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ElevateBio Supercharges Gene Editing and Therapeutic Product Development Capabilities Through Acquisition of Life Edit Therapeutics – Yahoo Finance

By Induced Pluripotent Stem Cells

- Life Edit's genome editing capabilities to be fully integrated with ElevateBio's other cell and gene enabling technologies, including induced pluripotent stem cells (iPSCs), viral vector, and cellular engineering

- Brings a broad array of editing modalities, including deletion, insertion, base editing, and CRISPRa/CRISPRi

CAMBRIDGE, Mass., October 27, 2021--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio), a cell and gene therapy technology company focused on powering transformative cell and gene therapies, today announced that it has acquired all of AgBiome Delta, LLCs (AgBiome) shares of Life Edit Therapeutics, Inc. (Life Edit). Life Edit offers a powerful suite of gene editing technologies that have the potential for any genomic sequence of interest to be removed, added, or altered. Life Edit holds one of the world's largest and most diverse arrays of novel RNA-guided nucleases and base editors that offer greater specificity and broad genome access. These nucleases were derived from AgBiome's proprietary non-pathogenic microbe collection, which could potentially reduce immunogenicity risks.

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"ElevateBios powerful suite of enabling technologies, which now includes Life Edits genome editing capabilities as well as our existing iPSC, viral vector, and cell engineering platforms, is designed to disrupt the rapidly advancing fields of cell and gene therapy," said David Hallal, Chairman and Chief Executive Officer of ElevateBio. "Our vision is to build a world-class center of excellence in genome engineering to push the boundaries of therapeutic development and drive innovation for our own therapeutic pipeline as well as provide access to these critical technologies to our growing number of industry partners."

"Genome editing is a central component of all cell and gene therapy development, and access to novel RNA-guided nucleases and base editors that offer specificity and broad genome coverage will be critical. We believe Life Edit's technology is one of the most versatile in the field, opening up enormous potential," Mitchell Finer, Chief Executive Officer of Life Edit and President, R&D of ElevateBio. "This integration will also enable Life Edit to have greater access to ElevateBios drug development and manufacturing capabilities as we build and advance the pipeline, which will initially focus on developing in vivo gene therapies to address neurologic conditions with high unmet need. In addition, by combining Life Edit's gene engineering capabilities with ElevateBios iPSC technology, our goal is to expand the number of therapeutic uses, including potentially making universal or hypoimmune cells that go undetected by the immune system."

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Life Edit was spun out of AgBiome in October 2020 and AgBiome continues to retain rights for gene editing outside of human therapeutics.

About Life Edit Therapeutics:Life Edit has one of the world's largest and most diverse arrays of novel RNA-guided nucleases and base editors active in mammalian cells. They were developed from a proprietary collection of non-pathogenic organisms and offer gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. Life Edit has a large and diverse library of RNA-guided nucleases, including Type II and Type V systems that encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with one of our proprietary deaminases. The company's nuclease collection has a broad range of Protospacer Adjacent Motifs (PAMs). These short sequences must accompany the DNA sequence for the enzyme to edit a gene, which offers unprecedented access to genomes. Life Edit has identified several classes of DNA modifying enzymes, including novel deaminases that can edit cytidine (C) or adenine (A). Many of the company's RGNs are smaller than widely used CRISPR-Cas systems, offering ease and flexibility for in vivo delivery and manufacturing.

Life Edit is headquartered in Morrisville, NC. Visit us at http://www.lifeeditinc.com, or follow Life Edit on LinkedIn and Twitter.

About ElevateBio:ElevateBio is a cell and gene therapy technology company built to power the development and manufacturing of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built world-class facilities, and integrated diverse technology platforms necessary for rapid innovation and commercialization of cell, gene, and regenerative therapies. The company has built an initial technology stack, including gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering, that can be leveraged across the entire portfolio and by strategic partners. At the center of the business model is ElevateBio BaseCamp, a centralized research and development (R&D) and manufacturing company that offers R&D, process development (PD), and Current Good Manufacturing Practice (CGMP) manufacturing capabilities. The company is focused on increasing long-term collaborations with industry partners while also developing its own highly innovative cell and gene therapies. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

ElevateBio is located in Waltham, MA. Visit us at http://www.elevate.bio, or follow ElevateBio on LinkedIn, Twitter, or Instagram.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211027005153/en/

Contacts

Investor: Catherine Hu chu@elevate.bio 646-535-8276

Media: Courtney Heath ScientPR Courtney@scientpr.com 617-872-2462

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ElevateBio Supercharges Gene Editing and Therapeutic Product Development Capabilities Through Acquisition of Life Edit Therapeutics - Yahoo Finance

ReNeuron enters cancer therapy collaboration with UCL – ShareCast

By Induced Pluripotent Stem Cells

Cell-based therapeutic specialist ReNeuron has entered a collaboration agreement with University College London (UCL), it announced on Tuesday, to conduct research into the generation of immune cells from induced pluripotent stem cells (iPSCs), for anti-cancer cell therapies.

