On May 9, 2024 MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on discovering, developing, manufacturing and commercializing innovative antibody-based therapeutics for the treatment of cancer, reported an update on its recent corporate progress and announced financial results for the quarter ended March 31, 2024 (Press release, MacroGenics, MAY 9, 2024, View Source [SID1234643002]).

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"We are very encouraged by the interim updated safety and preliminary efficacy data from the TAMARACK study of vobra duo in metastatic castration-resistant prostate cancer," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "We believe this interim data set helps validate our previously stated hypothesis that improved tolerability coupled with compelling biological activity could be achieved through dose reductions and a longer dosing interval. We believe vobra duo’s biological activity shown to date aligns well with the parameters we outlined at the outset of the study. Based on our evaluation of the interim data to date, we have initiated planning activities for a potential Phase 3 study that could commence next year. We anticipate sharing final safety, efficacy and durability data, including radiographic progression-free survival data, which is the primary endpoint of the study, in the second half of 2024. Furthermore, having preliminarily identified suitable vobra duo doses in mCRPC in the TAMARACK study, we have greater confidence in the molecule’s potential to help patients with a broad range of B7-H3-expressing cancers."

"The interim safety and anti-tumor activity observed to date in the TAMARACK study look very promising for patients with metastatic castration-resistant prostate cancer," said Johann DeBono, Regius Professor of Cancer Research and Professor in Experimental Cancer Medicine at The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust. "With the limited treatment options currently available to these patients, this novel ADC molecule could potentially become the first therapy targeting B7-H3 in patients with prostate cancer and would represent an important new treatment for this population."

Updates on Proprietary Investigational Programs

Recent progress and anticipated events related to MacroGenics’ investigational product candidates are highlighted below.

B7-H3-Directed Therapies

• Vobramitamab duocarmazine (vobra duo) is an antibody-drug conjugate (ADC) that targets B7-H3, an antigen with broad expression across multiple solid tumors and a member of the B7 family of molecules involved in immune regulation.

• MacroGenics completed enrollment of the TAMARACK Phase 2 study of vobra duo in November 2023. TAMARACK is being conducted in patients with metastatic castration-resistant prostate cancer (mCRPC) who were previously treated with one prior androgen receptor axis-targeted therapy (ARAT). Participants may have received up to one prior taxane-containing regimen, but no other chemotherapy agents. The TAMARACK study is designed to evaluate vobra duo at two different doses: 2.0 mg/kg or 2.7 mg/kg every four weeks (q4W).

• A new presentation of TAMARACK interim data, consisting of updated safety and preliminary efficacy data, all based on a data cut-off date of April 12, 2024, is available under "Events & Presentations" in the Investor Relations section of MacroGenics’ website or directly via this link. Below is a high-level summary of this interim data, which is subject to further updates:

Vobra Duo
2.0 mg/kg q4W Vobra Duo
2.7 mg/kg q4W
Patients Enrolled n=91 n=90
PSA Reduction Summary:
PSA Evaluable Patients n=82 n=71
Any PSA Reduction ≥50% 41 (50.0%) 36 (50.7%)
Confirmed PSA Reduction ≥50% 36 (43.9%) 26 (36.6%)
Tumor Response Summary:
RECIST Evaluable Patients with Measurable Disease at Baseline n=45 n=32
Disease Control Rate (CR+PR+SD) 41 (91.1%) 28 (87.5%)
Overall Response Rate (CR+PR, confirmed only) 8 (17.8%) 8 (25.0%)
Overall Response Rate (CR+PR, including unconfirmed) 11 (24.4%) 14 (43.8%)
Safety Summary:
Safety Population n=90 n=86
Treatment-Emergent Adverse Events All Grade 89 (98.9%) 86 (100.0%)
Treatment- Emergent Adverse Events Grade ≥3 49 (54.4%) 44 (51.2%)
TEAE Leading to Study Drug Discontinuation 10 (11.1%) 13 (15.1%)
TEAE Leading to Study Drug Dose Reduction 39 (43.3%) 44 (51.2%)
TEAE Leading to Study Drug Dose Interruption 38 (42.2%) 48 (55.8%)
Five Most Common TEAE All Grade Asthenia (46.7%)
Nausea (35.6%)
Oedema peripheral (32.2%) Decreased appetite (28.9%) Fatigue (25.6%) Asthenia (58.1%)
Decreased appetite (37.2%) Oedema peripheral (36.0%) Nausea (30.2%)
Pleural effusion (29.1%)
Pleural Effusions Grade 1=8.9%
Grade 2=8.9%
No Grade ≥ 3 event Grade 1=14.0%
Grade 2=14.0%
Grade 3=1.2%
Palmar-plantar Erythrodysaesthesia Syndrome Grade 1=11.1%
Grade 2=4.4%
No Grade ≥ 3 event Grade 1=12.8%
Grade 2=9.3%
Grade 3=1.2%

• The median number of cycles of vobra duo administered was five (range of 1-10).

