Prenatal vitamins might lower risk of second child with autism

Medical Xpress | February 27, 2019

Something as simple as taking prenatal vitamins during the first month of pregnancy might lower the odds of having a second child with autism. As researchers explain in a new report, once one child has been diagnosed with autism, any subsequent children face a higher risk of having the developmental disorder. But the study found that when moms in high-risk families took prenatal vitamins during the first month of pregnancy, their children had half the risk of developing an autism spectrum disorder (ASD). And in children who did develop autism, early prenatal vitamin use was linked to less severe autism symptoms, and higher thinking and memory skills.
"Mothers who used prenatal vitamins, especially in the first month of pregnancy, had a reduced risk of having another child with autism," said study author Rebecca Schmidt. She's an assistant professor of public health sciences at the MIND Institute of the University of California, Davis.

Spotlight

For the first time, US scientists are attempting to use gene editing to cure a man with a rare disease known as Hunter syndrome. Brian Madeux, a 44-year-old California man is the first person in the US to receive the experimental treatment which involves injecting billions of copies of a corrective gene along with zinc finger nucleases, which are enzymes designed to replace the faulty gene in his genome with the correct one.

Spotlight

For the first time, US scientists are attempting to use gene editing to cure a man with a rare disease known as Hunter syndrome. Brian Madeux, a 44-year-old California man is the first person in the US to receive the experimental treatment which involves injecting billions of copies of a corrective gene along with zinc finger nucleases, which are enzymes designed to replace the faulty gene in his genome with the correct one.

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AI

Iktos Partners with Kadmon to Use AI for New Drug Design

Iktos, Kadmon | May 19, 2021

Iktos, a company specializing in Artificial Intelligence for new drug design, announced today that it has signed a Research Collaboration Agreement with Kadmon, a clinical-stage biopharmaceutical company based in New York, USA, under which Iktos' generative modeling artificial intelligence (AI) technology will be used to allow the rapid and cost-effective design of novel drug candidates. Iktos will use its de novo structure-based generative modeling technologies to find novel compounds that meet a pre-defined target product profile as part of the deal, to speed up Kadmon's early-stage discovery efforts. Kadmon discovers, develops, and delivers small molecules and biologics for the treatment of human diseases. Intending to identify and develop new product candidates for significant unmet medical needs, Kadmon is expanding and incorporating novel drug discovery platforms. The AI technology developed by Iktos, which is focused on deep generative models, aids in the speed and efficiency of the drug discovery process. Iktos' technology creates virtual novel molecules that have all of the properties of a successful drug molecule automatically. This approach, which has been validated by Iktos' other collaborations, is an innovative approach to one of the most difficult problems in drug design: finding molecules that meet several important drug criteria at the same time, such as potency, selectivity, safety, and project-specific properties. Iktos' technology enables the creation of new hits with optimal protein-ligand interactions in early-stage discovery projects, as predicted by molecular modeling technology. This technique allows for a one-of-a-kind discovery of chemical space, as well as the development of innovative molecule designs with greater Freedom to Operate. Furthermore, allowing multi-parametric in silico optimization from the start of a project greatly reduces the hit finding and hit-to-lead optimization phases. About Iktos Iktos, a French start-up founded in October 2016, specializes in the development of artificial intelligence technologies for chemical research, especially medicinal chemistry, and new drug design. Iktos is working on a proprietary and innovative approach focused on deep learning generative models that allow users to build molecules in silico that follow all of the performance criteria of a small molecule discovery project using existing evidence. Iktos technology allows for significant efficiency gains in upstream pharmaceutical R&D. Iktos' software is utilized as both professional services and a SaaS software platform, Makya. Spaya, a synthesis planning software built on Iktos' proprietary AI technology for retrosynthesis, is also in the works.

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CELL AND GENE THERAPY

Imara Announces Primary Endpoint Change in the Ardent Phase 2b Clinical Trial of Tovinontrine (IMR-687) in Sickle Cell Disease

