PR Newswire | October 25, 2023
Flagship Pioneeringannounced the combination of Laronde and Senda Biosciences to launch Sail Biomedicines, a company pioneering the design and deployment of fully programmable medicines to transform patient care. Sail harnesses the power of first-in-category programmable payloads of Endless RNA™ (eRNA), first-in-category programmable nanoparticles, and emerging, proprietary AI technologies, to unlock the comprehensive programming of medicines for the first time. Guillaume Pfefer, Ph.D., MBA, who is also CEO-Partner at Flagship Pioneering, will become Sail's CEO and board member, while John Mendlein, Ph.D., who also serves as Executive Partner, Flagship Pioneering, will become the company's Executive Chairman.
Sail unites Laronde and Senda Biosciences, two companies that deliver more than eight years of combined data and multi-product platform building. Senda's platform was the first to leverage a universal chemical code of natural nanoparticles that enables directed and repeatable deployment of payloads, such as translatable RNA, directly to cells and tissues of choice. Laronde pioneered eRNA, a new class of synthetic, translatable RNA that can be programmed to express diverse proteins inside the body, with vast therapeutic potential.
"Endless RNA has the potential to create an entirely new class of programmable medicines across therapeutic areas that we will now be able to deliver directly to cells and tissues via deployment molecules with unique properties to confer specificity and greater tolerability," said Mendlein. "We believe these programmable medicines will be greatly enhanced via our proprietary generative AI technologies and rapid prototyping abilities to achieve breakthroughs currently beyond the grasp of the human mind. I look forward to working with the Sail Biomedicines team in this exciting new chapter."
"Our deployment platform utilizes natural nanoparticles to shuttle biomolecules into human cells, with unique tropism, potency, and redosability," said Pfefer. "I look forward to leading the integration of these two teams to accelerate the development of new product candidates, build strategic partnerships, and enable diverse value pools, with the goal of swiftly delivering life-changing vaccines and therapies for the people who need them."
"Sail Biomedicines builds on the progress made by two leading Flagship bioplatform companies and will enable integrative design for more effective programmable medicines," said Noubar Afeyan, Ph.D., Founder and CEO of Flagship Pioneering. "I am confident the combined leadership team and board will carry forward this company to realize its bold ambitions and ultimately deliver maximum impact for patients."
In addition to Mendlein and Pfeffer, the Sail Biomedicines Board of Directors will comprise all current members of the Laronde and Senda Biosciences boards, as follows
Pablo Cagnoni, M.D., President and Head of Research & Development, Incyte
Jose "Pepe" Carmona, MBA, Chief Financial Officer, ADC Therapeutics
Paula Hammond, Ph.D., Institute Professor, Massachusetts Institute of Technology, Head of Department of Chemical Engineering, Massachusetts Institute of Technology
Avak Kahvejian, Ph.D., General Partner, Flagship Pioneering
Ignacio Martinez, MBA, General Partner, Flagship Pioneering
Sheri McCoy, M.S., MBA, Former Vice Chairman, Johnson & Johnson
Mary Szela, MBA, CEO and President, TriSalus Life Sciences
About Sail Biomedicines
Sail Biomedicines is pioneering the integrative design and deployment of fully programmable medicines to transform patient care. Sail's platform combines first-in-class programmable circular RNA technology (Endless RNA™ or eRNA), and an industry-leading platform of programmable nanoparticles, utilizing natural components, to unlock comprehensive programming of medicines for the first time. By leveraging cutting-edge eRNA and nanoparticle deployment technology, Sail is building a wealth of data, enabling unparalleled use of generative AI techniques to identify and design fully programmable medicines that are potent, targeted, versatile, and tunable. Sail was founded by Flagship Pioneering.
About Flagship Pioneering
Flagship Pioneering is a biotechnology company that invents and builds platform companies, each with the potential for multiple products that transform human health or sustainability. Since its launch in 2000, Flagship has originated and fostered more than 100 scientific ventures, resulting in more than $90 billion in aggregate value. To date, Flagship has deployed over $3.4 billion in capital toward the founding and growth of its pioneering companies alongside more than $26 billion of follow-on investments from other institutions.
