Simcere and Idorsia enter into a licensing agreement for daridorexant in China

On November 15, 2022 Simcere Pharmaceutical Group Ltd (2096.HK, "Simcere"), an innovation and R&D-driven pharmaceutical company,and Idorsia Ltd (SIX: IDIA, "Idorsia"), specialized in the discovery, development and commercialization of small molecules to transform the horizon of therapeutic options, reported that they have entered into an exclusive licensing agreement for Idorsia’s daridorexant in China (Press release, Jiangsu Simcere Pharmaceutical Company, NOV 15, 2022, View Source [SID1234624119]).

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Jinsheng Ren,Chairman and CEO of Simcere commented:

"This licensing agreement represents an important and exciting step in pursuing Simcere’s mission of providing today’s patients with medicines of the future. There are over 200 million people in China who suffer from chronic insomnia that may benefit from daridorexant. Patients are eager for a better treatment option that improves quality of sleep and next day functioning. Simcere has a proven track record of successfully developing and marketing innovative Central Nervous System therapies in China. In collaboration with Idorsia, we hope to bring another impactful medicine to millions of people."

Under the agreement, Simcere will be granted an exclusive right to develop and commercialize daridorexant in the Greater China region (Mainland China, Hong Kong, and Macau), one of the world’s largest pharmaceutical markets. Simcere will be responsible for the funding and conducting of a local development program with Chinese patients. Simcere successfully commercializes Sanbexin for acute ischemic stroke and daridorexant will expand Simcere’s pipeline of Central Nervous System (CNS) products in China.

According to the terms of the agreement, Idorsia will receive a US$ 30 million upfront payment, and will be eligible to receive an additional milestone payment of US$ 20 million upon regulatory approval by the National Medical Products Administration, as well as commercial milestone payments and low double-digit tiered royalties based upon future sales.

Jean-Paul Clozel, MD and Chief Executive Officer of Idorsia commented:

"Daridorexant is on track to becoming a global success, helping the millions of sufferers of insomnia around the world. Marketed as QUVIVIQ, we expect daridorexant will soon be the leading branded insomnia medication in the US in terms of new prescriptions. It is also the first dual orexin receptor antagonist available to patients in Europe, and recent positive data in Japan gives me confidence that we can make it available there too. We have been convinced of Simcere’s shared enthusiasm for daridorexant, and I am confident that they are the right partner to join us on our mission of bringing an optimal treatment approach to patients with insomnia around the world by developing and commercializing daridorexant in China."

About daridorexant

Idorsia’s research team has been working on the science of orexin and orexin receptors since they were first described in 1998. The team’s initial work led to the conclusion that antagonism of the orexin system was the key to preserving a natural sleep architecture for patients with insomnia. With this as the target, the team designed a dual antagonist with the goal of a rapid onset of effect and a duration of action sufficient to cover the night but short enough to avoid any negative next-morning residual activity at optimally effective doses.

Daridorexant is a dual orexin receptor antagonist, which blocks the binding of the wake-promoting neuropeptides orexins. Rather than inducing sleep through broad inhibition of brain activity, daridorexant blocks only the activation of orexin receptors. Consequently, daridorexant decreases the wake drive, allowing sleep to occur, without altering the proportion of sleep stages. The results of a Phase 3 clinical development program have been reported by Mignot, E., et al. Safety and efficacy of daridorexant in patients with insomnia disorder: results from two multicentre, randomised, double-blind, placebo-controlled, phase 3 trials, in The Lancet Neurology 2022;21:125–39.

Notes to the editor

Global regulatory status of daridorexant

In January 2022, QUVIVIQ (daridorexant) was approved by the US Food and Drug Administration (FDA) and subsequently made commercially available in May 2022. For more information about QUVIVIQ in the US, see the Full Prescribing Information (PI and Medication Guide). In April 2022, marketing authorization of QUVIVIQ was granted by the European Commission and subsequently by the Medicines and Healthcare products Regulatory Agency (MHRA) in Great Britain via the European Commission Decision Reliance Procedure. For more information about QUVIVIQ in the EU, see the Summary of Product Characteristics. Launch preparations are underway in the major European markets and QUVIVIQ was made available in both Italy and Germany in November 2022. Daridorexant is currently under review with Swissmedic and Health Canada.

