Category Archives: Biotech Companies USA

AmorChem’s RNA Zipcode Delivery Technology Investment Exits via New York-Based Kodikaz Therapeutic Solutions, Inc.

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MONTREAL–(BUSINESS WIRE)–AmorChem II Fund L.P. (”AmorChem”) is delighted to announce that its RNA Zipcode delivery program has been acquired by Kodikaz Therapeutic Solutions, Inc. (”Kodikaz”), a New York City biotech focused on targeted delivery of cancer therapeutics. The RNA Zipcode program was the first university-based investment of AmorChem’s second fund and was developed by Professors Eric Lécuyer (Montreal Clinical Research Institute), Mathieu Blanchette (McGill University), and Jérôme Waldispühl (McGill University).

”We invested in this technology ahead of current trends and prior to the pandemic of 2020, when the promise of RNA-based therapeutics was crystallized in an effort that saved millions of lives. Focus on RNA-based therapeutics has grown steadily since, though one of the major issues remains delivery – not only to the appropriate tissue – but also to the correct subcellular localization. The work of Kodikaz on targeted delivery of therapeutics to cancer cells using their DNA zipcode technology made them a most attractive receptor for our assets, and in fact, both groups saw synergy in combining the two approaches. It’s truly exciting to see our RNA Zipcode program now move forward in collaboration between the researchers and the Kodikaz group, which includes a seasoned management team already in place,” says Kevin McBride, General Partner & CSO at AmorChem.

”The success of our RNA Zipcode program highlights our specialized investment strategy: digging deep into Quebec academia searching for novel therapeutic approaches that will benefit patients. This often means that AmorChem bets on nascent technologies that have not yet been proven. In the RNA Zipcode case, our team spearheaded the collaboration between three teams from two major research institutions and AmorChem’s investment leveraged additional financial support from several granting agencies. Our mobilization of the appropriate human and financial resources allowed us to attract a valuable partner. This is now bringing a transformative academic technology one step closer to patients while building Kodikaz’s pipeline to a full suite of complementary delivery tools,” comments Inès Holzbaur, Managing Partner at AmorChem.

”This is a great opportunity to partner with world-renowned inventors that bring expertise in artificial intelligence and RNA delivery. We are excited to bring in the new capability that permits us to further control delivery not only to the targeted cell but also within the cell with high specificity,” says Anthony Johnson, Kodikaz President & CEO.

The transaction was made possible by the great collaboration of Axelys and McGill University’s Office of Innovation + Partnerships. The RNA Zipcode program was generously supported by grants from the Genomic Applications Partnership Program (Genome Canada and Genome Quebec), as well as the Programme de soutien aux organismes de recherche et d’innovation (Ministère de l’Économie, de l’Innovation et de l’Énergie) and Médicament Québec.

About AmorChem

AmorChem (www.amorchem.com) is a leading early-stage venture capital fund launched in 2011 in Montreal. The AmorChem team utilizes its deep understanding of fundamental science to uncover its therapeutic potential and focuses its core expertise in translational research to accelerate therapeutic drug discovery and development across a broad spectrum of disease areas. The fund capitalizes on both its venture capital expertise and its entrepreneurial experience to spark the creation of start-up companies and help shape them into the next generation of biotech companies. With over $85M under management, AmorChem has financed over 30 university projects and started up several biotechnology companies from the fruits of this innovative research.

For more information on Kodikaz, visit www.kodikaz.com.

Contacts

Media contact
Inès Holzbaur

514 513-7453

ines@amorchem.com

Progentos Therapeutics Closes $65 Million Series A Financing to Advance Treatments for Multiple Sclerosis and Other Degenerative Diseases

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Founded by Chief Executive Officer Chris Loose, Ph.D. and Chief Scientific Officer, Sanjay Magavi, Ph.D., Progentos to Drive Clinical Development of Novel Therapeutics for Remyelination

Financing Led by Forbion with other Leading Life Science Investors including Alta Partners, Mission BioCapital, Longwood Fund, and Dolby Family Ventures

WATERTOWN, Mass.–(BUSINESS WIRE)–Progentos Therapeutics, a biotech company addressing the critical unmet need to regenerate myelin and restore function for patients with Multiple Sclerosis (MS) and other demyelinating diseases, today announced its launch and the closing of a $65 million series A round. This funding will enable Progentos to advance its MS program through human proof of concept studies and expand its pipeline in additional degenerative diseases.

Progentos is developing first-in-class small molecules designed to induce remyelination of axons affected by MS. In MS, disability is caused by demyelination, damage to the myelin sheaths that support the function and survival of axons. The Company’s proprietary molecules outperform previous approaches in differentiating oligodendrocyte progenitor cells (OPCs) to generate new oligodendrocytes and regenerate myelin in in vivo models.

“While there are many treatments that are highly effective at slowing the progression of disease, there is a significant unmet need for new approaches that can regenerate myelin and restore function for patients with MS,” said Dr. Chris Loose, CEO of Progentos. “I am thrilled with the support received from this group of investors and appreciate their recognition of the need to advance the current standard of care for the millions of individuals impacted by MS and other demyelinating diseases.”

The series A financing was led by Netherlands-based Forbion which was joined by Alta Partners, Mission BioCapital, Longwood Fund, and Dolby Family Ventures.

“We are truly excited to be part of Progentos’ journey towards delivering potentially disease-modifying drugs for diseases like multiple sclerosis, diseases with high unmet clinical needs. Chris and Sanjay are bringing a high level of technical expertise, entrepreneurship, and leadership and we are honored to support them in this endeavor,” said Forbion General Partner Dmitrij Hristodorov, Ph.D.

About Progentos Therapeutics

Progentos is developing first-in-class compounds to induce endogenous oligodendrocyte progenitor cells to remyelinate axons in patients suffering from MS and other demyelinating diseases. Combining expertise in chemistry, biology and in vivo models, Progentos discovers and develops novel small-molecule drugs to regenerate tissues in patients with degenerative diseases. The Company will have operations in Watertown, MA, USA and Naarden, The Netherlands. More information can be found at www.progentos.com

Contacts

Jason Glashow

Glashow Strategic Communications for Progentos Therapeutics

jason@glashowstrategic.com

Bristol Myers Squibb’s CAR T Cell Therapy Breyanzi Approved by the U.S. Food and Drug Administration for Relapsed or Refractory Follicular Lymphoma

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95.7% of patients responded to Breyanzi in the TRANSCEND FL trial

Breyanzi provided sustained clinical benefit with median duration of response not reached and the majority (77.1%) of responders in ongoing response at 18 months

Breyanzi is a personalized therapy with a differentiated profile, offering durable responses and a consistent safety profile across trials

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #Breyanzi—Bristol Myers Squibb (NYSE: BMY) today announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Breyanzi® (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Breyanzi is also now included in the National Comprehensive Cancer Network (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for B-cell Lymphomas as a Category 2A recommendation for third-line and subsequent therapy for relapsed or refractory FL.*

In relapsed or refractory FL, Breyanzi is delivered as a one-time infusion** with a single dose containing 90 to 110 x 106 CAR-positive viable T cells. Please see the Important Safety Information section below, including Boxed WARNINGS for Breyanzi regarding Cytokine Release Syndrome (CRS), Neurologic Toxicities, and Secondary Hematological Malignancies.

