On August 12, 2024 Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported that the Company has been awarded a $2 million grant from the National Institutes of Health (NIH) Small Business Innovation Research (SBIR) program to support the clinical investigation of MT-601 in patients with non-Hodgkin’s lymphoma (NHL) who have relapsed following anti-CD19 chimeric antigen receptor (CAR) T cell therapy (Press release, Marker Therapeutics, AUG 12, 2024, View Source [SID1234645733]).

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The SBIR grant has been awarded based in part on Marker’s preliminary clinical data in patients with lymphoma (Press Release, September 11, 2023) as well as non-clinical data demonstrating the anti-tumor activity of MT-601 on anti-CD19 CAR resistant lymphoma cells (Press Release, May 31, 2023). The proceeds of the grant will support the nationwide multi-center Phase 1 APOLLO study (ClinicalTrials.gov identifier: NCT05798897), evaluating the safety and efficacy of MT-601, a multi-tumor associated antigen (multiTAA)-specific T cell product, in patients with relapsed NHL including those previously treated with anti-CD19 CAR-T cell therapy. Including this SBIR grant, the Company has been awarded over $19 million in non-dilutive funding proceeds.

"We are pleased to receive the SBIR grant from the NIH to support our clinical Phase 1 study in CAR-relapsed patients with non-Hodgkin’s lymphoma," said Juan Vera, M.D., President and CEO of Marker Therapeutics. "Although anti-CD19 CAR-T cells are rapidly expanding as a treatment option in patients with hematological malignancies, approximately 40-60% of patients will relapse within the first year of therapy with currently no standard of care for patients post CD19-targeting CAR-T cells. The NIH award process is highly competitive, and we believe that the decision the NIH made suggests the potential scientific merit and the capacity of Marker’s APOLLO study to address an unmet medical need."

Dr. Vera continued: "While our results of the APOLLO study are preliminary, as we move through the dose escalation part of the study, we are encouraged by the objective responses observed in all three study participants treated at City of Hope (Press Release, April 8, 2024) and the lack of cytokine release syndrome (CRS) or immune effector cell associated neurotoxicity syndrome (ICANS) observed. We will continue to closely monitor all patients for long-term treatment effects and durability of response, and with the non-dilutive funding support from NIH we look forward to treating additional participants in this Phase 1 study."

"This grant award is a testimony of our continued commitment to apply for non-dilutive funding and allows us to leverage our data to drive innovation and growth while maximizing shareholder value. Obtaining non-dilutive funding is an ongoing effort, and we are actively applying for additional opportunities as they become available," concluded Dr. Vera.

About the NIH SBIR Program

The NIH Small Business Innovation Research (SBIR) Program sets aside more than $1.2 billion from its Research & Development Funding to specifically support early-stage small businesses throughout the United States. Many companies leverage the NIH SBIR funding to attract the partners and investors needed to take an innovation to market. The Small Business program focuses on a variety of high-impact technologies including research tools, diagnostics, digital health, drugs, and medical devices, and can provide the seed funding needed to bring scientific innovations from bench to bedside.

About MT-601

The Company’s lead product, MT-601, is a multi-tumor associated antigen (multiTAA)-specific T cell product that utilizes a non-genetically modified approach that specifically targets six different tumor antigens upregulated in lymphoma cells (Survivin, PRAME, WT-1, NY-ESO-1, SSX-2, MAGE-A4). Marker is currently investigating MT-601 in the Company-sponsored Phase 1 APOLLO trial (clinicaltrials.gov identifier: NCT05798897) for the treatment of lymphoma patients who are relapsed after or where CD19 CAR-T cell therapy is not an option.

About APOLLO

The APOLLO trial (clinicaltrials.gov Identifier: NCT05798897) is a Phase 1, multicenter, open-label study designed to evaluate the safety and efficacy of MT-601 in participants with lymphoma who relapsed after anti-CD19 CAR-T cell therapy or where anti-CD19 CAR-T cell therapy is not an option. The primary objective of this exploratory Phase 1 clinical trial is to evaluate the safety and preliminary efficacy of MT-601 in participants with various lymphoma subtypes. Under the APOLLO trial, it is anticipated that nine clinical sites across the United States will cumulatively enroll up to approximately 30 participants during the dose escalation phase.