The AIM-traded firm said it would be working alongside Dr Claire Roddie, associate professor at the UCL Cancer Institute, and the team at the UCL CAR-T cell cancer therapy programme.

It said it would provide UCL with iPSCs from its CTX immortalised neural progenitor cell line to be assessed for their ability to differentiate into functional T cells and natural killer (NK) cells.

If this first objective was met, the CTX-iPSC cell line would be further used to generate chimeric antigen receptors (CAR) T cells, or CAR-NK cells.

ReNeuron explained that CARs allow T and NK cells to target receptors present on the surface of cancer cells, allowing them to recognise and specifically kill tumour cells.

Dr Roddie and her team had extensive expertise in generating CAR cells, the company said, adding that both groups would work collaboratively to generate haematopoietic stem cells, lymphoid progenitors and cytotoxic T cells from the CTX-iPSCs.

We are very much looking forward to working with Dr Roddie and her team at UCL in this exciting and rapidly growing research space, said chief scientific officer Dr Stefano Pluchino.

This collaboration is another strong development for ReNeuron which demonstrates the uniqueness and strong translational potential of our proprietary iPSC technology platform and will allow us to further expand into the oncology space.

If this initial research is successful, significantly large numbers of cancer patients can be treated with next generation alternate cancer therapies.

At 1243 BST, shares in ReNeuron Group were up 4.91% at 119.6p.

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ReNeuron enters cancer therapy collaboration with UCL - ShareCast

Shah on the Potential Utility of NK Cells in Multiple Myeloma – OncLive

By Induced Pluripotent Stem Cells

Nina Shah, MD, discusses the potential utility of natural killer cells in multiple myeloma.

Nina Shah, MD, hematologist and oncologist, associate professor of medicine, Department of Medicine, University of California, San Francisco (UCSF) Helen Diller Family Comprehensive Cancer Center, discussesthepotential utility of natural killer (NK) cells in multiple myeloma.

NK cells are a potential type of allogeneic cellular therapy for patients with multiple myeloma, explains Shah. However, a challenge with NK cells is that there are not as many NK cells as other cells; therefore, NK cells need to be expanded and often engineered, Shah explains. One example of engineeringinducibleNK cells is being evaluated by Fate Therapeutics. The NK cells are engineered from a human induced pluripotent stem cellbank to then be cultured, Shah adds.

Moreover, the NK cells strongly express CD16 and Fc receptor on the cell surface. Additionally, CD38 is engineered so utilizing daratumumab (Darzalex) is an option with both engineering tactics, Shah continues. Additionally, the cells are engineered in the context of CAR so targeting BCMA is an option to kill cells. An IL-15 receptor fusion protein was also engineered to allow the NK cells to persist. Preclinical data have shown that the cells are active and persistent, Shah explains.

Overall, more data are anticipated, which will include patients with relapsed/refractory multiple myeloma who received NK cells with a monoclonal antibody, such as daratumumab, Shah explains. Moreover, this approach would be given off-the-shelf, which could open an accessible option to patients, Shah concludes.

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Shah on the Potential Utility of NK Cells in Multiple Myeloma - OncLive

Exacis Biotherapeutics Announces Strategic Partnership With CCRM For Specialty Manufacturing Of Services And Investment For Development Of…

By Induced Pluripotent Stem Cells

Cambridge, MA, - Exacis Biotherapeutics, Inc., a development-stage immuno-oncology company working to harness the immune system to cure cancer, today announced initiation of a strategic partnership with Toronto-based Centre for Commercialization of Regenerative Medicine (CCRM) for specialty manufacturing services related to the development of Exacis innovative, iPSC-derived mRNA-engineered NK cell products to treat cancer. The partnership also includes a cash investment into Exacis by CCRM Enterprises Holdings Ltd., the for-profit venture investment arm of CCRM, which will be used to fund operations.

