80 Clinical R&D DUTCH STEM CELL BIOTECH NEUROPLAST SECURES ¤10 MILLION (US$ 11.5 MILLION) IN SERIES B FUNDING TO FURTHER ADVANCE ITS TRANSFORMATIVE STEM CELL THERAPY FOR TRAUMATIC SPINAL CORD INJURY Dutch clinical phase biotech Neuroplast raised a total of ¤10 million (US$ 11.5 million) in funding from investors Lumana Invest, Brightlands Venture Partners, LIOF, and from the Innovation Credit from the Netherlands Enterprise Agency, to further advance the clinical development of a transformative treatment for Traumatic Spinal Cord Injury (TSCI). Neuroplast will use this Series B funding to obtain conditional EMA market approval for its Neuro-Cells® stem cell therapy. Annually, approximately 29,000 people across Europe and the USA suffer from acute Traumatic Spinal Cord Injury (TSCI), for which effective treatment is currently unavailable. Patients usually experience life-long disability and dependence, with a negative impact on quality of life. Furthermore, associated costs for society at large are estimated at over ¤11.4 billion ($13 billion) per year. With the aim of giving back perspective to people that suffer from primarily inflammation-driven neurological disorders, Neuroplast has developed Neuro-Cells®, a treatment that uses the patient's own stem cells to prevent (further) loss of function during the acute phase after sustaining damage to the spinal cord, to save mobility and independence. Marcel Kloosterman, Managing Partner at Brightlands Venture Partners (BVP), states, "The science behind Neuroplast's technology platform is truly groundbreaking. A future in which patients can make smart use of their own cells to put a stop to further neurological damages and improve potential regeneration is getting closer and closer. For BVP it is a thrill to be part of that fascinating journey." Neuroplast acquired a GMP[1] license and received a European Orphan Designation[2] that allows fast-track development. Furthermore, it has already successfully completed a clinical Phase I trial, in collaboration with Hospital Nacional de Parapléjicos in Spain, that confirmed safety and tolerability, without productrelated adverse events. The Dutch Limburg-based biotech is currently preparing for an international multi-center randomized placebocontrolled Phase II study.
SANOFI REITERATES CONFIDENCE IN STRONG GROWTH OUTLOOK AND SHOWCASES PIPELINE OF INNOVATIVE VACCINE CANDIDATES Sanofi has hosted a hybrid Vaccines Investor Event with key members of the Vaccines leadership team to discuss how the company applies the global Play to Win strategy to pursue growth opportunities and to build an industry-leading vaccines pipeline. Sanofi confirms its mid-term sales guidance announced at the 2019 Capital Markets Day of mid-tohigh single-digit growth1 for its Vaccines business. Sustained growth will be driven by four core franchises of influenza, meningitis, PPH3 & Boosters, as well as the planned launch of nirsevimab, a first-in-class monoclonal antibody for all infant protection against Respiratory Syncytial Virus. These core franchises are well positioned to capture market growth, such as in the global influenza market which Sanofi expects to exceed ¤15 billion by 2030. Sanofi has made significant progress in the field of mRNA technology with the recently established Center of Excellence and the integration of Translate Bio. Results from a first influenza mRNA clinical trial will be shared as well as initial successes in mRNA technology improvements. Of the 10 new vaccine candidates planned to enter the clinic by 2025, six will use mRNA technologies to target diseases with high unmet needs and disease burden such as chlamydia and acne. Sanofi will present the following assets in its growing R&D pipeline:
• A pneumococcal vaccine candidate covering 21 serotypes, with Phase 1/2 clinical trials underway in three target populations; results are expected by end of 2022. • A combination of MenQuadfi and a new meningitis B vaccine candidate to create a best-in-class pentavalent meningococcal vaccine with broader strain coverage; Phase 2 meningitis B read-out is expected by H2 2022 and the initiation of a Phase 1 trial for the pentavalent candidate is planned in 2023. • RSV vaccine candidates for toddlers and older adults to address a high unmet disease burden; Phase 1/2 trial results in toddlers are anticipated in 2022; in parallel, an mRNA vaccine candidate for older adults is planned to enter a Phase 1/2 study in H2 2022.
