Prestige Biopharma Receives FDA Fast Track Designation for PBP1510 in the Treatment of Pancreatic Cancer

Prestige Biopharma Receives FDA Fast Track Designation for PBP1510 in the Treatment of Pancreatic Cancer




Prestige Biopharma Receives FDA Fast Track Designation for PBP1510 in the Treatment of Pancreatic Cancer

SINGAPORE–(BUSINESS WIRE)–Prestige Biopharma has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for PBP1510 (International Non-proprietary name: Ulenistamab), in the treatment of unresectable or metastatic pancreatic adenocarcinoma (PDAC) that has relapsed following and/or is refractory to at least one line of prior therapy.

PBP1510 targets Pancreatic Adenocarcinoma Upregulated Factor (PAUF), a tumour-specific protein, found to be overexpressed in majority of pancreatic cancer cases. PAUF overexpression promotes key cellular functions, including proliferation, migration, invasion, and growth of pancreatic cancer cells, and contributes to the development of acquired resistance to chemotherapeutic agents. PBP1510 is designed to target these key biological mechanisms that results in limited effectiveness of current treatment options and rapid progression of pancreatic cancer.

By effectively inhibiting the tumorigenic effects of PAUF overexpression in preclinical models, PBP1510 represents a promising therapeutic strategy for addressing the unmet medical needs of pancreatic cancer patients. A global Phase 1/2a clinical trial is currently underway in the United States, Europe, and Asia, with the aim of bringing this innovative therapy to the clinic.

The first-in-human Phase 1/2a study is an open-label, multicentre, two-part study in patients with advanced/metastatic pancreatic cancer. Phase 1 is a dose-escalation phase, wherein PBP1510 will be administered, either as monotherapy or in combination with gemcitabine, in two separate dose-escalation cohorts. From Phase 1 part of the study a recommended Phase 2a dose (RP2D) will be determined based on the analysis of pharmacokinetics, safety, and efficacy data. Phase 2 is a dose-expansion phase, wherein PBP1510 at the RP2D in combination with gemcitabine will be administered to evaluate efficacy and safety of PBP1510.

Overall, the Phase 1/2a study aims to collect important safety data on the use of PBP1510 as a monotherapy or in combination with gemcitabine and explore the efficacy of a combined PBP1510 and gemcitabine regimen. The study will substantiate the preclinical findings of PBP1510’s synergistic antitumour activity in combination therapy with gemcitabine without increased toxicity, as anticipated from their distinct mechanisms of action.

With Fast Track designation from the FDA, PBP1510 represents a promising advancement in the treatment of pancreatic cancer. Prestige Biopharma intends to take full advantage of the benefits offered by the designation to provide faster access for patients in need.

Contacts

info@prestigebio.com

Top 6 Arizona bioscience startups in 2023

Top 6 Arizona bioscience startups in 2023




On the rise in 2023: Six Arizona bioscience startups

Phoenix area, Tucson companies selected for Flinn Foundation program

PHOENIX–(BUSINESS WIRE)–#Arizona–The Arizona bioscience startups representing the 10th cohort of the Flinn Foundation Bioscience Entrepreneurship Program are developing innovative products and devices to better treat cancer, improve mental health, provide long-lasting refrigeration of blood and medicine, and more efficiently and safely move patients.

The six early-stage firms competitively selected for the 2023 program include two companies each from Phoenix, Scottsdale, and Tucson.

Over the next year, the program participants will receive $30,000 in funding, a personalized learning plan with other support services facilitated by the Arizona Bioindustry Association (AZBio), and invitations to exclusive gatherings of Arizona bioscience and policy leaders as well as convenings with fellow entrepreneurs.

“These companies are developing cutting-edge diagnostics and treatments—for someone suffering from cancer to social isolation—which are critical to our physical and mental health,” said Tammy McLeod, Flinn Foundation president and CEO. “A record 48 firms applied for our program this year—a testament to Arizona’s vibrant bioscience startup culture.”

The 2023 Flinn Foundation Bioscience Entrepreneurship Program participants are:

Delta Development Team (Tucson)

Delta Development Team is a developer of ruggedized refrigeration systems, specializing in military applications. The Tucson-based company leveraged the knowledge and wisdom of military medical personnel to design its Autonomous Portable Refrigeration Unit, which provides days of use from one battery charge and is designed to withstand harsh weather conditions, so medical-care teams can take it with them no matter where their skills are needed.

EMR Data Cloud (Scottsdale)

Located in Scottsdale, EMR Data Cloud offers a platform for clinical genetic testing with electronic medical record (EMR) integration. Users can order genomic panels and other laboratory tests with just a few clicks on its platform. The company’s lab-integration technology bridges the gap between health care providers and diagnostic laboratories and quickly transfers information, including clinical notes, family history, medication lists, and insurance information in seconds.

Reference Medicine (Phoenix)

This Phoenix-based company’s mission is to become a one-stop shop for oncology biospecimens, enabling researchers to easily get specimens they need, at half the cost, to build cancer diagnostics. The company will address the gap in the oncology biospecimen market by combining partnerships with accredited teams worldwide with an innovative approach to specimen formatting. Its approach ensures that each set of specimens is fully utilized and creates a variety of formats, enabling the company to connect an individual donation to a diverse group of diagnostic research teams—increasing supply of critical specimens while driving down the cost for researchers.

Televeda (Phoenix)

Televeda is a mental-health platform used by public-health and community-based organizations to combat social isolation for vulnerable populations. The platform incorporates robust accessibility designs including a proprietary live-streaming user interface, an administration dashboard for CBOs to source content, hybrid-event management for broadcasting, data-reporting, and white-labeling. The Phoenix-based company helps streamline operations, saving clients 15-25 hours and $2,000-4,000 a month while improving their community-outreach metrics.

The Patient Company (Scottsdale)

The Patient Company is reimagining how patients are moved, starting with lateral patient transfer—moving a patient from one flat surface to another. The process currently takes a median of 22 minutes and is the second-leading cause of injury for health care employees. The Scottsdale-based company has developed SimPull™, a device that can be operated by a single clinical staff member, allowing for safe, efficient, and effective lateral transfer of patients, while decreasing the transfer time to 2 minutes.