Exacis CEO Gregory Fiore, MD, commented, We welcome CCRM as a key partner to allow us to rapidly advance our virus-free manufacturing processes to make novel NK cell products that are engineered for performance and to avoid rejection. CCRM is a recognized leader in iPSC-derived cell therapy development and manufacturing and we are thrilled to have them as a partner. Their confidence in Exacis is evidenced by the accompanying investment, by CCRM Enterprises Holdings Ltd., underscoring the unique value proposition offered by Exacis differentiated platform and approach to cell therapies. We look forward to partnering with CCRMs CDMO experts to apply our mRNA based technologies to develop best-in-class products to treat challenging hematologic and solid tumors.

Cynthia Lavoie, PhD, President and CIO of CCRM Enterprises Inc. added, We are pleased to support Exacis by way of an investment, and with our sector expertise and specialized infrastructure. This is a successful model that we have employed in the past to support promising technologies and together we will develop leading cell therapy products that utilize the substantial potential of the Exacis platform as it advances its iPSC-derived cell programs.

About Exacis Biotherapeutics

Exacis is a development stage immuno-oncology company focused on harnessing the human immune system to cure cancer by engineering next generation off-the-shelf NK and T cell therapies aimed at hematologic malignancies and solid tumors. Exacis was founded in 2020 with an exclusive global license to a broad suite of patents covering the use of mRNA based cell reprogramming and gene editing technologies for use in generating engineered NK and T cells for oncology applications. These patents were developed and are owned by Factor Bioscience.

Exacis differentiated cell therapy platform avoids the use of DNA and viruses and uses instead a proprietary mRNA based technology. Exacis uses the technology to generate iPSCs and to edit their genomes to create stealthed, potent allogeneic cell products, termed ExaNK, ExaCAR-T or ExaCAR-NK cells.

About CCRM

CCRM is a global, public-private partnership headquartered in Toronto, Canada. It receives funding from the Government of Canada, the Province of Ontario, and leading academic and industry partners. CCRM supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding and infrastructure. CCRM is the commercialization partner of the University of Toronto's Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About CCRMEnterprises Inc.

CCRMEnterprises Inc. is the for-profit venture investment arm of the Centre for Commercialization of Regenerative Medicine (CCRM).CCRM Enterprises invests in early stage ventures and projects developing therapeutics and enabling technologies. Through an extensive network of investors, it can bring together risk capital to support these early-stage ventures as they scale up along the development pathway.By tapping into CCRMs manufacturing infrastructure and expertise, CCRM Enterprises provides capital-efficient support to accelerate and de-risk these high potential, early-stage ventures, further enabling the development of an advanced therapies ecosystem. CCRM Enterprises Holdings Ltd. is the associated entity that holds shares in CCRM portfolio companies. Learn more about our investing strategy here.

About T and Natural Killer (NK) Cell Therapies

T and NK cells are types of human immune cells that are able to recognize and destroy cancer cells and can be modified through genetic engineering to target specific tumors.

Contact Exacis Biotherapeutics for further information and media:

Exacis Biotherapeutics info@exacis.com

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Exacis Biotherapeutics Announces Strategic Partnership With CCRM For Specialty Manufacturing Of Services And Investment For Development Of...

Team finds way to enhance stem cell therapy for CNS injuries – BioPharma-Reporter.com

By Stem Cell Treatment

The scientists, Christopher Rathnam and colleagues, say they have designed a way of controlling the formation of 3D spheroids made from stem cells, while enhancing the spheroids ability to differentiate into functional neurons.

The technology led to an increase in stem cell survival and differentiation two challenges with existing stem cell therapy systems in a mouse model of spinal cord injury, noted the team in a paper published inScience Advances

We believe that our technology platform is an ideal candidate for improving many other types of cell therapies that require high cell survival and effective control of cell fate, making it useful not only for treating [spinal cord injuries] but also for various other diseases and disorders, said the authors.

Although stem cell therapy holds enormous potential for treating debilitating injuries and diseases of the CNS, the team outlined how low survival and inefficient differentiation have restricted its clinical applications.

Recently, 3D cell culture methods, such as stem cellbased spheroids and organoids, have demonstrated advantages by incorporating tissue-mimetic 3D cell-cell interactions, said the experts.

However, a lack of drug and nutrient diffusion, insufficient cell-matrix interactions, and tedious fabrication procedures have compromised their therapeutic effects in vivo, they added.