Imara | November 22, 2021

Imara Inc. a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin and other serious diseases, today announced a change to the primary endpoint for the Ardent clinical trial, a Phase 2b study of tovinontrine (IMR-687) in patients with sickle cell disease (SCD), based on the recommendation of the U.S. Food and Drug Administration (FDA). Imara requested feedback from the FDA on the draft statistical analysis plan (SAP) for the Ardent trial in which fetal hemoglobin (HbF) response was the primary endpoint and annualized rate of vaso-occlusive crises (VOCs) was the key secondary endpoint. In reviewing the Ardent draft SAP and prior to any database lock for analysis, the FDA recommended that Imara change the primary endpoint to be annualized rate of VOCs. HbF response will continue to be evaluated as a key secondary endpoint. The endpoint revisions do not affect the conduct of the trial or operational aspects of the study. As part of its recommendation, the FDA suggested further interactions regarding the revised SAP and engagement on the potential of the current program for regulatory decision-making. “We welcome the FDA’s recommendations and are in the process of changing the primary endpoint of the Ardent trial to be annualized rate of VOCs and moving HbF response to be a key secondary endpoint. A reduction in VOC rate is an established approval endpoint, and we are engaging the FDA further on this and related topics, including possible streamlined paths to registration.” Rahul Ballal, Ph.D., President and Chief Executive Officer of Imara Dr. Ballal continued, “In light of this endpoint revision, the previously planned fourth quarter interim analysis will no longer occur. That interim analysis had been designed to have a focus on safety and pharmacodynamic biomarkers, including HbF, but did not include a review of VOCs. The first review of data from the Ardent trial, including annualized VOC rate, will be conducted when all subjects have completed assessment at Week 24 or terminated early, and is planned for the first quarter of 2022, subject to our upcoming discussions with the FDA. Final data analysis from the Ardent trial remains on track for the second half of 2022. In June 2021, we reported promising data from our Phase 2a and open label extension clinical trials in SCD that demonstrated reduced annualized rates of VOCs in patients treated with tovinontrine versus placebo. We expect to present updated 12-month VOC data from our ongoing Phase 2a open label extension clinical trial at the American Society of Hematology Annual Meeting in December 2021.” About the Ardent Phase 2b Clinical Trial The Ardent Phase 2b clinical trial is a fully-enrolled, global, randomized, double-blind, placebo-controlled, multicenter study with approximately 115 adult patients with sickle cell disease (SCD) enrolled. The planned primary efficacy objective will be to evaluate the annualized rate of vaso-occlusive crises (VOCs) in patients dosed with tovinontrine (IMR-687) as compared to placebo. A key secondary endpoint will be to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an absolute increase from baseline of at least 3% in HbF, as compared to placebo. Additional endpoints include the evaluation of the effect of tovinontrine versus placebo on other VOC-related outcome measures, HbF-associated biomarkers, markers of red blood cell hemolysis, white blood cell adhesion markers and quality of life measures over the course of a one-year treatment period. The FDA has granted Orphan Drug, Fast Track and Rare Pediatric Disease designations and the European Commission has granted Orphan Drug designation for tovinontrine for the treatment of SCD. About Tovinontrine (IMR-687) Tovinontrine is a highly selective and potent small molecule inhibitor of phosphodiesterase-9 (PDE9). PDE9 selectively degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology and hemoglobin production in red blood cells. Lower levels of cGMP are found in people with sickle cell disease (SCD) and beta-thalassemia and are associated with reduced blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide mediated vasodilation. Blocking PDE9 acts to increase cGMP levels, which is associated with several benefits including the potential reactivation of fetal hemoglobin (HbF), a natural hemoglobin produced during fetal development. Increased levels of HbF in RBCs have been demonstrated to improve symptomology and substantially lower disease burden in both patients with SCD and patients with beta-thalassemia. About Sickle Cell Disease Sickle cell disease (SCD), a hemoglobinopathy, is a rare inherited red blood cell disorder. The disease causes structural abnormalities in hemoglobin that cause red blood cells to become inflexible and elongated, ultimately blocking blood flow to organs, which can lead to vaso-occlusive crises (VOCs). SCD is characterized by debilitating pain, progressive multi-organ damage and early death. The global prevalence of SCD is estimated to be approximately 4.4 million patients, including an estimated 100,000 patients in the United States and 134,000 patients in the European Union. About Imara Imara Inc. is a clinical-stage biotechnology company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin and other serious diseases. Imara is advancing tovinontrine (IMR-687), a highly selective, potent small molecule inhibitor of PDE9 that is an oral, potentially disease-modifying treatment currently in clinical development for sickle cell disease and beta-thalassemia and preclinical development for heart failure with preserved ejection fraction, or HFpEF. Imara is also advancing IMR-261, an oral activator of nuclear factor erythroid 2–related factor 2, or Nrf2. Cautionary Note Regarding Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to (i) the Company’s plans to change the primary and secondary endpoints for the Ardent Phase 2b clinical trial of tovinontrine (IMR-687), (ii) the timing for reporting of additional data from the Ardent Phase 2b and open label extension clinical trials of tovinontrine in patients with sickle cell disease and (iii) the Company’s planned discussions with the FDA regarding the regulatory pathway for tovinontrine. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the impact of extraordinary external events, such as the risks and uncertainties resulting from the impact of the COVID-19 pandemic on the Company’s business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities and ability to readout data from the Ardent Phase 2b and open label extension clinical trials of tovinontrine in sickle cell disease; the Company’s ability to advance the development of tovinontrine under the timelines it projects in current and future clinical trials, demonstrate in any current and future clinical trials the requisite safety and efficacy of tovinontrine; and other factors discussed in the “Risk Factors” section of the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

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MEDICAL

Sotira Announces the Successful Completion of Second Phase of Pre-Clinical Testing for COVID-19 Therapeutic and Vaccine-Alternative

Sotira | November 25, 2020

Sotira, the Phoenix based biotech organization, is pleased to report the successful finish of the second period of pre-clinical testing for its COVID-19 therapeutic and vaccine-alternative, KEPTIDE™ COVID, confirming that this cutting-edge molecular therapy completely prevented of SARS-CoV2 in the lungs and kidney. These mice are viewed as a best quality level preclinical model to study COVID-19 on the grounds that the mice express human ACE2, the receptor utilized by the virus to gain entry to cells. The Sotira scientific group additionally found that the intranasal treatment of KEPTIDE™, 30 minutes before viral organization, secures wounds in lungs and kidney, yet additionally forestalls intense demise reaction in these humanized mice. Founded by James Keating, Sotira is a pharmaceutical and therapeutic company dedicated to developing cutting-edge molecular therapy to treat and prevent diseases. Led by its proprietary, patent-pending KEPTIDE™ technology, Sotira specializes in pharmaceutical preparations and substances for the treatment of infectious diseases, blood disorders, pain, inflammation, sepsis, alopecia, obesity, cognitive disorders, respiratory diseases, asthma, and of course the prevention of SARS-CoV2 (aka COVID-19).

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