“Breyanzi is a cornerstone of our cell therapy portfolio, providing a differentiated profile across a wide array of B-cell malignancies,” said Bryan Campbell, senior vice president, Head of Commercial, Cell Therapy, Bristol Myers Squibb. “Today’s approval of Breyanzi for relapsed or refractory FL provides an option with potential for lasting remission in a one-time infusion and a safety profile that allows for administration and monitoring in both the inpatient and outpatient setting in an increasing number of certified treatment centers in the U.S.”

Historically, FL has been considered an incurable disease, and patients frequently relapse following front-line therapy, with prognosis worsening after each subsequent relapse. Despite advances in treatment, there remains an unmet need for additional options that offer treatment-free intervals with durable, complete responses.

The Phase 2 TRANSCEND FL study included the largest primary analysis set of patients with relapsed or refractory FL of a clinical trial evaluating a CAR T cell therapy in this patient population. Based on the U.S. Prescribing Information (USPI), in patients treated with Breyanzi in the third-line plus setting and included in the primary efficacy analysis set (n=94), the overall response rate (ORR) was 95.7% (95% CI: 89.5-98.8). ORR was defined as the percentage of patients achieving a partial or complete response per Lugano criteria as assessed by an Independent Review Committee (IRC). The complete response (CR) rate was 73.4% (95% CI: 63.3-82.0) and required a negative bone marrow biopsy for confirmation. Responses were rapid and durable with a median time to response of one month (range: 0.6-3.3) and median duration of response (DOR) not reached (95% CI: 18.04-NR), with 80.9% of responders remaining in response at 12 months, and 77.1% of responders remaining in response at 18 months. Results from the primary analysis of TRANSCEND FL presented at the 2023 International Conference on Malignant Lymphoma showed an ORR of 97% (95% CI: 91.6-99.4; one-sided p<0.0001) in efficacy evaluable patients (n=101), with 94% of patients achieving a CR (95% CI: 87.5-97.8; one-sided p<0.0001).

“In the treatment of relapsed or refractory follicular lymphoma, patients often cycle through treatments with typically shorter responses with each new line of therapy. Those who have experienced early disease progression have notably poor prognosis,” said M. Lia Palomba, M.D., TRANSCEND investigator and lymphoma and cell therapy specialist, Memorial Sloan Kettering Cancer Center. “The FDA approval of liso-cel for patients with relapsed or refractory FL is an important advancement in addressing an ongoing unmet need in the FL treatment paradigm, providing patients a new option that has shown remarkably high response rates and an established safety profile.”

Breyanzi has exhibited a consistent safety profile and across clinical trials, any grade cytokine release syndrome (CRS) occurred in 53% of patients, including Grade >3 CRS in 4% of patients. The median time to onset was 5 days (range: 1 to 63 days). Any grade neurologic events (NEs) occurred in 31% of patients, with Grade >3 NEs occurring in 10% of patients. The median time to onset of NEs was 8 days (range: 1 to 63 days). The safety profile of Breyanzi allows for the option of outpatient treatment and management of patients. Patients in the TRANSCEND FL study were treated in the inpatient and outpatient setting.

“The lymphoma community has felt an urgent need for advancements in the treatment of relapsed or refractory follicular lymphoma,” said Meghan Gutierrez, chief executive officer, Lymphoma Research Foundation. “The approval of Breyanzi offers patients a new and meaningful treatment option that provides hope for lasting remission, and we are grateful to those who have contributed to this exciting milestone for patients.”

Bristol Myers Squibb offers various programs and resources to address the needs of patients and caregivers, and provides support that allows for access to therapies, including Breyanzi. Bristol Myers Squibb also supports the patient and physician treatment experience by providing Cell Therapy 360, a digital service platform, which optimizes access to relevant information, manufacturing updates, and patient and caregiver support.

*NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

**Treatment process includes leukapheresis, manufacturing, administration, and adverse event monitoring.

About TRANSCEND FL

TRANSCEND FL (NCT04245839) is an open-label, global, multicenter, Phase 2, single-arm study to determine the efficacy and safety of Breyanzi in patients with relapsed or refractory indolent B-cell non-Hodgkin lymphoma, including follicular lymphoma. The primary outcome measure is overall response rate, including best overall response of complete response or partial response as determined by an Independent Review Committee. Secondary outcome measures include complete response rate, duration of response, progression-free survival, and safety.

About FL

Follicular lymphoma (FL) is the second most common form of non-Hodgkin lymphoma (NHL) and the most common subtype of indolent NHL, accounting for 20 to 30 percent of all NHL cases. The average age of diagnosis for FL is 65 years of age. FL develops when white blood cells cluster together to form lumps in a person’s lymph nodes or organs. It is characterized by periods of remission and relapse, and the disease becomes more difficult to treat after relapse or disease progression.

About Breyanzi

Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR T cells. Breyanzi is made from a patient’s own T cells, which are collected and genetically reengineered to become CAR T cells that are then delivered via infusion as a one-time treatment.

Breyanzi is approved in the U.S. for the treatment of relapsed or refractory large B-cell lymphoma (LBCL) after at least one prior line of therapy and received accelerated approval for the treatment of relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma after at least two prior lines of therapy. Breyanzi is also approved in Japan, the European Union (EU), and Switzerland for the second-line treatment or relapsed or refractory LBCL, and in Japan, the European Union, Switzerland, the UK and Canada for relapsed and refractory LBCL after two or more lines of systemic therapy.

Bristol Myers Squibb’s clinical development program for Breyanzi includes clinical studies in other types of lymphoma. For more information, visit clinicaltrials.gov.

Indication

BREYANZI is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

  • adult patients with large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B, who have:
    • refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy; or
    • refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplantation (HSCT) due to comorbidities or age; or
    • relapsed or refractory disease after two or more lines of systemic therapy.

Limitations of Use: BREYANZI is not indicated for the treatment of patients with primary central nervous system lymphoma.

  • Adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
  • Adult patients with relapsed or refractory follicular lymphoma (FL) who have received 2 or more prior lines of systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

Important Safety Information

BOXED WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, AND SECONDARY HEMATOLOGICAL MALIGNANCIES

  • Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving BREYANZI. Do not administer BREYANZI to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab with or without corticosteroids.
  • Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving BREYANZI, including concurrently with CRS, after CRS resolution or in the absence of CRS. Monitor for neurologic events after treatment with BREYANZI. Provide supportive care and/or corticosteroids as needed.
  • T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI.
  • BREYANZI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the BREYANZI REMS.

