On May 21, 2024 MEI Pharma presented its corporate presentation (Presentation, MEI Pharma, MAY 21, 2024, View Source [SID1234643489]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On May 21, 2024 Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, proudly reported that its flagship program, RAG-01, has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) (Press release, Ractigen, MAY 21, 2024, View Source [SID1234643509]). This notable achievement marks a significant milestone in the advancement of saRNA technology and underscores Ractigen’s commitment to addressing critical unmet medical needs. The milestone establishes RAG-01 as the first saRNA drug worldwide to achieve FTD.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

RAG-01 is currently undergoing a Phase I clinical trial in Australia for the treatment of non-muscle invasive bladder cancer (NMIBC). The trial, initiated in December 2023, has successfully enrolled and dosed three patients, demonstrating the program’s progress in clinical development.

FDA’s recent approval of the Investigational New Drug (IND) application for RAG-01 further validates the therapeutic potential of this innovative saRNA therapy. This regulatory milestone not only paves the way for the expansion of clinical trials in the United States but also highlights the FDA’s recognition of RAG-01’s promise in addressing the urgent medical needs of NMIBC patients.

Fast Track Designation is granted to investigational drugs intended for the treatment of serious conditions with unmet medical needs, facilitating their expedited development and review process. With FTD, Ractigen gains enhanced opportunities for collaboration with the FDA, enabling closer communication and expedited guidance throughout the development and regulatory review process.

Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, expressed his enthusiasm about the FDA’s decision: "We are thrilled to receive Fast Track Designation for RAG-01, marking a significant milestone not only for our program but also for the saRNA field as a whole. This designation underscores the urgency and importance of advancing innovative therapies like RAG-01 to address critical medical needs. We remain dedicated to accelerating the development of innovative saRNA therapies to address a wide range of diseases, including cancer, genetic disorders, and chronic conditions. Through strategic collaborations and pioneering research efforts, the company aims to deliver transformative treatments that improve patient outcomes and quality of life."

About RAG-01: RAG-01 is a pioneering saRNA candidate engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. Traditionally considered "undruggable," p21 presents a unique opportunity for saRNA-based targeted activation. The drug, delivered through intravesical instillation using Ractigen’s proprietary LiCO delivery technology, has shown significant tumor suppression in mouse orthotopic bladder cancer models. Currently, the Phase I clinical trial of RAG-01 in Australia has successfully enrolled and dosed the first three patients. Its development marks a significant stride in RNAa based therapies, addressing the unmet needs of NMIBC patients.

About NMIBC: NMIBC represents 50-80% of all bladder cancer cases. Despite standard treatments like transurethral resection of bladder tumor (TURBT) followed by intravesical BCG or chemotherapy, recurrence rates remain high, estimated at 50-70% within the first five years. RAG-01’s development is a significant step towards addressing this substantial unmet need in bladder cancer therapy.

About RNAa: Pioneered by Dr. Long-Cheng Li and his team, RNAa is a clinically validated platform technology. It employs saRNA to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This technology has vast potential for developing therapeutic drugs across various diseases, especially where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.

On May 21, 2024 NANOBIOTIX (Euronext : NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering nanoparticle-based therapeutic approaches to expand treatment possibilities for patients with cancer and other major diseases, reported updates to its corporate strategy designed to enable long-term growth in view of significant progress in the Company’s collaboration for the co-development and global commercialization of lead therapeutic candidate NBTXR3 (Press release, Nanobiotix, MAY 21, 2024, View Source [SID1234643490]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Nanobiotix is developing three nanoparticle-based therapy platforms in sequence. The first therapeutic candidate from the first platform, potential first-in-class radioenhancer NBTXR3, is being developed and potentially commercialized in collaboration with NBTXR3 global licensee Janssen Pharmaceutica NV ("Janssen"), a Johnson & Johnson company. Nanobiotix plans to leverage the sustainable revenue it expects to come from the development and commercialization of NBTXR3 to further advance development of the two other platforms described in the conclusion of this announcement.

"Nanobiotix has long been known for the disruptive potential of NBTXR3 for patients with cancer around the world," said Laurent Levy, chief executive officer at Nanobiotix and chairman of the executive board. "The accomplishments we have achieved throughout our 20-year history stand as a testament to the hard work of our team, the potential value the biopharmaceutical industry at-large sees in our technology, as well as the tremendous faith and confidence our investors and other financiers hold in our vision and our management. Moreover, this global licensing agreement has us well-positioned to secure the path to market for NBTXR3—and our commitment to realize the radioenhancer’s medical and economic value remains our most important goal—but the time has also come for Nanobiotix to lead the next generation of nanoparticle-based therapies in healthcare."