GlobeNewswire | September 28, 2023
Repligen Corporation a life sciences company focused on bioprocessing technology leadership, announced that it has entered into a definitive agreement to acquire privately-held Metenova AB (Metenova) of Mölndal, Sweden. Metenova is projected to generate revenues of $24 million to $25 million for fiscal year 2023, led by the success of its magnetic mixing and drive train technologies that are widely used by global biopharmaceutical companies and contract development and manufacturing organizations (CDMOs). The company recently entered the single-use mixing market with the launch of its MixOne platform, leveraging the success of its existing stainless steel (repeat-use) product line.
Metenova is expected to contribute approximately $5 million in revenue to Repligen in the fourth quarter of 2023, and to contribute $25 million to $27 million in revenue in 2024, with 20%-25% revenue growth in 2025 forward. The acquisition is anticipated to be accretive to Repligen’s adjusted gross and operating margins in 2024, and to be accretive to adjusted earnings per share in 2025.
Over the past decade, Metenova has established market leadership through design innovation in magnetic mixing, with a state-of-the-art suite of both stainless steel and single-use impellers (mixer heads) and drive trains – key components of mixing vessels. The company offers a broad range of products, including high power solutions for efficient media and buffer preparation, and low shear solutions for gentle mixing of sensitive proteins. Metenova’s high performance aseptic mixing technologies are designed to minimize product damage and improve product yield and product consistency, contributing to improved ROI for customers. The company’s solutions are applicable to a wide range of small molecule and large molecule therapeutics and vaccines, including monoclonal antibodies, recombinant proteins, and cell and gene therapies.
Tony J. Hunt, President and Chief Executive Officer at Repligen said, “The addition of Metenova further strengthens our Fluid Management portfolio, with a product line that expands on the market success of our systems and fluid management assemblies and complements our recently acquired single-use bag business from FlexBiosys. We are excited to welcome the Metenova team to Repligen and we look forward to further developing and integrating their differentiated magnetic mixing technologies into our portfolio.”
Johan Westman, Chief Executive Officer at Metenova said, “We have made tremendous progress over the last ten years, building a market leading mixing portfolio. We are now at a stage in our development where we can benefit from more rapid expansion into the single-use market where many of our customers are scaling today. Repligen is the ideal partner for us to take this next step of growth and we look forward to working with our colleagues at Repligen to drive additional global demand for our single-use mixing products, while continuing to advance innovative single-use solutions for the industry.”
About Metenova AB
Metenova is a leading innovator and manufacturer of magnetic mixers for pharmaceutical and biotechnology use. We provide aseptic cutting-edge mixing technology for critical applications. Sales are mainly performed by distributors in over 30 countries with approximately one-third of sales in North America, one-third of sales in Europe and one-third of sales in Asia/ROW. Metenova recently entered the single-use mixing market with the launch of its MixOne platform, leveraging the success of its existing repeat-use (stainless steel) product line. Metenova is headquartered in Mölndal in the Gothenburg area and has approximately 50 employees.
About Repligen Corporation
Repligen Corporation is a global life sciences company that develops and commercializes highly innovative bioprocessing technologies and systems that enable efficiencies in the process of manufacturing biological drugs. We are “inspiring advances in bioprocessing” for the customers we serve; primarily biopharmaceutical drug developers and contract development and manufacturing organizations (CDMOs) worldwide. Our focus areas are Filtration and Fluid Management, Chromatography, Process Analytics and Proteins. Our corporate headquarters are in Waltham, Massachusetts, and the majority of our manufacturing sites are in the U.S., with additional key sites in Estonia, France, Germany, Ireland, the Netherlands and Sweden.