• A total of five events with fatal outcome occurred as follows: one Grade 5 event in the 2.0 mg/kg dosing cohort: acute myocardial infarction (considered unrelated to study drug by the investigator); three Grade 5 events in the 2.7 mg/kg dosing cohort: one cardiac arrest (considered unrelated to study drug by the investigator) and two events of pneumonitis. In addition, a patient in the 2.7 mg/kg dosing cohort had a Grade 3 pleural effusion that is recorded as having a fatal outcome. The latter three deaths are being investigated, as follow-up is incomplete on this ongoing trial.

• Additional data is provided in the presentation on the Company’s website and as filed with the Securities and Exchange Commission.

• Based on a current evaluation of this interim data, the Company is undertaking the initial steps necessary to prepare for the potential initiation of a Phase 3 study in mCRPC in 2025. The final decision to pursue such a Phase 3 study will be based on an analysis of the final data set, including rPFS, when available.

• The Company intends to share final safety, efficacy, and durability data, including the primary endpoint of radiographic progression-free survival, from the TAMARACK trial in the second half of 2024.

• MacroGenics plans to expand the TAMARACK study of vobra duo by enrolling patients with non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC), melanoma, squamous cell carcinoma of the head and neck (SCCHN) and anal cancer. The Company expects to initiate dosing in these additional cohorts in mid-2024.

• MacroGenics continues to enroll a Phase 1/2 dose escalation study of vobra duo in combination with lorigerlimab in patients with various advanced solid tumors. The Company anticipates commencing a dose expansion study of this combination in mCRPC and at least one additional indication in 2024.

• MGC026 is a clinical B7-H3-targeting ADC that is site-specifically conjugated to exatecan, a topoisomerase I inhibitor payload developed by Synaffix (a Lonza company). With distinct mechanisms of action, vobra duo and MGC026 may address different cancers, tumor stages, or be used in combination with alternate agents — or potentially with one another — to enhance their clinical utility. A Phase 1 dose escalation study of MGC026 in patients with advanced solid tumors is ongoing.

MGC026 preclinical data was presented recently at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. In preclinical studies, MGC026 was shown to have greater potency than B7-H3-directed antibodies conjugated to deruxtecan, or DXd, a topoisomerase-based payload utilized in other ADCs. In addition, the MGC026 payload has been shown to be less susceptible to multi-drug resistance (MDR) mechanisms than DXd and SN-38.

• Enoblituzumab is an Fc-optimized monoclonal antibody that targets B7-H3. The HEAT study, an investigator-sponsored, randomized Phase 2 clinical trial being conducted by MacroGenics’ academic collaborators, is ongoing. This study is being conducted to evaluate the activity of neoadjuvant enoblituzumab given prior to radical prostatectomy in up to 219 men with high-risk localized prostate cancer.

Lorigerlimab

• Lorigerlimab is a bispecific, tetravalent PD-1 × CTLA-4 DART molecule. In addition to the ongoing study of lorigerlimab in combination with vobra duo mentioned above, MacroGenics is enrolling LORIKEET, a randomized Phase 2 study of lorigerlimab in combination with docetaxel vs. docetaxel alone in second-line, chemotherapy-naïve mCRPC patients. A total of 150 patients are planned to be treated in the 2:1 randomized study. The current trial design includes a primary study endpoint of radiographic progression-free survival (rPFS). The Company anticipates completing enrollment of the study in 2024 and providing a clinical update in the first half of 2025.

Emerging ADC Pipeline

• MGC028 is a preclinical ADC incorporating an ADAM9-targeting antibody and represents the second MacroGenics ADC molecule that incorporates Synaffix’s novel site-specific linker and topoisomerase I inhibitor-based cytotoxic payload. ADAM9 (a disintegrin and metalloprotease domain 9) is a member of the ADAM family of multifunctional type 1 transmembrane proteins that play a role in tumorigenesis and cancer progression and is overexpressed in multiple cancers, making it an attractive target for cancer treatment. The Company currently anticipates submitting an investigational new drug (IND) application for MGC028 by the end of 2024.