TheraCea Pharma (Tucson)

Tucson-based TheraCea Pharma addresses the greatest challenge in the emerging $145 billion cancer immunotherapy market by providing clinical diagnostic products for patient selection and therapy guidance. TheraCea detects immunotherapy biomarkers using PET imaging. The company has developed a set of diagnostic agents that allow oncologists and pharmaceutical companies to select the right segment of patients who will be responsive to specific immunotherapy drugs.

The Flinn Foundation program attracted applicants from across the state. The six firms were selected following a two-stage review process, including virtual interviews with more than a dozen finalists.

The $30,000 in funding support each company receives is administered by AZBio, a trade association that promotes Arizona’s bioscience sector. Each participant receives a one-year membership to both AZBio and Arizona’s Bioscience Roadmap Steering Committee, which includes about 135 leaders from the public and private sectors in science, health care, business, academia, and policy who guide Arizona’s Bioscience Roadmap.

The Flinn Foundation has competitively selected 60 Arizona companies and provided $1.78 million in funding support since the inception of the program in 2014.

To learn more about the Flinn Foundation Bioscience Entrepreneurship Program and the selected firms, visit flinn.org/entrepreneur.

Contacts

Brian Powell, Flinn Foundation Communications Manager, (602) 744-6806, bpowell@flinn.org
Brad Halvorsen, Flinn Foundation Executive Vice President, (602) 744-6803, bhalvorsen@flinn.org

Top 6 Arizona bioscience startups in 2023

Tvardi Therapeutics Announces First Patients Dosed in Phase 2 Trial of TTI-101 in Metastatic Breast Cancer

Tvardi Therapeutics Announces First Patients Dosed in Phase 2 Trial of TTI-101 in Metastatic Breast Cancer




Tvardi Therapeutics Announces First Patients Dosed in Phase 2 Trial of TTI-101 in Metastatic Breast Cancer

The REVERT Trial is Evaluating STAT3 Inhibitor, TTI-101, Added to Palbociclib and an Aromatase Inhibitor to Overcome Resistance and Improve Clinical Outcomes for Metastatic Breast Cancer Patients

HOUSTON–(BUSINESS WIRE)–#IPF–Tvardi Therapeutics, Inc., a privately held, clinical-stage biopharmaceutical company focused on the development of STAT3 inhibitors, announced that the first breast cancer patients have been dosed in the Phase 1b/2 REVERT trial of TTI-101 added to palbociclib and aromatase inhibitor (AI) therapy in adult patients with HR+/HER2 palbociclib-resistant metastatic breast cancer. The first patients were dosed at the Washington University School of Medicine Siteman Cancer Center by Cynthia Ma, MD, PhD, Professor of Medicine, Clinical Director of the Breast Cancer Program, Section of Medical Oncology.

In the US, approximately 74,000 patients per year are diagnosed with HR+/HER2 metastatic breast cancer. These patients are generally treated with CDK4/6 inhibitors (such as palbociclib) and an AI. Unfortunately, the vast majority of these patients acquire resistance to palbociclib and AI therapy and their tumors continue to metastasize. Recent research has demonstrated that this resistance is driven by the activation of a protein in tumors known as STAT3. Tvardi has developed a STAT3 inhibitor, TTI-101, that has been well tolerated and has clinical activity across a broad range of tumors. The REVERT trial has been designed to add TTI-101 to palbociclib and AI therapy when breast cancer patients acquire resistance to standard therapy.

“I am excited the Washington University School of Medicine Siteman Cancer Center was the first to enroll patients to this important trial. The addition of TTI-101 to palbociclib and AI directly addresses the mechanism which leads to patients becoming resistant to the standard of care. TTI-101 has the potential to improve clinical outcomes for HR+/HER2 metastatic breast cancer patients,” said Cynthia Ma, MD, PhD.

“STAT3 is a well-known driver of tumor resistance. Based on Phase 1 trial data, TTI-101 specifically targets STAT3, and is well-positioned to reverse the resistance pathway for metastatic breast cancer,” said Imran Alibhai, PhD, CEO of Tvardi. “This is the first of three Phase 2 trials in metastatic breast cancer, advanced liver cancer, and idiopathic pulmonary fibrosis that Tvardi has initiated to address diseases driven by STAT3.”

For more information about the REVERT breast cancer trial that is enrolling at sites throughout the US, please visit ClinicalTrials.gov (NCT05384119).

About Tvardi Therapeutics

Tvardi is a privately held, clinical-stage biopharmaceutical company developing small molecule inhibitors of STAT3, a key regulatory protein positioned at the intersection of many signaling pathways integral to the survival and immune evasion of cancer cells. STAT3 also plays a central role in the pathogenesis of many inflammatory and fibrotic diseases. The company’s lead product, TTI-101, completed enrollment in its first-in-man Phase 1 trial of relapsed/refractory patients with advanced solid tumors. To date, TTI-101 monotherapy has been well-tolerated and has clinical activity across a broad range of tumors including multiple durable radiographic objective responses. The company has now initiated three Phase 2 clinical programs in hepatocellular carcinoma (NCT05440708), metastatic breast cancer (NCT05384119), and idiopathic pulmonary fibrosis (NCT05671835). To learn more, please visit https://tvarditherapeutics.com/.

Contacts

Tvardi Investor Relations

Sara Manning

ir@tvardi.com

Junshi Biosciences Announces Toripalimab in Combination with Chemotherapy for Treatment of Advanced Triple-negative Breast Cancer Met Primary Endpoint in Phase 3 Clinical Study

Junshi Biosciences Announces Toripalimab in Combination with Chemotherapy for Treatment of Advanced Triple-negative Breast Cancer Met Primary Endpoint in Phase 3 Clinical Study




Junshi Biosciences Announces Toripalimab in Combination with Chemotherapy for Treatment of Advanced Triple-negative Breast Cancer Met Primary Endpoint in Phase 3 Clinical Study

SHANGHAI, China, Feb. 20, 2023 (GLOBE NEWSWIRE) — Shanghai Junshi Biosciences Co., Ltd (“Junshi Biosciences”, HKEX: 1877; SSE: 688180) today announced that the pre-specified interim analysis has been completed for a randomized, double-blind, placebo-controlled, multi-center phase III clinical study (“TORCHLIGHT Study”, NCT04085276) examining the company’s product toripalimab in combination with paclitaxel for injection (albumin-bound) in patients with an initial diagnosis of stage IV or recurrent/metastatic triple-negative breast cancer. The Independent Data Monitoring Committee (IDMC) has determined that the primary endpoint met the pre-defined efficacy boundary. Junshi Biosciences will communicate with the regulatory authorities regarding matters related to the supplemental new drug application in the near future.

