Multiple Sulfatase Deficiency Disease

RAHWAY, N.J., Feb. 7, 2024 /PRNewswire/ -- Multiple sclerosis (MS) may be a common disorder, but the journey to diagnosis and understanding of the illness may still lead to confusion for some patients. In a new editorial from MerckManuals.com, Michael C. Levin, MD, College of Medicine, University of Saskatchewan, breaks down what patients and their families should know about MS, in order to educate themselves and prepare for conversations with a doctor. 1. Several factors increase the likelihood of being diagnosed with MS The cause of MS is unknown, but some studies point to early exposure to certain viruses in triggering the immune system to attack the body's own tissue. In fact, recent research looked at 955 active-duty military personnel diagnosed with MS and found the risk of MS increased 32-fold after infection with the Epstein-Barr virus, which is a type of herpes virus. 2. Signs and symptoms vary – and may go away on their own MS can affect any part of the brain or spinal cord, and symptoms can vary significantly from person to person and from episode to episode. Early symptoms may include: Tingling, numbness, pain, burning, and itching in the arms, legs, trunk, or face and sometimes a reduced sense of touch Loss of strength or dexterity in a leg or hand, which may become stiff Problems with vision Symptoms may come on (known as relapses) and lessen (known as remission), seemingly without rhyme or reason. 3. Treatment can significantly reduce relapses Individuals without a formal diagnosis may be tempted to ignore their illness, especially if symptoms have waned, says Levin. But he adds it's crucial to work to uncover the cause of symptoms and discuss them with a healthcare professional. Once MS is diagnosed, treatment can significantly extend periods of remission. On average, most individuals experience a relapse every one or two years. But with treatment, that can be extended to 10 or even 20 years between relapses, says Levin. Based on the condition and its symptoms, neurologists can guide patients toward the most effective treatments and medications. You can read more about MS in this editorial on MerckManuals.com. About The Merck Manuals and MSD Manuals First published in 1899 as a small reference book for physicians and pharmacists, The Merck Manual grew in size and scope to become one of the world's most widely used comprehensive medical resources for professionals and consumers. As The Manual evolved, it continually expanded the reach and depth of its offerings to reflect the mission of providing the best medical information to a wide cross-section of users, including medical professionals and students, veterinarians and veterinary students, and consumers. In 2015, The Manuals kicked off Global Medical Knowledge 2020, a global initiative with the goal to make the best current medical information accessible by up to three billion professionals and patients around the world by 2020. The Manuals achieved that goal, and today its medical information is available in nearly 250 countries and in 14 languages. It's continuing its ambitious mission through outreach, education and creating new reliable medical resources. For access to thousands of medical topics with images, videos and a constantly expanding set of resources, visit MerckManuals.com or MSDManuals.com and connect with us on social media: For Consumers in the U.S. and its territories: X (formerly Twitter) and Facebook. For Professionals in the U.S. and its territories: X (formerly Twitter) and Facebook About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn. SOURCE The Merck Manuals

Positive Phase 2b data announced for BNC210 on reduction of total symptom severity and multiple symptom clusters in post-traumatic stress disorder (PTSD), with a discussion with the U.S. Food & Drug Administration (FDA) anticipated in Q2’24. Positive End of Phase 2b (EoP2) meeting with FDA announced for BNC210 in Social Anxiety Disorder (SAD) following the Phase 3-enabling PREVAIL Phase 2 trial data readout. The Company is actively engaged in strategic partnership discussions with interested parties for multiple pipeline assets and is evaluating options to proceed with clinical development plans for BNC210 in both PTSD and SAD. ADELAIDE, Australia and CAMBRIDGE, Mass., Jan. 22, 2024 (GLOBE NEWSWIRE) -- Bionomics Limited (Nasdaq: BNOX) (Bionomics or Company), a clinical-stage biotechnology company developing novel, first-in-class, allosteric ion channel modulators to treat patients suffering from serious central nervous system (CNS) disorders with high unmet medical need, today provided a 2023 review and its outlook for 2024. “Bionomics continued its evolution into a U.S.-focused, late-stage clinical company last year. We finished 2023 with strong momentum from our positive readout in Phase 2b ATTUNE trial in PTSD and the successful EoP2 meeting with the FDA, paving the path to a registrational study in SAD. These two BNC210 programs generated significant interest from potential strategic partners, including the opportunity to co-develop and commercialize BNC210 in PTSD and SAD in the U.S. and abroad. We will explore such potential opportunities in parallel with other pathways that provide near and long-term value to the Company and its shareholders. Additionally, we’ve been receiving interest in our promising preclinical assets, KV3.1/3.2 channel activators and Pan Nav inhibitors, where we have identified lead candidates and continued our work on the 2nd generation α7 nicotinic receptor negative allosteric modulator (NAM) lead series,” commented Spyros Papapetropoulos, M.D., Ph.D., President and CEO of Bionomics. 