On August 10, 2023 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported a business update and provided second quarter 2023 financial results (Press release, Oncternal Therapeutics, AUG 10, 2023, View Source [SID1234634206]).

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"Oncternal is executing on its strategy to deliver significant value inflection points for our two clinical programs. The clearance of the IND for ONCT-534, our dual-action androgen receptor inhibitor, represents a major milestone for Oncternal and a new potential option for patients suffering from mCRPC. We have received support from top prostate cancer experts in the United States and Europe. We reiterate our guidance to dose the first patient later this year, with initial clinical data in the first half of 2024. Resistance to standard of care androgen receptor signaling inhibitors such as enzalutamide and abiraterone represents a significant unmet medical need. Pre-clinical studies have shown that ONCT-534 may directly address the most common resistance mechanisms, such as androgen receptor ligand-binding domain mutations and AR-V7 splice variants," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "The dosing of the first patient with ONCT-808, our ROR1 targeting autologous CAR T therapy, represents a significant milestone for patients suffering from aggressive B-cell lymphomas. We look forward to a data update later this year in this area of significant unmet medical need."

Recent Highlights

In August 2023, we received a ‘Study May Proceed’ letter from the U.S. Food and Drug Administration (FDA), less than 30 days after submitting our Investigational New Drug (IND) application for ONCT-534, our novel dual-action androgen receptor inhibitor (DAARI) to support a Phase 1/2 clinical trial in patients with metastatic castration-resistant prostate cancer (mCRPC) who are resistant to currently available androgen receptor (AR) inhibitor drugs such as enzalutamide and abiraterone
In June 2023, we announced that the first patient has been dosed in the Phase 1/2 dose escalation/dose expansion study of ONCT-808, our ROR1 targeting autologous CAR T cell therapy (NCT05588440)
Expected Upcoming Milestones

ONCT-808, our autologous ROR1-targeted CAR T cell therapy
Initial clinical data available by the end of 2023
Additional clinical readouts in 2024
ONCT-534, our dual-action androgen receptor inhibitor
Phase 1/2 clinical study initiation in the third quarter of 2023
Initial clinical data available in the first half of 2024

Second Quarter 2023 Financial Results

Our grant revenue was $0.1 million for the second quarter ended June 30, 2023. Our total operating expenses for the second quarter ended June 30, 2023 were $9.7 million, including $1.7 million in non-cash stock-based compensation expense. Research and development expenses for the quarter totaled $6.6 million, and general and administrative expenses for the quarter totaled $3.1 million. Net loss for the first quarter was $9.0 million, or a loss of $0.15 per share, basic and diluted. As of June 30, 2023, we had approximately 58.7 million shares of common stock outstanding, $45.5 million in cash, cash equivalents and short-term investments and no debt. We believe these funds will be sufficient to fund our operations into 2025.

On August 10, 2023 Onconova Therapeutics, Inc. (NASDAQ: ONTX), ("Onconova" or "the Company"), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported second quarter 2023 financial results and provided an update on recent pipeline progress (Press release, Onconova, AUG 10, 2023, View Source [SID1234634205]). Management plans to host a conference call and live webcast at 4:30 p.m. ET today to discuss these results.

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"We are very encouraged about the recent progress that the Onconova team has made for our two lead programs, narazaciclib, a differentiated multikinase CDK4/6 inhibitor targeting proteins involved in resistance pathways , and rigosertib, a cell signaling inhibitor, over the last few months, while effectively managing our financial resources. In addition, we are pleased that Victor Moyo, M.D., a highly experienced and successful clinical researcher and drug developer, has agreed to join the Company as Consulting Chief Medical Officer. We look forward to sharing several important updates in the coming months," said Steve Fruchtman, M.D., President and Chief Executive Officer.

Dr. Fruchtman continued, "For narazaciclib, our efforts have been dedicated to completing a Phase 1 program and defining a recommended Phase 2 dose to support evaluation of narazaciclib in a randomized trial. Onconova believes this CDK4/6 compound has the potential to provide differentiated efficacy based on targeting proteins that have been implicated in resistance mechanisms and the potential for an improved safety profile. We are pleased to see target engagement based on an assay measuring proliferation. We expect to report the results from our Phase 1 monotherapy and Phase 1/2 combination study with letrozole in Q4 2023. The readout will include safety, pharmacokinetics and the definition of a recommended Phase 2 dose."

