Inspirna to Present at the 42nd Annual J.P. Morgan Healthcare Conference

On January 4, 2024 Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class cancer therapeutics, reported that Chief Executive Officer Dr. Usman "Oz" Azam, M.D., will participate in a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11, 2024 at 8:30am PT / 11:30am ET (Press release, Inspirna, JAN 4, 2024, View Source [SID1234638975]).

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Inspirna Partners with Merck KGaA, Darmstadt, Germany, to Accelerate Global Development of Ompenaclid (RGX-202)

On January 4, 2024 Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class cancer therapeutics, reported a licensing agreement with Merck KGaA, Darmstadt, Germany, for ompenaclid (RGX-202), a first-in-class oral inhibitor of the creatine transport channel SLC6A8, currently in development for RAS mutated (RASm) second-line (2L) advanced or metastatic colorectal cancer (mCRC) and SLC6a8 targeting follow-on compounds (Press release, Inspirna, JAN 4, 2024, View Source [SID1234638974]).

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"We are excited to partner with Merck KGaA, Darmstadt, Germany, a leader in the oncology field with global drug development and commercial expertise in colorectal cancer specifically, to help bring our novel therapies to more patients in need," said Dr. Usman "Oz" Azam, M.D., Chief Executive Officer of Inspirna. "The data to date validates our belief in ompenaclid as a potential first-in-class therapy for advanced colorectal cancer and underscores the power of our proprietary target discovery platform RNA-DRIVEr. We look forward to working closely with Merck KGaA, Darmstadt, Germany, as we continue to evaluate ompenaclid in a Phase 2 randomized controlled trial."

"Over the past decade, the treatment paradigm for patients with RAS-mutated CRC, accounting for approximately 45% of second-line population, has not seen major innovation," said Victoria Zazulina, M.D., Head, Development Unit, Oncology for the Healthcare business of Merck KGaA, Darmstadt, Germany. "With our expertise in the treatment of CRC, and based on the encouraging early data for ompenaclid, this agreement with Inspirna offers the opportunity to advance a potential new first-in-class therapy that may improve outcomes for patients."

Under the terms of the license agreement, Merck KGaA, Darmstadt, Germany will receive an exclusive license to ompenaclid outside of the United States and an option to co-develop and co-promote ompenaclid in the US. Furthermore, the parties agreed to collaborate on Inspirna’s SLC6A8 follow-on compounds for which Inspirna will retain US co-development and co-commercialization rights. Inspirna will receive an upfront payment of $45 million. Upon the achievement of certain development and sales milestones for ompenaclid, Inspirna is eligible to receive milestone payments with tiered royalty rates in the low teens on net sales outside of the US. Inspirna is eligible to also receive development, regulatory and sales milestone payments for each follow-on compound targeting SLC6A8 along with up to double-digit royalties on net sales outside of the US.

Inspirna most recently presented clinical data from the Phase 1b/2 study of ompenaclid in combination with FOLFIRI and bevacizumab at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October 2023. Results as of the September 18, 2023 data cutoff showed encouraging efficacy and safety data, including a median progression-free survival of 10.2 months and median overall survival of 19.1 months across all 41 patients with 2L RASm mCRC. Of the 30 evaluable patients for response, the objective response rate was 37%, with 11 partial responses. Ompenaclid was well-tolerated, with no dose-limiting toxicities observed in the dose-escalation cohort and combination safety profile comparable to FOLFIRI plus bevacizumab backbone treatment.

Inspirna has initiated a Phase 2 double-blind randomized controlled trial in 2L RAS mutant advanced or metastatic mCRC comparing ompenaclid versus placebo plus FOLFIRI and bevacizumab.

IN8bio Highlights Recent Company Accomplishments and Outlines 2024 Pipeline Goals

On January 4, 2024 IN8bio, Inc. (NASDAQ: INAB), a leading clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported recent business updates and provided pipeline goals for 2024 (Press release, In8bio, JAN 4, 2024, https://investors.in8bio.com/news-releases/news-release-details/in8bio-highlights-recent-company-accomplishments-and-outlines [SID1234638973]).