About insomnia disorder

Insomnia disorder is defined as difficulty initiating or maintaining sleep, causing clinically significant distress or impairment in important areas of daytime functioning. This impact on sleep quantity or quality should be present for at least three nights per week, lasts for at least three months, and occurs despite an adequate opportunity to sleep.

Insomnia is a condition of overactive wake signaling and studies have shown that areas of the brain associated with wakefulness remain more active during sleep in patients with insomnia. It is a common problem with an estimated prevalence in China of 15% of the adult population.

Insomnia as a disorder is quite different from a brief period of poor sleep, and it can take its toll on both physical and mental health. It is a persistent condition with a negative impact on daytime functioning. Idorsia’s research has shown that poor quality sleep can affect many aspects of daily life, including the ability to concentrate, mood, and energy levels.

The goal of treatments for insomnia is to improve sleep quality and quantity, as well as daytime functioning, while avoiding adverse events and next-morning residual effects. Current recommended treatment of insomnia includes sleep hygiene therapy, cognitive behavioral therapy, and pharmacotherapy.

About the orexin system

Wake and sleep signaling is regulated by intricate neural circuitry in the brain. One key component of this process is the orexin system, which helps promote wakefulness. There are two forms of orexin neuropeptides – small protein-like molecules used by nerve cells (neurons) to communicate with each other in the brain – orexin A and orexin B.[7] Orexin promotes wakefulness through its receptors OX1R and OX2R. Together, these neuropeptides and receptors make up the orexin system. The orexin system stimulates targeted neurons in the wake system – leading to the release of several chemicals (serotonin, histamine, acetylcholine, norepinephrine) – to promote wakefulness. Under normal circumstances, orexin levels rise throughout the day as wakefulness is promoted and then fall at night. Overactivity of the wake system is an important driver of insomnia.

The daridorexant clinical development program

The Phase 3 registration program comprised two three-month studies, together with a long-term double-blind extension study. The program enrolled a total of 1,854 patients with insomnia disorder. As insomnia often presents later in life, and older adults are more susceptible to experience fragmented sleep, early awakening and daytime sleepiness, around 40% of the recruited population was at least 65 years of age.

The placebo-controlled studies investigated the effects of three doses of daridorexant (10 mg, 25 mg, and 50 mg) on sleep and daytime functioning parameters, objectively in a sleep lab by polysomnography and subjectively with a daily patient diary at home. The impact of insomnia on patients’ daytime functioning was measured daily using the sleepiness domain score from the Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ©) – a patient-reported outcome (PRO) instrument developed and validated according to the FDA Guidance for Industry.

More than 800 patients continued treatment in the 40-week extension study, which measured the effect of all three doses vs. placebo, generating data for long-term treatment of insomnia disorder.

Phase 3 data has been reported in The Lancet Neurology: The pivotal studies demonstrated that daridorexant 50 mg significantly improved sleep onset, sleep maintenance and self-reported total sleep time at months one and three compared to placebo. The largest effect was observed with the highest dose (50 mg), followed by 25 mg, while the 10 mg dose did not have a significant effect. In all treatment groups the proportions of sleep stages were preserved, in contrast to findings reported with benzodiazepine receptor agonists.

A major focus of the trials was to evaluate the impact of daridorexant on daytime functioning in patients with insomnia disorder, as assessed by the IDSIQ. IDSIQ is a patient-reported outcomes instrument specifically developed and validated according to FDA guidelines, to measure daytime functioning in patients with insomnia. The sleepiness domain score of the IDSIQ was evaluated as a key secondary endpoint in both pivotal studies and comparisons to placebo included type I error control for multiplicity. Daridorexant 50 mg demonstrated highly statistically significant improvement in daytime sleepiness at month one and month three. The sleepiness domain score was not significantly improved on 25 mg in either study at either timepoint.

The overall incidence of adverse events was comparable between treatment groups. The most frequently reported adverse reactions were headache and somnolence and, overall, the majority of adverse reactions were mild to moderate in intensity. There was no evidence of dose-dependent increases in adverse events across the dosing range. Further, no dependence, rebound insomnia or evidence of abuse or withdrawal symptoms indicative of physical dependence upon treatment discontinuation was observed in clinical studies.

