received a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.
In the pivotal study, 98% of patients with relapsed or refractory multiple myeloma responded to a one-off treatment with Carvykti, while 78% of patients who responded experienced a stringent complete response – a measure whereby a physician is unable to observe any signs or symptoms of disease through the use of imaging or other tests post-treatment.
Multiple myeloma is an incurable blood cancer that affects plasma cells, a type of white blood cell that are found in bone marrow. Although further developments for additional treatments have improved in recent years, many patients have a poor prognosis after the disease progresses.
Peter Lebowitz, global therapeutic area head, Oncology at Janssen Research & Development, said: “We are committed to harnessing our science, deep disease understanding and capabilities to bring forward cell therapies like Carvykti as we continue to focus on our ultimate goal of delivering a cure for multiple myeloma.”