2023 Highlights Key Clinical Highlights BNC210 in Social Anxiety Disorder and Post-Traumatic Stress Disorder A first and best-in-class α7 nicotinic, Phase 3-ready NAM of the α7 nicotinic receptor with a unique and differentiated psychoactive profile suitable for acute and chronic dosing in multiple CNS indications: Post-Traumatic Stress Disorder Positive topline results from the Phase 2b ATTUNE study were announced in September 2023, meeting the primary endpoint on reduction in total symptom severity and highlighting the potential of BNC210 to improve multiple PTSD symptom clusters with notable effects on thought intrusions, mood, trauma-related cognition, and sleep. The BNC210 program is the only non-psychedelic program with a novel mechanism of action to advance in late-stage development in recent years. Social Anxiety Disorder BNC210 may become the first intermittent “as-needed” non-sedating, non-habit forming, cognitively non-impairing anxiolytic for the treatment of anxiety in patients with SAD.In March 2023, the Company completed, on time and within budget, the full data analysis of the Phase 2b PREVAIL study demonstrating that BNC210 reduced anxiety during a public speaking challenge and provided support for further evaluation in Phase 3 clinical trials.A successful EoP2 meeting with the FDA in September 2023 confirmed the Phase 3 enabling nature of the PREVAIL dataset and clarified the plans for the registrational program for BNC210 in SAD, including the number of trials, trial designs, endpoints, sample sizes, size of the safety database and nonclinical requirements for registration. The results of the Phase 2b trials of BNC210 in SAD and PTSD, along with previous results observed in earlier trials in general anxiety disorder (GAD) and panic attacks, underscores BNC210’s potential in multiple, highly prevalent neuropsychiatric indications with significant market opportunity. Positive Allosteric Modulators (PAMs) of the α7 Nicotinic Acetylcholine Receptor Program with Merck & Co., Inc.The ongoing partnership with MSD* on α7 PAMs for the treatment of cognitive disorders such as Alzheimer's disease (AD) and cognitive impairment associated with schizophrenia (CIAS) has generated two clinical candidates that are currently in late Phase 1 clinical development after completing several Phase 1 safety, tolerability, pharmacokinetic and biomarkers studies. The partnership has a potential value of $500m in regulatory and clinical milestones with additional potential royalties from commercialization. Key Corporate Highlights In support of the Company’s transformation into a commercial-stage company, expanded the U.S.-based team with new highly experienced management appointments, positioning it to become a leading company in neuropsychiatry.Voluntarily delisted from the Australian Stock Exchange in August 2023 and is currently solely listed on the NASDAQ Global Market.Further reduced cash burn through efficient capital allocation.Maintained its current institutional investors’ support while adding new institutional investors to its register.Sustained strong analyst coverage that highlights the Company's potential future value.The Company has no outstanding debt, convertible securities, or warrants.Continued strengthening BNC210 intellectual property portfolio in the U.S. and abroad. Future Anticipated Milestones for BNC210 Clinical Program Bionomics continues to plan the initiation of its first Phase 3 study of BNC210 in patients with SAD.The Company plans to discuss a potential dose-ranging late-stage study of BNC210 in PTSD with the FDA; an update on the next steps is expected by the end of Q2'24.The Company is exploring the submission of a request for Breakthrough Therapy designation in PTSD in Q2’24.Leveraging the inbound demand, the Company is looking to secure a strategic partnership for BNC210. “We look forward to continuing to strongly execute, in a timely and fiscally efficient manner, and keep progressing our BNC210 programs in SAD and PTSD while also aiming to establish strategic partnerships in the coming months. Following a transformational 2023 where we delivered on all anticipated milestones, I am confident 2024 will be an exciting year for Bionomics as we keep making strides towards building shareholder value through the development of new treatment options for patients in two psychiatric disorders with significant unmet needs and market potential,” concluded Dr. Papapetropoulos. * Known as MSD outside the United States and Canada FOR FURTHER INFORMATION PLEASE CONTACT: GeneralRajeev Chandra Company SecretaryCoSec@bionomics.com.auInvestor RelationsKevin Gardnerkgardner@lifesciadvisors.comInvestor RelationsChris Calabreseccalabrese@lifesciadvisors.com About Bionomics LimitedBionomics (NASDAQ:BNOX) is a clinical-stage biotechnology company developing novel, first-in-class, allosteric ion channel modulators to treat patients suffering