To address these issues, the Rathnam led team developed a method in which biodegradable manganese dioxide nanosheets guide the rapid assembly of neural stem cells, derived from human induced pluripotent stem cells (iPSCs), into 3D spheroids.

The technique also enables controlled drug release inside the core of the spheroids, which could help to improve cell survival and differentiation, they said.

To evaluate the efficacy of the structures, which they termed synthetic matrix-assisted and rapidly templated (SMART) spheroids, the researchers implanted them at injury sites in a mouse model of spinal cord injury.

As controls, they injected cell suspensions and conventional neurospheres, formed without the use of their novel nanosheets, at the spinal cord injury sites, with the same total number of cells per animal and at the same concentrations.

They found significantly higher cell survival and improved neuronal differentiation efficiency for the SMART neurospheres compared with the controls both 7 days and 1 month after injection.

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Team finds way to enhance stem cell therapy for CNS injuries - BioPharma-Reporter.com

Bacterial Infections Linked with Unapproved Stem Cell Treatments – Contagionlive.com

By Stem Cell Treatment

Unproven products marketed as stem cell therapies could be risky, according to a new study from the Centers for Disease Control and Prevention, which analyzed bacterial infections from unapproved products derived from umbilical cord blood.

The case series study, published in JAMA Network Open, examined 20 bacterial infections in eight states. It involved reviewing medical records, sterility testing of products and whole-genome sequences of patient and product isolates for participants who developed bacterial infections after receiving stem cell treatments between August 2017 and September 2018.

The findings of this investigation show that stem cell therapies that are not FDA-approved or that are not used for the approved medical conditions can pose serious health risks to patients with no benefit, Kiran Mayi Perkins, MD, lead investigator with the CDCs Outbreak and Response Team, told Contagion. Currently, the only stem cell products derived from umbilical cord blood that are FDA-approved for use in the United States are approved for use in patients with disorders that affect the production of blood, but they are not approved for other uses. However, these products are often illegally marketed by clinics as being safe and effective for treating a wide range of diseases or conditions. Therefore, patients should be aware of the unproven benefits and the potential risks to their health when using unapproved and unproven stem cell products for conditions that they have not been shown to effectively treat.

All but one of the patients in the study required hospitalization after receiving stem cell treatment for conditions including osteoarthritis, rheumatoid arthritis and injury. The CDC performed sterility testing on vials of product and compared bacterial isolates with those from the patients.

We were surprised by the magnitude of bacterial contamination found in the vials that we tested; over half of the vials of the stem cell product that we tested were contaminated with bacteria, and many of these vials had very high bacterial counts, Perkins said.

Unapproved stem cell products have been marketed for conditions such as joint diseases, sports injuries and chronic pain and have become more prevalent as people seek products to prevent and treat COVID-19, the study noted. However, these uses are not approved by the US Food and Drug Administration (FDA).

ReGen Series products processed by Genetech and distributed by Liveyon were recalled after bacterial infections were reported in Texas and Florida in 2018, and a national investigation was launched. Information was gathered about patients, product administration, infection control practices and product manufacturing and distribution.

The bottom line is that many stem cell clinics are offering unproven products that have the potential to be dangerous, Perkins said. There is good research that is being done on stem cell therapies, but there are also a lot of clinics that are selling stem cells for unproven uses. To date, the only stem cell treatments approved by the FDA are products made from a donors umbilical cord blood that are used to treat certain cancers and disorders of the blood and immune system. If the cells are being used to treat other conditions such as pain, orthopedic conditions, autism, anti-aging, or COVID-19, they are not approved and may not be safe. We urge all patients and health care practitioners considering stem cell therapies to ensure that the stem cell product is being used for the approved indication or under an Investigational New Drug Application (IND) and is on FDAs list of approved products.

The states in which confirmed bacterial infections were identified as of March 2021 are Texas, Florida, California, Arizona, Kansas, Maine, Colorado and Massachusetts.

The treatments were injected into the patients knees, shoulders, spine or digits or administered through intravenous infusion or as a nasal spray. Infections included 10 at the injection site, five bloodstream infections and five with both injection site and bloodstream infections. Most common bacteria were Escherichia coli and Enterobacter cloacae.

CDC will continue to investigate any reports that it receives that are concerning for infectious risks to patients associated with the receipt of stem cell products and will report these to FDA, the agency that has regulatory oversight for these types of therapies, Perkins said.