Cytokine Release Syndrome (CRS)

Cytokine release syndrome (CRS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. In clinical trials of BREYANZI which enrolled a total of 614 patients with non-Hodgkin lymphoma (NHL), CRS occurred in 53% of patients, including Grade 3 or higher CRS in 4% of patients. The median time to onset was 5 days (range: 1 to 63 days). CRS resolved in 98% of patients with median duration of 5 days (range: 1 to 37 days). The most common manifestations of CRS (>10%) were fever, hypotension, tachycardia, chills, hypoxia and headache.

Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), cardiac arrest, cardiac failure, diffuse alveolar damage, renal insufficiency, capillary leak syndrome, hypotension, hypoxia, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS).

Ensure that 2 doses of tocilizumab are available prior to infusion of BREYANZI.

Neurologic Toxicities

Neurologic toxicities that were fatal or life-threatening, including immune effector cell-associated neurotoxicity syndrome (ICANS), occurred following treatment with BREYANZI. Serious events including cerebral edema and seizures occurred with BREYANZI. Fatal and serious cases of leukoencephalopathy, some attributable to fludarabine, also occurred.

In clinical trials of BREYANZI, CAR T cell-associated neurologic toxicities occurred in 31% of patients, including > Grade 3 cases in 10% of patients. The median time to onset of neurotoxicity was 8 days (range: 1 to 63 days). Neurologic toxicities resolved in 87% of patients with a median duration of 8 days (range: 1 to 119 days). Of patients developing neurotoxicity, 81% also developed CRS.

The most common neurologic toxicities (≥ 5%) included encephalopathy, tremor, aphasia, headache, dizziness, and delirium.

CRS and Neurologic Toxicities Monitoring

Monitor patients daily for at least 7 days following BREYANZI infusion at a REMS-certified healthcare facility for signs and symptoms of CRS and neurologic toxicities and assess for other causes of neurological symptoms. Monitor patients for signs and symptoms of CRS and neurologic toxicities for at least 4 weeks after infusion and treat promptly. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated. Manage neurologic toxicity with supportive care and/or corticosteroid as needed. Counsel patients to seek immediate medical attention should signs or symptoms of CRS or neurologic toxicity occur at any time.

BREYANZI REMS

Because of the risk of CRS and neurologic toxicities, BREYANZI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the BREYANZI REMS. The required components of the BREYANZI REMS are:

  • Healthcare facilities that dispense and administer BREYANZI must be enrolled and comply with the REMS requirements.
  • Certified healthcare facilities must have on-site, immediate access to tocilizumab.
  • Ensure that a minimum of 2 doses of tocilizumab are available for each patient for infusion within 2 hours after BREYANZI infusion, if needed for treatment of CRS.

Further information is available at www.BreyanziREMS.com, or contact Bristol Myers Squibb at 1-866-340-7332.

Hypersensitivity Reactions

Allergic reactions may occur with the infusion of BREYANZI. Serious hypersensitivity reactions, including anaphylaxis, may be due to dimethyl sulfoxide (DMSO).

Serious Infections

Severe infections, including life-threatening or fatal infections, have occurred in patients after BREYANZI infusion. In clinical trials of BREYANZI, infections of any grade occurred in 33% of patients, with Grade 3 or higher infections occurring in 12% of all patients. Grade 3 or higher infections with an unspecified pathogen occurred in 7%, bacterial infections in 4%, viral infections in 2%, and fungal infections in 0.7% of patients. One patient who received four prior lines of therapy developed a fatal case of John Cunningham (JC) virus progressive multifocal leukoencephalopathy four months after treatment with BREYANZI.

Febrile neutropenia developed after BREYANZI infusion in 8% of patients. Febrile neutropenia may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad spectrum antibiotics, fluids, and other supportive care as medically indicated.

Monitor patients for signs and symptoms of infection before and after BREYANZI administration and treat appropriately. Administer prophylactic antimicrobials according to standard institutional guidelines. Avoid administration of BREYANZI in patients with clinically significant, active systemic infections.

Viral reactivation: Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells. In clinical trials of BREYANZI, 26 of 29 patients with prior history of HBV were treated with concurrent antiviral suppressive therapy. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing. In patients with prior history of HBV, consider concurrent antiviral suppressive therapy to prevent HBV reactivation per standard guidelines.

Prolonged Cytopenias

Patients may exhibit cytopenias not resolved for several weeks following lymphodepleting chemotherapy and BREYANZI infusion. In clinical trials of BREYANZI, Grade 3 or higher cytopenias persisted at Day 29 following BREYANZI infusion in 34% of patients, and included thrombocytopenia in 24%, neutropenia in 22%, and anemia in 7% of patients. Monitor complete blood counts prior to and after BREYANZI administration.

Hypogammaglobulinemia

B-cell aplasia and hypogammaglobulinemia can occur in patients receiving treatment with BREYANZI. In clinical trials of BREYANZI, hypogammaglobulinemia was reported as an adverse reaction in 10% of patients. Hypogammaglobulinemia, either as an adverse reaction or laboratory IgG level below 500 mg/dL after infusion, was reported in 29% of patients. Monitor immunoglobulin levels after treatment with BREYANZI and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement as clinically indicated.

Live vaccines: The safety of immunization with live viral vaccines during or following BREYANZI treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during BREYANZI treatment, and until immune recovery following treatment with BREYANZI.

Secondary Malignancies

Patients treated with BREYANZI may develop secondary malignancies. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes. Monitor lifelong for secondary malignancies. In the event that a secondary malignancy occurs, contact Bristol Myers Squibb at 1-888-805-4555 for reporting and to obtain instructions on collection of patient samples for testing.

Effects on Ability to Drive and Use Machines

Due to the potential for neurologic events, including altered mental status or seizures, patients receiving BREYANZI are at risk for developing altered or decreased consciousness or impaired coordination in the 8 weeks following BREYANZI administration. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, for at least 8 weeks.

Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS)

Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis (IEC-HS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. Three of 89 (3%) safety evaluable patients with R/R CLL/SLL developed IEC-HS. Time to onset of IEC-HS ranged from 7 to 18 days. Two of the 3 patients developed IEC-HS in the setting of ongoing CRS and 1 in the setting of ongoing neurotoxicity. IEC-HS was fatal in 2 of 3 patients. One patient had fatal IEC-HS and one had ongoing IEC-HS at time of death. IEC-HS is a life-threatening condition with a high mortality rate if not recognized and treated early. Treatment of IEC-HS should be administered per current practice guidelines.

