On June 2, 2022 Pacira BioSciences, Inc. (NASDAQ: PCRX) reported that it will participate in an analyst-led fireside chat at the Jefferies Healthcare Conference at 9:00 AM ET on Thursday, June 9, 2022 in New York (Press release, Pacira Pharmaceuticals, JUN 2, 2022, View Source [SID1234615449]). Live audio of the event can be accessed by visiting the "Events" page of the company’s website at investor.pacira.com. A replay of the webcast will also be available for two weeks following the event.

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On June 2, 2022 Sarah Cannon Research Institute reported that it will highlight its latest cancer research insights through more than 140 abstracts and presentations at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place in Chicago and online from June 3-7, 2022 (Press release, Sarah Cannon Research Institute, JUN 2, 2022, View Source [SID1234615465]). Experts from across Sarah Cannon Research Institute’s network will join oncology leaders from around the globe to discuss the latest research findings that are accelerating progress in the fight against cancer. As a leader in drug development, Sarah Cannon Research Institute is presenting data from 75 early-phase studies.

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"Collaboration is essential to how we accelerate progress in the fight against cancer, and we look forward to coming together with our colleagues in Chicago to discuss the latest advances in oncology research," said Howard A. "Skip" Burris III, MD, FACP, FACSO, President, Clinical Operations & Chief Medical Officer, Sarah Cannon Research Institute. "With the variety of novel molecularly-targeted agents, immunotherapies, and cellular therapies in development, we are seeing a remarkable number of possibilities to truly transform care and personalize treatments for patients."

Presentations will cover a wide range of topics including how targeted agents and immunotherapies are performing in an array of disease settings, how molecular profiling is expanding access to clinical trials, and how researchers are advancing clinical research despite pressures in a post-pandemic environment.

Below are several highlighted presentations that Sarah Cannon principal investigators will lead during the Annual Meeting:

Gerald Falchook, MD, MS, Director, Drug Development, Sarah Cannon Research Institute at HealthONE, will deliver "Phase 1 Trial of TIM-3 Inhibitor Cobolimab Monotherapy and in Combination with PD-1 Inhibitors Nivolumab or Dostarlimab (AMBER)" in an oral presentation on June 4 from 1:15-4:15 p.m. CDT (2:27 p.m. CDT) in Hall B1.
Erika Hamilton, MD, Director, Breast Cancer and Gynecologic Cancer Research, Sarah Cannon Research Institute at Tennessee Oncology, will give two oral presentations – "Trastuzumab Deruxtecan (T-DXd) vs Trastuzumab Emtansine (T-DM1) in Patients (pts) with HER2-Positive (HER2+) Unresectable and/or Metastatic Breast Cancer (mBC): Safety Follow-Up of the Randomized, Phase 3 Study DESTINY-Breast03" on June 4 from 1:15-4:15 p.m. CDT (1:15 p.m. CDT) in Hall D1 and "Phase I, Two-Part, Multicenter, First-In-Human (FIH) Study of DS-6000a in Subjects with Advanced Renal Cell Carcinoma (RCC) and Ovarian Tumors (OVC)" on June 7 from 9:45 a.m. – 12:45 p.m. CDT (10:09 a.m. CDT) in S406.
Melissa Johnson, MD, Director, Lung Cancer Research, Sarah Cannon Research Institute at Tennessee Oncology, will present "Resistance to KRAS Inhibition and Future Combinations" in the Education Session, Development of Novel Inhibitors of the RAS Pathway, on June 7 from 8-9:15 a.m. CDT in S406. Dr. Johnson will also present "Dose Escalation of a Phase 1b/2 Study of Modakafusp Alfa, an Immune-Targeting Attenuated Cytokine, in Patients (pts) with Metastatic Solid Tumors" in an oral presentation on June 4 from 1:15-4:15 p.m. CDT (1:51 p.m. CDT) in Hall B1.
Andrew McKenzie, PhD, Vice President, Personalized Medicine, Sarah Cannon Research Institute; Scientific Director, Genospace, will present "Expanding Access to Clinical Trials Through Molecular Profiling Review and Telehealth Consultations" during the Education Session, Rethinking Cancer Clinical Trial Conduct With Telemedicine, on June 6 from 4:45-6 p.m. CDT in S504.
Jeremy Pantin, MD, Senior Investigator, Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial, will chair the Clinical Science Symposium, Beating Bad Blood: The Power of Immunotherapy in Hematologic Malignancies, as well as discuss "Refining the CAR-T’s Engine: How to Improve CD19-Directed CAR-T Performance" on June 6 from 8-9:30 a.m. CDT in S100bc.
David Spigel, MD, Chief Scientific Officer, Sarah Cannon Research Institute, is a speaker and panelist on the Education Session, The Business of Clinical Trials: The Impact of Funds Flow and the Shrinking Workforce on Trial Development, on June 5 from 8-9:15 a.m. CDT in S102.
Abstracts and presentations with Sarah Cannon experts as first authors will be presented by:

Dr. Howard Burris
Dr. Gerald Falchook
Lucio Gordan, MD, Chief Medical Officer, Sarah Cannon Research Institute at Florida Cancer Specialists
Dr. Erika Hamilton
Marilyn (Holt) Hammer, PhD, Program Specialist, Personalized Medicine, Sarah Cannon Research Institute
Lowell Hart, MD, FACP, Scientific Director, Clinical Research, Sarah Cannon Research Institute at Florida Cancer Specialists
Shekeab Jauhari, MD, Associate Director, Drug Development, Sarah Cannon Research Institute at Florida Cancer Specialists – Lake Mary
Dr. Melissa Johnson
William Kevin Kelly, DO, Senior Investigator, Drug Development, Sarah Cannon Research Institute at Sidney Kimmel Cancer Center
Meredith McKean, MD, MPH, Director, Melanoma & Skin Cancer Research, Sarah Cannon Research Institute at Tennessee Oncology
Dr. Andrew McKenzie (Chair)
Kathleen Moore, MD, Director, Drug Development, Sarah Cannon Research Institute at the Stephenson Cancer Center
Manish Patel, MD, Director, Drug Development, Sarah Cannon Research Institute at Florida Cancer Specialists – Sarasota
Meredith Pelster, MD, MSCI, Assistant Director, Gastrointestinal Research, Sarah Cannon Research Institute at Tennessee Oncology
Cesar Augusto Perez, MD, Director, Drug Development, Sarah Cannon Research Institute at Florida Cancer Specialists – Lake Nona
Russell Schilder, MD, Director, Drug Development, Sarah Cannon Research Institute at Sidney Kimmel Cancer Center
Emma Sturgill, PhD, Senior Program Specialist, Personalized Medicine, Sarah Cannon Research Institute
Susanna Ulahannan, MD, MMed, Associate Director, Phase 1 Program, Sarah Cannon Research Institute at The Stephenson Cancer Center (Co-Chair)

On June 2, 2022 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) reported new late-breaking clinical data accepted for oral presentation at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress (Press release, CRISPR Therapeutics, JUN 2, 2022, View Source [SID1234615416]). Vertex also announced three abstracts accepted for poster presentation at EHA (Free EHA Whitepaper).

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Late-breaking abstract #LB2367 entitled "Efficacy and Safety of A Single Dose of CTX001 For Transfusion-Dependent Βeta-Thalassemia and Severe Sickle Cell Disease," will be an oral presentation on Sunday, June 12 at 09:45‑11:15 CEST. The abstract from Vertex and CRISPR Therapeutics includes data on patients treated in CLIMB‑111 and CLIMB‑121 and followed in CLIMB‑131 with CTX001, now known as exagamglogene autotemcel (exa-cel). This abstract has been selected for the media briefing program and is therefore embargoed until Saturday, June 11 at 09:00 am CEST.

In addition, three real-world evidence and health economics abstracts from Vertex have been accepted for poster presentation.

Abstract #P1704 entitled "Projected Lifetime Economic Burden of Severe Sickle Cell Disease in the United States," will be a poster presentation on Friday, June 10 at 16:30‑17:45 CEST. The abstract posted online projects the per-patient lifetime direct health care cost of severe sickle cell disease (SCD) from a U.S. health care payer perspective using an economic model developed based on published model frameworks.