MGC028 preclinical data was presented recently at the AACR (Free AACR Whitepaper) Annual Meeting. In preclinical studies, MGC028 demonstrated specific antitumor activity in in vivo models representing gastric, lung, pancreatic, colorectal cancer, SCCHN and cholangiocarcinoma. In addition, in a non-human primate study, MGC028 was well tolerated at high dose levels, with mild, reversible side effects and no ocular toxicity, which is often a concern with tubulin-inhibitor-based ADCs. These promising preclinical results support the continued investigation of MGC028 as a therapeutic option for treating ADAM9-expressing solid cancers.

Partnered Program

• MGD024 is a next-generation, humanized CD123 × CD3 DART molecule designed to minimize cytokine-release syndrome, while maintaining anti-tumor cytolytic activity, and permitting intermittent dosing. MacroGenics continues to enroll patients in a Phase 1 dose-escalation study of MGD024 in patients with CD123-positive neoplasms, including acute myeloid leukemia and myelodysplastic syndromes. Under an October 2022 exclusive option and collaboration agreement, Gilead Sciences, Inc. has the option to license MGD024 at predefined decision points during the Phase 1 study.

First Quarter 2024 Financial Results

• Cash Position: Cash, cash equivalents and marketable securities balance as of March 31, 2024, was $184.2 million, compared to $229.8 million as of December 31, 2023.

• Revenue: Total revenue was $9.1 million for the quarter ended March 31, 2024, compared to total revenue of $24.5 million for the quarter ended March 31, 2023. The decrease was primarily due to a decrease in revenue from collaborative and other agreements, including a $15.0 million milestone received from Incyte in the quarter ended March 31, 2023.

• R&D Expenses: Research and development expenses were $46.0 million for the quarter ended March 31, 2024, compared to $45.9 million for the quarter ended March 31, 2023.

• SG&A Expenses: Selling, general and administrative expenses were $14.7 million for the quarter ended March 31, 2024, compared to $13.5 million for the quarter ended March 31, 2023. The increase was primarily related to increased stock-based compensation expense and other professional fees.

• Net Loss: Net loss was $52.2 million for the quarter ended March 31, 2024, compared to net loss of $38.0 million for the quarter ended March 31, 2023.

• Shares Outstanding: Shares of common stock outstanding as of March 31, 2024 were 62,560,502.

• Cash Runway Guidance: MacroGenics anticipates that its cash, cash equivalents and marketable securities balance of $184.2 million as of March 31, 2024, in addition to projected and anticipated future payments from partners and product revenues should extend its cash runway into 2026. The Company’s expected funding requirements reflect anticipated expenditures related to the Phase 2 TAMARACK clinical trial, the Phase 2 LORIKEET study as well as MacroGenics’ other ongoing clinical and preclinical studies.

Conference Call Information

To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call.

The listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of MacroGenics’ website at View Source A recorded replay of the webcast will be available shortly after the conclusion of the call and archived on MacroGenics’ website for 30 days following the call.

On May 9, 2024 LIXTE Biotechnology Holdings, Inc. ("LIXTE" or the "Company") (Nasdaq: LIXT and LIXTW), a clinical stage pharmaceutical company, reported it is co-sponsoring an international scientific workshop on "Therapeutic Over-Activation in Cancer" at Harvard University’s Dana Farber Cancer Institute on May 9 and 10, 2024 in Boston, Massachusetts (Press release, Lixte Biotechnology, MAY 9, 2024, View Source [SID1234643001]).

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This workshop will bring together leading experts from the pharmaceutical industry and academia to discuss a radically different approach to cancer therapy that is being spearheaded by LIXTE’s research team at the Netherlands Cancer Institute. This unconventional approach is based on the deliberate over-activation of oncogenic signaling in cancer cells as a therapeutic strategy using LIXTE’s lead compound, LB-100.

LIXTE is the only company that has a drug in clinical trials with demonstrated capacity to over-activate oncogenic signaling. The results obtained with LB-100 were recently posted online in a paper titled "Paradoxical Activation of Oncogenic Signaling as a Cancer Treatment Strategy" in the scientific journal Cancer Discovery, which will be published in the journal’s July 2024 issue. This study showed that LB-100 triggers hyper-activation of the signals that are responsible for the deregulated proliferation of cancer cells, thus leading to cell death. This approach is the opposite of most of the current generation of cancer therapies and opens potentially new treatment strategies. LIXTE currently has three ongoing clinical trials utilizing LB-100 to enhance various cancer therapies.