Adverse Reactions

The most common adverse reactions (incidence ≥ 30%) in:

  • LBCL are fever, cytokine release syndrome, fatigue, musculoskeletal pain, and nausea. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, platelet count decrease, and hemoglobin decrease.
  • CLL/SLL are cytokine release syndrome, encephalopathy, fatigue, musculoskeletal pain, nausea, edema, and diarrhea. The most common Grade 3-4 laboratory abnormalities include neutrophil count decrease, white blood cell decrease, hemoglobin decrease, platelet count decrease, and lymphocyte count decrease.
  • FL are cytokine release syndrome. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decreased, neutrophil count decreased, and white blood cell decrease.

Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.

Bristol Myers Squibb: Creating a Better Future for People with Cancer

Bristol Myers Squibb is inspired by a single vision—transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep understanding of causal human biology, cutting-edge capabilities and differentiated research platforms uniquely position the company to approach cancer from every angle.

Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. As a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

Learn more about the science behind cell therapy and ongoing research at Bristol Myers Squibb here.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements.

Contacts

Bristol Myers Squibb
Media Inquiries:
media@bms.com

Investors:
investor.relations@bms.com

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Nabla Bio Secures $26M Series A Financing and Collaborations with AstraZeneca, Bristol Myers Squibb and Takeda for Generative Protein Design

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➢ Company builds integrated AI and wet lab technologies to design developable, selective, and functionally active protein drugs against multipass membrane protein targets

➢ Radical Ventures leads new investment in Nabla Bio as company secures strategic pharmaceutical collaborations worth more than $550M total

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nabla Bio (aka “Nabla”), pioneers in generative protein design, today announced the close of a $26 million Series A financing, led by Radical Ventures with participation from all existing investors, and strategic collaborations with AstraZeneca, Bristol Myers Squibb Company and Takeda, worth more than $550 million in upfront and milestone payments, plus royalties.

Nabla develops integrated AI and wet-lab technologies that enable atomically precise drug design and high-throughput measurement of drug function, with an initial focus on antibodies targeting multipass membrane proteins, including for example, G protein-coupled receptors (GPCRs), ion channels, and transporters.

“We are unlocking new opportunities to build highly selective drugs against validated, but hard-to-drug targets with a degree of structural precision not previously possible,” said Nabla Co-Founder and CEO Surge Biswas. “We are excited to welcome Radical Ventures as our newest investor and to announce collaborations with three of the world’s largest pharmaceutical companies to help us bring our work closer to patients.”

Today, nearly all approved protein drugs are focused on soluble, extracellular targets, for which traditional brute force drug discovery technologies can yield antibodies with comparative ease. On the other hand, multipass membrane proteins are far more challenging to drug, due to their membrane-integrated nature, limited extracellular availability, conformational dynamics, and structural similarity across targets.

Given multipass membrane targets comprise two-thirds of all cell surface proteins and their central and varied involvement in controlling cell behavior, these targets have long been the “holy grail” of protein drug development. Nabla’s generative protein design platform is unlocking hundreds of these previously inaccessible targets by enabling the precise design of conformation- and target-selective antibody binders.

“With the technologies we’re developing, we could double the number of disease-relevant drug targets the industry goes after,” said Nabla Co-Founder Frances Anastassacos. “That’s an untapped therapeutic opportunity and why several leading pharmaceutical companies have collaborated with us.”

“We’re excited to be working with Nabla and using their generative antibody design and high throughput screening capabilities with the aim to design new drug candidates against challenging targets,” said Puja Sapra, Senior Vice President of Biologics Engineering and Oncology Targeted Delivery, AstraZeneca.

To maximize the patient impact of its platform, Nabla collaborates with leading pharmaceutical companies with a track record of successful drug development to expand their pipelines with high-quality drug candidates against some of the most challenging, high-impact targets. These collaborations also offer valuable insights into disease biology and platform development while offsetting a significant portion of R&D expense, enabling Nabla to grow quickly and sustainably.

Across its collaborations, Nabla has demonstrated the broad applicability of its platform, beyond drug design for multipass membrane proteins. This includes, for example, the design of novel cytokines, complex multi-domain antibodies, and receptor traps with greater in vitro activity and developability than leading drugs on the market. These results are an important step in translating generative protein design to clinical therapies and evidence of Nabla’s expanding capabilities.

“Generative AI and large language models are poised to master the language of proteins in the same way that they have mastered natural language in recent years, with profound implications for medicine,” said Radical Ventures Partner Rob Toews. “The world-class team at Nabla Bio helped pioneer this field. Their groundbreaking work on multipass membrane protein targets offers to revolutionize antibody therapeutics and has won them major commercial partnerships with several of the world’s largest pharmaceutical companies. We are thrilled to lead Nabla’s Series A.”

Nabla Bio launched in December 2021 with funding from Khosla Ventures and Zetta Venture Partners, to apply cutting-edge generative models to the design of protein-based therapies. The company builds on co-founder Surge Biswas’ work on the first protein language models published in a series of Nature Methods and Nature Biotechnology papers (2019, 2021, 2022) alongside colleagues in George Church’s Harvard Lab, which helped establish the field. Frances Anastassacos co-founded Nabla after working in biotech venture capital and completing her PhD at Harvard.

About Nabla Bio

Nabla Bio develops AI and wet-lab technologies that enable the rational design of developable, selective, and functional drugs against previously undruggable targets. Since launching in 2021 the company has raised $37 million with the backing of leading investors, including Radical Ventures, Khosla Ventures and Zetta Venture Partners. The company is headquartered in Cambridge, Mass. and continues to hire top machine learning and synthetic biology talent. For more information visit nabla.bio.

Contacts

Media:
Amanda Guisbond

Intersection: Health

amanda@nabla.bio

Chapter Raises $50 Million Led By XYZ Venture Capital to Help Millions More Seniors Maximize Their Medicare

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The fundraise will be used to accelerate product development for America’s retirees to get the most consumer-first Medicare guidance with the best available technology.

NEW YORK–(BUSINESS WIRE)–Chapter, the fastest-growing Medicare navigation platform in the country, announced today that it raised $50 million in Series C funding led by XYZ Venture Capital with participation from existing investors Narya Capital, Addition, Susa Ventures, and Maverick Ventures. This capital will accelerate product development and hiring as Chapter scales to support 10x more Americans in need of better healthcare coverage. Chapter is also proud to be the Medicare partner for the most forward-leaning healthcare and financial institutions in the country, and the new funding will expand Chapter’s ability to offer cutting-edge enterprise capabilities that drive growth for partners.

Selecting a Medicare plan is one of the most consequential decisions retirees face. Chapter ensures consumers get the right Medicare coverage for their needs at the lowest possible price. As the only unbiased Medicare navigation platform, Chapter aligns incentives between Medicare advisors and consumers by considering all options and making recommendations independent from economic relationships. The Company’s proprietary technology platform ensures the highest quality guidance at scale, allowing it to serve both individual members and organizations that support seniors, including financial advisories, health systems, and content publishers.

