PALM BEACH, Florida, April 10, 2019 /PRNewswire/ -- According to the Centers for Disease Control, autism affects an estimated 1 in 59 children in the United States today. The Autism Speaks website defines the main problem for researchers/practitioners as: "We know that there is not one autism but many subtypes, most influenced by a combination of genetic and environmental factors. Because autism is a spectrum disorder, each person with autism has a distinct set of strengths and challenges. Medicines for treating the three core symptoms of autism – communication difficulties, social challenges and repetitive behavior – have long represented a huge area of unmet need. Unfortunately, few drugs on the market today effectively relieve these symptoms and none of the options most often prescribed by practitioners work well for every individual." The Autism Speaks organization also express optimism about the present state of research: "The good news is that the range of medication options may soon change, thanks to recent advances in our understanding of the biology that produces autism's core symptoms. This has made it possible for researchers to begin testing compounds that may help normalize crucial brain functions involved in autism. Early experiments suggest that several compounds with different mechanisms of action have great potential for clinical use, and many are now in clinical trials. Active biotech and pharma companies in the markets this week include: Q BioMed Inc. (OTC:QBIO), PhaseBio Pharmaceuticals, Inc. (NASDAQ: PHAS), Insmed Incorporated (NASDAQ: INSM), Chimerix, Inc. (NASDAQ: CMRX), Geron Corporation (NASDAQ: GERN).
Another industry website adds: "… the diversity of Autism-related genes and the chronic nature of the disease present numerous challenges to the development of treatments. That may change through the rise of powerful new technologies and a greater understanding of the mechanisms that cause ASD. So there is a lot still to hope for! Companies willing to tackle these issues will be able to draw on new technologies and benefit from the increasing public awareness of this class of disorders, which seriously disrupt the lives of affected children and their families."
Q BioMed Inc. (OTCQB:QBIO) BREAKING NEWS: Q BioMed, a commercial stage biotech company, announced today the discovery of two novel biomarkers for pediatric nonverbal autism, identified in a subset of children with Autism Spectrum Disorder (ASD). This marks the first time a company has been able to identify biomarkers that hold the potential to stratify this subset of children. The study took into consideration 1,953 potential biomarkers and used Vineland II scores to stratify 240 children into three groups: verbal, semi-verbal, and nonverbal autism.
Q BioMed CEO, Denis Corin highlighted, "This is a major breakthrough for these children and their families. To date, very little attention and research has been focused on these nonverbal autistic children. In partnership with the clinical and advocacy community, QBioMed is leading the effort to better stratify this group, while also pursuing a treatment."
Children of this subset typically never develop the ability to speak, or commence with language progression between 9-14 months and then regress. Most psychologists and physicians feel comfortable providing a diagnosis of nonverbal around the age of 7 years. Each year, an estimated 18,000-20,000 newborns will go on to become autistic and nonverbal in the U.S.
Speaking to the benefit of the biomarker work completed to date by Q BioMed, Corin explained, "Children with ASD need safe, effective and targeted treatments that are tested under conditions that take their best interests into consideration. Today's announcement ensures we continue to take a tailored and patient-centric approach to find those children who will benefit the most from our efforts."
One of the highlights of the biomarker study was the identification of known genes and pathways that are directly implicated in speech impairment. The studies also revealed comorbidities that manifest serologically as early as 18 months (the lower limit of detection to date) even though the children do not yet manifest clinical symptoms of these diseases. Read this entire release and more news for QBIO at: https://www.financialnewsmedia.com/news-qbio/
Other recent developments in the biotech industry include:PhaseBio Pharmaceuticals, Inc.
(NASDAQ: PHAS) a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapies for orphan diseases, recently announced that the U.S. Food and Drug Administration ("FDA") has granted Breakthrough Therapy designation for PB2452, a novel reversal agent for the antiplatelet drug ticagrelor.
Breakthrough Therapy designation is designed to expedite the development and review of promising new drugs for serious or life-threatening conditions, when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. With all the features of the Fast Track program, Breakthrough Therapy designation also offers companies the opportunity for increased communication with FDA and an organizational commitment involving more intensive guidance from FDA senior managers. Companies may also be eligible for Accelerated Approval and Priority Review, if relevant criteria are met.
Insmed Incorporated (NASDAQ: INSM) a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, recently provided an update on the U.S. launch of ARIKAYCE® (amikacin liposome inhalation suspension), including preliminary net product sales for the first quarter of 2019. ARIKAYCE was granted accelerated approval by the U.S. Food and Drug Administration (FDA) on September 28, 2018, for the treatment of Mycobacterium aviumcomplex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options.
"We continue to be very pleased with the U.S. launch progress of ARIKAYCE and the positive feedback received from the refractory MAC lung disease community," said Will Lewis, Chairman and Chief Executive Officer of Insmed.
Chimerix, Inc. (NASDAQ: CMRX) recently named Michael Sherman as CEO. Formerly, Sherman led Endocyte. In addition to Sherman, the company has also named former Endocyte CFO Michael Andriole to the newly created position of chief business officer.
"We are delighted to welcome Mike Sherman and Mike Andriole to our executive management team," said Martha Demski, chair of the Chimerix board of directors. "This pair has a proven track record of successfully developing valuable clinical assets, and we believe Chimerix will significantly benefit from the strategic and operational experience they will bring the Company. This seasoned team has demonstrated their ability to unlock value while bringing important therapeutics to patients in need."
Geron Corporation (NASDAQ: GERN) closed down slightly in trading on Tuesday at $1.82 trading over 10.05 Million shares but did close up over 5% in after hours trading at $1.91. Geron is a late-stage clinical biopharmaceutical company focused on the development and potential commercialization of a first-in-class telomerase inhibitor, imetelstat, in hematologic myeloid malignancies.
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