On January 8, 2024 Jazz pharmaceuticals presented its corporate presentation (Presentation, Jazz Pharmaceuticals, JAN 8, 2024, View Source [SID1234639084]).

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On January 8, 2024 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported highlights of pipeline updates to be presented at the 42nd Annual J.P. Morgan Healthcare Conference on January 10, 2024 at 5:15 PM PST in San Francisco, California (Press release, Precigen, JAN 8, 2024, View Source [SID1234639100]).

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AdenoVerse Immunotherapies

"In the second quarter of this year, we anticipate presenting Phase 2 pivotal study data for our lead asset, PRGN-2012, in recurrent respiratory papillomatosis, RRP, and submitting the Company’s first BLA under an accelerated approval pathway in the second half of this year. In anticipation of a potential launch in 2025, we are actively preparing for commercial readiness. This is an exciting time for Precigen as we prepare to transition from a clinical to commercial stage biotechnology company. I am incredibly proud of the Precigen team for achieving the first breakthrough therapy designation and accelerated approval pathway so rapidly for an RRP treatment and for the life-changing potential that PRGN-2012 has for RRP patients," said Helen Sabzevari, PhD, President and CEO of Precigen.

· PRGN-2012 in RRP: PRGN-2012 is an investigational off-the-shelf AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of RRP. The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation and Orphan Drug Designation for PRGN-2012 for the treatment of RRP.

· PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP (clinical trial identifier: NCT04724980).

· PRGN-2012 demonstrated strong efficacy and favorable safety profile in the Phase 1 portion of the study with 50% of patients (N=12) in durable and ongoing Complete Response more than two years after PRGN-2012 treatment.

· A Phase 2 data presentation is anticipated in the second quarter of 2024.

· A planned Biologics License Application (BLA) submission under an accelerated approval pathway is anticipated in the second half of 2024.

· Commercial readiness preparations are underway for a potential launch in 2025.

· PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is an investigational off-the-shelf AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-associated cancers.

· The Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) is currently enrolling patients (clinical trial identifier: NCT05996523).

· The Phase 2 randomized, open-label study of PRGN-2009 in combination with pembrolizumab in patients with recurrent/metastatic cervical cancer is anticipated to initiate in the first quarter of 2024 (clinical trial identifier: NCT06157151).

UltraCAR-T Cell Therapies

"We continue to advance our UltraCAR-T clinical programs and remain enthusiastic about the data we are seeing in our Phase 1b expansion studies. Precigen’s UltraCAR-T cell therapies are engineered to specifically address the limitations of conventional CAR-T therapies by improving in vivo CAR-T expansion and persistence, adding a safety/kill switch to reduce the risk of toxicity and malignancy, utilizing a non-viral design to reduce the risk of malignant transformation associated with lentivirus and retrovirus vectors, eliminating long turnaround times for manufacturing and reducing the high cost of treatment," adds Sabzevari. "We look forward to sharing new results for these assets during the planned presentations for our PRGN-3006 and PRGN-3007 UltraCAR-T programs in 2024. In addition to ongoing clinical trials, we are excited by the preclinical data for a new and differentiated CD19 targeted UltraCAR-T, which has best-in-class potential for this validated target capitalizing on the unique advantages of the UltraCAR-T platform over conventional CAR-T."

· PRGN-3006 in AML/MDS: PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a safety/kill switch. PRGN-3006 has been granted Orphan Drug Designation in patients with acute myeloid leukemia (AML) and Fast Track Designation in patients with relapsed/refractory (r/r) AML by the FDA.

· PRGN-3006 is currently under evaluation in a Phase 1b clinical trial (clinical trial identifier: NCT03927261) for the treatment of patients with r/r AML or higher-risk myelodysplastic syndromes (MDS).

· The first-in-human, Phase 1 dose escalation portion of the study with lymphodepletion was completed in r/r AML and higher-risk MDS patients.

· Phase 1 dose escalation data showed that PRGN-3006 was well-tolerated with no dose-limiting toxicities (DLTs) and a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients infused following lymphodepletion.