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Bacterial Infections Linked with Unapproved Stem Cell Treatments - Contagionlive.com

Stem Cells Used to Treat Avascular Necrosis of the Femoral Head – Yale School of Medicine

By Stem Cell Treatment

Collapsed femoral heads caused by osteonecrosisotherwise known as avascular necrosis unfortunately represent the root cause for approximately 10% of all hip replacements nationwide. Daniel Wiznia, MD, is utilizing a stem cell treatment at Yale School of Medicine and integrating new techniques along with 3D imaging technology as part of a joint-preservation procedure.

Occurring in more than 20,000 Americans each year, osteonecrosis of the femoral head is commonly diagnosed in patients in their 30s and 40s. The disease is caused by injury of the blood supply to the head of the femur, which is the ball portion of the hips ball and socket joint.

If unaddressed, this disease may ultimately lead to the collapse of the femoral head, requiring the patient to undergo a hip replacement. For patients in this age group, a total hip replacement is not ideal as it likely will wear out and the patient will require more surgery.

The goal for each case is clear: prevent the femoral head from collapsing and the need for a hip replacement. As part of a surgical procedure, Wiznia harvests bone marrow from the patients pelvis. The individuals own stem cells are then isolated from the marrow, concentrated, and injected into lesions within the avascular portion of the femura treatment that is only taking place at some of the nations largest medical centers.

The key in these instances is to discover the avascular necrosis before the head collapses, Wiznia said. Because the vascular injury is usually a painless event, patients are generally unaware of the specific point in time when the vascular injury occurred, which is why cases are rarely discovered in time. However, we do know that 80% of patients who have avascular necrosis on one side of the hip have it on the opposite side. We usually are able to catch that second asymptomatic side in those situations and conduct the core decompression with stem cell treatment before it collapses.

According to Wiznia, this treatment reduces the risk of the head of the femur from collapsing, and the stem cell therapy has shown promising results. Soon after the procedure, many patients with avascular necrosis experience rejuvenated blood supply to the area and the bone is repopulated with new cells. This can additionally alleviate the short-term need for a hip replacement.

This novel stem cell therapy has demonstrated improved pain and function, and the stem cells decrease the risk of the femoral head from collapsing, Wiznia said. This translates into fewer young patients requiring hip replacements, and subsequent surgeries in their later years.

As an engineer himself, Wiznia works closely with the Yale School of Engineering & Applied Sciences and the Integrated 3D Surgical Team to better tailor this treatment to each specific patient.

One of the challenges of orthopaedic surgery in the human body is that surgeons are operating in a three-dimensional space and are often reliant on two-dimensional imagery such as X-rays, Wiznia added. Through the use of computer modeling, we are able to customize those images and create models that are specific to each patient, which, in turn, enhances outcomes and overall post-operative success rates.

Enhanced models and 3D imaging enable surgeons like Wiznia to better locate and target both the lesions and necrotized bone in these instances. Effectively doing so regenerates the femoral head and stimulates new osteoblast growth, which will heal the region, maintain the integrity of the joint, and decrease the chance of femoral head collapse and need for a hip replacement.

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Stem Cells Used to Treat Avascular Necrosis of the Femoral Head - Yale School of Medicine

Stem cell therapy can help combat common symptoms of aging – The Mountaineer

By Stem Cell Treatment

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Stem cell therapy can help combat common symptoms of aging - The Mountaineer

Vitti Labs Announces FDA Approval of IND Application for Phase II Clinical Trial of Combination Mesenchymal Stem Cell and Exosome Treatment of Novel…

By Stem Cell Treatment

LIBERTY, Mo., Oct. 26, 2021 /PRNewswire/ -- Vitti Labs (www.vittilabs.com), an AATB Accredited Tissue Bank focused on Life Science Research, Development and Manufacturing, announced today that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application to conduct Phase II Clinical Trials using a combination of Umbilical Cord Mesenchymal Stem Cell and Umbilical Cord Mesenchymal Stem Cell Exosomes for the treatment of Acute Respiratory Distress Syndrome (ARDS) associated with the Novel Corona Virus (COVID-19). This marks the very first time the FDA has approved an IND that uses both of these components together and so first of its kind therapy.

