Ionis’ FCS drug approved in EU, launch due in Germany

pharmaphorum | May 08, 2019

Ionis Pharmaceuticals’ RNA-targeted drug Waylivra has been conditionally approved in Europe for the devastating ultra-rare disease Familial Chylomicronemia Syndrome (FCS). The European Commission’s conditional approval means Ionis and its affiliate Akcea will have to re-apply for a marketing authorisation each year for Waylivra (volanesorsen), until they can present confirmatory data to the regulator. As part of the conditional marketing authorisation, Akcea and Ionis will conduct a non-interventional post-authorisation safety study (PASS) based on data from a registry. Waylivra’s approval is based on a positive opinion from the European Medicines agency’s Committee for Medicinal Products Human Use (CHMP) earlier this year. This ran contrary to a decision from the FDA, which rejected it earlier this year, setting up a situation where Waylivra will be available in Europe but not the US. There was no immediate word from Ionis about the price of the drug, and this is likely to vary depending from one member state to the next.

Spotlight

The developmental versatility of pluripotent stem cells (PSCs) offers a powerful approach for directing cell fate, and these cells are a promising source of progenitors and terminally differentiated somatic cells for cell replacement therapy and drug screening applications. In culture, PSCs have a unique trait of unlimited propagation while retaining their inherent potential to differentiate into cells from the three embryonic germ layers. When plated onto a non-adherent culture dish, these cells aggregate to form embryoid bodies (EBs) which undergo a process of spontaneous differentiation that closely imitates early human embryogenesis.

Spotlight

The developmental versatility of pluripotent stem cells (PSCs) offers a powerful approach for directing cell fate, and these cells are a promising source of progenitors and terminally differentiated somatic cells for cell replacement therapy and drug screening applications. In culture, PSCs have a unique trait of unlimited propagation while retaining their inherent potential to differentiate into cells from the three embryonic germ layers. When plated onto a non-adherent culture dish, these cells aggregate to form embryoid bodies (EBs) which undergo a process of spontaneous differentiation that closely imitates early human embryogenesis.

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RESEARCH

Nanoform Releases Next-Generation AI-based Drug selection Tool

Nanoform | March 23, 2021

Nanoform innovative nanoparticle medicine enabling company next generation of its STARMAP® AI artificial intelligence platform nanoparticle bioavailability excipient microns nm CESS technology silico pharma drugs lifecycle managementNanoform, an innovative nanoparticle medicine enabling company, has today dispatched the next generation of its STARMAP® AI (artificial intelligence) platform, v2.0. The technology uses scanty information AI to expand trial results from its CESS® nanoparticle designing process with detailed master information, permitting dependable expectations to be made in regards to accomplices' likely success of nanoforming their medication atoms. CESS® is a nanoparticle platform technology which produces unadulterated homogeneous medication particles from arrangement in a without excipient process. By diminishing the molecule size e.g., from 10 microns to 50 nm, the particular surface territory can be expanded by as much as 1000-crease, subsequently improving disintegration rate, solvency, and bioavailability. Subsequently, Nanoform can help pharma accomplices progress atoms into advancement that in any case might not have been conceivable. It additionally opens up energizing opportunities for a wide scope of novel medication conveyance applications. STARMAP® is a computerized form of the CESS® technology that empowers in silico tries in enormous amounts, making quick forecasts of which atoms ought to be nanoformed. This is significant since there are more potential medication atoms than particles in the known universe. STARMAP® can be a useful asset for pharma accomplices to pick appropriate medication contender for additional improvement from their huge libraries. The benefits may incorporate quicker way to advertise and additional opportunities for widening and extending drug pipelines while at the same time expanding the likelihood of medication improvement success. The STARMAP® platform can have wide materialness in drug disclosure and advancement just as in lifecycle management for existing promoted drugs and 505b2-like item improvement methodologies. "AI algorithms developed for big data have so far struggled to live up to expectations in pharma because the data, especially for early assets (drug discovery, drug screening), that is available to pharma is typically insufficient for generating reliable predictions. We believe sparse-data AI will work much better - in practice, this means augmenting experimental results with detailed expert knowledge, which can be used to prevent the AI from predicting outcomes that are nonsensical based on prior understanding. There is a lot of untapped potential in sparse-data AI for the pharma industry and the field continues to undergo rapid development in both academia and the industry in general," said Prof. Jukka Corander, Head of AI at Nanoform. "By determining which drug candidates are ideal for our CESS® process, the next-gen STARMAP® platform can potentially create new opportunities for our pharma partners. These can include both revisiting drug candidates unnecessarily discarded by AIs trained on old particle engineering techniques, and rapidly picking winners among new drug candidates. Ultimately, the benefit of more advanced AI will be felt by patients as new therapies are accelerated to market," commented Christian Jones, Chief Commercial Officer at Nanoform