According to GLOBOCAN 2020, breast cancer had the highest incidence rates worldwide, with 2.26 million new cases and 0.68 million deaths in 2020. In China, 0.42 million new cases and 0.12 million deaths due to breast cancer were reported in 2020, accounting for 18.4% and 17.1% of global cases, respectively. Amongst these breast cancer cases, triple-negative breast cancer (“TNBC”) accounted for approximately 15% to 20% of them. TNBC is a more aggressive type of tumor with a higher risk of recurrence and poor prognosis. Advanced TNBC is insensitive to targeted therapy and endocrine therapy, and there are currently no specific treatment methods available.

In recent years, oncology immunotherapy drugs represented by PD-(L)1 inhibitors have achieved a series of breakthroughs in treating various types of tumors. However, to this day, no immunotherapy drugs have been approved for advanced TNBC in China, and chemotherapy remains the primary treatment option. Alternative drugs include anthracyclines, taxanes, platinum-based drugs, etc. Both mono-chemotherapy and combined chemotherapy have poor efficacy, with a median survival time of about nine to 12 months and a 5-year survival rate of less than 30%.

The TORCHLIGHT study is the first Phase III registration study in China to achieve a positive outcome in an advanced triple-negative breast cancer immunotherapy. This randomized, double-blind, placebo-controlled, multi-center Phase III clinical study was designed to compare the safety and efficacy of toripalimab combined with paclitaxel for injection (albumin-bound) and placebo combined with paclitaxel for injection (albumin-bound) in patients with an initial diagnosis of stage IV breast cancer or recurrent/metastatic triple-negative breast cancer.

The interim analysis of this study demonstrated that, compared with paclitaxel for injection (albumin-bound), toripalimab in combination with paclitaxel for injection (albumin-bound) in patients with initial diagnosis of stage IV or recurrent metastatic triple-negative breast cancer can significantly prolong the progression-free survival (PFS) of PD-L1 positive patients. Meanwhile, the overall survival (OS), one of the secondary endpoints, also showed a clear trend of improvement in PD-1 positive patients as well as in all patients regardless of PD-1 status. The safety data of toripalimab is consistent with known risks, and no new safety signals were identified.

“The TNBC subtype of breast cancer is the most aggressive and has the worst prognosis,” said Professor Zefei JIANG from the Department of Oncology at the Chinese People’s Liberation Army General Hospital, the Vice President and Secretary General of the Chinese Society of Clinical Oncology (CSCO) and Principal Investigator of the TORCHLIGHT study. “Advanced TNBC patients have limited survival rates and lack effective treatment methods. Aiming to improve patient survival, a group of Chinese researchers successfully conducted the first phase III study on immune-oncology for advanced TNBC patients, even amidst the COVID-19 pandemic, and achieved breakthrough results. These results demonstrate that the combined use of the monoclonal antibody, toripalimab, with traditional chemotherapy significantly prolonged the PFS of patients, and this has the potential to become a new standard treatment for patients with an initial diagnosis of stage IV TNBC as well as those with recurrent/metastatic TNBC, offering patients new hope!”

“I’m extremely pleased that TORCHLIGHT research has been successful, and its success is a result of the dedicated efforts of patients, researchers, and development teams,” said Dr. Jianjun ZOU, the President of Global Research and Development at Junshi Biosciences. “We will work closely with regulatory authorities to ensure that relevant indications are approved as soon as possible. We hope that toripalimab will provide better treatment options for patients and address unmet medical needs.”

About Toripalimab

Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system’s ability to attack and kill tumor cells.

More than thirty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally by Junshi Biosciences, including in China, the United States, Southeast Asia, and European countries. Ongoing or completed pivotal clinical trials evaluating the safety and efficacy of toripalimab cover a broad range of tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney and skin.

In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI®). Currently, there are six approved indications for toripalimab in China:

  1. unresectable or metastatic melanoma after failure of standard systemic therapy;
  2. recurrent or metastatic NPC after failure of at least two lines of prior systemic therapy;
  3. locally advanced or metastatic urothelial carcinoma that failed platinum-containing chemotherapy or progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy;
  4. in combination with cisplatin and gemcitabine as the first-line treatment for patients with locally recurrent or metastatic NPC;
  5. in combination with paclitaxel and cisplatin in first-line treatment of patients with unresectable locally advanced/recurrent or distant metastatic esophageal squamous cell carcinoma (“ESCC”);
  6. in combination with pemetrexed and platinum as the first-line treatment in EGFR mutation-negative and ALK mutation-negative, unresectable, locally advanced or metastatic non-squamous non-small cell lung cancer (“NSCLC”).

The first three indications have been included in the National Reimbursement Drug List (NRDL) (2022 Edition). Toripalimab is the only anti-PD-1 monoclonal antibody included in the NRDL for treatment of melanoma.

In terms of international layout, the Biologics License Application (BLA) for toripalimab in combination with gemcitabine/cisplatin, for the first-line treatment of patients with advanced recurrent or metastatic NPC and toripalimab monotherapy for the second-line or later treatment of recurrent or metastatic NPC after platinum-containing chemotherapy is under review by the U.S. Food and Drug Administration (FDA). In December 2022 and February 2023, the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) accepted the marketing authorization application (MAA) for toripalimab in combination with cisplatin and gemcitabine for the first-line treatment of patients with locally recurrent or metastatic NPC, and toripalimab in combination with paclitaxel and cisplatin for the first-line treatment of patients with unresectable locally advanced/recurrent or metastatic esophageal squamous cell carcinoma, respectively.

About Junshi Biosciences
Founded in December 2012, Junshi Biosciences (HKEX: 1877; SSE: 688180) is an innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics. The company has established a diversified R&D pipeline comprising over 50 drug candidates, with five therapeutic focus areas covering cancer, autoimmune, metabolic, neurological, and infectious diseases. Junshi Biosciences was the first Chinese pharmaceutical company that obtained marketing approval for anti-PD-1 monoclonal antibody in China. Its first-in-human anti-BTLA monoclonal antibody for the treatment of various cancers was the first in the world to be approved for clinical trials by the FDA and NMPA and has since entered Phase Ib/II trials in both China and the US. Its anti-PCSK9 monoclonal antibody was the first in China to be approved for clinical trials by the NMPA.