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"2023 was a year marked by strong execution for IN8bio. We delivered on several clinical milestones, reporting positive data updates across our pipeline of innovative gamma-delta T cell therapies at many prestigious medical meetings, and secured the financing to extend our runway into 2025," said William Ho, CEO and co-founder of IN8bio. "We continue to be excited by the data from the Phase 1 trial of INB-100, which showed that all evaluable patients remained in durable complete remission, with six patients remaining relapse free beyond one year. Importantly, we are excited that we are seeing in vivo expansion and persistence of allogeneic gamma-delta T cells, now out to 365 days. This persistence clearly differentiates our results from the data presented on other allogeneic cellular therapies to date and we believe this data underscores the far-reaching potential of gamma-delta T cells to help provide durable relapse-free periods in patients with hematologic malignancies undergoing haploidentical stem cell transplantation. We believe we are well positioned to execute on our clinical programs and are poised for many exciting updates in 2024."

Recent Company Updates

Announced positive clinical update demonstrating continued durable complete remissions in 100% of evaluable patients (n=10) in the Phase 1 trial of INB-100 in leukemia patients undergoing haploidentical stem cell transplantation. The data was presented at the ASH (Free ASH Whitepaper) Annual Meeting in December.
Presented data demonstrating INB-200’s extended progression-free survival in glioblastoma multiforme (GBM) patients and potential for INB-400 to treat GBM at the SNO Annual Meeting.
Initiated enrollment for the Phase 2 trial of INB-400 in GBM (NCT05664243).
Announced a private placement totaling up to $46.9 million in gross proceeds. The initial closing of $14.4 million is expected to support operational execution and extended the Company’s cash runway into 2025 with the potential for up to $32.5 million in additional capital at increasing valuations.
Appointed internationally recognized immuno-oncology expert Dr. Corinne Epperly, M.D., M.P.H., to IN8bio’s Board of Directors.
Anticipated 2024 Pipeline Goals

INB-100: Enroll an additional 10 patients in an expansion cohort at the recommended Phase 2 dose (RP2D) and report Phase 1 long-term follow-up results at multiple medical meetings throughout 2024; potentially submit IND for Phase 3 randomized control trial in 2024.
INB-200: Report Phase 1 long-term follow up results at multiple medical meetings in 2024.
INB-300: Present additional preclinical data demonstrating proof-of-concept for the nsCAR platform targeting CD33 and CD123 at American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2024.
INB-400: Dose first patient and treat up to 15 patients at multiple sites across the United States in the Phase 2 trial in newly diagnosed GBM; potentially submit IND for allogeneic Phase 1b in relapsed GBM in 2024.

Halia Therapeutics to Attend the 42nd JP Morgan Healthcare Conference and Present at Biotech Showcase in San Francisco

On January 4, 2024 Halia Therapeutics, a clinical-stage biopharmaceutical company, is pioneering a novel class of small molecule medications designed to combat inflammation, reported that its leadership team will attend the 42nd JP Morgan Healthcare Conference (January 8 – 11) and present at Biotech Showcase 2024 (January 8–10) in San Francisco (Press release, Halia Therapeutics, JAN 4, 2024, View Source [SID1234638972]).

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Biotech Showcase, produced by Demy-Colton and EBD Group, is an investor conference focused on driving advances in therapeutic development by providing a sophisticated networking platform for executives and investors that fosters investment and partnership opportunities.

At the Showcase, Halia Therapeutics will discuss its lead product, HT-6184, which inhibits chronic inflammation associated with multiple diseases. A Phase II clinical trial to evaluate HT-6184 was recently initiated to treat patients with lower-risk myelodysplastic syndromes. This year, registered attendees can view Halia’s presentation live and access a recorded version currently available.