“The Medicare industry is in the midst of rapid transformation at every level: policy and regulation, consumer expectations, and underlying plan design. Chapter brings a fiduciary approach to Medicare guidance and advocates for policies that protect consumers,” says Chapter CEO Cobi Blumenfeld-Gantz “I’m so excited to deepen our partnerships across the industry to move faster toward every American getting the Medicare guidance and experience they deserve.”

This fundraise comes on the heels of the most significant regulatory change to how Medicare will reach consumers in over a decade. As the only broker invited to testify before Congress on necessary improvements to Medicare, Chapter pushed for realigning incentives to benefit consumers — a point of view that has since helped shape the newly-issued rules. As one example, the Centers for Medicare and Medicaid Services (CMS) will now prohibit lead generation websites from selling consumer data to multiple buyers simultaneously. This is a welcomed change that will reduce unwanted calls and harassment for America’s seniors. All of this aligns with Chapter’s view of the industry, making it one of the only players in the market advocating for consumer-centric service in compliance with these new rules.

Chapter has partnered with the leading financial institutions, health systems, and content publishers to grow quickly and efficiently. With over 4x year-over-year growth and 5x less churn than the industry standard, Chapter is the frontrunner in the category, influencing how both regulators and industry leaders believe Medicare should be delivered in the future.

“Chapter has designed state-of-the-art technology that makes a truly best-in-class experience work at scale for seniors, while still providing deep, granular information on providers, drug pricing, and more,” says Ross Fubini, Managing Partner at XYZ Venture Capital. “They’re also seeing this incredible pull from the ecosystem at large. Investment advisors, hospital groups, physicians — the biggest content sites devoted to seniors — they’re all eager to deliver better value to their customers and see Chapter as the way to do it. They’re exactly the type of team that makes us so excited to invest in the public sector, and work with folks like them from their earliest innings.”

In addition to this funding announcement, Chapter is excited to share the launch of its new Over The Counter (OTC) product, enabling consumers to quickly and easily redeem OTC benefits in their plans. Many Medicare plans include a host of benefits, but these features are often difficult to navigate and access. The Chapter OTC product allows consumers to scan their insurance card and quickly get the benefits they’ve been promised. This is just one example of the new products the Chapter team has been building, with more to come on the roadmap.

Chapter is excited to bring on new partners, who are already seeing significant organic growth and retention by partnering with Chapter for Medicare guidance. Whether they are healthcare systems, financial services institutions, or senior-facing content publishers, their connections with Chapter have allowed them to amplify the positive impact they too can make on their patients, clients, and readers — all contributing to a better, more holistic system of care for our aging population.

Contacts

For more information:

Jordan McElwain

media@askchapter.org

NanoVibronix Announces Distribution Agreement with VA Supplier CB Medical for the Distribution of UroShield

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TYLER, Texas–(BUSINESS WIRE)–NanoVibronix, Inc. (Nasdaq: NAOV) (“NanoVibronix,” or the “Company”), a medical device company that produces the UroShield®, PainShield® and WoundShield® Surface Acoustic Wave (SAW) Portable Ultrasonic Therapeutic Devices, today announced that it has entered into a distribution agreement with CB Medical, LLC (“CB Medical”) for the sale and distribution of the Company’s UroShield.

UroShield is an ultrasound-based product designed to prevent bacterial colonization and biofilm on indwelling urinary catheters and increase antibiotic efficacy, ultimately reducing the incidence of catheter-associated urinary tract infections (CAUTI). UroShield is also intended to decrease pain and discomfort associated with urinary catheter use.

CB Medical is a U.S. Department of Veteran Affairs (“VA”) certified and Service-Disabled Veteran-Owned Small Business (“SDVOSB”) with two decades of experience serving veterans. Founded by an Iraq War veteran, CB Medical is committed to helping veterans and active-duty members live pain free. Under the terms of the multi-year agreement, CB Medical has the right to sell and distribute UroShield to its customers throughout the VA system in the United States.

Brian Murphy, Chief Executive Officer of NanoVibronix, Inc., commented, “This new agreement provides us with another distribution channel for UroShield within the VA system of providers. CB Medical has a national presence and is committed to helping veterans and active-duty members gain access to beneficial treatments. With millions of urinary catheters used both in and outside of hospitals every year, the risk of infection can be high. UroShield not only prevents infection but can also reduce the pain associated with urinary catheters. We are pleased to be able to add UroShield to CB Medical’s catalog for the benefit of veterans and to advance our efforts towards achieving broader distribution.”

Shawn Pinkston, President and Chief Executive Officer of CB Medical, commented, “We are committed to bringing world-class medical supplies and treatments to beneficiaries of the VA system. There are many veterans, particularly those with spinal injuries, who live with indwelling catheters and are therefore at constant risk of infection. We are pleased to now offer UroShield as both a preventative device for infection and to help reduce the discomfort of living with a urinary catheter.”

About NanoVibronix

NanoVibronix, Inc. (Nasdaq: NAOV) is a medical device company headquartered in Tyler, Texas, with research and development in Nesher, Israel, focused on developing medical devices utilizing its patented low intensity surface acoustic wave (SAW) technology. The proprietary technology allows for the creation of low-frequency ultrasound waves that can be utilized for a variety to medical applications, including for disruption of biofilms and bacterial colonization, as well as for pain relief. The devices can be administered at home without the assistance of medical professionals. The Company’s PainShield® product is a portable device suitable for administration at home without assistance of medical professionals. Additional information about NanoVibronix is available at: www.nanovibronix.com.

Forward-looking Statements

This press release contains “forward-looking statements.” Such statements may be preceded by the words “intends,” “may,” “will,” “plans,” “expects,” “anticipates,” “projects,” “predicts,” “estimates,” “aims,” “believes,” “hopes,” “potential” or similar words. Forward-looking statements are not guarantees of future performance, are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control, and cannot be predicted or quantified; consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with: (i) market acceptance of our existing and new products or lengthy product delays in key markets; (ii) negative or unreliable clinical trial results; (iii) inability to secure regulatory approvals for the sale of our products; (iv) intense competition in the medical device industry from much larger, multinational companies; (v) product liability claims; (vi) product malfunctions; (vii) our limited manufacturing capabilities and reliance on subcontractor assistance; (viii) insufficient or inadequate reimbursements by governmental and/or other third party payers for our products; (ix) our ability to successfully obtain and maintain intellectual property protection covering our products; (x) legislative or regulatory reform impacting the healthcare system in the U.S. or in foreign jurisdictions; (xi) our reliance on single suppliers for certain product components, (xii) the need to raise additional capital to meet our future business requirements and obligations, given the fact that such capital may not be available, or may be costly, dilutive or difficult to obtain; (xiii) our conducting business in foreign jurisdictions exposing us to additional challenges, such as foreign currency exchange rate fluctuations, logistical and communications challenges, the burden and cost of compliance with foreign laws, and political and/or economic instabilities in specific jurisdictions; and (xiv) market and other conditions. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the Securities and Exchange Commission (“SEC”), including the Company’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC’s web site at: http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events, or otherwise, except as required by law.

