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.

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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.

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]).

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

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On May 21, 2024 Imvax, Inc., a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies, reported the completion of enrollment in its randomized, multicenter, double-blind, placebo-controlled Phase 2b clinical trial of IGV-001 in patients with newly diagnosed glioblastoma (ndGBM) (Press release, Imvax, MAY 21, 2024, View Source;utm_medium=rss&utm_campaign=imvax-announces-completion-of-enrollment-in-phase-2b-clinical-trial-of-igv-001-and-successful-financing [SID1234643488]). IGV-001 is derived from Imvax’s proprietary Goldspire immuno-oncology platform, which is designed to use a patient’s own tumor cells to induce a broad and durable immune response against tumors. The primary endpoint of the Phase 2b trial is Progression Free Survival (PFS) and top-line data is expected by mid-2025.

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Separately, the Company reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to IGV-001 for the treatment of ndGBM based on the Company’s existing Phase 1b data supporting its therapeutic potential in this underserved and life-threatening condition.

Imvax also announced the closing of an approximately $35 million financing round, with half of the capital coming from new investors. Over the past 15 months, the Company has raised $57 million to support the completion of the Phase 2b trial.

"We are very pleased to announce full enrollment of the trial in less than 14 months, which speaks to both the clinical interest in this potential therapy and the efficient execution of our novel approach to treating GBM patients," said John P. Furey, Chief Executive Officer. "We are grateful to the investigators, patients, and their families for their commitment to this study and look forward to seeing top-line data next year. We are also delighted that the FDA has granted Fast Track designation to IGV-001. The last significant advancement in the standard of care for GBM – about an 8-week improvement in PFS – was nearly 20 years ago, highlighting the need for continued innovation and treatment options. We believe IGV-001 may offer meaningful clinical benefit for patients diagnosed with this deadly disease."

Mr. Furey continued, "We are grateful for the support of both existing and multiple new investors in our recent fundraising, which reflects their belief in the promise of IGV-001 and their commitment to patients facing a GBM diagnosis. We now have sufficient resources to support the completion of the trial and carry us beyond the reporting of top-line results in mid-2025."

About the Phase 2b Trial
The Phase 2b clinical trial is a randomized, multicenter, double-blind, placebo-controlled study (NCT04485949) designed to assess the safety and efficacy of IGV-001, an autologous biologic-device combination product, in ndGBM patients. The trial enrolled approximately 100 participants in a 2:1 randomization across 20 sites in the United States. Approximately 48 hours after surgical resection, participants in the IGV-001 arm were implanted with biodiffusion chambers containing a combination of personalized whole tumor-derived cells with an antisense oligonucleotide (IMV-001); in the placebo arm, the chambers contained an inactive solution only. In both arms, the biodiffusion chambers were explanted approximately 48 hours later, and after six weeks all patients were treated with standard of care (adjuvant radiotherapy and temozolomide followed by maintenance temozolomide). The primary endpoint of the trial is progression-free survival (PFS) and key secondary endpoints include overall survival (OS) and safety.

About IGV-001
IGV-001 is an autologous biologic-device combination product derived from Imvax’s proprietary Goldspire immuno-oncology platform for solid tumors, which involves a unique approach to inducing a broad and durable immune response against tumors. Phase 1 studies showed that IGV-001 was safe and well tolerated, and a Phase 1b ndGBM study also yielded several efficacy signals, including significant improvements in PFS, OS, radiographic evidence of tumor response, and multiple biomarker changes that supported the presence of an immune response (Andrews DW, et al., Clin Cancer Res. 2021;27(7):1912-1922). In ten Stupp-eligible ndGBM patients in the highest dose cohort treated with IGV-001, median PFS was 17.1 months, compared with 6.5 months in historical standard-of-care (SOC) treatment, and median OS was 38.2 months, compared with 16.2 months in historical SOC.

About Fast Track Designation
Fast Track is a process designed by the FDA to facilitate the development and expedite the review of investigational treatments that have the potential to treat serious conditions and address recognized areas of unmet medical need. Programs with Fast Track designation can benefit from early and more frequent interactions with the FDA to discuss the product candidate’s development plan in addition to a rolling submission of the marketing application. Product candidates with Fast Track designation also may be eligible for priority review and accelerated approval.

On May 21, 2024 GT Biopharma, Inc. (NASDAQ: GTBP) (the "Company"), reported that it has entered into a definitive securities purchase agreements for the purchase and sale of 740,000 shares of the Company’s common stock at a purchase price of $4.35 per share of common stock in a registered direct offering priced at-the-market under Nasdaq rules (Press release, GT Biopharma, MAY 21, 2024, View Source [SID1234643487]). In a concurrent private placement, the Company will issue warrants to purchase up to 740,000 shares of common stock. The warrants have an exercise price of $4.35 per share, will be exercisable immediately and will have a term of five years following the date of issuance. The closing of the offering is expected to occur on or about May 23, 2024, subject to the satisfaction of customary closing conditions.

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Roth Capital Partners is acting as the exclusive placement agent for the offering.

The gross proceeds to the Company from this offering are expected to be approximately $3.2 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. The Company intends to use the net proceeds from this offering for general corporate purposes.

The securities in the offering described above (but not the warrants issued in the concurrent private placement or the shares of common stock underlying the warrants) are being offered by the Company pursuant to a "shelf" registration statement on Form S-3 (File No. 333-267870) previously filed with the Securities and Exchange Commission (the "SEC") and declared effective by the SEC on October 20, 2022. The offering is being made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement, relating to the offering that will be filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at View Source or by contacting Roth Capital Partners, LLC at 888 San Clemente Drive, Newport Beach CA 92660, by phone at (800) 678-9147.

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.