Nrf2

Plus, news about Takeda, Foresee Pharmaceuticals and Capricor Therapeutics: GSK, Cambridge University team up on immune diseases: The UK drugmaker is pouring £50 million ($65 million) into a five-year partnership with the university and its hospitals to advance R&D for respiratory and kidney disease treatments. The collaboration will make use of AI and machine learning, with a focus on traditionally challenging indications. — Ayisha Sharma Angelini Pharma signs licensing deal with Korean biotech: The deal covers a Cureverse program called CV-01, which is an Nrf2 agonist designed to treat “a range of brain health disorders, including epilepsy.” Cureverse will get an undisclosed upfront payment and can receive up to $360 million in milestones as well as additional royalties. The deal covers global rights outside of South Korea, China, Hong Kong, Macau and Taiwan. — Max Gelman Takeda announces more layoffs in Massachusetts: The workforce reduction will impact 45 employees in Cambridge and 34 in Lexington, according to a WARN notice filed last week. Takeda has already laid off hundreds of employees in the state this year, including 641 in May and another 220 in July . The company declined to comment on the types of roles impacted. — Nicole DeFeudis Foresee Pharmaceuticals touts early topline data for MMP-12 inhibitor: Healthy patients taking the inhibitor, called linvemastat, saw no serious adverse events, and the most common treatment-emergent adverse events were mild nausea and headaches. The company expects to start Phase 2 studies in asthma and inflammatory bowel disease next year. — Katherine Lewin Capricor Therapeutics brought in $86.3 million from a public offering, up from the $75 million it planned to raise. — Jaimy Lee

The agreement gives Angelini Pharma an option to exclusive licensing rights to develop and commercialize CV-011, a clinical Phase 1 investigational novel compound with broad potential to treat brain health diseasesCureverse receives undisclosed upfront payments and is eligible for future milestone-dependent payments and royalties, while retaining rights to commercialize CV-01 in the Republic of Korea, China, Hong Kong, Macau and TaiwanThe collaboration builds on Angelini Pharma’s strong portfolio and deep therapeutic expertise in brain health as well as Cureverse’s early development know-how and capabilities ROME, Oct. 21, 2024 (GLOBE NEWSWIRE) -- Angelini Pharma, part of the privately owned Angelini Industries, and Cureverse Inc., an early-stage research and development company, announced today that they entered into an exclusive global option agreement for the development and commercialization of Cureverse’s innovative brain health asset CV-01. Under the terms of the agreement, Angelini Pharma will lead all the development efforts for CV-01 and, following an initial option period, will have the right to obtain the global exclusive development and commercialization license for the compound, outside of the Republic of Korea, China, Hong Kong, Macau and Taiwan. CV-01 is a novel and innovative investigational compound that activates protective pathways regulated by the nuclear factor erythroid 2-related factor 2 (Nrf2) and has a strong potential to be a medication with disease-modifying properties across a range of brain health disorders, including epilepsy.i CV-01 is an investigational compound currently undergoing a Phase 1 clinical trial in the Republic of Korea and is being developed by Cureverse for Alzheimer’s Disease. Cureverse will receive an upfront payment and will also be eligible to receive additional payments upon pre-defined development and commercial milestones of up to approx. US$360 million, as well as tiered royalties on post-approval net sales. “Our strategic collaboration with Cureverse further strengthens Angelini Pharma’s position as an emerging leader in brain health,” said Jacopo Andreose, Chief Executive Officer of Angelini Pharma. “Neurological conditions such as epilepsy are among leading causes of disease burden worldwide.ii While significant progress has been achieved in the past 15 years, many people living with epilepsy are still unable to reach seizure control despite combination treatment of several anti-seizure medications.iii Through the development of CV-01 and potentially other compounds, we aim to provide much-needed solutions for people living with brain health conditions across the world.” “Our research has unveiled the remarkable potential of CV-01 to transform the landscape of brain health, from epilepsy to Alzheimer’s and Parkinson’s Disease,” said Sung Jin Cho, Chief Executive Officer at Cureverse Inc. “We are truly delighted to be partnering with Angelini Pharma on this journey. Their deep therapeutic expertise and commitment to brain health perfectly align with our vision. Together, we are not just developing a treatment—we are