Presentation details at Biotech Showcase:

Category Listing: Inflammation, Oncology, Neurology

Speaker: Margit Janat-Amsbury, M.D., Ph.D., Chief Medical Officer of Halia Therapeutics

Date: January 9, 2024

Time: 10:15 a.m. PT

Location: Hilton San Francisco Union Square, 333 O’Farrell St, San Francisco, CA 94102 (Franciscan-B)

Fusion Pharmaceuticals Announces Clinical Program and Manufacturing Updates

On January 4, 2024 Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, reported significant progress with its FPI-2265 development program, an update on FPI-1434 Phase 1 Cohort 2 data and the production of the first clinical doses at the Company’s proprietary manufacturing facility (Press release, Fusion Pharmaceuticals, JAN 4, 2024, View Source [SID1234638971]).

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"We begin 2024 with strong momentum, given a potential registration-enabling path for FPI-2265, encouraging results in our FPI-1434 program, including first signs of antitumor activity, and a fully operational TAT manufacturing facility that has already begun to produce clinical doses for our actinium-based PSMA lead program," said Chief Executive Officer John Valliant, Ph.D.

"We achieved alignment with the U.S. Food and Drug Administration (FDA) on a protocol and development plan for FPI-2265, providing our team with a potential path to registration and positioning FPI-2265 to be the first actinium-based PSMA targeting radioligand therapy to market, if approved. Given the significant and growing market for PLUVICTO, we believe that FPI-2265 will address an important unmet need for patients who progress on or after lutetium-based therapy."

FPI-2265 Phase 2/3 Development Plan in mCRPC

The Company announced today that it has aligned with the FDA on its submitted Phase 2/3 protocol for FPI-2265, a targeted alpha therapy (TAT) targeting prostate specific membrane antigen (PSMA) for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC) with progressive disease. The updated development plan includes a Phase 2 dose optimization lead-in, expected to complete enrollment by the end of 2024, and a Phase 3 registrational trial expected to begin in 2025.

The Phase 2 portion of the protocol is designed to evaluate the safety and efficacy of FPI-2265 across three dosing regimens in approximately 60 patients with mCRPC with progressive disease after 177Lu-based PSMA radioligand therapy, such as PLUVICTO. Based on literature and TATCIST data reported to date, 100 kBq/kg administered every 8 weeks is known to be a safe and active dose regimen. In order to further optimize the benefit/risk ratio of FPI-2265, Fusion will explore alternate regimens with higher dosing frequency while keeping cumulative dose and total duration of treatment the same. Additional regimens to be evaluated will include a dose of 50 kBq/kg every 4 weeks and 75 kBq/kg every 6 weeks. The primary endpoints are safety and the proportion of patients with ≥ 50% decline in PSA level with key secondary endpoints of objective response rate (ORR) and radiographic progression free survival (rPFS). The Phase 2 trial is expected to initiate in the second quarter of 2024 with enrollment completed by year-end. The Company will seek to hold an End of Phase 2 meeting with the FDA to determine the recommended Phase 3 dosing regimen based on analysis of the Phase 2 data.

"We believe evaluating dosing regimens that deliver the same total dose over the same duration of treatment in the Phase 2 portion of the study allows us to optimize our Phase 3 clinical trial dose in alignment with FDA guidance and determine the best potential regimen of FPI-2265," said Chief Medical Officer, Dmitri Bobilev, M.D.

The Phase 3 portion of the trial is designed to be a registration-enabling global trial evaluating the efficacy and safety of FPI-2265 compared with standard of care in approximately 550 patients with mCRPC with progressive disease who have previously been treated with a 177Lu-based PSMA radiotherapy. The primary endpoint will evaluate rPFS. Key secondary endpoints include PFS, ORR, OS, PSA50 and duration of response. The Company plans to initiate the Phase 3 trial in 2025.

