The National Institute for Health and Care Excellence (NICE) has issued a draft Appraisal Consultation Document that does not recommend CSL Behring’s Hemgenix (etranacogene dezaparvovec) gene therapy for haemophilia B. The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors. In its guidance, NICE’s evaluation committee said that while there is clear evidence that the therapy reduces the number of bleeding episodes haemophilia B patients have each year, there is not enough evidence on how well it works in the long term, meaning cost-effectiveness estimates are “uncertain”. It also cited problems with the indirect comparison between the gene therapy and factor IX replacement therapies. Haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting factor IX, a protein needed to produce blood clots to stop bleeding. Patients ...
The National Institute on Alcohol Abuse and Alcoholism (NIAAA) in the US has awarded a $2m grant to Amygdala Neurosciences to support investigational new drug (IND) application and early development of the aldehyde dehydrogenase (ALDH2) inhibitor for the treatment of alcohol use disorder. According to the 2021 survey by the NIAAA, 29.5 million people aged 12 years and older have experienced alcohol use disorder. Current treatments include behavioural therapy, medication, support groups, and counselling. ANS-858 is an investigational selective and reversible ALDH2 inhibitor, which has been reported to reduce craving and drug-seeking behaviour in preclinical studies. Amygdala CEO Brent Blackburn said in the press release: “This award will enable the development of ANS-858 through pre-clinical evaluation required of an investigational new drug (IND) application to the FDA.” There are multiple drugs currently in development for treating alcohol use disorder. These mainly investigate the use of psychedelic compounds, including Beckly Psytech’s ...
The US Food and Drug Administration (FDA) has granted NS Pharma’s NS-089/NCNP-02 breakthrough therapy designation in a move that will help ramp up the future treatment options for Duchenne Muscular Dystrophy (DMD). The news follows another recent FDA win for NS Pharma, which is owned by Japanese pharmaceutical company Nippon Shinyaku, after its therapy was awarded a rare paediatric disease (RPD) designation earlier in July. A first-in-human clinical trial (NCT04129294) in six Japanese patients with DMD amenable to exon 44 skipping showed that the therapy increased dystrophin protein expression, and improved the motor function of patients as measured by The North Star Ambulatory Assessment. NS-089/NCNP-02 is an antisense nucleotide that helps cells skip over a particular exon. In the case of DMD patients, this is the mutated – and therefore dysfunctional – exon 44 in the DMD gene. Nippon Shinyaku discovered NS-089/NCNP-02 in a joint research project with the National ...
A trial investigating desensitisation immunotherapy delivered as toothpaste for adults with peanut allergy reached a final milestone after Intrommune Therapeutics completed the last patient’s last visit. The Phase I OMEGA clinical trial (NCT04603300) is a multi-centre, randomised, placebo-controlled double-blind study involving splitting 32 participants with peanut allergy into two groups to receive either an escalating dose of the company’s candidate INT301 or placebo. The primary outcome is the safety of the drug compared to placebo. Safety is measured by the percentage of participants able to tolerate the highest dose and incidence of adverse reactions. Intrommune Therapeutics’ candidate utilises the company’s oral mucosal immunotherapy (OMIT) platform to deliver the therapy in fully functional toothpaste. OMIT’s simple administration allows for improved adherence, which is important for desensitising a patient to a specific allergy. Allergy immunotherapy requires consistent exposure. Peanut allergy is one of the most common allergies that ...
Roche has entered into a new partnership with Alnylam Pharmaceuticals to develop and commercialise a potential treatment for high blood pressure, with the deal worth up to $2.8bn. The candidate, zilebesiran, is an investigational, subcutaneously administered RNAi therapeutic currently in phase 2 development to treat hypertension in populations with high-unmet needs. Under the terms of the agreement, which provides Roche with exclusive commercialisation rights to the therapy outside of the US and joint commercialisation rights within the country, Alnylam will receive an upfront payment of $310m and is also eligible to receive certain milestone payments. Hypertension, the leading cause of cardiovascular disease, affects more than 1.2 billion adults worldwide. While several therapies exist, a significant unmet need remains, especially for high-risk patients. Zilebesiran works by preventing the production of angiotensinogen, a protein that plays a key role in raising blood pressure, in the liver. ...