Delivering Potentially Curative Outcomes for Patients with Locally Advanced and Metastatic Cancers

NBTXR3 is being evaluated as a therapeutic candidate that could integrate into the cancer treatment paradigm for the 60% of patients with cancer who receive radiotherapy ("RT") as part of their therapeutic regimen each year. To date, NBTXR3 has been injected in hundreds of patients by leading physicians at several pre-eminent clinical research institutions in the United States, Europe, and Asia.

The current NBTXR3 development program is initially focused on the completion of NANORAY-312, an ongoing pivotal phase 3 study evaluating the radioenhancer for elderly patients with locally advanced head and neck cancer ("LA-HNSCC"), and the launch of a randomized Phase 2 study evaluating NBTXR3 for patients with stage 3 non-small cell lung cancer ("NSCLC") for which Janssen is fully responsible.

Preparing NANORAY-312 for Potential Regulatory Submission
In preparation for potential regulatory submission in the event of positive trial results, Nanobiotix and Janssen aligned at the collaboration’s Joint Strategy Committee ("JSC") to transfer the global sponsorship of the Phase 3 pivotal head and neck cancer trial. Nanobiotix will continue to support Janssen in execution of NANORAY-312 during and after the sponsorship transfer is complete. Study operations will remain ongoing during the transfer.

Following discussions that began at the end of 2023, and in view of the intended transfer of the sponsorship, the JSC recently aligned to a protocol amendment that would remove the planned futility analysis in light of robust, positive final data from the expansion part of Study 102, a Phase 1 study evaluating NBTXR3 in a similar population. Given that the Study 102 results provided satisfactory support for the NANORAY-312 trial design, the futility analysis was deemed unnecessary. This amendment is subject to review and approval by health authorities in all countries where NANORAY-312 is operational as well as by the sites participating in the study. To date, the amendment has been accepted by health authorities in several major regions in the study.

Additionally, Nanobiotix and Janssen have agreed to a change in approach to the planned interim analysis such that interim data will analyzed and reported after both the requisite number of events have been observed and the last patient has been recruited in 1H2026, rather than immediately after the requisite number of events as originally planned. This revised approach helps to ensure that potentially positive trial results do not influence recruitment prior to completion of the study. As such, Nanobiotix now expects the interim analysis to be reported after the last patient is recruited in 1H2026.

"Bringing a therapeutic candidate from concept to global registration is an exciting, yet daunting proposition for any biotechnology company, even those working within well-known asset classes with well-worn development and regulatory pathways," said Louis Kayitalire, MD, chief medical officer at Nanobiotix. "That is why our global licensing agreement with Janssen is great news for patients, for healthcare professionals, and for all other supporters of NBTXR3’s potential. We are confident that the leadership of the JSC will optimize the probability of success for NANORAY-312."

Nanobiotix is currently vetting options for extension of the Company’s cash runway beyond the current expectation of 3Q2025.

Advancing the NBTXR3 Collaboration

Nanobiotix and Janssen have made significant progress in establishing development operations in the collaboration since execution of the global licensing agreement in July 2023.

Operational progress highlights:

Established a JSC comprised equally of executives from both companies to drive strategic decision making for the global development program
Activated Joint Working Teams staffed by both companies with an initial focus on manufacturing and preparing NANORAY-312 for global regulatory submission in the event of a positive interim analysis
Began replication of Nanobiotix manufacturing processes to equip Janssen to produce NBTXR3 in parallel with ongoing production activities at Nanobiotix facilities
Began allocation of discretionary in-kind contributions from Janssen to Nanobiotix along with payments for manufacturing technical assistance and other product development support services
Achieved the first operational milestone in NANORAY-312, triggering a $20M milestone payment from Janssen to Nanobiotix
Received US FDA "Study May Proceed" letter for a Janssen-led Phase 2 NSCLC study after review of the Investigational New Drug application
Began elaboration of the overall clinical development plan for NBTXR3 including new indications beyond LA-HNSCC and stage 3 NSCLC
Consolidated global development and commercialization rights with Janssen via the Janssen purchase of rights to China and other Asian Markets from former Nanobiotix partner LianBio
Evolving the NBTXR3 Development Pipeline