JANUARY 2022 • HPN | HOSPITALPROFESSIONALNEWS.IE
• Next generation influenza programs to deliver differentiated flu vaccines that set a new standard of care; the influenza mRNA QIV program will enter Phase 1/2 trial in 2022 and a Phase 3 trial is planned to start in 2023; Sanofi also deploys proprietary machine-learning to explore the potential for better strain selection in the future. • Opening new growth areas: o Chlamydia: Sanofi has initiated a program to prevent chlamydia infection, a silent cause of infertility in women; a Phase 1 trial of an mRNAbased candidate is planned to start in 2023. o Acne: Sanofi strives to leverage its vaccine technology to develop the first immunotherapy against acne and to enter the clinic in 2023. SANTHERA AND REVERAGEN ANNOUNCE POSITIVE TOPLINE RESULTS WITH VAMOROLONE Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc (US: private) have announced new topline results after completion of the VISION-DMD study at week 48. As previously reported, the FDA considered the safety and efficacy data of vamorolone at 24 weeks (period 1) sufficient for an NDA filing. Efficacy assessments at the now reported completion at week 48 (period 2) included Time to Stand (TTSTAND) velocity, 6-Minute Walk Test (6MWT), Time to Run/ Walk 10 meters (TTRW) velocity, and North Star Ambulatory Assessment (NSAA). Vamorolone 6 mg/kg/day showed maintenance of efficacy across all parameters until end of study at week 48, and was statistically superior to 2 mg/kg/day for TTSTAND velocity and 6MWT but not for TTRW velocity or NSAA. For subjects who continued on the same dose of vamorolone throughout the study, the safety profile was consistent between periods with no increase in frequency or severity of adverse events being observed over time. Subjects switching from 24-week treatment with prednisone to vamorolone 6 mg/kg/day showed no loss of efficacy through to the end of the study. In addition, vamorolone treatment at both doses reversed the growth impairment seen during prednisone treatment and was associated with fewer adverse events, including those associated with corticosteroid use. VISION-DMD was a pivotal double-blind Phase 2b study designed to demonstrate efficacy
and safety of vamorolone compared to placebo and prednisone (active control) in the treatment of DMD [1, 2]. In the first 24-weeks (period 1), 121 ambulant boys aged 4 to <7 years were randomized to receive vamorolone (2 or 6 mg/kg/day) or prednisone (0.75 mg/kg/day) or placebo. 114 subjects continued for another 24 weeks (period 2), where those on vamorolone 2 and 6 mg/kg/day continued to end of study on these doses, and those on prednisone and placebo had been previously randomized to receive vamorolone 2 or 6 mg/kg/day after a 4-week tapering period. 112 subjects completed the study. Efficacy of vamorolone established at 24 weeks was maintained over 48-week treatment period Efficacy at week 48 was assessed for measures including TTSTAND velocity, 6MWT, TTRW velocity and NSAA. The size of effect (change from baseline) observed at the primary endpoint at week 24 for vamorolone 6 mg/kg/day was maintained at week 48 for TTSTAND velocity (0.052 vs 0.045 rises/s), NSAA (3.4 vs 3.5 points), TTRW velocity (0.29 vs 0.23 m/s) and improved for 6MWT (37 vs 48 meters). Vamorolone 2 mg/kg/day showed clinically relevant and robust efficacy at week 24 for the predefined hierarchical endpoints of TTSTAND velocity and 6MWT, as well as NSAA which was an exploratory endpoint. At week 48, vamorolone 6mg/kg/day was statistically superior to vamorolone 2 mg/kg/day in TTSTAND velocity (p=0.010) and 6MWT (p=0.047) but not for TTRW velocity (p=0.37) and NSAA (p=0.60). The efficacy benefit seen with prednisone in period 1 was maintained when subjects were switched to vamorolone 6 mg/kg/ day during period 2: TTSTAND velocity (0.28 vs 0.27 rises/s), 6MWT distance (407 vs 408 meters), TTRW velocity (2.20 vs 2.20 m/s) and NSAA (25.6 vs 26.2 points), all absolute values. Vamorolone at both doses was generally safe and well tolerated Of the 114 subjects who entered into period 2, two subjects discontinued treatment (one adverse event, one withdrawn consent). Three serious adverse events thought to be unrelated to study drug were reported during vamorolone treatment. For subjects who continued on the same dose of vamorolone throughout the study, the safety profile was consistent at week 48 compared to the results previously reported at week 24.
