CELL AND GENE THERAPY
Imara | November 22, 2021
Imara Inc. a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin and other serious diseases, today announced a change to the primary endpoint for the Ardent clinical trial, a Phase 2b study of tovinontrine (IMR-687) in patients with sickle cell disease (SCD), based on the recommendation of the U.S. Food and Drug Administration (FDA).
Imara requested feedback from the FDA on the draft statistical analysis plan (SAP) for the Ardent trial in which fetal hemoglobin (HbF) response was the primary endpoint and annualized rate of vaso-occlusive crises (VOCs) was the key secondary endpoint. In reviewing the Ardent draft SAP and prior to any database lock for analysis, the FDA recommended that Imara change the primary endpoint to be annualized rate of VOCs. HbF response will continue to be evaluated as a key secondary endpoint. The endpoint revisions do not affect the conduct of the trial or operational aspects of the study. As part of its recommendation, the FDA suggested further interactions regarding the revised SAP and engagement on the potential of the current program for regulatory decision-making.
“We welcome the FDA’s recommendations and are in the process of changing the primary endpoint of the Ardent trial to be annualized rate of VOCs and moving HbF response to be a key secondary endpoint. A reduction in VOC rate is an established approval endpoint, and we are engaging the FDA further on this and related topics, including possible streamlined paths to registration.”
Rahul Ballal, Ph.D., President and Chief Executive Officer of Imara
Dr. Ballal continued, “In light of this endpoint revision, the previously planned fourth quarter interim analysis will no longer occur. That interim analysis had been designed to have a focus on safety and pharmacodynamic biomarkers, including HbF, but did not include a review of VOCs. The first review of data from the Ardent trial, including annualized VOC rate, will be conducted when all subjects have completed assessment at Week 24 or terminated early, and is planned for the first quarter of 2022, subject to our upcoming discussions with the FDA. Final data analysis from the Ardent trial remains on track for the second half of 2022. In June 2021, we reported promising data from our Phase 2a and open label extension clinical trials in SCD that demonstrated reduced annualized rates of VOCs in patients treated with tovinontrine versus placebo. We expect to present updated 12-month VOC data from our ongoing Phase 2a open label extension clinical trial at the American Society of Hematology Annual Meeting in December 2021.”
About the Ardent Phase 2b Clinical Trial
The Ardent Phase 2b clinical trial is a fully-enrolled, global, randomized, double-blind, placebo-controlled, multicenter study with approximately 115 adult patients with sickle cell disease (SCD) enrolled. The planned primary efficacy objective will be to evaluate the annualized rate of vaso-occlusive crises (VOCs) in patients dosed with tovinontrine (IMR-687) as compared to placebo. A key secondary endpoint will be to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an absolute increase from baseline of at least 3% in HbF, as compared to placebo. Additional endpoints include the evaluation of the effect of tovinontrine versus placebo on other VOC-related outcome measures, HbF-associated biomarkers, markers of red blood cell hemolysis, white blood cell adhesion markers and quality of life measures over the course of a one-year treatment period.
The FDA has granted Orphan Drug, Fast Track and Rare Pediatric Disease designations and the European Commission has granted Orphan Drug designation for tovinontrine for the treatment of SCD.
About Tovinontrine (IMR-687)
Tovinontrine is a highly selective and potent small molecule inhibitor of phosphodiesterase-9 (PDE9). PDE9 selectively degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology and hemoglobin production in red blood cells. Lower levels of cGMP are found in people with sickle cell disease (SCD) and beta-thalassemia and are associated with reduced blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide mediated vasodilation. Blocking PDE9 acts to increase cGMP levels, which is associated with several benefits including the potential reactivation of fetal hemoglobin (HbF), a natural hemoglobin produced during fetal development. Increased levels of HbF in RBCs have been demonstrated to improve symptomology and substantially lower disease burden in both patients with SCD and patients with beta-thalassemia.
About Sickle Cell Disease
Sickle cell disease (SCD), a hemoglobinopathy, is a rare inherited red blood cell disorder. The disease causes structural abnormalities in hemoglobin that cause red blood cells to become inflexible and elongated, ultimately blocking blood flow to organs, which can lead to vaso-occlusive crises (VOCs). SCD is characterized by debilitating pain, progressive multi-organ damage and early death. The global prevalence of SCD is estimated to be approximately 4.4 million patients, including an estimated 100,000 patients in the United States and 134,000 patients in the European Union.