About multiTAA-specific T cells

The multi-tumor associated antigen (multiTAA)-specific T cell platform is a novel, non-genetically modified cell therapy approach that selectively expands tumor-specific T cells from a patient’s/donor’s blood capable of recognizing a broad range of tumor antigens. Unlike other T cell therapies, multiTAA-specific T cells allow the recognition of hundreds of different epitopes within up to six tumor-specific antigens, thereby reducing the possibility of tumor escape. Since multiTAA-specific T cells are not genetically engineered, Marker believes that its product candidates will be easier and less expensive to manufacture, with an improved safety profile, compared to current engineered T cell approaches, and may provide patients with meaningful clinical benefits.

On August 12, 2024 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported financial results for the second quarter ended June 30, 2024 (Press release, Leap Therapeutics, AUG 12, 2024, View Source [SID1234645732]).

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Leap Highlights:

· Overall response rate (ORR) increases to 33% across all evaluable patients and 38% across evaluable patients with left-sided CRC in updated data from Part A of the Phase 2 DeFianCe study evaluating DKN-01 in combination with bevacizumab and chemotherapy in second-line patients with advanced colorectal cancer (CRC)
· Expanded the randomized controlled Part B of the DeFianCe study to 180 patients; enrollment expected to be completed by end of September 2024 with data expected in mid-2025
· Patient follow-up continues in the randomized controlled Part C of the Phase 2 DisTinGuish study evaluating DKN-01 in combination with tislelizumab and chemotherapy in first-line patients with advanced gastroesophageal junction (GEJ) and gastric cancer; data expected in Q4 2024 or early 2025
· Completed $40 million private placement with new and existing investors, including Gilead Sciences, Inc.

"With the momentum provided by our $40 million private placement, we are positioned to achieve our critical company milestones," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "We have executed well on our two DKN-01 randomized controlled trials and on preparatory activities for registrational studies. We look forward to sharing initial data from both randomized controlled studies over the next 12 months as we strive to deliver new treatments for patients fighting against cancer."

DKN-01 Development Update

· ORR increases in updated data from Part A of the DeFianCe Study. The DeFianCe study (NCT05480306) is a Phase 2 study evaluating DKN-01 in combination with bevacizumab and chemotherapy in second-line patients with advanced microsatellite stable CRC. Preliminary results from Part A of the study were previously reported at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in January 2024. In April 2024, a ninth Part A patient was identified as having a partial response (PR). This patient, who has left-sided Consensus Molecular Subtype 4 CRC with APC and TP53 mutations and KRAS wildtype genetics, had been previously treated with cetuximab and chemotherapy. The patient enrolled in Part A in March 2023 and had a best response of stable disease (SD) for over a year before the tumor reduction deepened into a PR. The patient remains on study with a confirmed PR.

· Key Updated Part A Findings (as of June 7, 2024 data cut-off):

o Across all patients enrolled (n=33):

§ ORR among response-evaluable patients (n=27) was 33% and disease control rate (DCR) was 93%, including 9 PRs and 16 patients with a best response of SD
§ Median progression-free survival (PFS) was unchanged at 6.3 months

o Enhanced activity in patients with left-sided tumors (n=25), a group that has more frequent activation of the Wnt pathway modulated by DKK1

§ 38% ORR and 100% DCR in response-evaluable population (8 PRs, 13 SDs)
§ Median PFS was unchanged at 8.6 months

o DKN-01 plus bevacizumab and chemotherapy was well-tolerated, with a majority of DKN-01 related events being low grade (Grade 1/2)

· Enrollment in Part B of the DeFianCe Study in CRC patients is ongoing and expected to be completed by the end of September 2024. The Company expanded the randomized controlled Part B of the DeFianCe study from 130 to 180 patients and included PFS in the subpopulation of patients with left-sided CRC as an additional primary endpoint. As of August 9, 2024, 161 patients have enrolled in Part B. The Company expects to complete enrollment by the end of September 2024, with data expected mid-2025.