Contacts

Investor Contacts:
Brett Maas, Managing Principal, Hayden IR, LLC

brett@haydenir.com
(646) 536-7331

4M Therapeutics : Novel Preclinical Data Demonstrating Potent Effects of Inhibitors of GSK3β for the Treatment of Bipolar Disorder

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– GSK3 inhibitors showed successful CNS target engagement and efficacy comparable to lithium at significantly lower doses in models of bipolar disorder

SKILLMAN, N.J.–(BUSINESS WIRE)–4M Therapeutics Inc., an early-stage biotechnology company developing treatments for neuropsychiatric and neurodegenerative conditions, today announced the presentation of a poster at the Society of Biological Psychiatry (SOBP) Annual Meeting, that occurred from May 9-11, 2024, in Austin, Texas. The poster, titled “The Effects of Novel Small Molecules in a Preclinical Model of Positive Affective State,” was presented on May 11, 2024.

“Our presentation underscored the capability of our novel small molecules to selectively target GSK3β and demonstrate efficacy for the treatment of bipolar disorder and acute mania,” said Dr. Pablo Lapuerta, Chief Executive Officer and Co-Founder of 4M Therapeutics. “We continue to accelerate the development of much-needed therapeutic options that have the potential to significantly improve upon the safety and efficacy of the standard of care for patients with neuropsychiatric conditions. We remain on track to initiate a Phase 1 clinical trial for our lead investigational compound in 2025.”

The poster examined the effects of 4M Therapeutics’ glycogen synthase kinase 3 beta (GSK3β) inhibitors, 4MT-A and 4MT-B, on amphetamine (AMP)-induced positive appetitive ultrasonic vocalizations (USVs), a validated model of positive affective state. Marked increases in USVs when treated with AMP were attenuated by lithium, and equal or greater reduction in USVs was observed when treated with 4MT-A and 4MT-B at 1/500th to 1/1000th the concentration of lithium. Similar patterns were observed across USV categories and treatment groups in both the first and second hour after AMP dosing. In addition, supporting data showed target engagement for 4MT-A through the reduction of pCRMP2 in vitro in excitatory cortical neurons and in vivo in rat brain. These statistically significant reductions in pCRMP2 demonstrated GSK3β inhibition affecting a key biomarker.

About 4M Therapeutics Inc.

4M Therapeutics Inc. (4MTx) is advancing treatments for neuropsychiatric and neurodegenerative conditions. The Company focuses on targets for a wide array of disorders and indications. 4MTx applies unique insights from its living human brain cell platform, which was developed through a collaboration between Harvard, MIT, and the University of Washington to identify and design more effective and safer therapeutics. The Company’s pipeline includes potential breakthrough treatments for bipolar mania, agitation in Alzheimer’s disease, neurodegeneration, and other CNS disorders. For more information, visit www.4mtx.net.

Contacts

For investor and media inquiries, please contact:
Kimberly Lee, DO

Chief Business Officer

ir@4mtx.net

Cardurion Pharmaceuticals Presents Positive Clinical Results from CARDINAL‑HF Phase 2a Clinical Trial of PDE9 Inhibitor in Patients With Heart Failure

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– PDE9 inhibitor, CRD-740, demonstrated favorable safety profile and achieved statistical significance for the trial’s primary endpoint, median increase in plasma cyclic guanosine monophosphate (cGMP) –

– This clinical trial confirms that high levels of PDE9 inhibition lead to increases in cGMP, reflecting increased activation of the myocardial natriuretic peptide (NP) signaling pathway –

– Targeting PDE9 represents a novel approach to activating the NP signaling pathway, a highly validated pathway with established clinical benefits in heart failure –

– Clinical results presented today at the Annual Congress of the Heart Failure Association of the European Society of Cardiology –

BURLINGTON, Mass.–(BUSINESS WIRE)–Cardurion Pharmaceuticals, Inc. (“Cardurion”), a clinical-stage biotechnology company developing next-generation therapeutics for the treatment of cardiovascular diseases, today announced the presentation of positive clinical data from CARDINAL‑HF, the first Phase 2 proof-of-concept clinical trial of a phosphodiesterase-9 (PDE9) inhibitor in patients with heart failure. These data were presented today at the Annual Congress of the Heart Failure Association of the European Society of Cardiology taking place on May 11-14 in Lisbon, Portugal.

In the CARDINAL-HF Phase 2a trial, CRD-740 met the primary endpoint in patients with heart failure with reduced ejection fraction (HFrEF), demonstrating a statistically significant median increase in plasma cyclic guanosine monophosphate (cGMP) after four weeks of treatment. Plasma cGMP is a biomarker for intracellular cGMP whose levels reflect the activity of the protective myocardial natriuretic peptide (NP) signaling pathway, which has proven clinical benefits in heart failure. CRD-740 was generally well tolerated in the trial.

“These very promising data from the first Phase 2 proof-of-concept clinical trial of a PDE9 inhibitor in patients with heart failure represent an important next step in the development of this novel mechanism,” said James Udelson, MD, Chief of Cardiology at Tufts Medical Center and Principal Investigator for the CARDINAL-HF trial. “The results of this trial are impressive, showing robust PDE9 inhibition with significant increases in plasma and urinary cGMP, along with a favorable safety profile, both of which support moving into further clinical testing in heart failure.”

“Therapeutic targeting of the NP signaling pathway is precedented by today’s standard of care treatment for patients with heart failure, and PDE9 inhibition represents a new mechanism for activating this pathway to seek improved patient outcomes. Significant unmet needs remain, as heart failure is a prevalent and growing chronic condition that causes significant morbidity and mortality for millions of people,” said Scott D. Solomon, MD, Professor of Medicine at Harvard Medical School.