Imara Inc. is a clinical-stage biotechnology company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin and other serious diseases. Imara is advancing tovinontrine (IMR-687), a highly selective, potent small molecule inhibitor of PDE9 that is an oral, potentially disease-modifying treatment currently in clinical development for sickle cell disease and beta-thalassemia and preclinical development for heart failure with preserved ejection fraction, or HFpEF. Imara is also advancing IMR-261, an oral activator of nuclear factor erythroid 2–related factor 2, or Nrf2.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to (i) the Company’s plans to change the primary and secondary endpoints for the Ardent Phase 2b clinical trial of tovinontrine (IMR-687), (ii) the timing for reporting of additional data from the Ardent Phase 2b and open label extension clinical trials of tovinontrine in patients with sickle cell disease and (iii) the Company’s planned discussions with the FDA regarding the regulatory pathway for tovinontrine. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the impact of extraordinary external events, such as the risks and uncertainties resulting from the impact of the COVID-19 pandemic on the Company’s business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities and ability to readout data from the Ardent Phase 2b and open label extension clinical trials of tovinontrine in sickle cell disease; the Company’s ability to advance the development of tovinontrine under the timelines it projects in current and future clinical trials, demonstrate in any current and future clinical trials the requisite safety and efficacy of tovinontrine; and other factors discussed in the “Risk Factors” section of the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
PharmAbcine | December 08, 2020
PharmAbcine Inc., a clinical-stage biotech organization focusing in on the improvement of completely human antibody therapeutics, declared today that the organization will introduce the refreshed break information from the continuous stage Ib trial of olinvacimab and pembrolizumab combination therapy in metastatic triple-negative breast cancer (mTNBC) patients at the 2020 San Antonio Breast Cancer Symposium (SABCS 2020) occurring for all intents and purposes over December 8-11, 2020.
Positive data from the progressing security and tolerability study will feature wellbeing and certain viability information including ORR (Overall Response Rate) and DCR (Disease Control Rate) from 11 patients diagnosed with mTNBC.
"We are excited to present highly encouraging data from our ongoing trial in the biggest breast cancer symposium. These data offer important insights into future development of the combo therapy," said Dr. Jin-San Yoo, CEO of PharmAbcine.
TurtleTree | October 04, 2021
TurtleTree has officially opened its new R&D facility in California's state capital. Located in West Sacramento, this 24,000-square-foot building will house a world-class research hub showcasing the company's extensive array of innovations and cell-based technologies.
Representing a milestone in TurtleTree's US-based expansion plans, the research facility will spur the development of precision fermentation technology currently used to produce valuable milk ingredients like lactoferrin. This marks a significant stride towards the production of consumer-ready milk and dairy, reaffirming TurtleTree's status as a leading player in the field of sustainable nutrition.
In addition to improving these capabilities, the establishment of this groundbreaking facility will also further TurtleTree's mission of uplifting the world beyond the creation of sustainable food. To adequately staff the facility, the company will create approximately 40 new tech-related jobs in and around Greater Sacramento. These will span diverse fields from food science to engineering, yielding fresh opportunities for seasoned professionals and fresh graduates alike.
Referencing this positive impact, TurtleTree's CEO and co-founder, Fengru Lin, stated: "Establishing this R&D facility in Greater Sacramento marks the first step towards bringing our unique products from development to market—one that we're very glad will help unlock new opportunities within the local community. We're eagerly looking forward to working with the brilliant talent in the area and building a future generation of nutrition that we can all enjoy for decades to come."
Echoing similar sentiments, various West Sacramento representatives have also offered their support for the new facility. President and CEO of the Greater Sacramento Economic Council, Barry Broome, stated: "We want to congratulate TurtleTree for locating their R&D and manufacturing facility in West Sacramento. This move signals market validation of why Greater Sacramento is world-class in biotech, specifically alternative proteins research."
West Sacramento is honored to have been chosen by TurtleTree as the location for their much-anticipated research facility. The city looks forward to assisting the TurtleTree leadership to achieve their goals, with respect to the facility, through an accelerated occupancy permit process, TurtleTree is making significant contributions to a sustainable, global food supply and is also creating opportunities to prepare future generations to continue this important scientific work through our student internship program, which will be of great benefit to the city's youth.
-West Sacramento mayor, Martha Guerrero
Moving forward, TurtleTree remains committed to its role in developing the food and agriculture ecosystem in Northern California. Aside from functioning as a research hub, the R&D facility will also serve as an integral touchpoint for TurtleTree to connect and collaborate with educational institutions, research-focused partners, strategic partners, future customers, and investors.
TurtleTree is a biotech company that's using cell-based technologies to create sustainable food and dairy. In spite of this, we earnestly believe that what we do extends beyond the dining table and into the heart of humanity. At our core, we seek to uplift the world by providing a new generation of nutrition that's better for the planet, better for the animals, and better for people everywhere.
About the Greater Sacramento Economic Council (GSEC)
The Greater Sacramento Economic Council is the catalyst for innovative growth strategies in the Capital Region of California. The organization spearheads community-led direction to retain, attract, grow, and scale tradable sectors, develop advanced industries, and create jobs and investment throughout a six-county region. Greater Sacramento represents a collaboration between local and state governments, market leaders, influencers, and stakeholders, with the sole mission of driving inclusive economic growth. The Greater Sacramento region was founded on discovery, built on leadership, and fueled by innovation.
About the City of West Sacramento
West Sacramento is located minutes from the state capital, midway between San Francisco and Lake Tahoe, in the heart of California's Great Central Valley. West Sacramento has abundant land, high-quality water, and nearby agricultural production combined with market access and proximity to the world-renowned food and agricultural research of U.C. Davis. Over 60 companies in food and ag-tech research, production, and distribution have established West Sacramento's Global Food Industry Hub, including Origin Materials, TOMRA Sorting Solutions, NuSeed Americas, Nor-Cal Beverage, Bayer Crop Science, and Nippon Shokken U.S.A.