In the face of the pandemic, Junshi Biosciences’ response was strong and immediate, joining forces with Chinese and international scientific research institutions and enterprises to develop an arsenal of drug candidates to combat COVID-19, taking the initiative to shoulder the social responsibility of Chinese pharmaceutical companies by prioritizing and accelerating COVID-19 R&D. Among the many drug candidates is JS016 (etesevimab), China’s first neutralizing fully human monoclonal antibody against SARS-CoV-2 and the result of the combined efforts of Junshi Biosciences, the Institute of Microbiology of the Chinese Academy of Science and Lilly. JS016 administered with bamlanivimab has been granted Emergency Use Authorizations (EUA) in over 15 countries and regions worldwide. As of December 3 2021, over 700,000 patients have been treated with bamlanivimab or bamlanivimab and etesevimab, potentially preventing more than 35,000 hospitalizations and at least 14,000 deaths. Meanwhile, VV116, a new oral nucleoside analog anti-SARS-CoV-2 drug designed to hinder virus replication, has been approved for marketing in China and Uzbekistan. The JS016 and VV116 programs are a part of the company’s continuous innovation for disease control and prevention of the global pandemic.

Junshi Biosciences has more than 3,100 employees in the United States (San Francisco and Maryland) and China (Shanghai, Suzhou, Beijing, Guangzhou, etc). For more information, please visit: http://junshipharma.com.

Junshi Biosciences Contact Information
IR Team:
Junshi Biosciences
info@junshipharma.com
+ 86 021-6105 8800

PR Team:
Junshi Biosciences
Zhi Li
zhi_li@junshipharma.com
+ 86 021-6105 8800

Ryne Biotechnology Awarded $4 Million from the California Institute for Regenerative Medicine

Ryne Biotechnology Awarded $4 Million from the California Institute for Regenerative Medicine




Ryne Biotechnology Awarded $4 Million from the California Institute for Regenerative Medicine

SAN DIEGO–(BUSINESS WIRE)–Ryne Biotechnology, Inc. (Ryne Bio), a therapeutics company leveraging induced pluripotent stem cell (iPSC) technology to discover and develop a platform of off-the-shelf neuron replacement therapies for neurological disorders, today announced that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4 million Clinical Stage Research Program (CLIN1) grant. This funding will enable the company to advance its lead candidate RNDP-001, an iPSC-derived dopamine neuron progenitor for the treatment of both inherited and idiopathic forms of Parkinson’s disease, through submission of an Investigational New Drug (IND) application within the next 12 months.

RNDP-001 has completed preclinical efficacy and safety studies. This CIRM award will allow Ryne Bio to finalize its IND-package including the production of GMP-grade materials to enable the evaluation of RNDP-001 in Phase 1 clinical trials for both inherited and idiopathic forms of Parkinson’s disease.

“We appreciate CIRM’s partnership in our vision to reverse degenerative conditions of the brain by developing off-the-shelf cell replacement therapies,” said Nick Manusos, Chief Executive Officer of Ryne Bio. “A dramatic shift in the standard of care for patients with neurodegenerative disease is long overdue. We are thrilled to be developing groundbreaking therapies for patients in need of better treatment options.”

Beyond RNDP-001, Ryne Bio is developing a platform of drug candidates, including next-generation, gene-modified programs that have the potential to modify and reverse disease progression in Parkinson’s disease and other moderate to severe central nervous system disorders.

“The underlying cause of Parkinson’s disease is progressive degeneration of a patient’s dopamine neurons. Ryne Bio is able to directly replace dopamine neurons that have been lost by utilizing precision manufacturing techniques,” said Howard Federoff, M.D., Ph.D., Ryne Bio’s Chief Medical Officer, scientific co-founder and Principal Investigator on the CLIN1 award.

In addition to funding from CIRM, Ryne Bio was launched and seeded in 2022 by Saisei Ventures, an emerging venture capital firm focused on building revolutionary advanced medicine companies. “The potential of off-the-shelf cell replacement therapies is on the cusp of being realized for complex and intractable disease,” said Jonathan Yeh, Managing Partner of Saisei Ventures. “This funding decision from CIRM provides robust validation of the Ryne Bio approach, and supports the delivery of this best-in-class therapy to patients.”

About Ryne Bio

Ryne Bio is a biotechnology company pioneering next-generation approaches to cure neurological disorders. The company utilizes induced pluripotent stem cell (iPSC) technology, a Nobel-winning breakthrough that enables scientists to manufacture any human cell, in order to advance Ryne Bio’s off-the-shelf neuron replacement therapeutics. By focusing on an iPSC technology platform, and forging partnerships with global leaders in surgical delivery and clinical development, Ryne Bio is dedicated to advancing a best-in-class pipeline targeting neurological diseases. For additional information, visit ryne-bio.com.

About the California Institute for Regenerative Medicine (CIRM)

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information go to www.cirm.ca.gov.

About Saisei Ventures

Saisei Ventures is a leading venture capital firm dedicated to building next-generation companies in the healthcare sector. It aims to partner with passionate bio-entrepreneurs to develop and implement business strategies that will generate strong proof of concept, clinical validation, and market value. With operations in Japan and the United States, Saisei aims to enhance the value of its portfolio companies by leveraging its unique networks in and the institutional advantages of both countries. Saisei’s first fund, Saisei Bioventures L.P., is focused on building revolutionary cell and gene therapy and regenerative medicine companies from foundational technologies originating from Japan. For additional information, visit saiseiventures.com.

Contacts

Ryne Bio Investor Relations

ir@ryne-bio.com

Ryne Biotechnology Awarded $4 Million from the California Institute for Regenerative Medicine

U.S. FDA Approves Trodelvy® in Pre-treated HR+/HER2- Metastatic Breast Cancer

U.S. FDA Approves Trodelvy® in Pre-treated HR+/HER2- Metastatic Breast Cancer




U.S. FDA Approves Trodelvy® in Pre-treated HR+/HER2- Metastatic Breast Cancer

— First Trop-2 Directed ADC to Demonstrate Overall Survival Benefit in HR+/HER2- Metastatic Breast Cancer Patients who had Received Prior Endocrine-based Therapy and at Least Two Chemotherapies —

— Trodelvy has Now Improved Survival in both Pre-Treated HR+/HER2- Metastatic Breast Cancer and in Second-Line Metastatic Triple-Negative Breast Cancer

FOSTER CITY, Calif.–(BUSINESS WIRE)–Gilead Sciences, Inc. (Nasdaq: GILD) today announced the U.S. Food and Drug Administration (FDA) has approved Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with unresectable locally advanced or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting. The approval is based on statistically significant and clinically meaningful progression-free survival and overall survival data from the Phase 3 TROPiCS-02 study. Trodelvy is now also recommended as a Category 1, preferred treatment for metastatic HR+/HER2- breast cancer by the National Comprehensive Cancer Network® (NCCN®) as defined in the Clinical Practice Guidelines in Oncology (NCCN Guidelines®)i.