The oral presentation of these results, entitled, “A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Tolerability and Pharmacodynamic Effects of CRD-740, a PDE9 Inhibitor, in Participants with Chronic Heart Failure,” were presented today by James Udelson, M.D., Principal Investigator of CARDINAL-HF and Chief of Cardiology at Tufts Medical Center, in the “Late-Breaking Clinical Trials: Medical Therapy” session at the Annual Congress of the Heart Failure Association of the European Society of Cardiology. Key findings presented include:

  • CRD-740 was generally well-tolerated in patients with HFrEF, a disease representing approximately one-half of patients with chronic heart failure.
  • PDE9 inhibition with CRD-740 achieved statistically significant median increases in plasma cGMP at four weeks, compared to placebo, which was the primary endpoint in the trial. Statistically significant median increases in urinary cGMP were also observed in the patients who received CRD-740, compared to placebo.
  • Increases in cGMP of the magnitude seen in the trial demonstrate that CRD-740 achieves high levels of PDE9 inhibition, which prevents cGMP metabolism by the PDE9 enzyme and leads to increased activation of the NP signaling pathway. Activation of the clinically validated NP signaling pathway has been shown to benefit patients with chronic heart failure in outcome studies with sacubitril/valsartan.
  • These increases in plasma and urinary cGMP were observed in patients who received CRD‑740 with and without background treatment with sacubitril/valsartan, supporting the potential for CRD-740 as a monotherapy, and as a new approach to augment efficacy in the setting of sacubitril/valsartan therapy and to further activate the NP signaling pathway.

Based on the results of the CARDINAL-HF Phase 2a trial, Cardurion has launched two Phase 2 clinical trials in 640 patients, including a dose-ranging trial in patients with HFrEF and a proof-of-concept trial in patients with heart failure with preserved ejection fraction (HFpEF).

“These findings from Cardurion’s pioneering program in PDE9 support our conviction that PDE9 inhibition presents an important new mechanism for independently targeting and further activating the well-validated NP signaling pathway. The data from this trial suggest that PDE9 inhibition has the potential to provide benefit to patients when administered alone or in combination with guideline directed medical therapy, and ultimately become standard of care for patients with both types of heart failure,” said Peter Lawrence, Chief Executive Officer of Cardurion Pharmaceuticals.

“We are delighted to share these clinical results for CRD-740 as our team advances PDE9 inhibition as a novel approach to addressing the unmet needs of patients with chronic heart failure,” said Howard Surks, MD, Chief Medical and Scientific Officer of Cardurion Pharmaceuticals. “We thank our patients and investigators for their partnership and look forward to continuing the development of our PDE9 inhibitors to improve outcomes for patients.”

About the CARDINAL-HF clinical trial

CARDINAL-HF is the first Phase 2 proof-of-concept trial of a PDE9 inhibitor for the treatment of patients with heart failure. The Phase 2a clinical trial is a randomized, placebo-controlled trial of 60 chronic, stable patients with heart failure with reduced ejection fraction (HFrEF) who were receiving guideline-directed medical therapy (GDMT). The primary endpoints of the trial are safety and tolerability of CRD-740 and changes in plasma cGMP at four weeks, a precedented biomarker for activation of the NP signaling pathway. Other endpoints include changes in urinary cGMP and N-terminal pro b-type natriuretic peptide (NTpro-BNP) and effect of CRD-740 administration on the Kansas City Cardiomyopathy questionnaire (KCCQ), which measures symptoms, physical and social limitations, and quality of life in patients with heart failure.

The Executive Committee for the CARDINAL-HF trial includes Dr. James Udelson, Principal Investigator and Chief of Cardiology at Tufts Medical Center; Dr. Scott Solomon, Professor of Medicine at Harvard Medical School; and Dr. John McMurray, Professor of Medical Cardiology and Deputy Director of the Institute of Cardiovascular and Medical Sciences at the University of Glasgow. Drs. Udelson, Solomon and McMurray, along with Dr. Eugene Braunwald, the Distinguished Hersey Professor of Medicine at Harvard Medical School, also lead Cardurion’s Clinical Advisory Board. The CARDINAL-HF Steering Committee is further comprised of leading international heart failure clinical investigators.

About PDE9 inhibition

PDE9 inhibitors target phosphodiesterase (PDE) 9, an enzyme whose elevated activity in patients with heart failure reduces the beneficial effects of the natriuretic peptide (NP) pathway, fundamental to cardiovascular homeostasis. The beneficial effects of the NP signaling pathway in the heart muscle cell are mediated by cyclic guanosine monophosphate (cGMP) and activation of its downstream signaling molecule protein kinase G1. PDE9 is an enzyme that selectively degrades cGMP; therefore, by inhibiting PDE9, our goal is to preserve cGMP generation and enhance the beneficial natriuretic peptide signaling within heart muscle cells.

About Chronic Heart Failure

Heart failure is a prevalent and growing condition that is responsible for substantial morbidity and mortality. Approximately 6.5 million people in the United States suffer from heart failure, and 50 percent of these patients die from the condition within five years of diagnosis. One in five patients with heart failure are hospitalized annually, and 25% of these patients will be admitted again within a month of discharge. Approximately half of all patients with heart failure have reduced ejection fraction (HFrEF) and half have preserved ejection fraction (HFpEF). Despite the availability of drugs indicated to reduce morbidity and mortality in patients with both types of heart failure, substantial unmet medical need remains.

About Cardurion Pharmaceuticals

Cardurion Pharmaceuticals is a clinical-stage biotechnology company focused on the discovery and development of novel, next-generation therapeutics for the treatment of cardiovascular diseases. Cardurion was founded by physician-scientists with world-class expertise in cardiovascular signaling pathways, and a shared passion to find and develop a pipeline of novel treatment options to improve the lives of patients. Cardurion has two groundbreaking clinical programs in development, a PDE9 inhibitor targeting heart failure and the first ever CaMKII inhibitor in clinical development targeting multiple cardiovascular indications.

Cardurion Pharmaceuticals has facilities in Burlington, Massachusetts and Shonan, Japan. For more information, please visit the company’s website at https://cardurion.com.

Contacts

Kathryn Morris

The Yates Network

kathryn@theyatesnetwork.com

Be Biopharma Announces New Preclinical Data for Novel B Cell Medicine for the Potential Treatment of Hypophosphatasia

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  • Preclinical research demonstrates that CRISPR/Cas9-based precision B cell gene engineering coupled with artificial intelligence-guided protein design produces active tissue-nonspecific alkaline phosphatase (“ALP”)
  • Data presented at American Society of Gene & Cell Therapy 27th Annual Meeting

Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), today presented results from new preclinical research demonstrating production of active ALP by a BCM, which highlights BCMs as a potential treatment for Hypophosphatasia (HPP). Researchers used CRISPR/Cas9 precision gene engineering and artificial intelligence-guided protein design to modify primary human B cells to produce ALP, an enzyme deficient in people living with HPP. The findings were presented during a poster presentation at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting on Friday, May 10, at 12:00pm ET.

HPP is a rare genetic disease caused by loss of function mutations in the ALPL gene which leads to deficient ALP activity. People living with HPP can experience wide ranging systemic complications, with impaired bone mineralization, such as rickets/osteomalacia, being a major clinical hallmark of severe disease. The only approved therapy for HPP is an enzyme replacement therapy, asfotase alfa, which requires multiple injections every week and is approved only for use in patients with pediatric-onset forms of HPP.