Abstract #P1703 entitled "Economic Burden of Transfusion‑Dependent Beta‑Thalassemia in the United States," will be a poster presentation on Friday, June 10 at 16:30‑17:45 CEST. The abstract posted online estimates the economic burden of transfusion-dependent beta thalassemia (TDT) using administrative claims data to estimate the costs and health care utilization associated with disease management in the U.S.

Abstract #P1482 entitled "Patients With Severe Sickle Cell Disease on Standard-of-Care Treatment Are Very Unlikely to Become VOC‑Free for One Year: A Cohort Study of Medicaid Enrollees," will be a poster presentation on Friday, June 10 at 16:30‑17:45 CEST. The abstract posted online contextualizes the efficacy of exa‑cel in eliminating vaso‑occlusive crises (VOCs) in patients with SCD using nationwide U.S. Medicaid claims data from 2000 to 2014 to assess the proportion of patients with recurrent VOCs who became VOC‑free during a 1‑year follow up on standard of care.
The accepted abstracts are now available online on the EHA (Free EHA Whitepaper) website.

Exa‑cel is being investigated in multiple ongoing clinical trials as a potential one-time therapy for patients with either TDT or SCD.

About exagamglogene autotemcel (exa-cel)

Exa‑cel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 gene‑edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent VOCs, in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen‑carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exa‑cel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, exa‑cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene‑editing approaches being evaluated for TDT and SCD, exa‑cel is the furthest advanced in clinical development.

About CLIMB‑111 and CLIMB‑121

The ongoing Phase 1/2/3 open‑label trials, CLIMB‑111 and CLIMB‑121, are designed to assess the safety and efficacy of a single dose of exa‑cel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exa‑cel infusion. Each patient will be asked to participate in CLIMB‑131, a long‑term follow‑up trial.

About CLIMB-131

This is a long‑term, open‑label trial to evaluate the safety and efficacy of exa‑cel in patients who received exa‑cel in CLIMB‑111, CLIMB‑121, CLIMB‑141 or CLIMB‑151. The trial is designed to follow participants for up to 15 years after exa‑cel infusion.

About CLIMB‑141 and CLIMB‑151

The ongoing Phase 3 open-label trials, CLIMB‑141 and CLIMB‑151, are designed to assess the safety and efficacy of a single dose of exa‑cel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to ages 2 to less than 5 years of age at a later date. Each trial will enroll up to 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a long‑term follow‑up trial.

About the Gene‑Editing Process in These Trials

Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patient’s cells will be edited using the CRISPR/Cas9 technology. The edited cells, exa‑cel, will then be infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT), a process which involves a patient being treated with myeloablative busulfan conditioning. Patients undergoing HSCT may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of exa‑cel. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of exa‑cel on multiple measures of disease and for safety.

About the Vertex‑CRISPR Collaboration

Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exa‑cel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exa‑cel and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics.

On June 2, 2022 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, reported that senior management will participate in a fireside chat at the Goldman Sachs 43rd Annual Global Healthcare Conference on Tuesday June 14, 2022 at 10:00 a.m. P.T./ 1:00 p.m. E.T (Press release, Sarepta Therapeutics, JUN 2, 2022, View Source [SID1234615450]). The fireside chat will be held at the Terranea Resort, Rancho Palos Verdes, CA.

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The presentation will be webcast live under the Events & Presentations section of the investor relations section of Sarepta’s website at View Source and will be archived there following the presentation for 90 days. Please connect to Sarepta’s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

On June 2, 2022 Massive Bio, Inc., a leader in precision medicine and artificial intelligence (AI) enabled patient centric clinical trial enrollment, reported that it raised $9 million co-led by Revo Capital and Kenan Turnacioğlu, Chair of Board of Directors PaigeAI, with additional participation from DEG – Deutsche Investitions- und Entwicklungsgesellschaft mbH, the German Development Finance Institution (Press release, Massive Bio, JUN 2, 2022, View Source [SID1234615466]).