Despite decades of advances, people living with pre-treated HR+/HER2- metastatic breast cancer need new treatment options. Nearly all people with this type of breast cancer will eventually develop resistance to endocrine-based therapies and progress on available chemotherapies,” said Hope S. Rugo, MD, Professor of Medicine and Director, Breast Oncology and Clinical Trials Education at the UCSF Helen Diller Family Comprehensive Cancer Center, U.S. and principal investigator of the TROPiCS-02 study. “This approval is significant for the breast cancer community. We have had limited options to offer patients after endocrine-based therapy and chemotherapy, and to see a clinically meaningful survival benefit of more than three months with a quality of life benefit for these women is exceptional.”

In the TROPiCS-02 study, Trodelvy demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit of 3.2 months versus comparator single-agent chemotherapy (treatment of physician’s choice; TPC) (median OS: 14.4 months vs. 11.2 months; hazard ratio [HR]=0.79; 95% CI: 0.65-0.96; p=0.02). Trodelvy also demonstrated a 34% reduction in risk of disease progression or death (median PFS: 5.5 versus 4.0 months; HR: 0.66; 95% CI: 0.53-0.83; p=0.0003). Three times as many people treated with Trodelvy were progression free at one year versus those treated with chemotherapy (21% versus 7%). In a post-hoc analysis, data demonstrated Trodelvy’s efficacy across HER2-low and IHC0 status in pre-treated metastatic breast cancer patients in the TROPiCS-02 trial.

Trodelvy also significantly improved additional secondary endpoint measures, including objective response rate and time to deterioration (TTD) assessed by the Global Health Status/Quality of Life and Fatigue scale per EORTC-QLQ-C30. No statistically significant difference in TTD in Pain Scale was observed.

The FDA approval is an important step forward for both women and men living with metastatic breast cancer, especially for those individuals whose tumor is no longer responding to endocrine-based therapies and who are facing a poor prognosis,” said Laura Carfang, Executive Director, SurvivingBreastCancer.org. “We need to combat this terrible disease, and all options that potentially slow its progress and extend life for those living with metastatic breast cancer are welcomed.”

We are pleased that Trodelvy could now provide new hope for people living with pre-treated HR+/HER2- metastatic breast cancer, building on the transformative role that Trodelvy is already playing for people with metastatic triple-negative breast cancer,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. “We thank the physicians, patients and their families who put their trust in the TROPiCS-02 study and helped make this milestone possible.”

The safety profile for Trodelvy was consistent with prior studies, with no new safety signals identified in this patient population. In TROPiCS-02 the most frequent serious adverse reactions (>1%) were diarrhea (5%), febrile neutropenia (4%), neutropenia (3%), abdominal pain, colitis, neutropenic colitis, pneumonia, and vomiting (each 2%). The most common Grade 3-4 lab abnormalities (incidence ≥25%) in the TROPiCS-02 study were reduced neutrophils and leukocytes. No patients treated with Trodelvy experienced interstitial lung disease.

This review by the FDA was conducted under Project Orbis and granted Priority Review.

The European Medicines Agency has also validated a Type II Variation Marketing Authorization Application for Trodelvy in HR+/HER2- metastatic breast cancer.

Trodelvy has a Boxed Warning for severe or life-threatening neutropenia and severe diarrhea; please see below for additional Important Safety Information.

About HR+/HER2- Metastatic Breast Cancer

Hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) breast cancer is the most common type of breast cancer and accounts for approximately 70% of all new cases. Almost one in three cases of early-stage breast cancer eventually become metastatic, and among patients with HR+/HER2- metastatic disease, the five-year relative survival rate is 30%. As patients with HR+/HER2- metastatic breast cancer become resistant to endocrine-based therapy, their primary treatment option is limited to single-agent chemotherapy. In this setting, it is common to receive multiple lines of chemotherapy regimens over the course of treatment, and the prognosis remains poor.

About the TROPiCS-02 Study

The TROPiCS-02 study is a global, multicenter, open-label, Phase 3 study, randomized 1:1 to evaluate Trodelvy versus physicians’ choice of chemotherapy (eribulin, capecitabine, gemcitabine, or vinorelbine) in 543 patients with HR+/HER2- metastatic breast cancer who were previously treated with endocrine therapy, CDK4/6 inhibitor and two to four lines of chemotherapy for metastatic disease. The primary endpoint is progression-free survival per Response Evaluation Criteria in Solid Tumors (RECIST 1.1) as assessed by blinded independent central review (BICR) for participants treated with Trodelvy compared to those treated with chemotherapy. Secondary endpoints include overall survival, overall response rate, clinical benefit rate and duration of response, as well as assessment of safety and tolerability and quality of life measures. In the study, HER2 negativity was defined per American Society of Clinical Oncology (ASCO) and the College of American Pathologists (CAP) criteria as immunohistochemistry (IHC) score of 0, IHC 1+ or IHC 2+ with a negative in-situ hybridization (ISH) test. More information about TROPiCS-02 is available at https://clinicaltrials.gov/ct2/show/NCT03901339.

About Trodelvy

Trodelvy® (sacituzumab govitecan-hziy) is a first-in-class Trop-2 directed antibody-drug conjugate. Trop-2 is a cell surface antigen highly expressed in multiple tumor types, including in more than 90% of breast and bladder cancers. Trodelvy is intentionally designed with a proprietary hydrolyzable linker attached to SN-38, a topoisomerase I inhibitor payload. This unique combination delivers potent activity to both Trop-2 expressing cells and the microenvironment.

Trodelvy is approved in more than 40 countries, with multiple additional regulatory reviews underway worldwide, for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease.