The US Food and Drug Administration (FDA) granted SN Bioscience an orphan drug designation for its small cell lung cancer (SCLC) drug SNB-101, a polymer nanoparticle drug. The South Korea-headquartered company has received approval to run Phase I clinical trials in the US and South Korea, per a 20 July press release. The US trial (NCT04640480) is with solid tumours for any type of cancer. The orphan drug designation programme is an FDA initiative to support the development of new treatments for rare diseases. The classification gives sponsors tax credits from clinical trials, exemption from user fees, and a potential seven years of market exclusivity after approval. SNB-101 is an intravenously or intratumorally administered drug that acts as a topoisomerase I inhibitor. The therapy causes DNA breaks, inhibiting DNA replication and shutting down the cell cycle to prevent tumour growth. The drug uses the active ...
Sanofi and Scribe Therapeutics have announced an expanded collaboration agreement worth over $1.2bn to advance in vivo genetic medicines for sickle cell and other genomic diseases. The agreement follows the launch of the companies’ existing partnership focused on developing ex vivo editing of natural killer cell therapies for cancer. Under the latest deal, Sanofi will gain an exclusive licence to use Scribe’s CRISPR X-Editing genome editing technologies to develop in vivo therapies. In exchange, Scribe will receive a $40m upfront payment and will also be eligible for more than $1.2bn based on the successful completion of certain development and sales milestones. Sanofi will largely be responsible for any resulting candidates, but Scribe will have the opportunity to opt into sharing the development costs and profits on one future programme in the US. Christian Mueller, global head of Genomic Medicine Unit at Sanofi, said: “We’re ...
Acumen Pharmaceuticals has presented positive topline results from an early-stage study of its investigational Alzheimer’s disease therapy at this year’s Alzheimer’s Association International Conference (AAIC). The drug, ACU193, works by targeting and binding to amyloid beta oligomers, a toxic and soluble version of the amyloid protein that forms brain plaques associated with Alzheimer’s disease. Results from the phase 1 randomised, placebo-controlled INTERCEPT-AD study of 62 patients with early Alzheimer’s disease showed the candidate was generally well tolerated throughout both single-ascending and multiple-ascending dose cohorts. Patients who received higher doses of the drug also showed a reduction in amyloid plaque after six to 12 weeks, the company said, adding that the study suggests the drug can be given as a monthly intravenous infusion. Commenting on the positive results, Acumen’s president and chief executive officer, Daniel O’Connell, said: “ACU193’s observed dose-related central target engagement, rapid reduction of ...
As a shortage of key chemotherapy drugs continues to upend cancer treatment across the country, the FDA has resorted to securing some of the meds from China.After approving the importation of four lots back in May, the agency has now cleared 10 more lots of cisplatin for shipment to the U.S. from Chinese company Qilu Pharmaceuticals. Canada’s Apotex will distribute the drugs, which are expected to arrive this week, a company spokesperson told Fierce Pharma. Apotex is “very pleased” to aid in addressing the shortage, the spokesperson added. Cisplatin, along with fellow platinum-based chemotherapy carboplatin, are commonly used as a standard of care across many types of cancer. The newest import was first reported by Bloomberg. After the FDA flagged quality issues at a facility that produced the drug, a “ripple effect” ensued across the supply chain, the FDA’s Oncology Center of Excellence chief Richard Pazdur, M.D., told The Cancer ...
A new talking therapy for depression has shown encouraging early signs of being more effective and cheaper to deliver than the current best practice of Cognitive Behavioural Therapy (CBT). A pilot trial from the University of Exeter, funded by the National Institute of Health and Care Research (NIHR) and published in Lancet EClinical Medicine, has found Augmented Depression Therapy (ADepT) could be a significant advance in depression care. A core feature of depression is anhedonia (reduced interest or pleasure) and wellbeing deficits, but current depression psychotherapies like CBT fail to adequately target these components. ADepT has been developed to pay just as much attention to building wellbeing as it does reducing depressive symptoms. Professor of Clinical Psychology, Barney Dunn, from the University of Exeter led the trial and said: “Depression is widespread and a significant contributor to global disability, resulting in extensive social and economic costs. Only ...
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