The global licensing agreement between Nanobiotix and Janssen established a framework for potential, complementary co-development of relevant indications in the near, medium, and long term. This framework includes the potential for Nanobiotix to lead new randomized Phase 2 NBTXR3 studies. The JSC will ultimately determine the next indications beyond the immediate operational priorities in LA-HNSCC and stage 3 NSCLC. Nanobiotix expects the current pipeline, as detailed below, to play an important role in the JSC decision-making process:

Validating the safety and efficacy of RT-activated NBTXR3 for patients with LA-HNSCC and stage 3 NSCLC in the near-term

Pivotal Phase 3 Study Evaluating RT + NBTXR3 with or without cetuximab for Elderly and Frail Patients with LA-HNSCC who are Ineligible for Cisplatin Chemotherapy (NANORAY-312)
Lead: Nanobiotix (Intent to transfer global sponsorship aligned)
Status: Ongoing
Next Step: Last patient recruited 1H2026
Randomized Phase 2 Study Evaluating NBTXR3 + Chemoradiation + anti-PD-L1 for Patients with Inoperable, Stage 3 NSCLC
Lead: Janssen
Status: "Study May Proceed" letter received
Next Step: First patient randomized

Exploring the safety and efficacy of RT-activated NBTXR3 for patients with recurrent or metastatic (R/M) HNSCC that is naïve or resistant to anti-PD-1 in the medium term

Phase 1 Study Evaluating RT + NBTXR3 + Anti-PD-1 for Adult Patients with Recurrent or Metastatic ("R/M") HNSCC Naïve to Anti-PD-1 (Study 1100 Cohort 1)
Lead: Nanobiotix
Status: Dose escalation part completed and dose expansion part ongoing
Next Step: Presentation of first expansion part data at ASCO (Free ASCO Whitepaper) 2024
Phase 1 Study Evaluating RT + NBTXR3 + Anti-PD-1 for Adult Patients with Recurrent or Metastatic R/M HNSCC Resistant to Anti-PD-1 (Study 1100 Cohort 2)
Lead: Nanobiotix
Status: Dose escalation part completed and dose expansion part ongoing
Next Step: Presentation of first expansion part data at ASCO (Free ASCO Whitepaper) 2024

Establishing potential expansion opportunities in additional settings in the medium to long term

Phase 1 Study Evaluating RT + NBTXR3 for Adult Patients with Locally Advanced Pancreatic Cancer
Lead: The University of Texas MD Anderson Cancer Center ("MD Anderson")
Status: Dose escalation part completed and dose expansion part ongoing
Next Step: Presentation of updated data 2H2024
Phase 1 Study Evaluating RT + NBTXR3 for Adult Patients with Inoperable, Recurrent NSCLC Amenable to Re-irradiation
Lead: MD Anderson
Status: Dose escalation part completed and dose expansion part ongoing
Next Step: Presentation of first data 1H2025
Phase 1 Study Evaluating RT + NBTXR3 + Chemotherapy for Adult Patients with Locally Advanced Esophageal Cancer
Lead: MD Anderson
Status: Dose escalation part ongoing
Next Step: Completion of dose escalation part in 2024, initiation of dose expansion part, and presentation of first data 2025
Phase 1 Study Evaluating RT + NBTXR3 + Anti-PD-1 for Adult Patients with Multiple Advanced Tumors and Metastases Resistant to Anti-PD-1 (Study 1100 Cohort 3)
Lead: Nanobiotix
Status: Dose escalation part completed and dose expansion part ongoing
Next Step: Completion of dose expansion part and presentation of first data 2025
Phase 1 Study Evaluating RT + NBTXR3 for Adult Patients with Locally Advanced Liver Cancer and/or Liver Metastases
Lead: Nanobiotix
Status: Phase 1 completed with primary endpoints met
Next Step: Potential new indication for further development in medium to long term
Phase 1 Study Evaluating RT + NBTXR3 + Chemotherapy for Adult Patients with LA-HNSCC
Status: Completed with primary endpoints met
Next Step: Potential new indication for further development in medium to long term
Phase 1 Study Evaluating RT + NBTXR3 + Chemotherapy for Adult Patients with Locally Advanced Rectal Cancer
Status: Phase 1 completed with primary endpoints met
Next Step: Potential new indication for further development in medium to long term
Validating the safety and efficacy of RT-activated NBTXR3 by moving to global registration in LA-HNSCC remains the immediate priority of Nanobiotix and accounts for the majority of operational and financial resource allocation.

The Company expects to provide an updated global development plan for NBTXR3 after alignment at the JSC.