paving the way for a future where patients with neurological conditions have access to truly life-changing therapies.” About Brain HealthBrain health as a therapeutic area includes both neurological and mental health conditions.iv Brain health is defined by the World Health Organization as a state of brain functioning across cognitive, sensory, social-emotional, behavioral and motor domains, allowing a person to realize their full potential over the course of their life, irrespective of the presence or absence of disorders.v Brain health represents an urgent global health priority. An estimated 179 million Europeans are living with brain health conditionsvi and more than 18% of all health loss around the world is associated with brain conditions.vii Additionally, brain health conditions are responsible for considerable individual, economic and societal impact, including loss of independence, reduced productivity, strained relationships, increased risk of suicide as well as high healthcare costs.viii There is an urgent need to raise awareness, and reduce stigma, prejudice and discrimination, to ensure that brain health is valued, promoted and protected for all.v About EpilepsyEpilepsy is one of the most widespread neurological diseases in the world, affecting globally approximately 50 million people of all ages.ix In Europe, up to six million people are estimated to be living with this disease.x Epilepsy can have multiple potential causes, including structural, metabolic, genetic and other factors, though approximately half of cases worldwide do not have a known cause.ix The complications associated with epilepsy are severe, with a risk of premature mortality up to three times higher than the general population.ix The recurrent seizures associated with this condition also have wide-ranging effects on a person’s broader physical and mental health, education and employment opportunities and other quality of life factors. ix Treatments are available to help reduce seizures and improve quality of life, however approximately 40% of people living with epilepsy are still uncontrolled despite the treatment with two anti-seizure medications.iii About Angelini PharmaAngelini Pharma is an international pharmaceutical company, part of the privately owned multi-business Angelini Industries. The Company researches, develops and commercializes health solutions with a focus on the areas of Brain Health, including Mental Health and Epilepsy, and Consumer Health. Founded in Italy at the beginning of the 20th century, Angelini Pharma operates directly in 20 countries, employing more than 3,000 people. Its products are marketed in over 70 countries through strategic alliances with leading international pharmaceutical groups. For more information about Angelini Pharma please visit https://www.angelinipharma.com. About Angelini IndustriesAngelini Industries is a multinational industrial group founded in Ancona in 1919 by Francesco Angelini. Today, Angelini Industries represents a solid and diversified industrial reality that employs approximately 5,800 employees and operates in 21 countries around the world with revenues of over 2 billion euros, generated in the health, industrial technology, and consumer goods sectors. A targeted investment strategy for growth; constant commitment to research and development; deep knowledge of markets and business sectors, make Angelini Industries one of the Italian companies of excellence in the sectors in which it operates. To learn more visit www.angeliniindustries.com. About Cureverse Inc.Founded in 2021, Cureverse has rapidly become a key innovator in the Korean biotech landscape, with a bold vision to revolutionize brain health on a global scale. Specializing in cutting-edge small-molecule therapies, Cureverse focuses on tackling central nervous system (CNS) disorders like Alzheimer’s disease (AD), aiming to bring hope to millions worldwide. While many AD drugs have made it to market, a true breakthrough has yet to be realized—until now. At the heart of our innovation is CV-01, a 'First-in-Class' therapy that uniquely targets the Keap1/Nrf2 pathway, activating Nrf2 through precise modulation. This approach offers unprecedented selectivity, effectively controlling neuroinflammation with precision unmatched by existing treatments. Alongside CV-01, Cureverse is actively advancing CV-02, a biased S1P1 agonist that is now IND-ready, as well as several promising follow-up non-clinical programs. With recent IND approvals and strong clinical progress, Cureverse is well-positioned to transform the way we treat neurological conditions and improve patients' lives across the globe. Media Contact Chiara AntoniucciGlobal Head of Brand and Media Communications, Angelini PharmaPhone: +39 3477133926Email: chiara.antoniucci@angelinipharma.com Sung Jin ChoChief Executive Officer, Cureverse Inc.Email: sjcho@cureverse.co.kr, sjcho@kist.re.kr ________________________________ 1 CV-01 is an investigational compound, not approved for use by regulatory authorities i Data on file from Cureverse.ii Institute for Health Metrics and Evaluation. Global burden of conditions affecting the nervous system. Available at: https://www.healthdata.org/research-analysis/library/global-burden-conditions-affecting-nervous-system Last accessed: October 2024.iii Chen Z, Brodie MJ, Liew D, Kwan P. Treatment Outcomes in Patients With Newly Diagnosed Epilepsy Treated With Established and New Antiepileptic Drugs: A 30-Year Longitudinal Cohort Study. JAMA Neurol. 