· An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024.

· PRGN-3005 in Ovarian Cancer: PRGN-3005 is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a safety/kill switch.

· The Phase 1b dose expansion portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT03907527).

· PRGN-3007 in Advanced ROR1+ Hematological and Solid Tumors: PRGN-3007 is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a safety/kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression.

· The Phase 1 dose escalation portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT05694364).

· A preliminary Phase 1 dose escalation data presentation is anticipated by the end of 2024.

· UltraCAR-T Targeting CD19: Preclinical data for the Company’s UltraCAR-T targeting CD19 (a validated target) have demonstrated significant potential and the Company is preparing to initiate a Phase 1 study to support a potential best-in-class CD19 CAR-T leveraging the unique advantages of the UltraCAR-T platform.

Precigen’s 42nd Annual J.P. Morgan Healthcare Conference presentation will be available on the Company website in the Events & Presentations section following the presentation.

On January 8, 2024 ConcertAI, LLC, a leading oncology real-world evidence data and AI technology company, and NeoGenomics, Inc., a leading oncology testing services company, reported a broad collaboration to advance large-scale hematological research solution to investigate real-world clinical practice and outcomes in hematological malignancies (Press release, NeoGenomics Laboratories, JAN 8, 2024, View Source;generative-ai-solutions-at-ash-2023-302028062.html [SID1234639118]).

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Few diseases are as complex as hematological malignancies. The number of alternative treatments considered for patients at different points throughout their care is more varied and individualized than what is seen in diagnosis and management of solid tumors. Hematological malignancies require surveillance of patients over multiple time periods with numerous clinical and diagnostic measures to assess sustained response to treatment or relapse.

Combining ConcertAI’s longitudinal clinical data with NeoGenomics’ comprehensive biomarkers derived from hundreds of hematological tests, we are able to establish a robust and definitive real-world evidence (RWE) hematology solution. The collaboration advances molecular and genetic data solutions for the entire drug development lifecycle, from early clinical development planning to post-approval epidemiological studies. The scale and volume of the combined dataset, which covers over one million patient lives across over 1,000 oncology clinics, provides coverage of key biomarkers throughout the entire patient treatment journey and across multiple lines of therapy, which enables high-value research and quality of care insights. This is the first population-scale hematology data set, which is both large and broad enough to minimize selection bias, to offer an actionable representation of the hematological prevalent disease in the US. The multi-modal combination of Electronic Medical Record (EMR) and rich biomarker data allows for the latest causal inference methodologies, increasingly preferred by the U.S. FDA and other regulatory bodies, and clinical AI methodologies with assurance of high representativeness and generalizability.

"ConcertAI has spent the last three years with biopharma researchers, medical societies, patient advocacy groups, and academic teams to define requirements for truly definitive, insight-enabling research solutions for hematological malignancies," said Jeff Elton, PhD, CEO of ConcertAI. "We did this with the collective goal of having a decade or more of data, richness, consistency in biomarker coverage and the ability to integrate across clinical data and diagnostic modalities. NeoGenomics captures the majority of all U.S. hematologic malignancy testing being sent out to a reference laboratory. It was obvious to both teams that combining our data and capabilities could solve the legacy challenges of depth, breadth, and limited longitudinality."

With rich cytogenetics, morphology, flow cytometry, FISH, and molecular hematological data, this collaboration now has the potential to apply the latest Generative Artificial Intelligence and other complementary approaches that are historically limited by data set sizes and lack of standardization. Hematological diseases can be stable under treatment for years and then enter a period of non-response and relapse. New AI approaches offer the potential to define predictable patterns linked to specific biomarker patterns, aligned to different treatment approaches, and directly associated with outcomes, which can inform new therapeutic programs, clinical trial designs, and treatment strategies.

"We are excited to see the collaboration produce a rich and meaningful dataset to fuel life science research, clinical trial planning and real-world data application," said Lindsey Gasparini, Vice President of Informatics at NeoGenomics. "Our expansive footprint in hematologic testing will enable an important view into clinical work up, diagnosis, and monitoring through longitudinal and multi-modal testing for the community oncologist. ConcertAI has an impressive collection of research-grade clinical data in oncology and hematology, and the power in linking that with the depth and breadth of NeoGenomics data is transformational," Gasparini continued.