This therapy is designed to suppress the pro-inflammatory processes in the pulmonary system that occurs in COVID patients, while simultaneously alleviating pulmonary distress, such as acute lung injury and inflammation as seen in ARDS (Acute Respiratory Distress Syndrome). In addition to its anti-inflammatory and anti-fibrotic properties, the combination therapy aims to reduce oxidative stress. This Phase II study will evaluate the utility of intravenous umbilical cord mesenchymal stem cells and exosomes in mitigating the pulmonary consequences of COVID-19. As this treatment has already shown tremendous promise in the treatment for ARDS associated with COVID-19, the completion of this Phase II Clinical Trial is expected to conclude in Q1 of 2022.

Philipp Vitti, Chief Scientist of Vitti Labs, stated, "We are very excited to be the first FDA Approved IND to have Umbilical Cord Mesenchymal Stem Cells and their Exosomes being utilized together as a multi-dose combination therapy for IV use. The preliminary trials have been overwhelmingly successful. Mesenchymal Stem Cells and their exosomes have unique therapeutic benefits, and together they create advanced therapeutic properties. This application approval is a great advancement in the ongoing progress to utilize Umbilical Cord Mesenchymal Stem Cells and Exosomes for different disease models, and Vitti Labs is excited and proud to contribute their resources to finding effective treatment options for the worldwide pandemic."

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About Vitti Labs

Vitti Labs is a cGMP certified, AATB accredited Tissue bank in Liberty, Missouri. They harvest biomaterials from the umbilical cord and placenta to utilize their properties to activate and support the repair of the body. Vitti Labs has a commercial division which focuses on human cellular tissue products. Vitti Labs' research and development division focuses on understanding various disease models and utilizing umbilical cord and placental derived Mesenchymal Stem Cells and Exosomes for therapies of those diseases.

For any media requests/inquiries: Miriam McKinney, 816-200-7959, 322286@email4pr.com

http://www.vittilabs.com

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Vitti Labs Announces FDA Approval of IND Application for Phase II Clinical Trial of Combination Mesenchymal Stem Cell and Exosome Treatment of Novel...

Biohaven Enrolls Phase 1a/1b Clinical Trial of BHV-1100, Lead Asset from its ARM (Antibody Recruiting Molecule) Platform, in Combination with NK Cell…

By Stem Cell Treatment

-- Biohaven initiates a clinical trial of the novel antibody recruiting molecule BHV-1100 to assess safety, tolerability, and exploratory clinical activity in Multiple Myeloma

Published: Oct. 27, 2021 at 7:30 AM EDT|Updated: 18 hours ago

NEW HAVEN, Conn., Oct. 27, 2021 /PRNewswire/ --Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), announced the enrollment of the first patient in a Phase 1a/1b trial in Multiple Myeloma using the ARM, BHV-1100, in combination with autologous cytokine induced memory-like (CIML) natural killer (NK) cells and immunoglobulin (Ig) to target and kill multiple myeloma cells expressing the cell surface protein CD38 (Figure 1). BHV-1100 is the lead clinical asset from Biohaven's ARM Platform, developed from a strategic alliance with PeptiDream Inc. This clinical trial will assess the safety and tolerability, as well as exploratory efficacy endpoints, in newly diagnosed multiple myeloma patients who have tested positive for minimal residual disease (MRD+) in first remission prior to autologous stem cell transplant (ASCT).

NK cells are part of the innate immune system, which is designed to recognize and destroy "non-self" or diseased cells in the body. However, tumor cells can evade detection by immune effector cells, allowing the tumor to advance. BHV-1100 targets a cell-surface protein, CD38, that is heavily overexpressed on multiple myeloma and binds to it, recruiting primed autologous cytokine induced memory-like (CIML) natural killer (NK) cells to destroy the tumor.

Charlie Conway, Ph.D., Chief Scientific Officer at Biohaven commented, "While many recent advances have been made to benefit multiple myeloma patients, most patients will unfortunately still relapse. We are excited to investigate BHV-1100 for its ability to recruit autologous CIML NK cells to the site of the tumor. Based on preclinical data from Biohaven Labs, we anticipate that our CD38 targeting ARM-enabled NK cells will kill CD38-positive multiple myeloma cells, and recruit other immune effector cells to assist in reducing the tumor burden."

Biohaven has initiated enrollment in the clinical trial and plans to enroll 25 patients for this single-center, open-label study (ClinicalTrials.gov Identifier: NCT04634435; https://clinicaltrials.gov/ct2/show/NCT04634435). The study will enroll newly diagnosed multiple myeloma patients who have minimal residual disease (MRD+) in first remission prior to an autologous stem cell transplant (ASCT).