2018 Mar 1;75(3):279-286.iv Winkler AS, et al. Lancet Glob Health. 2024;12(5):e735-e736.v World Health Organization. Optimizing brain health across the life course: WHO position paper. Geneva: World Health Organization; 2022.vi Nutt D, et al. Eurohealth (Int) 2017;23:21–5.vii Institute for Health Metrics and Evaluation. Understanding brain health around the world. Available at: https://brainhealthatlas.org/ Last accessed: October 2024.viii World Health Organization. World mental health report: transforming mental health for all. Geneva: World Health Organization; 2022.ix WHO. “Epilepsy Key Facts.” N.p., 9 Feb. 2023. Web. 24 Aug. 2023.x Behr et al. Epidemiology of epilepsy. Revue Neurologique 2016 Jan; 172(1):27-36. doi: 10.1016/j.neurol.2015.11.003.

Friedreich’s ataxia is a rare, debilitating and life-shortening neuromuscular disease caused by genetic factors 1 If approved, omaveloxolone would be the only disease-specific treatment option for patients living with this progressive and challenging disease This New Drug Submission reflects Biogen’s commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients TORONTO , Sept. 17, 2024 /CNW/ - Biogen Canada Inc. (Biogen) is pleased to announce today that Health Canada has accepted for priority review its New Drug Submission (NDS) for omaveloxolone, an innovative therapy for Friedreich’s ataxia for the treatment of adults and adolescents aged 16 years and older. This once-daily oral medication, if approved, would represent the only therapy specifically targeted for Friedreich’s ataxia, addressing a critical unmet treatment need in this patient population. “Friedreich’s ataxia is a devastating diagnosis that profoundly impacts the lives of patients and their families,” said Eric Tse , General Manager at Biogen Canada. “The omaveloxolone NDS acceptance marks a significant step forward for Canadian patients, offering hope and potential for a therapy that could help manage this debilitating neurodegenerative disorder. We look forward to working with Canadian health authorities to bring forward this promising treatment for those in need.” Friedreich’s ataxia is a rare, inherited condition that affects about 1 in 40,000 people and has a profound impact on their lives, causing a slow, progressive loss of muscle coordination, slurred speech, weakness, and sensory loss. 2 Symptoms typically begin in childhood or adolescence, and while the progression varies, it often leads to significant disability over time 2 with individuals becoming wheelchair-bound 8 to 10 years after disease onset. 1 The NDS for omaveloxolone is supported by efficacy and safety data from the placebo-controlled MOXIe Part 2 trial, which indicated that omaveloxolone significantly improved modified Friedreich Ataxia Rating Scale (mFARS) scores at 48 weeks relative to placebo. Omaveloxolone received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration, as well as Orphan Drug status by the European Commission. 3,4 It has received regulatory approval for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older in both the United States and the European Union, with strong support from the medical and patient communities. 3,4 In Canada , priority review submissions follow an accelerated timeline where the standard review period is shortened to 180 days. 5 A regulatory decision on the New Drug Submission for omaveloxolone is anticipated in early 2025. About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment, to deliver long-term growth. Biogen has been proudly serving Canadian patients for more than 25 years. For information about Biogen Canada, please visit . References: Ataxia Canada . Friedreich’s Ataxia . Available at: Accessed September 2024 . AboutKidsHealth. Friedreich ataxia . Jan. 2020 . Available at: . Accessed September 2024 . U.S. Food and Drug Administration. (2023, February 28 ) FDA approves first treatment for Friedreich’s ataxia [press release] Accessed September 2024 . Biogen Inc. (2024, February 12 ) Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia [press release] Accessed September 2024 . Health Canada . Priority Review of Drug Submissions (Therapeutic Products) Available at: Accessed September 2024 . SOURCE Biogen Canada Inc.