Ablexis and AlivaMab Discovery Services Announce Expansion of Antibody Drug Discovery and Engineering Capabilities

On November 15, 2022 Ablexis, LLC ("Ablexis"), a biopharmaceutical company focused on licensing its AlivaMab Mouse technology for antibody drug discovery, and AlivaMab Discovery Services, LLC ("ADS"), a leader in the discovery of superior antibody-based therapeutics, which together are leading providers of solutions for the discovery of superior antibody-based therapeutics, reported an expansion of platforms and capabilities in antibody drug discovery and engineering (Press release, AlivaMab Discovery Services, NOV 15, 2022, View Source [SID1234624118]). These innovative new offerings include:

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Molecularly diverse panels of lead-quality antibodies ready for reformatting into multispecific formats of the partner’s choice, including a large panel of T-cell-engaging CD3 antibodies that potently eliminate cancer cells with minimal cytokine release.
New strains of AlivaMab XKL Mice offered by Ablexis that further expand the molecular and epitope diversity of the immune response to promote even broader responses against the increasingly complex types of antigens at the cutting edge of antibody drug discovery.
NGS-based hit-expansion capabilities that expand upon ADS’ existing advanced hybridoma-based approaches, increasing captured diversity.
Immune repertoire display technology offered by ADS, which enhances the capture of alternative diversities of the immune response, downstream screening, and antibody drug engineering.
"Ablexis and AlivaMab Discovery Services continue to invest in new technologies that maximize value and flexibility for our partners," said Larry Green, PhD, Chief Executive Officer of Ablexis and AlivaMab Discovery Services. "Our partners enjoy an edge on their competition, not only through access to the expanding suite of AlivaMab Mouse transgenic mice for antibody drug discovery and ADS’ expanding offerings of validated technologies in antibody discovery and engineering, but also through the intellectual contributions of our scientific leadership team and deep bench of scientists. The result is that we outperform with unsurpassed speed and efficiency in the delivery of molecularly diverse, drug-quality human antibodies, even for the most challenging drug targets, enabling our partners to discover and advance better antibody drug candidates faster."

Data on these new capabilities will be highlighted in two presentations by Jane Seagal, PhD, Vice President of Antibody Discovery at ADS, at PEGS Europe, to be held at the InterContinental Barcelona from November 14 to 16, 2022.

A Platform for Tuning Therapeutic Efficacy of T-Cell-Engaging Bispecific Antibodies – November 15 at 3:35 pm CET.
Discovery of Diverse Antibody Panels Using AlivaMab Mouse: The Foundation for Successful Antibody Therapeutics – November 16 at 12:15 pm CET.

DermTech and Sonora Quest Laboratories Expand Access to Innovative Melanoma Detection Test

On November 15, 2022 DermTech, Inc. (NASDAQ: DMTK) ("DermTech" or the "Company"), a leader in precision dermatology enabled by a non-invasive skin genomics platform, reported an agreement with Sonora Quest Laboratories (Sonora Quest), Arizona’s leading diagnostic laboratory, to expand access to enhanced melanoma detection (Press release, DermTech International, NOV 15, 2022, View Source [SID1234624117]). As a result of the agreement, Sonora Quest will be the exclusive laboratory in Arizona to offer the DermTech Melanoma Test to its vast network of healthcare providers.

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The DermTech Melanoma Test uses a non-invasive Smart Sticker to lift skin cells from the surface of a patient’s skin to be tested for select genomic markers associated with melanoma. It rules out melanoma with over 99% reliability. The agreement between DermTech and Sonora Quest will increase the availability of the DermTech Melanoma Test to Arizonans through a visit to their healthcare providers within the Sonora Quest network. Not only will this provide additional patient populations with heightened access to the DermTech Melanoma Test, but it could also reduce the number of unnecessary biopsies and referrals to specialists and reduce costs to the healthcare system.

"This agreement with Sonora Quest is a testament to our continued commitment to helping end deaths from melanoma," said John Dobak, M.D., CEO of DermTech. "Given the frequency of patients asking their healthcare providers about suspicious moles, introducing a simple way for providers to rule out melanoma with confidence is key. We look forward to working with Sonora Quest to reach more patients who visit their healthcare providers as a first line of defense."