PR Newswire | October 10, 2023
Immunic, Inc. a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases, today announced positive interim data from its phase 2 CALLIPER trial of nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), in patients with progressive multiple sclerosis (PMS). The Company believes that this data shows biomarker evidence that vidofludimus calcium's activity extends beyond the previously observed anti-inflammatory effects, thereby further reinforcing its neuroprotective potential.
Serum NfL responses were consistently observed for vidofludimus calcium across progressive MS disease and all subpopulations. In the overall PMS population at 24 weeks (N=203), vidofludimus calcium was associated with a 6.7% reduction from baseline in serum NfL, compared to a 15.8% increase over baseline in placebo (p=0.01, post hoc). At 48 weeks (N=79), vidofludimus calcium reduced serum NfL by 10.4% from baseline, compared to a 6.4% increase in placebo. Substantial reductions were also seen across all PMS subtypes, as well as in patients that show or do not show disease and/or magnetic resonance imaging (MRI) activity.
Although early, interim GFAP data also showed a promising signal: at 24 weeks (N=203), GFAP increased by 3.7% for vidofludimus calcium, and 4.4% for placebo. At 48 weeks (N=79), the change was only 2.7% for vidofludimus calcium, with a 6.4% increase for placebo. Progression of GFAP response is generally thought to evolve more slowly than NfL, and the Company believes that a longer follow-up may further strengthen this signal.
"Serum NfL has been consistently shown to capture disease activity and to predict future disability in MS. Vidofludimus calcium shows a separation in serum NfL over placebo in this interim analysis, an effect also seen across different subgroups," stated Prof. Jens Kuhle, M.D., Ph.D., Senior Physician, Head of Neuroimmunology Unit and Multiple Sclerosis Centre, University Hospital Basel, Switzerland. "Particularly remarkable, the non-active progressive MS population, which represents the highest unmet medical need in MS, also showed differences in NfL levels over this relatively short observation period in favor of vidofludimus calcium. Meanwhile, although longer follow-up is needed, the GFAP data set also shows a potential promising early signal. Overall, the interim biomarker data further support vidofludimus calcium's possible activity beyond an anti-inflammatory effect, which may be related to its potent Nurr1 activation."
"The clear separation observed in serum NfL for vidofludimus calcium over placebo in the PMS patient population represents another major step forward for, what potentially could be, a first-in-class Nurr1 activator for MS," commented Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "Although no head-to-head data is available, it is encouraging to see that vidofludimus calcium's improvement in NfL over placebo appears at least as good as, and is in fact numerically higher than that observed with historical studies of other therapeutic approaches for PMS. We believe that, if the top-line CALLIPER data, expected in April of 2025, continue to show a neuroprotective effect, we may be able to position vidofludimus calcium as the first oral treatment option for non-active SPMS. Additionally, the drug's first-in-class ability to activate Nurr1, a known neuroprotective target, should also significantly benefit our ongoing phase 3 ENSURE program in relapsing MS where prevention of disability progression independent of relapse activity (PIRA), serves as a key outcome."
"We are very pleased to see such strong improvements in serum NfL for vidofludimus calcium over placebo in the overall PMS population of this interim analysis, as well as across all PMS subtypes and in patients with and without disease activity, and with and without MRI activity. We even saw evidence in non-active SPMS, a population where the medical need for new therapies is high as there is currently no relevant treatment available in the US," added Andreas Muehler, M.D., Chief Medical Officer of Immunic. "Finally, we were also excited to see an encouraging early signal with GFAP. This is a newer biomarker which is thought to evolve more slowly and with lower amplitude than NfL, and longer follow-up will hopefully allow us to see even stronger results."
The Company believes that these results corroborate separate findings from its phase 2 EMPhASIS trial in relapsing-remitting multiple sclerosis (RRMS), where vidofludimus calcium was associated with a decrease in serum NfL at 24 weeks (-17.0% for 30 mg and -20.5% for 45 mg) as compared to baseline values, as contrasted with a 6.5% increase in serum NfL over baseline among placebo patients.
