Medical
PR Newswire | October 05, 2023
Capsida Biotherapeutics Inc. and Kate Therapeutics announced a strategic partnership to leverage Capsida's expertise and adeno-associated virus (AAV) manufacturing capabilities to enable KateTx's initial internal portfolio of muscle and heart disease programs.
Under the terms of the agreement, Capsida will provide GMP manufacturing of KateTx's gene therapies as the medicines advance through preclinical and clinical development. In return, Capsida will receive undisclosed funding for the term of the agreement.
KateTx's publicly disclosed internal programs include myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy, which are two of the leading causes of adult-onset muscular dystrophy.
"Capsida possesses end-to-end expertise – which includes AAV capsid engineering, preclinical, clinical, and state-of-the-art manufacturing," said Peter Anastasiou, chief executive officer (CEO) of Capsida Biotherapeutics. "We are thrilled to be able to leverage our established capabilities in AAV manufacturing and support a peer company to reach patients in a complementary space, while continuing to advance our own internal and partnered pipeline in CNS and ophthalmology."
"KateTx is leading the way in developing capsid and cargo combinations to address muscle and heart diseases in ways that have not been previously possible," said Kevin Forrest, Ph.D., president and CEO of Kate Therapeutics. "Capsida's exceptional AAV manufacturing capabilities allow us to build our pipeline, de-risk our programs, and advance them into the clinic in the most rapid manner possible, so we can bring much-needed therapies to patients quickly."
"This collaboration between Capsida and KateTx leverages complementary expertise and overlapping needs across both innovative companies to advance programs in a capital-efficient manner. Capsida has established first-in-class engineered AAV manufacturing capabilities applicable across broad therapeutic areas, and this partnership with KateTx aims to bring these next-generation gene therapies to patients sooner than with traditional approaches," said Beth Seidenberg, M.D., co-founding managing director at Westlake Village BioPartners, member of the board of Capsida, and chair of the board at KateTx.
About Capsida Biotherapeutics
Capsida Biotherapeutics is an integrated gene therapy company developing treatments for rare and common diseases across all ages. Capsida's new class of engineered adeno associated virus (AAV)-based delivery technologies enables a broad range of therapeutic modalities, including DNA, RNA, gene editors, vectorized antibodies and other genetic cargoes, to be systemically delivered to specific organ systems while simultaneously limiting exposure to non-targeted organs. Capsida's proprietary capsid engineering and broad IP estate allows for identification and development of capsids with tropism for any tissue or cell type. The company's initial focus has been delivering therapeutic cargoes intravenously to the central nervous system (CNS) with high transduction levels to the desired tissues and cells. The company is developing a pipeline of both wholly owned programs in CNS and partnered programs with AbbVie (CNS and broad range of ophthalmic disorders), Lilly (CNS), and CRISPR Therapeutics (CNS). Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech.
About Kate Therapeutics
Kate Therapeutics (KateTx) is a patient-focused biotechnology company developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined muscle and heart diseases. The company is applying novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation. These breakthroughs have the potential to improve the efficacy and safety of gene therapies and enable the pursuit of a broader set of targets that are otherwise difficult to drug with current technologies.
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Medical
PR Newswire | October 17, 2023
Ginkgo Bioworks which is building the leading platform for cell programming and biosecurity, and Zenfold Sustainable Technologies, a company focused on developing and manufacturing specialty ingredients using sustainable technologies and precision fermentation, today announced a collaboration to leverage Ginkgo Enzyme Services in its effort to discover an enzyme critical to the manufacturing of veterinary products. This partnership aims to replace traditional chemical processes in veterinary active ingredient production with a sustainable biological method.
"This collaboration marks a significant step forward in Zenfold's mission to bring sustainable manufacturing technology to the veterinary medicine industry," said Dr. BSV Prasad, CEO and Managing Director of Zenfold Sustainable Technologies. "By utilizing Ginkgo's expertise and innovation, we are well-positioned to develop a sustainable solution that will have a lasting impact on the Indian market and beyond."