In February 2023, Fusion acquired an IND for the ongoing Phase 2 clinical trial (the TATCIST trial) evaluating FPI-2265 (225Ac-PSMA I&T). The TATCIST trial was designed to evaluate patients with mCRPC with progressive disease, including patients who are naïve to PSMA-targeted radiopharmaceuticals and those who have been pre-treated with 177Lu-based PSMA radiopharmaceutical therapy. Fusion intends to report data from approximately 25 to 30 patients in April 2024 and then prioritize enrollment in the new Phase 2/3 trial.

The Company is also pursuing the opportunity to potentially move the therapy candidate into earlier lines of treatment with combinations of FPI-2265 and olaparib. Fusion expects to initiate a combination trial in the first half of this year.

FPI-1434 Cohort 2 Data & Next Steps

Fusion announced today encouraging early findings from Cohort 2 in the ongoing FPI-1434 Phase 1 clinical trial. No dose limiting toxicities (DLTs) were observed to date in the 25 kBq/kg dose cohort. Two out of three patients completed the DLT period, and one pancreatic cancer patient discontinued treatment due to disease progression.

One heavily treated patient with Ewing sarcoma showed evidence of anti-tumor activity after a single 25 kBq/kg dose of FPI-1434. The second patient received four cycles of therapy and showed stable disease as best response. FPI-1434 was well tolerated, with no DLTs and transient Grade 1 or less thrombocytopenia at the 25 kBq/kg dose level.

The Company plans to complete and further evaluate results from Cohort 2 and hold a Safety Review Committee (SRC) meeting to evaluate the emerging data. Fusion plans to share more details on the data and the FPI-1434 development program in mid-2024.

Dr. Valliant continued, "We continue to believe alpha emitters represent the evolution of the toxin used in antibody-drug conjugates (ADCs) and hold the potential to improve the potency of naked antibodies. There is significant untapped potential to use the precision targeting of antibodies to deliver the potent payload of actinium directly to tumor cells. While early, we are encouraged by the results showing good safety and evidence of antitumor activity at low doses of FPI-1434."

In June 2023, Fusion reported results from three patients at 15 kBq/kg in Cohort 1 of the cold/hot dosing regimen. In Cohort 1, cold/hot dosing was observed to be generally well tolerated with no treatment-related serious adverse events (SAEs) or dose DLTs. Pre-administration of cold antibody demonstrated improved tumor uptake while also reducing hematological toxicity observed in the hot only dosing arm. Two heavily pre-treated patients from the cold/hot dosing arm received three and five cycles of treatment, with both achieving durable stable disease as their best response.

Manufacturing Facility Update

Fusion reported today that it has completed validation of its state-of-the-art good manufacturing practice (GMP) manufacturing facility and produced the first clinical dose of a TAT.

Dr. Valliant continued, "The initiation of production at our own facility, and the diversification afforded by our external partnerships, positions us for execution on our multiple clinical programs. We built Fusion on a foundation of end-to-end manufacturing expertise, including experience with global radiopharmaceutical logistics and distribution. We also now have one of the first in-house TAT manufacturing facilities with access to a generator technology that allows for convenient onsite production of actinium-225, providing us with additional capacity and flexibility in our manufacturing programs."

Fusion’s facility, which has clinical and commercial scale manufacturing capabilities, is designed to support the Company’s growing pipeline of TATs and expected to be capable of producing up to 100,000 doses per year. Doses produced out of Fusion’s manufacturing facility are expected to support FPI-2265 manufacturing and are expected to be expanded to include Fusion’s other proprietary and partnered programs.

Financials

On a pro forma basis as of September 30, 2023, Fusion’s cash, cash equivalents and investments were approximately $287 million, after taking into account subsequent proceeds of approximately $65 million from sales under the Company’s at-the-market equity offering program and an expected $15 million draw down under the Company’s existing debt facility. Fusion expects its cash, cash equivalents and investments will now be sufficient to fund operating expenses and capital expenditure requirements into the fourth quarter of 2025.