"Arizonans spend significant time outdoors, potentially putting us at higher risk of skin cancer," said David A. Dexter, president and CEO for Sonora Quest. "Early detection is key and our agreement with DermTech will provide greater access to this innovative testing method to detect melanoma. We’re dedicated to continually investing in innovative technology like this to make health care more convenient and provide better outcomes to all Arizonans."

One of the deadliest forms of skin cancer, melanoma is most treatable when diagnosed early – in fact, the survival rate is 99% when detected early and only 27% once it spreads distantly.

NorthStar Medical Radioisotopes Expands Business Platform with New, Patient-focused, Radiopharmaceutical Contract Development and Manufacturing Organization (CDMO) Services Unit

On November 15, 2022 NorthStar Medical Radioisotopes, LLC, a global innovator in the development, production and commercialization of radiopharmaceuticals used for therapeutic applications and medical imaging, reported a significant expansion of its business platform with the formation of a new, patient-focused Radiopharmaceutical Contract Development and Manufacturing Organization (CDMO) services unit (Press release, NorthStar Medical Radiostopes, NOV 15, 2022, View Source [SID1234624116]). Based on the success of its diagnostic imaging and therapeutic radioisotope production programs, NorthStar has created the services unit to further advance development and commercialization of radiopharmaceuticals to treat patients who have cancer and other serious diseases. The CDMO will provide collaborator companies with a full range of customized radiopharmaceutical development and commercialization services, and serve NorthStar in progressing its own radiopharmaceutical programs. Ground has been broken on a new facility, located alongside production facilities on the Company’s Beloit, Wis. campus. Upon completion, NorthStar will be the first and only U.S. company housing commercial-scale, multi-radioisotope production and radiopharmaceutical development services on the same campus, enabling collaborator companies to realize logistical, regulatory and cost benefits.

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"Establishing this new radiopharmaceutical CDMO services unit at NorthStar marks an important inflection point in our growth plan to facilitate efficient patient supply of game-changing radiopharmaceuticals," said Stephen Merrick, Chief Executive Officer of NorthStar Medical Radioisotopes. "NorthStar’s industry-leading reputation is grounded in technological innovation, successful execution and proven expertise. Our track record of success includes production and expanded capacity of U.S.-produced, non-uranium based molybdenum-99 (Mo-99) for use in diagnostic imaging. We are poised to be the first commercial-scale producer of non-carrier added (n.c.a.) therapeutic radioisotopes actinium-225 (Ac-225) and copper-67 (Cu-67) using advanced, environmentally preferable electron accelerator technology. With our depth and breadth of technical knowledge in the development and production of medical radioisotopes, we are now expanding to help companies progress their own radiopharmaceutical development programs. NorthStar’s CDMO will enable us to share our radiopharmaceutical development and commercialization expertise with pharmaceutical or biotechnology companies that may require additional infrastructure, resources or the specialized knowledge required for complex radiopharmaceutical development, to help them develop and deliver products with the potential to improve care for even more patients with serious disease."

"We are excited that NorthStar will be able to offer a full spectrum of radiopharmaceutical CDMO services to facilitate the radiopharmaceutical drug development process," said Frank Scholz, Ph.D., President and Chief Operating Officer of NorthStar Medical Radioisotopes. "We plan to collaborate with pharmaceutical and biotechnology companies to provide customized input and support across all phases of program development, from early stage through commercialization and product life cycle management. NorthStar’s radiopharmaceutical CDMO services will be customized to accommodate each customer’s needs and in compliance with rigorous regulations and attention to safety. They will include radionuclide procurement and formulation, dedicated suites for compounding and filling operations, quality assurance and quality control, analytical services, regulatory support, scale-up and commercialization activities and logistical support with packaging and shipping. NorthStar’s highly talented workforce possesses the specialized expertise to help support our radiopharmaceutical CDMO activities, and we plan to hire additional employees in association with this expansion. We have begun design, construction and fitting-out of clean rooms, laboratories and other required infrastructure for the dedicated 36,000 square foot facility, and expect that it should be available to deliver services in late 2024, pending appropriate licensure and regulatory approvals."