"We are excited to partner with Zenfold, which brings a powerful vision of sustainable biotechnology to veterinary active ingredient development. This collaboration will enable us to apply our state-of-the-art enzyme discovery module to enable more innovation in the veterinary medicine space," said Jennifer Wipf, SVP, Head of Commercial, Cell Engineering at Ginkgo. "The project involves creating a broad library of cDNA candidates from a metagenomic collection of billions of enzyme sequences. That's the kind of scale visionary projects like this need, and Ginkgo is committed to scaling up this solution for the betterment of animal health and the environment."
About Ginkgo Bioworks
Ginkgo Bioworks is the leading horizontal platform for cell programming, providing flexible, end-to-end services that solve challenges for organizations across diverse markets, from food and agriculture to pharmaceuticals to industrial and specialty chemicals. Ginkgo's biosecurity and public health unit, Concentric by Ginkgo, is building global infrastructure for biosecurity to empower governments, communities, and public health leaders to prevent, detect and respond to a wide variety of biological threats.
About Zenfold
Zenfold Sustainable Technologies is a pioneer in the field of sustainable biology, with a focus on veterinary actives and enzyme supply. This collaboration with Ginkgo Bioworks marks Zenfold's expansion into the global veterinary market and further emphasizes their commitment to environmental responsibility and innovative biological solutions.
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Medical
globenewswire | September 12, 2023
Nurix Therapeutics, Inc. a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with hematologic malignancies and solid tumors, today announced that it has entered into a multi-year, multi-target strategic collaboration agreement with Seagen Inc. to advance a new class of medicines called Degrader-Antibody Conjugates (DACs) for use in cancer. The collaboration between the two companies will focus on an innovative approach to combine two powerful technologies to target cancer—antibody-drug conjugation (ADC) and targeted protein degradation (TPD)—with the goal of creating drugs with new mechanisms of action as well as improved specificity and anti-cancer activity.
“By combining the tissue and tumor specificity of antibodies with highly potent and catalytic targeted degradation of cancer driver proteins, we believe that DACs may represent a next generation of cancer medicine for a wide range of solid tumors and hematologic malignancies,” said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. “With Seagen, our strategic goal is to advance ADC technology to the next level to provide patients with new DAC drugs that deliver greater anti-tumor efficacy and safety compared to currently available agents.”
“The targeted protein degrader modality provides unique advantages over payloads currently employed across the ADC field,” said Gwenn M. Hansen, Ph.D., chief scientific officer of Nurix. “This collaboration is a new application of our DELigase technology, and we are delighted to work with Seagen, a pioneer in the development and commercialization of ADC therapeutics, to create a new generation of drugs to fight cancer.”
Under the terms of the agreement, Nurix will receive an upfront payment of $60 million and has the potential to receive up to approximately $3.4 billion in research, development, regulatory and commercial milestone payments across multiple programs. In addition, Nurix will be eligible for mid-single to low double digit tiered royalties on future sales, and Nurix retains an option for U.S. profit sharing and co-promotion on two products arising from the collaboration. As part of the multi-year collaboration, Nurix will use its proprietary DELigase platform to develop a suite of targeted protein degraders against multiple targets nominated by Seagen that are suitable for antibody conjugation. Seagen will be responsible for conjugating these degraders to antibodies to make DACs and advancing these DAC drug candidates through preclinical and clinical development and commercialization. Given the potential to conjugate multiple antibodies to unique degraders, several DAC drugs may be developed and commercialized within this collaboration.
With the receipt of the $60 million upfront payment, Nurix expects that its existing cash, cash equivalents and marketable securities, excluding any future potential milestones from collaborations, will be sufficient to fund its operating activities into the second quarter of 2025.
About Nurix Therapeutics, Inc.
Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative medicines based on the modulation of cellular protein levels as a novel treatment approach for cancer and other challenging diseases. Leveraging extensive expertise in E3 ligases together with proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform, to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurix’s drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin-proteasome system to selectively decrease or increase cellular protein levels. Nurix’s wholly owned, clinical stage pipeline includes targeted protein degraders of Bruton’s tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B, an E3 ligase that regulates activation of multiple immune cell types including T cell and NK cells. Nurix is headquartered in San Francisco, California.
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Medical
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.
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