Marinus Pharmaceuticals | July 31, 2020
Marinus Pharmaceuticals, Inc., a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD), a rare refractory form of pediatric epilepsy.
“We are pleased that ganaxolone has received Rare Pediatric Disease Designation from the FDA and are enthusiastic that the timing of this news aligns with our continued expectation to report top line data this quarter from our pivotal Phase 3 Marigold Study evaluating oral ganaxolone in patients with CDD,” said Scott Braunstein, M.D., Chief Executive Officer at Marinus. “This designation for ganaxolone for CDD underscores the significant unmet medical need for children and young adults with this serious and rare genetic disease, which causes early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Our goal is to advance a pipeline of programs that can treat rare seizure disorders, including CDD, tuberous sclerosis complex and PCDH19-related epilepsy, and bring much-needed medicines to patients as soon as possible.”
Algernon Pharmaceuticals, Charles River Laboratories | February 11, 2021
Algernon Pharmaceuticals Inc. a clinical stage pharmaceutical development company is pleased to announce that is has signed an agreement with Charles River Laboratories for preclinical studies of AP-188 ("N,N-Dimethyltryptamine or DMT") for the Company's stroke clinical research program. Algernon's preclinical study of DMT will be conducted at the Charles River research facility in Finland.
Charles River Laboratories, Inc. is an American corporation specializing in a variety of preclinical and clinical laboratory services for the pharmaceutical, medical device and biotechnology industries. It also supplies assorted biomedical products and research and development outsourcing services for use in the pharmaceutical industry.
Algernon recently established a clinical research program for the treatment of stroke zeroed in on DMT, a known psychedelic compound that is part of the tryptamine family. The Company plans to be the principal company globally to seek after DMT for stroke in humans and is planning to begin a clinical trial as soon as conceivable in 2021.
The Company's decision to investigate DMT, called "The Spirit Molecule," and move it into human trials for stroke, is based on various independent, positive preclinical studies demonstrating that DMT advances neurogenesis as well as structural and functional neural plasticity. These are key factors involved in the brain's ability to form and reorganize synaptic connections, which are needed for healing following a brain injury.
Unlike other companies recently researching psychedelic drugs, Algernon will be focusing on a sub-hallucinogenic, or microdose of DMT provided by continuous intravenous administration. By pursuing a continuous active microdose, the goal will be to provide patients with the therapeutic benefits of DMT, without having a psychedelic experience. This is an important element when considering treating a patient who has quite recently endured a stroke, wherein medications that cause a hallucinogenic response would cause unwanted confusion and stress.
N,N-Dimethyltryptamine, or DMT, is a hallucinogenic tryptamine drug producing impacts similar to those of other psychedelics like LSD, ketamine, psilocybin and psilocin. DMT happens naturally in many plant species and animals and has been utilized in strict ceremonies as a traditional spiritual medicine by indigenous individuals in the Amazonian basin. DMT can also be synthesized in a laboratory.
At higher portions, DMT has a rapid onset, intense psychedelic impacts, and a relatively short duration of action with an estimated half-existence of under fifteen minutes. Like other hallucinogens in the tryptamine family, DMT binds to serotonin receptors to deliver euphoria and psychedelic impacts. Because the impacts of DMT don't last long, it has been alluded to in certain circles as the "businessman's outing".
Named the "Spirit Molecule" by Dr. Rick Strassman, an American clinical associate professor of psychiatry and DMT research pioneer, and Algernon consultant, DMT has been shown to induce neuroplasticity in a number of key preclinical studies DMT is believed to activate pathways involved with forming neuron connections and has been shown in studies to increase the number of dendritic spines on cortical neurons. Dendritic spines form synapses (connections) with other neurons and are a major site of molecular activity in the brain.
While Dr. Strassman's Phase 1 bolus intravenous human study identified the sub-hallucinogenic portion of DMT in humans, another preclinical animal study demonstrated this same portion level actually retains the neuroplastic impact seen in higher hallucinogenic dosages.
Algernon will be investigating an intravenous sub-hallucinogenic portion of DMT in its research and clinical studies.
About Algernon Pharmaceuticals Inc.
Algernon is a drug re-purposing company that investigates safe, already approved drugs, including naturally occurring compounds, for new disease applications, moving them efficiently and safely into new human trials, developing new formulations and seeking new regulatory approvals in global markets. Algernon specifically investigates compounds that have never been approved in the U.S. or Europe to avoid off label prescription writing.
Pfizer | July 22, 2021
Pfizer Inc. and BioNTech SE today announced the signing of a letter of intent with The Biovac Institute (Pty) Ltd, also known as "Biovac," a South African biopharmaceutical business headquartered in Cape Town, to produce the Pfizer-BioNTech COVID-19 Vaccine for sale inside the African Union.
Biovac will manufacture and distribute COVID-19 vaccines as part of Pfizer and BioNTech's worldwide COVID-19 vaccine supply chain and production network, which currently spans three continents and includes more than 20 facilities. Technical transfer, on-site development, and equipment installation operations will begin immediately to enable Biovac's participation in the process.
Biovac's Cape Town plant is expected to be integrated into the vaccine supply chain by the end of 2021, according to Pfizer and BioNTech. Biovac will acquire drug substances from European facilities, and final dosage manufacturing will begin in 2022. Annual production will reach 100 million finished dosages at full operating capacity. All dosages will be delivered solely inside the African Union's 55 member states.
Quality, compliance, safety track record, technical capability, capacity availability, highly trained workforce, project management abilities, prior working relationship, and commitment to working with flexibility through a fast-paced program are all factors that Pfizer and BioNTech consider when selecting contract manufacturers. Since 2015, Pfizer and Biovac have collaborated on the Prevenar 13 vaccine's sterile formulation, fill, finish, and distribution.
Pfizer and BioNTech have delivered more than 1 billion doses of COVID-19 vaccine to more than 100 nations and territories across the globe. The companies are firmly committed to ensuring that all people around the world have equitable and affordable access to COVID-19 vaccines, actively working with global governments and global health partners to provide 2 billion doses to low and middle-income countries in 2021 and 2022 – 1 billion each year. This comprises a non-profit arrangement to provide 500 million doses to the US government, which the government will then give to the African Union and the COVAX 92 Advanced Market Commitment (AMC) nations, as well as a 40 million dose direct supply deal with the COVAX facility.
Both BioNTech and Pfizer worked together to create the Pfizer-BioNTech COVID-19 Vaccine, which is based on BioNTech's unique mRNA technology. In addition to the European Union, BioNTech has emergency use authorizations or equivalent in the United States (jointly with Pfizer), Canada, and other countries in advance of a planned application for full marketing authorizations in these countries.
The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the US Food and Drug Administration (FDA), but it has been approved for emergency use by FDA under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID-19), which is caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in people aged 12 and up. Unless the declaration is ended or the permission withdrawn sooner, the emergency use of this product is only allowed for the duration of the declaration that conditions exist justifying the authorization of emergency use of the medical product under Section 564 (b) (1) of the FD&C Act.