David Spiegel M.D., PhD, inventor of the ARM technology and Professor of Chemistry and Pharmacology at Yale University, commented, "This is an important milestone in the development of the ARM therapeutic platform taking a novel technology from 'benchtop to bedside'. It also highlights Biohaven's commitment to benefit patients in need."

About ARMs: Antibody Recruiting Molecules

ARMs,antibody recruiting molecules, are engineered with modular components that are readily interchangeable, giving the platform tremendous flexibility and rapid development timelines. ARM compounds are being developed at Biohaven Labs to redirect a patient's own antibodies for therapeutic effect with multiple benefits over traditional monoclonal antibody therapies, including the potential for oral dosing. For BHV

1100, the ARM platform is being used to provide antigen targeting to NK cell-based therapies without genetic engineering. This NK cell targeting approach is also being investigated with allogeneic, or 'off-the-shelf', immune cell-based therapies.

About Multiple Myeloma Multiple myeloma is a type of blood cancer of the plasma cell that develops in the bone marrow, the soft tissue inside our bones. Healthy plasma cells produce antibodies, which are critical for the immune system's ability to recognize disease-causing entities, such as bacteria, viruses and tumor cells. In multiple myeloma, however, genetic abnormalities in a single plasma cell cause it to divide uncontrollably. This leads to the over-production of a single (monoclonal) antibody protein, referred to as an "M protein". Also, these cancerous cells divide to the point of crowding out normal, healthy cells that reside in the bone marrow. Many patients are diagnosed due to symptoms such as bone pain or fractures, kidney failure (thirst, dehydration, confusion), nerve pain, fever, and weakness. The American Cancer Society estimates that approximately 34,920 new cases will be diagnosed, and 12,410 deaths will occur in 2021 from multiple myeloma.

About BiohavenBiohaven is a commercial-stage biopharmaceutical company with a portfolio of innovative, best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders and areas of unmet need. Biohaven's neuro-innovation portfolio includes FDA-approved NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder, Alzheimer's disease, and spinocerebellar ataxia; and MPO inhibition for amyotrophic lateral sclerosis. More information about Biohaven is available atwww.biohavenpharma.com.

About PeptiDream PeptiDream Inc. is a public(Tokyo Stock Exchange 1st Section 4587) biopharmaceutical company founded in 2006 employing their proprietary Peptide Discovery Platform System (PDPS), a state-of-the-art highly versatile discovery platform which enables the production of highly diverse (trillions) non-standard peptide libraries with high efficiency, for the identification of highly potent and selective hit candidates, which then can be developed into peptide-based, small molecule-based, or peptide-drug-conjugate-based therapeutics. PeptiDream aspires to be a world leader in drug discovery and development to address unmet medical needs and improve the quality of life of patients worldwide. Further information regarding PeptiDream can be found at: http://www.peptidream.com.

Forward-looking Statement This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of Biohaven's management about BHV-1100 as a treatment for multiple myeloma. Forward-looking statements include those related to: Biohaven's ability to effectively develop and commercialize BHV-1100, delays or problems in the supply or manufacture of BHV-1100, complying with applicableU.S.regulatory requirements, the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials, the timing of planned interactions and filings with the FDA, the timing and outcome of expected regulatory filings, the potential commercialization of Biohaven's product candidates, the potential for Biohaven's product candidates to be firstin class or best in class therapies and the effectiveness and safety of Biohaven's product candidates. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of Biohaven's Annual Report on Form 10-K for the year ended December 31, 2020, filed with the Securities and Exchange Commission onMarch 1, 2021, and Biohaven's subsequent filings with the Securities and Exchange Commission. The forward-looking statements are made as of this date and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

NURTEC and NURTEC ODT are registered trademarks of Biohaven Pharmaceutical Ireland DAC. Neuroinnovation is a trademark of Biohaven Pharmaceutical Holding Company Ltd.

ARM is a trademark of Kleo Pharmaceuticals, Inc.

Biohaven Contact Dr. Vlad Coric Chief Executive Officer Vlad.Coric@biohavenpharma.com

Media Contact Mike Beyer Sam Brown Inc. mikebeyer@sambrown.com 312-961-2502

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Originally posted here:
Biohaven Enrolls Phase 1a/1b Clinical Trial of BHV-1100, Lead Asset from its ARM (Antibody Recruiting Molecule) Platform, in Combination with NK Cell...

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