Professor René Bernards, Ph.D., of the Netherlands Cancer Institute (NKI), a leader in the field of molecular carcinogenesis and a member of LIXTE’s Board of Directors, is co-leading the workshop. The NKI presentation will focus on findings of a recent pre-clinical study showing that LIXTE’s lead compound, LB-100, can force cancer cells to give up their cancer-causing properties.

LIXTE’s lead compound, LB-100, is part of a pioneering effort in an entirely new field of cancer biology – activation lethality – that is advancing a new treatment paradigm.

On May 9, 2024 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported its first quarter 2024 financial and operating results and will host a conference call at 4:30 p.m. Eastern Time to discuss these results and to provide a business update (Press release, Lineage Cell Therapeutics, MAY 9, 2024, View Source [SID1234643000]).

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"The quarter was highlighted by significant milestones and data updates on our lead program," stated Brian M. Culley, Lineage CEO. "A key area of attention for investors is our partnership with Roche and Genentech, and we are pleased to announce a new services agreement which reflects an additional commitment by Genentech for the benefit of the OpRegen program. We believe this agreement will enable our partners to take advantage of our cell transplant expertise to more fully investigate the promising potential of the OpRegen program and do so in a cost-effective manner. We also are planning to bring our second cell transplant program, OPC1, into the clinic this year for a condition with growing awareness of its unmet need and commercial opportunity. Lastly, we continue to build value through the advancement of our early-stage pipeline, which can help create value by capitalizing on the continued validation of our cell transplant approach."

Recent Operational Highlights

RG6501 (OpRegen)
Established new services agreement with Genentech, a member of the Roche Group, to support ongoing development of OpRegen. Under this new agreement, Lineage will provide additional clinical, technical, training and manufacturing services that further support the ongoing advancement and optimization of the OpRegen program. These additional services will be fully funded by Genentech and include: (i) activities to support the ongoing Phase 1/2a study and currently-enrolling Phase 2a study; and (ii) additional technical training and materials related to Lineage’s cell therapy technology platform to support commercial manufacturing strategies.
Continued execution under our collaboration with Roche and Genentech across multiple functional areas, including support for the ongoing Phase 2a clinical study in patients with GA secondary to AMD.
Positive clinical data from long-term follow-up of patients from the Phase 1/2a clinical study of OpRegen presented by David Telander, MD, PhD, Retinal Consultants Medical Group, at the 2024 Retinal Cell & Gene Therapy Innovation Summit.
Mean BCVA gain of 5.5 letters at 24 months in Cohort 4 patients (less advanced geographic atrophy)
Mean BCVA gains greater among patients with improvement in outer retinal structure (+7.4 letters)
Maintenance or increases in external limiting membrane (ELM) and retinal pigment epithelium (RPE) layers at 24 months observed in patients with extensive coverage of OpRegen across the areas of GA
Data suggests OpRegen may counteract RPE cell dysfunction and cell loss secondary to geographic atrophy by providing support to remaining retinal cells, with multi-year effects observed following a single administration
Preclinical results from a surgical development study of OpRegen presented by Rachel N. Andrews, DVM, PhD, DACVP, Genentech, a member of the Roche Group, at 2024 Association for Research in Vision and Ophthalmology Annual Meeting (2024 ARVO).
OPC1
DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) clinical study for the treatment of subacute and chronic spinal cord patient start-up activities underway.
Received an Education Conference II Grant from the California Institute for Regenerative Medicine (CIRM), to support the 2nd Annual Spinal Cord Injury Investor Symposium, hosted in partnership with the Christopher & Dana Reeve Foundation, to be held on June 26 and 27, 2024 at the Sanford Consortium for Regenerative Medicine in La Jolla, CA.
Corporate Updates
Appointed veteran industry executive Dr. Charlotte Hubbert as Vice President of Corporate Development. Dr. Hubbert previously served as Partner and Head of Gates Foundation Venture Capital, an initiative at the Bill and Melinda Gates Foundation Strategic Investment Fund, and most recently served in the leadership team at NanoString Technologies. She currently serves on the Board of Directors of the Beckman Research Institute at the City of Hope and is a Strategic Director at Madrona Venture Group.
Balance Sheet Highlights

Cash, cash equivalents, and marketable securities of $43.6 million as of March 31, 2024, is expected to support planned operations into Q3 2025.

First Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from collaboration revenues and royalties. Total revenues for the three months ended March 31, 2024 were approximately $1.4 million, a net decrease of $1.0 million as compared to $2.4 million for the same period in 2023. The decrease was primarily driven by lower collaboration and licensing revenue recognized from deferred revenues under the collaboration and license agreement with Roche.