“This study demonstrates how BCMs coupled with artificial intelligence-guided protein design broaden the potential of our medicines to express highly effective conjugated therapeutic proteins such as ALP-Fc fusion proteins,” said Rick Morgan, Chief Scientific Officer. “BCMs are designed to provide constant and durable protein levels without preconditioning, are redosable, and can be applied to a wide range of diseases, including a potential first-in-class medicine for people living with HPP.”

In this study, primary human B cells were expanded and precision engineered by CRISPR/Cas9 genome editing with AAV-mediated homology directed repair (HDR) to insert an ALP gene expression cassette into various loci, including CCR5 (a safe harbor locus). Guided by an artificial intelligence-based ALP protein structure design engine, protein constructs were optimized for activity and stability of ALP-Fc fusion proteins. Engineered BCMs secreted active ALP proteins up to 200 ng/1e6 cells/24hr. Robust in vitro phenotypic correction using ALP secreting BCMs (ALP-BCMs) was demonstrated in the MC3T3 osteoblast precursor mineralization model.

About Engineered B Cell Medicines – A New Class of Cellular Medicines

The B cell is a powerful cell that produces thousands of proteins per cell per second at constant levels, and over decades. Precision genome editing can now be used to engineer B Cells that produce therapeutic proteins of interest, driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs) – with the potential to be durable, allogeneic, redosable, and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics with broad application — across protein classes, patient populations and therapeutic areas.

About Be Biopharma

Be Biopharma (“Be Bio”) is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020 by B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., from Seattle Children’s Research Institute. Be Bio is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute and others. Since our founding, Be Bio’s investors have committed over $180 million to enable the Company to re-imagine medicine based on the power of B cell therapy. For more information, please visit us at Be.Bio and our LinkedIn page.

Contacts

Investors:
ir@be.bio

Media:
media@be.bio

Sanofi and Novavax announce co-exclusive licensing agreement to co-commercialize COVID-19 vaccine and develop novel flu-COVID-19 combination vaccines

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Sanofi and Novavax announce co-exclusive licensing agreement to co-commercialize COVID-19 vaccine and develop novel flu-COVID-19 combination vaccines

  • Agreement provides patients with broader access to a protein-based non-mRNA adjuvanted COVID-19 vaccine through combined commercial strength, from 2025 onwards
  • Accelerates potential for development of a novel flu-COVID-19 combination product based on authorized vaccines with demonstrated efficacy and tolerability, potentially offering patients enhanced convenience and protection

Paris and Gaithersburg, Md., United States. May 10, 2024. As part of Sanofi’s commitment to developing a diverse portfolio of best-in-class vaccines, the company has entered into a co-exclusive licensing agreement with Novavax, a biotechnology company headquartered in Maryland, US.

The terms of the agreement include: a co-exclusive license to co-commercialize Novavax’s current stand-alone adjuvanted COVID-19 vaccine worldwide (except in countries with existing Advance Purchase Agreements and in India, Japan, and South Korea where Novavax has existing partnership agreements); a sole license to Novavax’s adjuvanted COVID-19 vaccine for use in combination with Sanofi’s flu vaccines; and a non-exclusive license to use the Matrix-M adjuvant in vaccine products. In addition, Sanofi will take a minority (<5%) equity investment in Novavax.

Jean-Francois Toussaint
Global Head of Vaccines R&D
“With flu and COVID-19 hospital admission rates now closely mirroring each other, we have an opportunity to develop non-mRNA flu-COVID-19 combination vaccines offering patients both enhanced convenience and protection against two serious respiratory viruses. We’re excited by the prospect of combining Novavax’s adjuvanted COVID-19 vaccine that has shown high efficacy and favorable tolerability, with our rich portfolio of differentiated flu vaccines that have demonstrated superior protection against flu and its serious complications. Improved tolerability and thermostability, without compromise on efficacy, are what regulators, recommending bodies, and patients will demand.”

John Jacobs
CEO, Novavax

“This collaboration is important for Novavax and for global public health. Our new partnership combines Novavax’s proprietary recombinant protein and nanoparticle technologies, Matrix™ adjuvant, and R&D expertise with Sanofi’s world-class leadership in launching and commercializing innovative vaccines. Together, we can broaden access to both our COVID-19 vaccine and our adjuvant to ensure more individuals can benefit from the protection vaccines can provide. Novavax is now in a stronger position to refocus our efforts on leveraging our technology platform and novel adjuvant in research and development and pipeline expansion to help advance our mission of developing life-saving vaccines to fight infectious diseases.” 

Under the terms of the licensing agreement:

  • Novavax will receive an upfront payment of $500 million and up to $700 million in development, regulatory and launch milestones, up to $1.2 billion in total.
  • Starting in 2025, Sanofi will book sales of Novavax’s adjuvanted COVID-19 vaccine and will support certain R&D, regulatory, and commercial expenses.
  • Novavax will receive tiered double-digit percentage royalty payments on sales by Sanofi of COVID-19 vaccines and flu-COVID-19 combination vaccines.
  • Sanofi will be solely responsible for development and commercialization of any novel flu-COVID-19 combination vaccine containing a Sanofi flu vaccine.
  • Outside of the collaboration, each party may develop and commercialize their own flu-COVID-19 vaccines and adjuvanted products at their own cost.
  • Novavax is entitled to additional launch and sales milestones opportunities of up to $200 million plus mid-single digit royalties for each additional Sanofi vaccine product developed under a non-exclusive license with Novavax’s Matrix-M adjuvant technology.
  • In addition, Sanofi will take a minority (<5%) equity investment in Novavax.

About Novavax
Novavax, Inc. (Nasdaq: NVAX) promotes improved health by discovering, developing and commercializing innovative vaccines to help protect against serious infectious diseases. Novavax, a global company based in Gaithersburg, Md., U.S., offers a differentiated vaccine platform that combines a recombinant protein approach, innovative nanoparticle technology and Novavax’s patented Matrix-M adjuvant to enhance the immune response. The Company’s portfolio includes its COVID-19 vaccine, and its pipeline includes CIC and stand-alone influenza vaccine candidates. In addition, Novavax’s adjuvant is included in the University of Oxford and Serum Institute of India’s R21/Matrix-M malaria vaccine. Please visit novavax.com and LinkedIn for more.

About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

Media Relations
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com
Sally Bain | + 1 617 834 6026 | sally.bain@sanofi.com
Evan Berland | + 1 215 432 0234 | evan.berland@sanofi.com

Nicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.com

Investor Relations
Thomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.com
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com
Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com
Corentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com
Tarik Elgoutni| + 1 617 710 3587 | tarik.elgoutni@sanofi.com
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com

Sanofi Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2023. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

Attachment