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This investment will enable the company to further scale its operations globally, invest in marketing, and launch additional non-status quo products in oncology clinical trial enrollment with its data-rich platform. The company’s clinical research business launched in late 2019, and the financing round builds on another exponential growth year for Massive Bio, with 60,000 cancer patients and 26 enterprise customers. 88% of those customers are large pharmaceutical companies and CROs. Massive Bio has expanded to 12 countries with a global workforce of 70 employees in the past 12 months.

Massive Bio Co-founder and CEO Selin Kurnaz stated, "We are building a category-defining company in the oncology ecosystem by engineering patient enrollment value chain, and we are excited by the unwavering support from our new and existing investors, who have seen our exponential organic growth, strong ROI to our clients and unique value proposition to patients and oncology eco-system." Dr. Kurnaz added, "We are significantly different than the companies at our stage, cost-efficient and generating consistent revenue and client growth despite the economic downturn, relying mostly on customer demand to fuel our growth. Our passion, dedication, perseverance, and smart work has led to the company’s success and expansion in 12 markets without a sales force or marketing. Having the financial support, healthcare expertise and global coverage of investors such as DEG and Kenan, along with heavy technology expertise of our existing investors like Revo Capital will help us execute our goals globally and at accelerated scale."

With tens of thousands active cancer clinical trials globally, and dozens of biomarkers and precision oncology molecules, Massive Bio aims to use AI and patient-centric concierge services at scale to reach cancer patients across the world and accelerate the oncology research and development infrastructure. Massive Bio had recently announced the launch of its NASA-style Oncology Clinical Trial Command Center (OCTCC) to disrupt and accelerate trial enrollment, the launch of its 100K Cancer Clinical Trial Singularity Program aimed at matching 100,000 cancer patients in real-time to cutting-edge clinical trials using its AI-based technology, website, and apps across iOS and Android platforms, and was included in the 2022 NYC Digital Health 100 which showcases the most exciting health start-ups in the New York region.

"Massive Bio has an impressive track record of landing and expanding on pharmaceutical customers while being relentless on execution. We were impressed with highly motivated and mutually complementary founders’ team that has the courage to solve large and complex problems with minimal capital and resources and we felt that we can bring their dream into action together in an accelerated fashion with our capital, domain expertise and highly qualified network. They have the potential to transform the oncology research industry and become a world-changing company," said Kenan Turnacioğlu, who is the co-lead of the round and new board member of Massive Bio.

"Our AI-enabled clinical trial and precision oncology therapy-finding technology along with our real-time patient-empowering approaches are key to solve the problem in clinical trial enrollment in cancer pharmaceutical biotech pipeline, and we have successfully proven the necessary market fit, product deliverables, ongoing demand and expansive network effects to meet our clients’ and investors’ confidence in our platform’s success," said Arturo Loaiza-Bonilla, M.D., Co-Founder of Massive Bio. "This vote of trust from investors is the product of our relentless efforts for constant growth, best use of technology and data to deliver to each client, and to optimize systems to automate processes efficiently, while expanding our innovation stack, network development and portfolio verticals, with a keystone strategy of being a hub to allow the sustainability of the digital networks we keep and thrive upon," added Loaiza-Bonilla.

"After working with Massive Bio for the past two years and seeing how its AI-powered end-to-end platform streamlined clinical trial recruitment while providing important insights to its pharma customers, we are proud to lead the round for the company’s next phase," said Cenk Bayrakdar, Founding Partner and Managing Director at Revo Capital.

DEG’s Dr. Tilman Kruse, Global Head of Early Growth & Venture Capital, and Anna Winter, Investment Manager Private Equity & Venture Capital and deal team leader, commented, "At DEG, we are impressed how Massive Bio has created a successful technology-driven business which has an enormous impact on people: As a global investor with a mandate to improve health and living conditions as part of the UN-Sustainable Development Goals (SDG), we are particularly proud to support Massive Bio’s further international expansion to improve access to new cancer treatments and clinical trials around the world, helping cancer patients globally. We are grateful to the founders and the entire cap table for smooth, trustful, and efficient cooperation on this round which enables Massive Bio to scale up and address its global Total Addressable Market (TAM)."