· Randomized controlled Part C of the DisTinGuish study in patients with GEJ and gastric cancer is ongoing, with initial data expected in Q4 2024 or early 2025. The DisTinGuish study (NCT0436380) is a Phase 2, randomized, open-label, multicenter study of DKN-01 in combination with tislelizumab and chemotherapy in first-line, HER-2 negative patients with GEJ and gastric cancer. Part C enrolled 170 patients randomized 1:1 to evaluate DKN-01 in combination with tislelizumab and chemotherapy, compared to tislelizumab and chemotherapy alone. The Company expects to report initial data from Part C of the DisTinGuish study in Q4 2024 or early 2025.

Business Update:

· Completed a $40 million private placement. In April 2024, Leap entered into a securities purchase agreement with a select group of new and existing investors including Gilead Sciences, Inc., a life sciences-focused investor, Samsara BioCapital, LP, 683 Capital Partners, LP, Laurion Capital Management LP, and Rock Springs Capital Management LP. Gross proceeds from the private placement were approximately $40 million. The net proceeds from this financing, combined with existing cash, cash equivalents and marketable securities, are expected to fund Leap’s operating and capital expenditures into the second quarter of 2026.

Selected Second Quarter 2024 Financial Results

Net Loss was $20.4 million for the second quarter 2024, compared to $13.4 million for the same period in 2023. The increase was primarily due to an increase in research and development expenses.

Research and development expenses were $17.9 million for the second quarter 2024, compared to $11.1 million for the same period in 2023. The increase of $6.8 million was primarily due to an increase of $5.7 million in clinical trial costs due to patient enrollment, the duration of patients on study, the enhancement of correlative studies, increase in site activity associated with Part C of the DisTinGuish study, and the expansion of the size of Part B of the DeFianCe study. There was also an increase of $0.6 million in manufacturing costs related to clinical trial material and manufacturing campaigns and an increase of $0.5 million in payroll and other related expenses due to an increase in headcount of our R&D full-time employees.

General and administrative expenses were $3.4 million for the second quarter 2024, compared to $3.6 million for the same period in 2023. The decrease was due to a decrease of $0.3 million in professional fees associated with our business development activities, partially offset by a $0.1 million increase in payroll and other related expenses.

Cash and cash equivalents totaled $78.5 million at June 30, 2024.

On August 12, 2024 IN8bio, Inc. (Nasdaq: INAB), a leading clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies for cancer, reported updated positive clinical data from both of the Company’s Phase 1 investigator-sponsored trials of INB-100 for hematological malignancies and INB-200 for GBM (Press release, In8bio, AUG 12, 2024, View Source [SID1234645731]). The Company has also completed a Type B meeting with the FDA and received guidance on the registrational path to advance INB-100 for the treatment of AML.

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Every AML patient treated with INB-100 remains in complete remission (CR), and patients across both trials have exceeded expected progression-free survival (PFS) to date. These data continue to demonstrate the broad clinical potential of gamma-delta T cells for difficult-to-treat cancers and provides support for the advancement of these therapies into Phase 2 trials.

As of August 1, 2024, no new relapses have been reported since the clinical updates provided at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the European Hematology Association (EHA) (Free EHA Whitepaper) annual meetings.

"Our gamma-delta T cell therapies, engineered with our industry-leading manufacturing technology, continue to demonstrate their potential to eliminate residual cancer cells and to revolutionize cancer treatment," said William Ho, CEO and co-founder of IN8bio. "The safety profile of gamma-delta T cells has been manageable and well-tolerated across both indications with no significant cell therapy-related toxicities reported to date in any patients across these Phase 1 trials."

Program Details as of August 1, 2024:

INB-100 for AML

FDA Guidance on Registrational Program: Following a Type B meeting with the FDA earlier this summer, IN8bio received regulatory guidance on advancing INB-100 for the treatment of AML as a post-transplant maintenance therapy, with relapse-free survival as the primary endpoint. To date, 100% of AML patients treated with INB-100 are in long-term CR, providing a promising path for the registrational trial. IN8bio plans to submit an Investigational New Drug (IND) application to the FDA in Q1 2025. Pending clearance, the Company could initiate a registrational trial for AML in 2025.
100% 1-year Relapse-Free Survival: All patients dosed in the Phase 1 investigator-sponsored trial continue to demonstrate relapse-free survival beyond one year. These patients are mostly classified as high-risk, a category where ~25% would typically be expected to relapse within 100 days post-transplant and up to 50% by one year.
AML Patient Outcomes: 100% of AML patients remain relapse-free after receiving their dose of INB-100. There have been no new relapses reported since the last update with a data cut-off on May 15, 2024. The previously reported patients with other leukemic diagnoses (ALL and MDS/MPN overlap with concurrent TP53 mutations) who relapsed are still alive. The proposed Phase 2 registrational trial will only include patients with AML, a highly aggressive leukemia with high relapse rates, where Phase 1 results to date have shown the most promising long-term responses.
Expansion Cohort: Enrollment in expansion cohort is ongoing, and all treated patients remain in CR, with several having been evaluated for at least 90 days post-transplant and the longest nearing seven months. Full enrollment of the 10-patient expansion cohort is expected by the end of 2024, with long-term follow up results anticipated in 2025.
Gamma-Delta T Cell Persistence: A significant increase in dose-dependent long-term expansion and persistence of circulating gamma-delta T cells continues to be observed up to day 365 post-infusion. This marks the first instance of an allogeneic cellular therapy demonstrating both persistence and expansion over this extended time frame. Cell persistence potentially allows for the gamma-delta T cells to conduct longer immune surveillance to prevent relapse.
INB-200 for GBM

Novel Cellular Therapy Approach: IN8bio’s proprietary drug-resistant immunotherapy (DRI) technology combines standard-of-care chemotherapy with gene-edited, chemotherapy-resistant gamma-delta T cells. Initial data points to a potential dose response across the three cohorts with dose-escalation ranging from a single dose in cohort 1, three doses in cohort 2, and up to six repeat doses in cohort 3. All patients in cohort 1 eventually relapsed. There have been no new relapses with a range of remission from 9.5 to 37.9 months in cohorts 2 and 3 to date. Multiple patients in these higher repeat dose cohorts have now exceeded the overall survival expected with standard-of-care alone relative to historical data.

MGMT-unmethylated GBM patients: Several patients in this group, who are typically poor responders and generally unresponsive to chemotherapy, have remained in remission longer than expected. Notably, one patient who received six doses of INB-200 has been in remission for over a year. Updated clinical data from this trial is expected to be presented in Q4 2024.

INB-400 in Phase 2 trial: This study is investigating six doses of autologous gamma-delta T cells in front-line GBM treatment in combination with standard-of-care. The trial is actively enrolling and treating patients at multiple leading cancer centers across the United States.
Mr. Ho, also commented, "These therapies take advantage of the gamma-delta T cells’ natural ability to target the heterogeneity of cancers, prevent immune escape and disease relapse. Multiple patients have now remained in progression-free remissions longer than expected with many now exceeding expected overall survival, based on historical data. The safety profile and long-term remissions observed with both INB-100 and INB-200, now exceeding three years, across two difficult indications, suggest a significant potential advancement for cellular therapies for cancer. With these compelling results to date, IN8bio stands at the forefront of innovation in oncology and gamma-delta T cell development."

Conference Call Details

IN8bio will host a conference call and webcast today, Monday, August 12, 2024, at 8:30 am ET. The webcast can be accessed by clicking this link and can also be accessed on the Events & Presentations page of the Company’s website. To participate in the live call, please register using this link. It is recommended that participants register at least 15 minutes in advance of the call. Once registered, participants will be informed of the dial-in number and will be provided a unique PIN.

On August 12, 2024 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on the development of first-in-class and best-in-class targeted oncology therapies, reported financial results for the second quarter ended June 30, 2024, and provided a business update (Press release, Immunome, AUG 12, 2024, View Source [SID1234645730]).

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"Immunome is focused on establishing and developing a broad pipeline of differentiated oncology therapies. We continue to advance work required to prepare AL102 for regulatory submissions, and we are moving towards submitting INDs for IM-1021 and IM-3050," said Clay B. Siegall, Ph.D., President and Chief Executive Officer. "These programs are supported by an expanding team of drug developers, including leaders who previously contributed to the successful development and commercialization of ADCs and small molecules."

Dr. Siegall continued, "In addition to our clinical pipeline, we have identified multiple promising ADC targets, all of which offer first-in-class potential. We believe that cost-effective business development efforts combined with focused research can accelerate the expansion of our pipeline and that rigorous science provides the foundation for transformative cancer therapies."