Trodelvy is also approved in the U.S. to treat certain patients with pre-treated HR+/HER2- metastatic breast cancer and has an accelerated approval for treatment of certain patients with second-line metastatic urothelial cancer; see below for full indication statements.

Trodelvy is also being developed for potential investigational use in other TNBC, HR+/HER2- and metastatic UC populations, as well as a range of tumor types where Trop-2 is highly expressed, including metastatic non-small cell lung cancer (NSCLC), metastatic small cell lung cancer (SCLC), head and neck cancer, and endometrial cancer.

U.S. Indications for Trodelvy

In the United States, Trodelvy is indicated for the treatment of adult patients with:

  • Unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease.
  • Unresectable locally advanced or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting.
  • Locally advanced or metastatic urothelial cancer (mUC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

U.S. Important Safety Information for Trodelvy

BOXED WARNING: NEUTROPENIA AND DIARRHEA

  • Severe or life-threatening neutropenia may occur. Withhold Trodelvy for absolute neutrophil count below 1500/mm3 or neutropenic fever. Monitor blood cell counts periodically during treatment. Consider G-CSF for secondary prophylaxis. Initiate anti-infective treatment in patients with febrile neutropenia without delay.
  • Severe diarrhea may occur. Monitor patients with diarrhea and give fluid and electrolytes as needed. At the onset of diarrhea, evaluate for infectious causes and, if negative, promptly initiate loperamide. If severe diarrhea occurs, withhold Trodelvy until resolved to ≤Grade 1 and reduce subsequent doses.

CONTRAINDICATIONS

  • Severe hypersensitivity reaction to Trodelvy.

WARNINGS AND PRECAUTIONS

Neutropenia: Severe, life-threatening, or fatal neutropenia can occur and may require dose modification. Neutropenia occurred in 64% of patients treated with Trodelvy. Grade 3-4 neutropenia occurred in 49% of patients. Febrile neutropenia occurred in 6%. Neutropenic colitis occurred in 1.4%. Withhold Trodelvy for absolute neutrophil count below 1500/mm3 on Day 1 of any cycle or neutrophil count below 1000/mm3 on Day 8 of any cycle. Withhold Trodelvy for neutropenic fever. Administer G-CSF as clinically indicated or indicated in Table 1 of USPI.

Diarrhea: Diarrhea occurred in 64% of all patients treated with Trodelvy. Grade 3-4 diarrhea occurred in 11% of patients. One patient had intestinal perforation following diarrhea. Diarrhea that led to dehydration and subsequent acute kidney injury occurred in 0.7% of all patients. Withhold Trodelvy for Grade 3-4 diarrhea and resume when resolved to ≤Grade 1. At onset, evaluate for infectious causes and if negative, promptly initiate loperamide, 4 mg initially followed by 2 mg with every episode of diarrhea for a maximum of 16 mg daily. Discontinue loperamide 12 hours after diarrhea resolves. Additional supportive measures (e.g., fluid and electrolyte substitution) may also be employed as clinically indicated. Patients who exhibit an excessive cholinergic response to treatment can receive appropriate premedication (e.g., atropine) for subsequent treatments.

Hypersensitivity and Infusion-Related Reactions: Serious hypersensitivity reactions including life-threatening anaphylactic reactions have occurred with Trodelvy. Severe signs and symptoms included cardiac arrest, hypotension, wheezing, angioedema, swelling, pneumonitis, and skin reactions. Hypersensitivity reactions within 24 hours of dosing occurred in 35% of patients. Grade 3-4 hypersensitivity occurred in 2% of patients. The incidence of hypersensitivity reactions leading to permanent discontinuation of Trodelvy was 0.2%. The incidence of anaphylactic reactions was 0.2%. Pre-infusion medication is recommended. Have medications and emergency equipment to treat such reactions available for immediate use. Observe patients closely for hypersensitivity and infusion-related reactions during each infusion and for at least 30 minutes after completion of each infusion. Permanently discontinue Trodelvy for Grade 4 infusion-related reactions.

Nausea and Vomiting: Nausea occurred in 64% of all patients treated with Trodelvy and Grade 3-4 nausea occurred in 3% of these patients. Vomiting occurred in 35% of patients and Grade 3-4 vomiting occurred in 2% of these patients. Premedicate with a two or three drug combination regimen (e.g., dexamethasone with either a 5-HT3 receptor antagonist or an NK1 receptor antagonist as well as other drugs as indicated) for prevention of chemotherapy-induced nausea and vomiting (CINV). Withhold Trodelvy doses for Grade 3 nausea or Grade 3-4 vomiting and resume with additional supportive measures when resolved to Grade ≤1. Additional antiemetics and other supportive measures may also be employed as clinically indicated. All patients should be given take-home medications with clear instructions for prevention and treatment of nausea and vomiting.

Increased Risk of Adverse Reactions in Patients with Reduced UGT1A1 Activity: Patients homozygous for the uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1)*28 allele are at increased risk for neutropenia, febrile neutropenia, and anemia and may be at increased risk for other adverse reactions with Trodelvy. The incidence of Grade 3-4 neutropenia was 58% in patients homozygous for the UGT1A1*28, 49% in patients heterozygous for the UGT1A1*28 allele, and 43% in patients homozygous for the wild-type allele. The incidence of Grade 3-4 anemia was 21% in patients homozygous for the UGT1A1*28 allele, 10% in patients heterozygous for the UGT1A1*28 allele, and 9% in patients homozygous for the wild-type allele. Closely monitor patients with known reduced UGT1A1 activity for adverse reactions. Withhold or permanently discontinue Trodelvy based on clinical assessment of the onset, duration and severity of the observed adverse reactions in patients with evidence of acute early-onset or unusually severe adverse reactions, which may indicate reduced UGT1A1 function.

Embryo-Fetal Toxicity: Based on its mechanism of action, Trodelvy can cause teratogenicity and/or embryo-fetal lethality when administered to a pregnant woman. Trodelvy contains a genotoxic component, SN-38, and targets rapidly dividing cells. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with Trodelvy and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with Trodelvy and for 3 months after the last dose.

ADVERSE REACTIONS

In the pooled safety population, the most common (≥ 25%) adverse reactions including laboratory abnormalities were decreased leukocyte count (84%), decreased neutrophil count (75%), decreased hemoglobin (69%), diarrhea (64%), nausea (64%), decreased lymphocyte count (63%), fatigue (51%), alopecia (45%), constipation (37%), increased glucose (37%), decreased albumin (35%), vomiting (35%), decreased appetite (30%), decreased creatinine clearance (28%), increased alkaline phosphatase (28%), decreased magnesium (27%), decreased potassium (26%), and decreased sodium (26%).