The companies will be launching a hematology-focused collaborative version of ConcertAI’s Clinical Trial Optimization solution later in Q1, supporting study design and optimizing all aspects of trial planning, with multiple clinical development initiatives planned.

On January 8, 2024 Eikon Therapeutics, Inc., a pioneering biotechnology company that leverages advanced engineering to enhance drug discovery and development, reported that the company will provide an overview of clinical and preclinical progress in a presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8th at 9:00 AM Pacific Time (Press release, Eikon Therapeutics, JAN 8, 2024, View Source [SID1234639135]).

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"Eikon has made enormous progress in the development of advanced imaging tools that aid the discovery of important new therapeutics. Our clinical pipeline now includes an immunomodulatory agent, active both by itself and in combination with PD-(L)1 inhibitors, which is entering global Phase 2 studies, as well as a highly selective PARP1 inhibitor for which a Phase 1 program is now enrolling appropriate patients," said Roger M. Perlmutter, M.D., Ph.D., CEO and Board Chair of Eikon Therapeutics. "Beyond these important clinical studies, we have a set of promising development candidates that are expected to advance into the clinic over the next 12 to 24 months. Our progress showcases the integrated clinical development capabilities of Eikon’s team and demonstrates that our proprietary instruments can identify novel chemical matter with especially desirable pharmaceutical properties. These achievements offer the promise of improved treatment outcomes in cancer patients, and in patients afflicted with intractable inflammatory or neurodegenerative conditions."

Key updates:

EIK1001 Advances to Phase 2: Eikon’s most advanced candidate, EIK1001, a systemically administered TLR 7 and 8 co-agonist, has received Investigational New Drug Application (IND) clearance from the U.S. Food and Drug Administration (FDA) and has proceeded into the first of several planned Phase 2 trials focused, in this case, on patients with advanced lung cancer when administered in combination with standard-of-care pembrolizumab plus chemotherapy. The Phase 2 investigation of EIK1001 is supported by compelling data from a Phase 1 trial of over 300 patients, demonstrating its promise both as a standalone agent and in rational combinations with PD-(L)1 inhibitors.
EIK1003 Now Enrolling Patients: Eikon’s selective PARP1 inhibitor, EIK1003, has entered a Phase 1/2 clinical trial at multiple U.S. sites assessing safety and tolerability in adult patients that have received at least one prior PARP inhibitor treatment for specific solid tumors expressing selected deleterious genetic mutations. International sites will join the trial early in 2024. Preclinical studies of EIK1003 (formerly IMP1734) have shown remarkable biochemical and cellular selectivity, positioning it as a potential therapeutic option with less hematologic toxicity when compared to existing PARP1/2 inhibitors. This program is led by Eikon with its collaboration partner, Impact Therapeutics, which is responsible for trial oversight in greater China.
IND-enabling Studies Begin for Brain-Penetrant PARP1 Inhibitor, IMP17307: Collaborating with Impact Therapeutics, Eikon has initiated IND-enabling studies for a brain-penetrant PARP1 candidate and is planning for a Phase 1 clinical trial in 2025.
Discovery Pipeline Progress: Using its proprietary platform, Eikon is conducting lead optimization for several programs including those targeting the androgen receptor and its clinically relevant variants, as well as several targeting key proteins in the DNA damage repair pathway including Werner (WRN). The company anticipates nominating a new clinical candidate in early 2024.
Eikon Systems Business Unit Established: Eikon has developed a suite of instruments that permits detailed, automated characterization of protein motion in living cells. Since most proteins function by interacting with other cellular structures (including other proteins), analysis of the dynamic behavior of individual protein molecules yields information regarding the function and regulation of biologic systems. These tools are increasingly sought after by academic investigators through Eikon’s Technology Access Program. The company expects that instruments from Eikon Systems will help to catalyze the use of protein motion measurements routinely as a means of elucidating biological processes.
An archived recording of Eikon’s presentation at the J.P. Morgan Healthcare Conference can be accessed at: www.EikonTx.com/#news.