Operating Expenses: Operating expenses are primarily comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended March 31, 2024 were $8.1 million, a decrease of $0.9 million as compared to $9.0 million for the same period in 2023.

R&D Expenses: R&D expenses for the three months ended March 31, 2024 were $3.0 million, a net decrease of $1.2 million as compared to $4.2 million for the same period in 2023. The net decrease was primarily driven by $0.4 million for our OPC1 program, $0.3 million for our preclinical programs, and $0.2 million for our OpRegen program. Another $0.3 million of the decrease was attributable to other research and development expenses, primarily related to reduced manufacturing activities.

G&A Expenses: G&A expenses for the three months ended March 31, 2024 were $5.0 million, a net increase of $0.3 million as compared to approximately $4.7 million for the same period in 2023. The increase was primarily driven by $0.2 million in stock-based compensation expenses, and an overall increase in costs incurred for consulting services.

Loss from Operations: Loss from operations for the three months ended March 31, 2024 were $6.7 million, an increase of $0.1 million as compared to $6.6 million for the same period in 2023.

Other Income/(Expenses), Net: Other income (expenses), net for the three months ended March 31, 2024 was comprised of other income of $0.1 million, compared to other income of $0.4 million for the same period in 2023. The net decrease was primarily driven by the employee retention credit recognized in the prior year, partially offset by exchange rate fluctuations related to Lineage’s international subsidiaries.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended March 31, 2024 was $6.5 million, or $0.04 per share (basic and diluted), compared to a net loss attributable to Lineage of $4.4 million, or $0.03 per share (basic and diluted), for the same period in 2023.

Conference Call and Webcast

Interested parties may access the conference call on May 9th, 2024, by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through May 15th, 2024, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 1330332.

On May 9, 2024 Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence ("AI") company developing targeted and transformative cancer therapies using its proprietary RADR AI and machine learning ("ML") platform with multiple clinical-stage drug programs, reported operational highlights and financial results for the first quarter 2024, ended March 31, 2024 (Press release, Lantern Pharma, MAY 9, 2024, View Source [SID1234642999]).

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"Our company made meaningful progress across multiple clinical trials and in furthering our AI platform this past quarter while advancing our internal capabilities to both support data-driven, precision oncology trials and accelerate the cost-effective development of drug-conjugates. Our team is at the forefront of demonstrating how combining emerging AI technologies, cancer biology and biomarker expertise along with focused clinical operations holds the promise of transforming timelines and costs in biopharma development." said Panna Sharma, President and CEO of Lantern Pharma.

Sharma continued, "Over the past several months, we have experienced growing interest in machine-learning enabled drug development and, in our RADR, AI platform. Our team is energized by the growing desire to adopt and leverage AI-driven innovations in biopharma and in the meaningful progress we are making with our own drug-candidates. We are excited about the opportunities we have in front of us to drive increased collaborations on our AI platform this year, and also propel the streamlined development of our own portfolio of high-value, high-impact drug-candidates."

Highlights of AI-Powered Pipeline:

LP-184 – Five cohorts of patients, comprised of dose levels 1 thru 5, have been enrolled and dosed – in escalating doses – in the ongoing Phase 1A clinical trial. This is a first-in-human Phase 1 trial across multiple solid tumor indications that are advanced and refractory to existing standard-of-care therapies. The trial is actively enrolling and dosing patients at dose level 6 that have relapsed/refractory advanced solid tumors, such as pancreatic cancer, glioblastoma (GBM), lung, triple-negative breast cancer, and multiple other solid tumor types. There have not been any observed dose-limiting toxicities to date.

The company believes that enrollment should be complete this summer and on-track for a readout of data in late summer or early fall. Current efforts are underway to focus enrollment efforts on cancer patients with tumors that have DDR(1) (DNA damage repair) deficiency. DDR deficient tumors have been observed to have higher sensitivity to LP-184. The company has also submitted a dose optimization and expansion protocol (Supplement A) to the FDA related to LP-184 in non-CNS solid tumors, including TNBC (triple negative breast cancer) with DDR alterations. Additionally, the company in collaboration with Starlight Therapeutics, has also submitted to the FDA a dose optimization and expansion protocol in recurrent IDH wild-type high grade gliomas (Supplement B).