CG Oncology Raises $120 Million in Oversubscribed Series E Financing to Advance Clinical-Stage Urologic Oncology Pipeline

On November 15, 2022 CG Oncology, Inc., an oncolytic immunotherapy company focused on developing novel therapeutics for patients with urologic cancers, reported the close of an oversubscribed $120 million Series E financing round, co-led by ORI Capital, Longitude Capital and Decheng Capital, with participation from RA Capital Management, Acorn Bioventures, Malin Corporation, Ally Bridge Group and Sirona Capital (Press release, CG Oncology, NOV 15, 2022, View Source [SID1234624115]). The company has secured a total of over $200 million in funding to date. In connection with the financing, Brian Liu, M.D., Principal from Longitude Capital, will join the company’s board of directors.

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The proceeds from the Series E financing will be used to advance the company’s lead clinical programs in bladder cancer towards FDA approval as well as broaden the scope of its pipeline to address unmet medical needs in urologic cancer, such as the first-line setting in Bacillus Calmette-Guérin (BCG)-naïve, intermediate-risk non-muscle invasive bladder cancer (NMIBC) patients.

"We are delighted by the support from leading global investors to help us accomplish our mission of developing innovative therapeutics for patients suffering from urologic cancers," said Arthur Kuan, Chief Executive Officer, CG Oncology. "Our continued clinical progress in bladder cancer is a testament to our lead candidate CG0070 in both monotherapy and combination therapy studies, setting the stage to further expand development across additional urologic oncology indications."

CG0070 is an intravesically delivered oncolytic immunotherapy agent in an ongoing Phase 3 trial (BOND3) with CG0070 as a monotherapy for the treatment of BCG-unresponsive NMIBC, and a fully enrolled combination Phase 2 study (CORE1) of CG0070 with anti-PD-1 therapy KEYTRUDA (pembrolizumab) in the same indication. CG Oncology has a clinical collaboration with Merck (known as MSD outside the United States and Canada) to evaluate the combination of CG0070 with pembrolizumab in CORE1. Interim Phase 2 data for CORE1 announced at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2022 Annual Meeting on November 10, 2022, continue to show both strong anti-tumor activity and tolerability of CG0070 in combination with pembrolizumab for patients with BCG-unresponsive NMIBC.

"We have strong conviction in CG Oncology which continues to generate compelling data to develop potential bladder-sparing innovative therapeutics for patients suffering from bladder cancer," said Simone Song, Founder and Senior Partner of ORI Capital. "We’ve been strong supporters of the CG Oncology team from an early stage by leading the Series C round, and we are particularly proud to co-lead this round with Longitude Capital and Decheng Capital, as we see tremendous potential for CG0070 to be a game changer for patients with NMIBC."

"In advanced clinical studies CG0070 has continued to demonstrate potentially transformational results in patients with bladder cancer unresponsive to BCG, a difficult-to-treat patient population," said Brian Liu, M.D., Principal, Longitude Capital. "I am excited to join CG Oncology’s board of directors during this significant expansion trajectory for the company and am looking forward to working with the leadership team to bring this therapy to patients in need."

CG0070 has been administered to over 200 patients for the treatment of bladder cancer. The investigational therapy has exhibited potential best-in-class efficacy and safety in monotherapy as well as strong response rates when combined with checkpoint inhibitors. Given CG0070’s combination results with strong complete response rates in heavily pre-treated BCG-unresponsive, high-risk NMIBC patients, there has been significant demand within the urologic community for CG0070 to also be investigated in the BCG-naïve NMIBC setting, where limited treatment options have been exacerbated by the global BCG shortage impacting both providers and patients. In response, CG Oncology has decided to investigate the activity of CG0070 as a first-line treatment of BCG-naïve, intermediate-risk NMIBC in a Phase 2 study to expand clinical development for additional patients with bladder cancer.

About CG0070

Our lead candidate, CG0070, is an intravesically delivered oncolytic immunotherapy agent in a Phase 3 trial for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. CG0070 is also in a Phase 2 study in combination with KEYTRUDA (pembrolizumab) in the same indication. Other types of bladder cancer are being evaluated with CG0070 in combination with OPDIVO (nivolumab).