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BUSINESS INSIGHTS

ILC Dover Becomes a Provider of Ascent / Entry Spacesuits for Boeing's Starliner

ILC Dover | May 27, 2022

ILC Dover, the leading provider of spacesuits since the Apollo era, announced that it was selected to be one of two providers of Boeing's Ascent/Entry Suit for the company's Commercial Crew Program and has developed a bespoke AES spacesuit for CST-100 Starliner crews. "We are proud to utilize our expertise to create a new AES for the Starliner spacecraft. Having worked with the Boeing team on the Starliner's landing airbags, which provide a safe, cushioned arrival to Earth from space, we understood the importance of delivering another quality product for this program." Patty Stoll, President of Space Systems and Engineered Solutions at ILC Dover The Boeing AES is based off ILC Dover's commercial Launch, Entry, and Abort suit, SOL™. ILC Dover worked with Boeing to tailor SOL for the Starliner spacecraft to provide protection for astronauts during the most critical phases of spaceflight, including launch, docking, re-entry and landing. With over 50 years of spacesuit experience, the AES suit was designed to provide maximum mobility to operate, enter and exit the spacecraft, as well as provide protection for astronauts in case of an emergency. "Our success is measured by those we've helped return to Earth safely for over 50 years of space travel and we will continue to provide spacesuits and soft solutions that will keep astronauts safe in the harsh environment of space," said Stoll. Working with ILC Dover's in-house fashion designer, the cover layer of the spacesuit was designed with a unique look for Starliner crew members. A replica of the Starliner AES will be on display on Florida's Space Coast at the Kennedy Space Center Visitor Complex's new attraction called Gateway: The Deep Space Launch Complex. ILC Dover and Boeing worked with Adam Savage, television personality and longtime special effects designer and fabricator, to create the replica. About ILC Dover ILC Dover is a world-leader in the innovative design and production of solutions for biopharmaceutical, pharmaceutical, medical device markets as well as a leading supplier for the space industries. Our customers will attest to our relentless dedication to high value products, advanced technology, and responsive service, as our visionary solutions have improved efficiency while safeguarding people, product, and infrastructure in hazardous conditions through flexible protective solutions since 1947.

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PHARMACY MARKET

Virpax Announces MMS019 Manufacturing and Supply Agreement

Virpax Pharmaceuticals | August 27, 2021

Virpax® Pharmaceuticals, Inc., a company specializing in developing product candidates for pain management, CNS and anti-viral indications, today announced that it has entered into a commercial manufacturing and supply agreement with Seqens, an integrated global leader in pharmaceutical solutions with 24 manufacturing sites worldwide and seven research and development facilities throughout the U.S. and Europe. The agreement with Seqens provides for both the supply material for Virpax’s clinical studies as well as the long-term commercial supply of MMS019. Seqens will conduct process development and validation of additional large scale commercial quantities of MMS019 at its facilities in Devens and Newburyport, Massachusetts. “Establishing a collaboration with a strong partner capable of supplying clinical and commercial scale quantities of MMS019 is another important advancement in our MMS019 product development strategy. Seqens has a demonstrated expertise in developing and manufacturing highly-complex molecules for large scale production,” said Anthony Mack, Chairman and CEO of Virpax. “Importantly, we expect this collaboration to support future development and supply additional Molecular Envelope Technology programs under development, including Envelta™ and PES200, our post-traumatic stress disorder product candidate,” concluded Mr. Mack. About MMS019 MMS019 is a drug product candidate based on a type of nanotechnology that enables the exclusive delivery of a metabolically labile peptide drug into the brain via intranasal delivery. MMS019 is manufactured using industrially relevant equipment and processes (high pressure homogenization and spray drying). There is pharmacological evidence of activity of molecular envelope technology (MET) enabled enkephalin in morphine-tolerant animals. The MET nanoparticles are well tolerated via the nasal route at the dose administered. MMS019 demonstrated comparable preclinical activity to morphine in all animal pain models tested without the drug seeking and tolerance associated with opioids. About Seqens SEQENS is an integrated global leader in pharmaceutical solutions and specialty ingredients. With 24 production sites and 7 R&D centers in Europe, North America and Asia, SEQENS develops and manufactures tailor-made solutions and ingredients for the most demanding industries such as healthcare, electronics, cosmetics, food and home care. Driven by a culture of excellence and a strong entrepreneurial spirit, our 3,200 employees are committed to providing our customers with the highest level of service and quality while acting ethically in accordance with our corporate social responsibility program.” About Virpax Pharmaceuticals Virpax is developing branded, non-addictive pain management product candidates using its proprietary technologies to optimize and target drug delivery. Virpax is initially seeking FDA approval using its three patented drug delivery platforms. Epoladerm™ is a topical diclofenac metered-dose spray film formulation being developed to manage acute musculoskeletal pain and osteoarthritis. Probudur™ is a single injection liposomal bupivacaine formulation being developed to manage post-operative pain. Envelta™ is an intranasal molecular-envelope enkephalin formulation being developed to manage acute and chronic pain, including pain associated with cancer. Virpax is also using its intranasal Molecular Envelope Technology (MET) to develop its PES200 product candidate to manage post-traumatic stress disorder (PTSD) and its MMS019 product candidate to inhibit viral replication caused by influenza or SARS-CoV-2.

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