The dosage and safety data obtained in the Phase 1a and 1b trials are expected to be used to advance the central nervous system (CNS) indications for a future Phase 2 trial to be sponsored by Lantern’s wholly owned subsidiary, Starlight Therapeutics. The Phase 1a data will also inform other anticipated later phase trials in select solid tumors, most likely with genomic signatures signifying DDR (DNA damage repair) deficiency, that have shown responsiveness to LP-184. Genomic identification of these patients and biomarker characterization of their underlying tumor is central to our focus of personalizing treatment and developing efficient later stage clinical trials. To further this effort, Lantern has also initiated the development of a PCR-based molecular diagnostic test that may help in identifying cancer patients with the best likelihood of response and benefit from treatment with LP-184.

AI and preclinical studies are ongoing to further refine drug combination studies supporting the use of LP-184 to improve the durability or overall response rates in combination with FDA approved drugs that are widely used in cancer treatment. Globally, the aggregate annual market potential of LP-184’s target indications is estimated to be approximately $12+ billion, consisting of $4.5+ billion for CNS cancers and $7.5+ billion for solid tumors.

LP-284 – The initial two cohorts of patients have been dosed, and no dose-limiting toxicities have been observed in the Phase 1a clinical trial. The company expects to open additional sites in the US throughout the second quarter with the potential to advance to Phase 1b/2 by the close of 2024. LP-284 has shown nanomolar potency across multiple published in vitro and in vivo studies, including mantle cell lymphoma (MCL), double hit lymphoma (DHL), and other advanced NHL cancer subtypes and certain sarcomas with DDR deficiencies, notably those with compromised functioning of the ataxia-telangiectasia mutated (ATM) gene due to mutations or deletions. Nearly all MCL, DHL, and HGBL patients relapse from the current standard-of-care agents and there is an urgent and unmet need for novel improved therapeutic options for these patients. In the US and Europe, MCL, DHL, and HGBLs are diagnosed in 16,000-20,000 patients each year and have an estimated annual market potential of over USD 3+ billion.

LP-300 – The phase 2 Harmonic clinical trial sites in the US are continuing to screen for patients and have also increased the pace of enrollment. This past quarter we also received approval to proceed with the Phase 2 clinical trial in Japan and Taiwan. This is expected to accelerate the collection of patient and response data needed for the next-stage of evaluation and development of LP-300, an investigational therapeutic for the treatment of relapsed and inoperable primary adenocarcinoma of the lung given in combination with chemotherapy Additionally, it may also bring a needed therapeutic option for LCINS (Lung Cancer In Never Smokers) diagnosed patients in Japan and Taiwan, where one-third of all lung cancer diagnoses are made among those who have never smoked.

Dr. Yashushi Goto, a physician and researcher focused on lung cancer at the National Cancer Center of Japan, will be leading the phase 2 trial in Japan, where the incidence of non-small cell lung cancer (NSCLC) in never-smokers is double or more than that of the United States. Lantern believes that this improves the positioning for drug-candidate LP-300 to develop collaborative and co-development partnerships with global biopharma companies with a primary focus in serving the Asian markets.

The Harmonic trial is assessing the effect of LP-300 in combination with standard-of-care chemotherapy (carboplatin and pemetrexed) in LCINS patients with relapsed NSCLC. Globally, LCINS patients are a growing population of patients and do not respond well to PD-1/PD-L1-based therapies or the available chemotherapy doublets, leaving them with reduced treatment options. In the US it is estimated that LP-300 has an annual market potential of $1.5 billion, and a global estimated annual market potential of over $2.6 billion. LCINS is the eighth leading cause of cancer-related mortality in the USA and the fifth most common cause of cancer-related deaths worldwide.

RADR Platform Growth and Development:

RADR continues to advance in size, scope, and capabilities and is progressing towards becoming a standard for AI-driven drug development in oncology – for both early-stage development and later-stage patient biomarker and combination therapy identification. The company recently announced a artificial intelligence (AI)-driven collaboration to optimize the development of a protein disulfide isomerase (PDI) inhibitor drug candidate, XCE853, for a variety of novel and targeted cancer indications. The collaboration is leveraging RADR’s AI-based capabilities, including 200+ machine learning (ML) algorithms and foundational models for oncology drug development to uncover biomarkers and molecular correlates of efficacy and define potential combination regimens to sharpen and accelerate XCE853’s drug development strategy. Lantern Pharma is receiving equal IP co-ownership and drug development rights in newly discovered biomarkers, novel indications, and use for new pharmacological strategies for XCE853.

The scope of RADR’s data has broadened with a strategic focus on additional classes of compounds, including drug-conjugates such as ADCs and inclusion of detailed data on chemical and biochemical features and drug-interaction data. Additionally, data from clinical studies such as those being obtained from liquid biopsy, and data from preclinical combination studies that aim to define drug interaction and optimal dosage are being incorporated into the datapoints and data sets powering RADR. Lantern expects to pursue additional biopharma and technology partnerships during 2024 to further advance and commercialize the RADR AI platform.