CALLIPER is a multicenter, randomized, double-blind, placebo-controlled phase 2 trial which enrolled 467 patients with primary PMS or active or non-active secondary PMS at more than 70 sites throughout North America as well as Western, Central and Eastern Europe. Patients were randomized to either 45 mg daily doses of vidofludimus calcium or placebo, and the trial's primary endpoint is the annualized rate of percent brain volume change up to 120 weeks. Key secondary endpoints include the annualized rate of change in whole brain atrophy and time to 24-week confirmed disability progression based on the expanded disability status scale (EDSS).
Anticipated MS Clinical Milestones
Top-line data from the phase 2 CALLIPER trial of vidofludimus calcium in PMS is expected in April of 2025.
Data from the interim analysis of the phase 3 ENSURE program of vidofludimus calcium in relapsing MS is expected in late 2024, with the top-line readout of the first of the ENSURE trials at the end of 2025.
The interim data of the phase 2 CALLIPER trial of vidofludimus calcium in PMS will be filed on a Form 8-K and discussed during the management presentation to be held tomorrow at 8:00 am ET. The presentation will also be accessible on the "Events and Presentations" section of Immunic's website at: https://ir.imux.com/events-and-presentations.
About Progressive Multiple Sclerosis
Multiple sclerosis (MS) is an autoimmune disease that affects the brain, spinal cord and optic nerve. In MS, myelin, the coating that protects the nerves, is attacked and damaged by the immune system. Thus, MS is considered an immune-mediated demyelinating disease of the central nervous system. Progressive multiple sclerosis (PMS) includes both primary progressive MS (PPMS) and secondary progressive MS (SPMS). PPMS is characterized by steadily worsening neurologic function from the onset of symptoms without initial relapse or remissions. SPMS is identified following an initial relapsing-remitting course, after which the disease becomes more steadily progressive, with (active SPMS) or without (non-active SPMS) other disease activity present.
About Vidofludimus Calcium (IMU-838)
Vidofludimus calcium is a small molecule investigational drug in development as an oral next-generation treatment option for patients with multiple sclerosis and other chronic inflammatory and autoimmune diseases. The selective immune modulator activates the neuroprotective transcription factor nuclear receptor related 1 (Nurr1), which is associated with direct neuroprotective properties. Additionally, vidofludimus calcium is a known inhibitor of the enzyme dihydroorotate dehydrogenase (DHODH), which is a key enzyme in the metabolism of overactive immune cells and virus-infected cells. This mechanism is associated with the anti-inflammatory and anti-viral effects of vidofludimus calcium. Vidofludimus calcium has been observed to selectively act on hyperactive T and B cells while leaving other immune cells largely unaffected and enabling normal immune system function, e.g., in fighting infections. To date, vidofludimus calcium has been tested in more than 1,400 individuals and has shown an attractive pharmacokinetic, safety and tolerability profile. Vidofludimus calcium is not yet licensed or approved in any country.
About Immunic, Inc.
Immunic, Inc. is a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases. The company's lead development program, vidofludimus calcium (IMU-838), is currently in phase 3 and phase 2 clinical trials for the treatment of relapsing and progressive multiple sclerosis, respectively, and has shown therapeutic activity in phase 2 clinical trials in patients suffering from relapsing-remitting multiple sclerosis and moderate-to-severe ulcerative colitis. Vidofludimus calcium combines neuroprotective effects, through its mechanism as a first-in-class nuclear receptor related 1 (Nurr1) activator, with additional anti-inflammatory and anti-viral effects, by selectively inhibiting the enzyme dihydroorotate dehydrogenase (DHODH). IMU-856, which targets the protein Sirtuin 6 (SIRT6), is intended to restore intestinal barrier function and regenerate bowel epithelium, which could potentially be applicable in numerous gastrointestinal diseases, such as celiac disease, where it is currently in preparations for a phase 2 clinical trial. IMU-381, which currently is in preclinical testing, is a next generation molecule being developed to specifically address the needs of gastrointestinal diseases.