Updated NBTXR3 Global Development Pipeline Visualization (as of May 21, 2024)

A chart accompanying this announcement is available at View Source

*Janssen assumed responsibility for operations in Asia after securing global rights to NBTXR3 in China and Other Asian markets from former Nanobiotix collaborator LianBio. Nanobiotix and Janssen have also aligned to intent to transfer the global sponsorship.

On May 21, 2024 Dragonfly Therapeutics, Inc., a clinical stage biotechnology company developing novel immunotherapies, reported that it has entered into a clinical collaboration with Merck (known as MSD outside the US and Canada), to evaluate DF9001, Dragonfly’s EGFR immune engager, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in patients with advanced solid tumors expressing EGFR (Press release, Dragonfly Therapeutics, MAY 21, 2024, View Source [SID1234643510]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Merck, a trusted leader in drug development, has been a wonderful collaborator with Dragonfly since we signed our first collaboration agreement in 2018," said Bill Haney, co-founder and CEO of Dragonfly Therapeutics. "We are pleased to enter into this agreement with Merck for our ongoing, investigational Phase 1 trial of DF9001. In preclinical models, DF9001 engages multiple immune effector cells to drive anti-tumor activity and induces PD-L1 expression in tumor cells, sensitizing cold tumors to checkpoint inhibitors. We are hopeful that DF9001 in combination with KEYTRUDA will drive potent anti-tumor activity in a broad range of indications and are excited to further accelerate progress in the clinic to benefit patients in need."

Dragonfly is the study sponsor and first patients are expected to receive DF9001 in combination with pembrolizumab in Q4 2024. Clinical trial sites are currently open for monotherapy dosing in the U.S., with additional sites in North America and Europe expected to open in 2024. Additional information about the trial, including eligibility criteria, can be found at: View Source (ClinicalTrials.gov Identifier: NCT 05597839).

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About DF9001
DF9001 is an investigational first-in-class multi-specific drug candidate that targets EGFR and potently redirects natural killer (NK) cells, gamma-delta T cells, and CD8 T cells by engaging activating receptors NKG2D and CD16. DF9001 was discovered and developed using Dragonfly’s TriNKET platform. DF9001 is being evaluated in adult patients for the treatment of advanced solid EGFR-positive tumors. DF9001 has the potential to stimulate anti-tumor immunity in patients who are not eligible or not adequately responding to current therapies. DF9001 is the second wholly owned drug candidate in a pipeline of TriNKETs that Dragonfly is developing to address high unmet needs for patients across a broad range of disease areas.

On May 21, 2024 Pheon Therapeutics (Pheon), a leading Antibody-Drug Conjugate (ADC) specialist developing next generation ADCs for a wide range of hard-to-treat cancers, reported the completion of a $120m Series B financing to fund the development of its pipeline of differentiated ADCs (Press release, Pheon Therapeutics, MAY 21, 2024, View Source [SID1234643491]). The financing was led by TCGX with participation from other new investors BVF Partners, Lightspeed and Perceptive Advisors, alongside existing investors Atlas Venture, Brandon Capital, Forbion, and Research Corporation Technologies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The new financing will be used to further advance Pheon’s differentiated ADC pipeline through clinical proof of concept. The first three assets are aimed at an undisclosed novel target which is highly overexpressed in a wide range of solid tumors. The first program has demonstrated an unprecedented preclinical therapeutic index while utilizing a DAR8 Topoisomerase-1 inhibitor linker-payload, whereas the next two ADCs utilize other linker-payload technologies to mine the broad potential of this target. The company expects to start its first Phase 1 clinical trial in 2024 and rapidly advance towards dose expansion cohorts. The capital will also enable the expansion of Pheon’s suite of in-house technology platforms to generate optimized ADC constructs.

Cyrus Mozayeni MD, Chief Executive Officer of Pheon, said: "This raise is a critical step as we transition into a clinical-stage company. The proceeds will fund a robust clinical development pathway for our first three ADC assets, which are based on an exceptional novel target. We are excited to be working on these promising candidates and look forward to sharing their potential therapeutic benefits with patients."

Cariad Chester, Managing Partner of TCGX, commented: "The recent clinical successes of optimized ADC constructs validate the promise of this therapeutic modality to treat solid tumors. Continued progress against cancer is predicated upon innovative approaches to new targets. Pheon has an exciting pipeline of first and best-in-class ADC programs and I look forward to working with the company as it enters into the next stage of growth and development."

As part of the financing, Cariad Chester, Managing Partner of TCGX, will join the board of directors.