Starlight Therapeutics:

Starlight Therapeutics, a wholly owned subsidiary of Lantern Pharma focused on CNS and brain cancers with STAR-001, continues advancements with the filing of a clinical trial protocol for the Phase 1B dose optimization and expansion cohort in recurrent IDH wild-type high grade gliomas. IDH wild-type glioblastomas are the most malignant glial tumors with median survival of only 14–16 months after diagnosis; patients aged ≥ 65 years have reportedly worse outcomes.(3) Lantern formed a wholly-owned subsidiary, Starlight Therapeutics Inc. ("Starlight"), in early 2023 for the clinical development of drug candidate LP-184’s central nervous system (CNS) and brain cancer indications – including GBM, brain mets., and several rare pediatric CNS cancers. Starlight will refer to the molecule LP-184, as it is developed in CNS indications, as "STAR-001". The indications and mechanistic insights powering the creation of Starlight and the identification of multiple CNS tumors that can be potentially impacted were largely driven by insights and analysis from the RADR AI platform.

During Q4 of 2023 Lantern announced that it had hired a CMO, Dr. Marc Chamberlain, who will focus on Starlight’s clinical trials, development of personnel to execute on the planned clinical trials and overall support in corporate development activity. Starlight and Lantern expect to initiate Phase 1b/2 clinical trials during the second half of 2024. The market potential for the currently planned indications for Starlight’s synthetically-lethal, cancer-cell DNA damaging agent – STAR-001 – is estimated to be 4.5 billion to 5+ billion USD across both adult and pediatric primary and secondary CNS cancers.

ADC & Drug Conjugate Programs:

During the first quarter, Lantern, in collaboration with academic research partners in Germany, advanced the development, synthesis, and preclinical proof-of-concept of a novel, highly potent, cryptophycin-based ADC (cpADC). The cpADC has shown picomolar potency in a wide range of solid tumors tested in preclinical development and is being further evaluated for clinical potential in six solid tumor indications. In preclinical work, the cpADC produced an 80% cancer cell kill rate which was more than other commonly used approved ADCs, including in a cancer sub-type, medium and low HER-2 expression cancers, which is an area of critical patient need. Lantern expects to move towards IND development of its ADC program during 2024 with a focus on select solid tumors that are unresponsive or refractory to current therapies.

Additionally, Lantern has advanced its AI module for differentiated, machine-learning based ADC development, characterization, analysis and bioactivity prediction. The ADC module is being developed as an extension to RADR and leverages the data and biomarker insights curated by and generated in RADR. Lantern has plans to further advance the development through partnerships and collaborations with both technology and biopharma companies.

Additional Operational Highlights:

A new publication on the lethal activity of LP-184, inducing elevated levels of DNA double-strand breaks, in HR deficient (HRD) cancer cells was published by research scientists at Lantern Pharma in collaboration with Georgetown University Medical Center in Cancer Research Communications. The publication showcased that depletion of key HR components BRCA2 or ataxia telangiectasia mutated (ATM) in cancer cells conferred up to 12-fold increased sensitivity to LP- 184 and that LP-184 showed nanomolar potency in a diverse range of HRD cancer models. A link to the publication titled "LP-184, a Novel Acylfulvene Molecule, Exhibits Anticancer Activity against Diverse Solid Tumors with Homologous Recombination Deficiency" can be accessed here.

New data and scientific findings for LP-284 and the ongoing clinical trial were presented at AACR (Free AACR Whitepaper) (American Association for Cancer Research) during the 2024 Annual Meeting in San Diego – Phase 1a/1b clinical trial of LP-284, a highly potent TP53 mutation agnostic DNA damaging agent, in patients with refractory or relapsed lymphomas and solid tumors (NCT06132503)

The company also announced during April that it will hosting a series of educational and informative webinars focused on updates on Lantern’s areas of research, clinical trials and AI efforts titled Webinar Wednesdays. The first of these webinars was held on April 24th and featured Dr. Joseph Treat, a Professor in the Department of Hematology and Oncology, Vice Chair of Education, and Medical Director of Ambulatory Care at Fox Chase Cancer Center discussing LP-300 and the Harmonic clinical trial focused on LCINS patients.

First Quarter 2024 Financial Highlights

Balance Sheet: Cash, cash equivalents, and marketable securities were approximately $38.4 million as of March 31, 2024, compared to approximately $41.3 million as of December 31, 2023. The quarterly cash burn rate continues to reflect our capital-efficient, collaborator-centered business model.