In the ASCENT study (locally advanced or metastatic triple-negative breast cancer), the most common adverse reactions (incidence ≥25%) were fatigue, diarrhea, nausea, alopecia, constipation, vomiting, abdominal pain, and decreased appetite. The most frequent serious adverse reactions (SAR) (>1%) were neutropenia (7%), diarrhea (4%), and pneumonia (3%). SAR were reported in 27% of patients, and 5% discontinued therapy due to adverse reactions. The most common Grade 3-4 lab abnormalities (incidence ≥25%) in the ASCENT study were reduced neutrophils, leukocytes, and lymphocytes.

In the TROPiCS-02 study (locally advanced or metastatic HR-positive, HER2-negative breast cancer), the most common adverse reactions (incidence ≥25%) were diarrhea, fatigue, nausea, alopecia, and constipation. The most frequent serious adverse reactions (SAR) (>1%) were diarrhea (5%), febrile neutropenia (4%), neutropenia (3%), abdominal pain, colitis, neutropenic colitis, pneumonia, and vomiting (each 2%). SAR were reported in 28% of patients, and 6% discontinued therapy due to adverse reactions. The most common Grade 3-4 lab abnormalities (incidence ≥25%) in the TROPiCS-02 study were reduced neutrophils and leukocytes.

In the TROPHY study (locally advanced or metastatic urothelial cancer), the most common adverse reactions (incidence ≥25%) were diarrhea, fatigue, nausea, any infection, alopecia, decreased appetite, constipation, vomiting, rash, and abdominal pain. The most frequent serious adverse reactions (SAR) (≥5%) were infection (18%), neutropenia (12%, including febrile neutropenia in 10%), acute kidney injury (6%), urinary tract infection (6%), and sepsis or bacteremia (5%). SAR were reported in 44% of patients, and 10% discontinued due to adverse reactions. The most common Grade 3-4 lab abnormalities (incidence ≥25%) in the TROPHY study were reduced neutrophils, leukocytes, and lymphocytes.

DRUG INTERACTIONS

UGT1A1 Inhibitors: Concomitant administration of Trodelvy with inhibitors of UGT1A1 may increase the incidence of adverse reactions due to potential increase in systemic exposure to SN-38. Avoid administering UGT1A1 inhibitors with Trodelvy.

UGT1A1 Inducers: Exposure to SN-38 may be reduced in patients concomitantly receiving UGT1A1 enzyme inducers. Avoid administering UGT1A1 inducers with Trodelvy.

Please see full Prescribing Information, including BOXED WARNING.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including Gilead’s ability to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing or additional clinical trials, including those involving Trodelvy; uncertainties relating to regulatory applications for Trodelvy and related filing and approval timelines, including the risk that the European Commission may not approve the pending marketing authorization application for Trodelvy in HR+/HER2- metastatic breast cancer, and pending or potential applications for the treatment of metastatic TNBC, mUC, HR+/HER2- breast cancer, NSCLC, SCLC, head and neck cancer, endometrial cancer and other cancers, in the currently anticipated timelines or at all; Gilead’s ability to receive regulatory approvals for such indications in a timely manner or at all, and the risk that any such approvals may be subject to significant limitations on use; the possibility that Gilead may make a strategic decision to discontinue development of Trodelvy for such indications and as a result, Trodelvy may never be commercialized for these indications; the risk that physicians may not see the benefits of prescribing Trodelvy for treatment of HR+/HER2- metastatic breast cancer; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gilead’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead, and Gilead assumes no obligation and disclaims any intent to update any such forward-looking statements.

U.S. Prescribing Information for Trodelvy including BOXED WARNING, is available at www.gilead.com.

Trodelvy, Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies.

For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@GileadSciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.

UC Disclaimer

The information stated above was prepared by Gilead Sciences, Inc., and reflects solely the opinion of the corporation. Nothing in this statement shall be construed to imply any support or endorsement of Gilead, or any of its products, by The Regents of the University of California, its officers, agents and employees.

i Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Breast Cancer Version 1.2023. © National Comprehensive Cancer Network, Inc. 2023. All rights reserved. Accessed January 2023. To view the most recent and complete version of the guideline, go online to NCCN.org. 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.

Contacts

Jacquie Ross, Investors

investor_relations@gilead.com

Meaghan Smith, Media

public_affairs@gilead.com

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

Autophagy is a catabolic process which degrades a cellular own component through the lysosomal machinery. The lysosome, at the heart of the autophagy sytem is important in various processes (cancer, infection…).

Genoscience Pharma - new approaches disrupting cancer cell lysosomal functions

In Cancer, they are required in tumor cells for cellular adhesion, motility and signaling, exocytosis, angiogenesis and overall survival, growth, aggressiveness, metastatic potential and drug resistance. Because of their high metabolic rates, rapidly dividing and invasive cancer cells require increased new biomass production to survive. The lysosome is also important for adaptation to nutrient stress as it contains hydrolytic enzymes that play a major role in the degradation of intracellular macromolecules and catabolic (such as autophagy) and anabolic growth. This busy lysosomal behavior leads to alterations in lysosomal structure and function, which, paradoxically, renders cancer cells more sensitive to lysosomal destabilization. In addition, lysosomal enzyme activity is elevated in many tumors compared to adjacent normal tissue, and several reports suggest that lysosomes in tumor cells are more fragile than normal lysosomes. Therefore, lysosome seems to be a target of interest in the fight against cancers. Targeting lysosomes triggers apoptotic and lysosomal cell death pathways.

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

In virology, It has been shown that autophagy is activated during virus and bacterial infection and that some viruses can use the autophagy system to facilitate their own replication . Some viruses, such as Coronaviruses are single stranded, positive sense RNA viruses, which induce the rearrangement of cellular membranes upon infection of a host cell. This provides the virus with a platform for the assembly of viral replication complexes, improving efficiency of RNA synthesis. Genoscience Pharma focuses on new approaches disrupting cancer cell lysosomal functions.