On January 8, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported its anticipated 2024 milestones and value-driving catalysts through 2026 that support the company’s mission to transform patient outcomes in rare diseases (Press release, Agios Pharmaceuticals, JAN 8, 2024, View Source [SID1234639052]). Agios will present at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 7:30 a.m. PT, and a live webcast will be available at investor.agios.com.

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"We were pleased to announce positive topline data from the Phase 3 study of our lead PK activator, mitapivat, in non-transfusion-dependent alpha- or beta-thalassemia last week, a segment of the population with no currently approved therapeutic options in the U.S. We look forward to data readouts from four additional Phase 3 studies across our industry-leading PK activator franchise by the end of 2025," said Brian Goff, chief executive officer at Agios. "This robust series of near-term catalysts positions Agios for potential launches of a first- and best-in-class therapy in thalassemia in 2025 and in sickle cell disease in 2026, and we look forward to maximizing the commercial opportunities ahead of us. Supported by our strong cash position, Agios is poised for significant progress in the next 12-24 months, and we look forward to the opportunity to deliver a novel oral treatment option for two additional hematologic diseases with high unmet need."

2023 Highlights

Thalassemia: Completed enrollment in the Phase 3 ENERGIZE and ENERGIZE-T studies of mitapivat in non-transfusion-dependent and transfusion-dependent thalassemia, respectively
Sickle Cell Disease: Announced positive data from the Phase 2 portion of the RISE UP study of mitapivat and dosed the first patients in the Phase 3 portion
Pediatric PK Deficiency: Completed enrollment in the Phase 3 ACTIVATE kids-T study of mitapivat in children with PK deficiency who are regularly transfused. Enrolled more than half of patients in the Phase 3 ACTIVATE-kids study of mitapivat in children with pediatric PK deficiency who are not regularly transfused
Lower-risk Myelodysplastic Syndromes (LR-MDS): Announced clinical proof-of-concept in Phase 2a study of AG-946, supporting continued development in Phase 2b
Earlier-stage Pipeline: Filed an Investigational New Drug Application (IND) for PAH stabilizer for the treatment of phenylketonuria (PKU)
Business Development: Announced exclusive worldwide license agreement with Alnylam for novel siRNA targeting TMPRSS6 for the potential treatment of polycythemia vera (PV)
Data Presentations: Presented broad set of clinical and translational data at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, including positive data from the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease
Anticipated 2024 Milestones

Thalassemia: Following the announcement of topline data from the Phase 3 ENERGIZE study last week, Agios plans to report topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year) and submit a New Drug Application (NDA) for mitapivat in thalassemia (year-end)
Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat (year-end)
Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat (mid-2024). Report topline data from Phase 3 ACTIVATE kids-T study (year-end)
Lower-risk Myelodysplastic Syndromes (LR-MDS): Dose first patient in Phase 2b study of AG-946 (mid-year)
Earlier-stage Pipeline: Dose the first patient in the Phase 1 study of PAH stabilizer for the treatment of PKU (H1 2024)
Four Additional Phase 3 Readouts and Two Potential New Indication Approvals Expected by End of 2026

2024

Data readout from Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia (announced January 3, 2024)
Data readout from Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year)
Data readout from Phase 3 ACTIVATE kids-T study of mitapivat in pediatric PK deficiency (year-end)
2025

Data readout from Phase 3 portion of the RISE UP study of mitapivat in sickle cell disease
Data readout from Phase 3 ACTIVATE kids study of mitapivat in pediatric PK deficiency
Potential FDA approval for mitapivat in thalassemia
2026

Potential FDA approval for mitapivat in sickle cell disease
Potential FDA approval for mitapivat in pediatric PK deficiency
Presentation at 42nd Annual J.P. Morgan Healthcare Conference

Agios will webcast its corporate presentation from the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10 at 7:30 a.m. PT. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.