R&D Expenses: Research and development expenses were approximately $4.3 million for the quarter ended March 31, 2024, compared to approximately $2.6 million for the quarter ended March 31, 2023. This increase was largely driven by an increase in clinical trial activity and clinical trial site activations.

G&A Expenses: General and administrative expenses were approximately $1.5 million for the quarter ended March 31, 2024, compared to approximately $1.7 million for the quarter ended March 31, 2023.

Net Loss: Net loss was approximately $5.4 million (or $0.51 per share) for the quarter ended March 31, 2024, compared to a net loss of approximately $3.9 million (or $0.36 per share) for the quarter ended March 31, 2023.

Warrant Exercises: Lantern issued 20,132 shares of common stock during Q1 2024, relating to the cashless exercise of warrants to purchase 79,021 shares. Also, in Q1 2024, Lantern issued 17,481 shares of common stock for aggregate proceeds of approximately $55,000, relating to the exercise of warrants for cash. Following these exercises, there remain 81,496 warrants outstanding to purchase Lantern common stock at a weighted average exercise price of $16.55 per share.

Earnings Call and Webinar Details:

Lantern will host its 1st quarter 2024 earnings call and webinar today, May 9, 2024, at 4:30 p.m. ET. A link to register can be accessed at: Lantern 1st Quarter 2024 Earnings Call & Webinar Link

Related presentation materials will be accessible at: View Source
A replay of the 1st quarter 2024 earnings call and webinar will be available at: View Source

On May 9, 2024 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to developing small molecule therapeutics that address cancers and other diseases driven by deregulated transcription, reported recent business progress and financial results for the first-quarter of 2024 (Press release, Kronos Bio, MAY 9, 2024, View Source [SID1234642998]).

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"We have continued to make good progress towards our goals. We are looking forward to sharing a clinical update on the KB-0742 data to date at ASCO (Free ASCO Whitepaper) in June," said Nobert Bischofberger, Ph.D., president and chief executive officer of Kronos Bio, Inc. "We expect to complete the IND-enabling studies for KB-9558 by the end of 2024 and expect to dose our first patients in the first half of 2025. Our collaboration with Genentech and our internal discovery programs continue to advance and we look forward to sharing our progress later this year."

Recent Company News
•At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2024, the Company presented three posters, including the first public presentation of data from its p300 program. The posters include:
◦p300 catalytic inhibition selectively targets IRF4 oncogenic activity in multiple myeloma (link to poster)
◦A dose escalation and cohort expansion study of the CDK9 inhibitor KB-0742 in relapsed, refractory and transcriptionally addicted solid tumors (link to poster)
◦KB-0742, an oral highly selective CDK9 inhibitor, demonstrates preclinical activity in transcription factor fusion driven adenoid cystic carcinoma patient-derived models (link to poster)

•The Company announced that they will be presenting updated study data from KB-0742-1001, the ongoing Phase 1/2 trial of KB-0742 at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper):
Title: Study update of the oral CDK9 inhibitor KB-0742 in relapsed or refractory transcriptionally addicted advanced solid tumors
Presenter: Brian A. Van Tine, M.D., Ph.D., Washington University in St. Louis
Abstract ID#: 3102
Poster Session: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Location: Hall A, McCormick Place, Chicago, Illinois
Poster Board #: 247
Date and Time: Saturday, June 1, 2024, from 9:00 a.m. to 12:00 p.m. CDT

First Quarter 2024 Financial Highlights

•Cash, Cash Equivalents and Investments: With its ongoing and currently planned clinical programs and $152.0 million in cash, cash equivalents and investments as of March 31, 2024, the Company anticipates sufficient resources to fund its planned operations into the second half of 2026.

•R&D Expenses: Research and development expenses were $14.2 million for the first quarter of 2024, which includes non-cash stock-based compensation expense of $1.2 million.

•G&A Expenses: General and administrative expenses were $7.5 million for the first quarter of 2024, which includes non-cash stock-based compensation expense of $2.2 million.

•Impairment of Long-lived Assets and Restructuring: The Company incurred impairment of long-lived assets expense of $6.6 million for the first quarter of 2024. The Company also incurred restructuring expense of $6.2 million for the first quarter of 2024, which includes non-cash stock-based compensation of $4.4 million.

•Net Loss: Net loss for the first quarter of 2024 was $30.0 million, or $0.50 per share, including non-cash stock-based compensation expense of $7.7 million.