Genoscience Pharma was founded in 2001 by Pr Philippe Halfon, a world renowned medical expert on viral diseases, especially on Human Immunodeficient Virus (HIV) and Hepatitis C Virus (HCV). The company was initially focused on the development of anti-HCV agents. Two protease inhibitors were thus developed and out-licensed to BioLineRX. A new scientific direction was taken in 2012 after the discovery of a new chemical family: autophagy inhibitors. Following promising preliminary in vitro and in vivo results from these small molecules, including against cancer stem cells, Genoscience Pharma has decided to take the opportunity to make the difference in Oncology, especially in cancers where medical needs are still unmet. Now, Genoscience Pharma is a clinical stage biopharmaceutical company focused on translating novel scientific insights into medicines for patients with cancer.

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

More info: https://www.genosciencepharma.com/

Keywords : lysosomal functions , cancer, oncology , Genoscience Pharma , lysosomes , infectious diseases  , virus , Covid19, leukemia , pancreatic cancer , hepatocarcinoma , small molecules

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARMGO Pharma is a privately held biopharmaceutical company dedicated to applying original, targeted science to the discovery and development of novel small-molecule therapeutics to treat debilitating cardiac, musculoskeletal, and neurological disorders. The company’s proprietary drugs, known as Rycals®, are a new class of oral agents that repair calcium leaks through the Ryanodine Receptor (RyR).

ARMGO Pharma - developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARM210 is a potential disease modifying therapy for genetic diseases, which targets the Ryanodine Receptor calcium channel (RyR), an intracellular calcium-release channel that becomes leaky in these and other diseases. Intracellular calcium leaks via mutant RyR1 channels impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle, causing the symptoms in RYR1-RM.

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential disease modifying therapy for RYR1-RM. Muscle biopsies of the patients in this trial have been previously shown to respond biochemically to ARM210 in vitro. This trial will evaluate the safety and explore the biochemical effect of oral administration of ARM210 in these same patients.

ARMGO Pharma has been awarded an exclusive, worldwide license from Columbia University for its RyR technology based on the research of founding scientist Andrew R. Marks, M.D. In December 2021, ARMGO Pharma raises a $35 million Series B  funding round to progress clinical studies of lead molecule ARM210 in cardiac and skeletal muscle diseases.

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

More info : www.armgo.com

Keywords : ARMGO Pharma , small molecule , therapeutics , RyR technology , ARM210 , cardiovascular , musculoskeletal disorders , neurological disorders , CNS , neuroscience , Rycals , Ryanodine Receptor , RyR , Ryanodine Receptor modulators

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

Nanostics is focused on development and commercialization of novel, non-invasive diagnostic tests for cancer. Its core technology is an advanced liquid biopsy platform that can accurately diagnose cancer from a single drop of blood. Its lead product, ClarityDx Prostate, is the most accurate diagnostic test to diagnose aggressive prostate cancer, and is positioned to emerge as the world’s leading diagnostic tool for prostate cancer.

Nanostics - micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

EVs carrying disease-specific biomarkers like nucleic acid and proteins are continuously released from cells and can be found in biological fluids including blood, urine, semen, and cerebrospinal fluid.  The levels of disease-specific EVs are closely related to disease progression making EVs promising targets for minimally invasive diagnostic assays.

Technology designed to accurately detect and measure EVs has the potential to greatly improve liquid biopsy diagnostics. At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

The ability to detect and measure EVs is transforming the diagnostic landscape in immunology, neurology, cardiology, and oncology. The ClarityDX® platform technology is well-positioned for future pipeline products including tests for screening, early diagnosis, complementary diagnosis, monitoring, and management for many diseases and medically-related events. Nanostics’ R&D team continues to expand the product pipeline through in-house and partnered projects to include additional liquid biopsy diagnostic tests to improve patient care.

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

More info : http://www.nanosticsdx.com

Keywords : machine learning, exosomes, extracellular vesicles, diagnostics, oncology, cancer, prostate cancer, cardiovascular, neuroscience, biofluids , liquid biopsy , micro-flow cytometry

Alpha Cognition – developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis (ALS).

Alpha Cognition - developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

ALPHA-1062, a patented new chemical entity that has demonstrated safety and improved ALPHA-1062, is a patented new chemical entity being developed as a new generation acetylcholinesterase inhibitor for the treatment of Alzheimer’s disease, with expected minimal gastrointestinal side effects. ALPHA-1062’s active metabolite is differentiated from donepezil and rivastigmine in that it binds neuronal nicotinic receptors, most notably the alpha-7 subtype, which is known to have a positive effect on cognition. ALPHA-1062 is also being developed in combination with memantine to treat moderate to severe Alzheimer’s dementia and as an intranasal formulation for traumatic brain injury.

ALPHA-0602 (Progranulin) is expressed in several cell types in the central nervous system and in peripheral tissues, promotes cell survival, regulates certain inflammatory processes, and plays a significant role in regulating lysosomal function and microglial responses to disease. Its intended use for the treatment of neurodegenerative diseases has been patented by the Company and Alpha-0602 has been granted an Orphan Drug Designation for the treatment of Amyotrophic Lateral Sclerosis by the FDA.

ALPHA-0702 and ALPHA-0802 are Granulin Epithelin Motifs, or GEMs, derived from full length progranulin which have therapeutic potential across multiple neurodegenerative diseases. Progranulin is comprised of 7½ Granulin Epithelin Motifs (GEMs). GEMs are important in regulating cell growth, survival, repair, and inflammation. Individual granulin domain peptides are approximately 60 amino acids in length (~7kDa). GEMs are metabolically stable and resistant to proteolysis (long half-life). Evidence suggests that not all GEMs are equally potent at promoting cell survival signaling or reducing proteinopathy, and may compete (e.g., binding to effectors) with active GEMs and effectively decrease their potency. Specific GEM combinations have been shown to be more effective than full length progranulin in promoting Cathepsin D maturation. GEMs have been shown to be important in regulating cell growth, survival, repair, and inflammation. Alpha-0702 and ALPHA-0802 are designed to deliver this with potentially lower toxicity, and greater therapeutic effect.

More info : http://www.alphacognition.com/

Keywords : Alpha Cognition , neurodegenerative diseases , dementia , Alzheimer’s Dementia , Alzheimer Disease , Amyotrophic Lateral Sclerosis , acetylcholinesterase inhibitor , orphan drug , CNS , neuroscience , traumatic brain injury , Granulin Epithelin Motifs , Gene Therapy