from serious central nervous system (“CNS”) disorders with high unmet medical need. Bionomics is advancing its lead drug candidate, BNC210, an oral, proprietary, selective negative allosteric modulator of the α7 nicotinic acetylcholine receptor, for the acute treatment of Social Anxiety Disorder (SAD) and chronic treatment of Post-Traumatic Stress Disorder (PTSD). Beyond BNC210, Bionomics has a strategic partnership with Merck & Co., Inc. (known as MSD outside the United States and Canada) with two drugs in early-stage clinical trials for the treatment of cognitive deficits in Alzheimer’s disease and other central nervous system conditions. Bionomics’ pipeline also includes preclinical assets that target Kv3.1/3.2 and Nav1.7/1.8 ion channels being developed for CNS conditions of high unmet need. www.bionomics.com.au Forward-Looking StatementsBionomics cautions that statements included in this press release that are not a description of historical facts are forward-looking statements. Words such as “may,” “could,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “intend,” “predict,” “seek,” “contemplate,” “potential,” “continue” or “project” or the negative of these terms or other comparable terminology are intended to identify forward-looking statements. These statements include the Company’s plans to advance the development of its product candidates, the timing of achieving any development or regulatory milestones, and the comparability and potential of such product candidates, including to achieve any benefit or profile or any product approval or be effective. The inclusion of forward-looking statements should not be regarded as a representation by Bionomics that any of its plans will be achieved. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in the Company’s business and other risks described in the Company’s filings with the Securities and Exchange Commission (SEC), including, but not limited to, the Company’s Annual Report on Form 20-F filed with the SEC, and its other reports. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Bionomics undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. Further information regarding these and other risks is included in Bionomics’ filings with the SEC which are available from the SEC’s website (www.sec.gov) and on Bionomics’ website (www.bionomics.com.au) under the heading “Investor Center.” All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.

Nearly all cervical cancer cases are linked to infection with high-risk human papillomaviruses (HPV). Image credit: Shutterstock/Naeblys. The US Food and Drug Administration (FDA) has expanded Keytruda’s use (pembrolizumab) in combination with chemoradiotherapy to certain advanced types of cervical cancer. Patients with FIGO (International Federation of Gynecology and Obstetrics) 2014 Stage III-IVA cervical cancer will now be eligible for MSD’s blockbuster immunotherapy. MSD states the FDA nod means its drug is the only anti-PD-1 therapy and chemoradiotherapy combination approval for newly diagnosed US patients with high-risk locally advanced cervical cancer, according to a 12 January press release. Keytruda already has two FDA approvals in cervical cancer for patients who express PD-L1 tumours. One as a first-line therapy in combination with chemotherapy, with or without bevacizumab, the other as a single agent second-line therapy in patients for whom chemotherapy has not worked. The agency based its decision on results from the Phase III KEYNOTE-A18 trial (NCT04221945). Patients who received the combo therapy demonstrated a progression-free survival (PFS) – the risk of disease progression or death was reduced by 41%. MSD said the FIGO 2014 Stage III-IVA disease sub-group was the main driver of PFS improvement. The trial enrolled 1,0060 cervical cancer patients who had not received surgery, radiation, or systemic therapy for treatment. Cervical cancer is the fourth most common cancer in women globally, according to the World Health Organisation. Most cases are linked to infection with high-risk human papillomaviruses (HPV). MSD Research Laboratories’ global clinical development vice president Dr. Gursel Aktan said: “This approval provides newly diagnosed patients with an anti-PD-1-based treatment option that has the potential to reduce the risk of disease progression or death compared to chemoradiotherapy alone.” Keytruda is MSD’s best-selling drug and is one of the most lucrative drugs worldwide. In the US, it is now approved in 39 indications.  In 2022, Keytruda generated $20.9bn in global sales for MSD, up 22% from the year before. Keytruda is set to lose its patent protection in 2028, however, with the market likely to become flooded with lower-cost biosimilars, echoing what happened with AbbVie’s Humira (adalimumab). GlobalData’s Pharma Intelligence Centre forecasts peak sales of $33.7bn in 2028. GlobalData is the parent company of Pharmaceutical Technology . MSD has been busy shoring up future revenue streams in oncology as a result, with the big pharma acquiring T-cell engager specialist Harpoon Therapeutics for $680m in January 2024. In October 2023, the company also entered an agreement to develop and commercialise three of Daiichi Sankyo ’s antibody drug conjugate (ADC) in a deal that could be worth up to $22bn .