Pipeline Highlights

Full enrollment for the Phase 3 RINGSIDE Part B study of AL102 for the treatment of desmoid tumors was completed in February 2024, and Immunome continues to expect to report topline data for RINGSIDE Part B in the second half of 2025. In parallel, Immunome is performing additional manufacturing and pharmacology work required to support a new drug application filing for AL102.

Immunome also anticipates submitting INDs for IM-1021 and IM-3050 in the first quarter of 2025, as previously disclosed.

These programs are complemented by robust discovery efforts centered on next-generation ADCs and active business development activity, including recent transactions with Atreca, Nectin Therapeutics, Bluefin Biomedicine, and OncoResponse intended to expand Immunome’s ADC toolbox.

Second Quarter 2024 Financial Results

· As of June 30, 2024, cash, cash equivalents and marketable securities totaled $278.4 million. Immunome’s current cash runway is expected to extend into 2026.

· Research and development expenses for the quarter ended June 30, 2024 were $29.1 million, including stock-based compensation costs of $1.0 million.
· In-process research and development expenses for the quarter ended June 30th, 2024 were $6.3 million. These expenses were related to Immunome’s business development activity.
· General and administrative expenses for the quarter ended June 30, 2024 were $7.0 million, including stock-based compensation expense of $2.2 million.
· Immunome reported a net loss of $36.1 million for the quarter ended June 30, 2024.

On August 12, 2024 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), reported its financial results for the second quarter ended June 30, 2024 and provided a business update (Press release, Gossamer Bio, AUG 12, 2024, View Source [SID1234645729]).

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"Between the continued progress made in the enrollment of the Phase 3 PROSERA Study and the transformational collaboration agreement with the Chiesi Group, Gossamer has made great strides in the second quarter to advance the clinical development and strengthen the market potential of seralutinib," said Faheem Hasnain, Co-Founder, CEO, and Chairman of Gossamer Bio.

"Additionally, we look forward to announcing seralutinib presentations and posters at major medical meetings in the coming months, which continue to support the potential of seralutinib as a novel agent in the treatment of patients with PAH and PH-ILD."

Seralutinib (GB002): Inhaled PDGFR, CSF1R and c-KIT Inhibitor for PAH and PH-ILD
•Enrollment is ongoing in the PROSERA Study, a global registrational Phase 3 clinical trial in patients with WHO Functional Class II and III PAH. The primary endpoint is change in six-minute walk distance (6MWD) from baseline at week 24. Topline results from the PROSERA Study are expected in the fourth quarter of 2025.
•In mid-2025, after engaging and discussing with global regulatory authorities, we expect to commence a global registrational Phase 3 clinical trial of seralutinib for the treatment of patients with PH-ILD.
•On May 6, Gossamer Bio and the Chiesi Group announced a development and co-commercialization collaboration for seralutinib. Gossamer and Chiesi will split global development costs for seralutinib, except for the PROSERA Study, for which Gossamer will remain solely responsible. In the United States, Gossamer and Chiesi will split profits and losses, and Chiesi will be solely responsible for commercialization of seralutinib outside of the United States, for which Gossamer will receive mid-to-high teens royalties.

Financial Results for Quarter Ended June 30, 2024
•Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of June 30, 2024, were $354.5 million. The Company expects the combination of current cash, cash equivalents and marketable securities will be sufficient to fund its operating and capital expenditures into the first half of 2027.
•Revenue from Sale of Licenses and from Contracts with Collaborators: For the quarter ended June 30, 2024, revenue from the sale of licenses was $88.8 million and revenue from contracts with collaborators was $7.1 million. Our revenue consists of a one-time development cost reimbursement payment for licenses related to the collaboration with Chiesi and ongoing payments for research and development services related to the collaboration with Chiesi.
•Research and Development (R&D) Expenses: For the quarter ended June 30, 2024, R&D expenses were $35.1 million, compared to $36.3 million for the same period in 2023.
•General and Administrative (G&A) Expenses: For the quarter ended June 30, 2024, G&A expenses were $8.7 million, compared to $10.0 million for the same period in 2023.
•Net Income (Loss): Net income for the quarter ended June 30, 2024, was $49.2 million, or $0.22 basic net income per share, compared to a net loss of $42.5 million, or $0.45 basic net loss per share, for the same period in 2023.