Dr. Fruchtman concluded, "For rigosertib, we continue to believe this rigosertib’s unique action on cell signaling pathways, including K-RAS and PLK-1, combined with an acceptable safety profile, could position it as an attractive anti-cancer agent. In June, we had a constructive Type B meeting with the FDA for the use of rigosertib monotherapy in the lead, ultra-rare indication of RDEB-associated squamous cell carcinoma. Based on that meeting and the impressive clinical responses in previously refractory patients we have seen and presented at major medical meetings, we plan to design a registrational trial and will look to provide an update on next steps in H1 2024. In the meantime, we continue to support two investigator sponsored studies for rigosertib, underway in melanoma and KRAS mutated non-small cell lung cancer which includes any KRAS mutation that may be present."

Second Quarter Financial Results

Cash and cash equivalents as of June 30, 2023, were $29.7 million, compared to $38.8 million as of December 31, 2022. The Company believes that its cash and cash equivalents will be sufficient to fund ongoing clinical trials and business into the second quarter of 2024.

Research and development expenses were $2.5 million for the second quarter of 2023, compared with $2.0 million for the second quarter of 2022.

General and administrative expenses were $2.2 million for the second quarter of 2023, compared with $2.1 million for the second quarter of 2022.

Net loss for the second quarter of 2023 was $4.3 million, or $0.20 per share on 21.0 million weighted shares outstanding, compared with a net loss of $4.0 million, or $0.19 per share for the second quarter of 2022 on 20.9 million weighted shares outstanding.

Conference Call and Webcast Information

Interested parties who wish to participate in the conference call may do so by dialing:

· (800) 715-9871 for domestic and
· (646) 307-1963 for international callers and
· Using conference ID 9506701

Those interested in listening to the conference call via the internet may do so by visiting the investors and media page on the Company’s website at www.onconova.com and clicking on the webcast link. In addition to the live webcast, a replay will be available on the Onconova website for 90 days following the call.

On August 10, 2023 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported pipeline progress and highlighted second quarter 2023 financial results (Press release, Nuvalent, AUG 10, 2023, View Source [SID1234634204]).

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"As we enter the second half of 2023, we are well positioned to continue advancing our pipeline of novel kinase inhibitors," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "The focused efforts of our team in the first half of 2023 have laid the foundation for a number of meaningful development milestones, including the planned reporting of preliminary Phase 1 data at a medical meeting in the fourth quarter of the year from the dose-escalation portion of our ALKOVE-1 Phase 1/2 trial of NVL-655. Additionally, progress continues towards the selection of a recommended Phase 2 dose and transition to Phase 2 in our ARROS-1 Phase 1/2 trial of NVL-520, and the submission of a new IND application for NVL-330 for the treatment of patients with HER2 exon 20 insertion-positive cancers. I’m incredibly proud of what this team has accomplished to date and look forward to providing further updates later on this year."

Pipeline Highlights and Key Anticipated Milestones

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Preliminary Data from ALKOVE-1 Phase 1/2 Trial of NVL-655 Anticipated at a Medical Meeting in the Fourth Quarter of 2023: NVL-655 is a brain-penetrant ALK-selective inhibitor designed with the aim to address the clinical challenges of emergent treatment resistance, central nervous system (CNS)-related adverse events, and brain metastases that may limit the use of first-, second-, and third-generation ALK inhibitors. Our ALKOVE-1 clinical trial is a first-in-human Phase 1/2, multicenter, open-label, dose-escalation and expansion study evaluating NVL-655 as an oral monotherapy in patients with advanced ALK-positive NSCLC and other solid tumors. The Phase 1 dose-escalation portion of the study is enrolling patients with previously treated ALK-positive solid tumors and will evaluate the overall safety and tolerability of NVL-655. Additional objectives include determination of the recommended Phase 2 dose (RP2D), characterization of the pharmacokinetic profile, and evaluation of preliminary anti-tumor activity. The company anticipates reporting preliminary dose-escalation data from this trial at a medical meeting in the fourth quarter of 2023.
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ARROS-1 Phase 1/2 Trial of NVL-520 Progressing Toward Selection of RP2D: NVL-520 is a novel ROS1-selective inhibitor designed with the aim to address the clinical challenges of emergent treatment resistance, CNS-related adverse events, and brain metastases that may limit the use of currently available ROS1 inhibitors. Our ARROS-1 clinical trial is a first-in-human Phase 1/2, multicenter, open-label, dose-escalation and expansion study evaluating NVL-520 as an oral monotherapy in patients with advanced ROS1-positive NSCLC and other solid tumors. Enrollment of patients with previously treated ROS1-positive solid tumors is ongoing in the Phase 1 portion of ARROS-1 and the company is advancing toward the selection of an RP2D.
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NVL-330 Continuing to Advance through IND-enabling Studies: NVL-330, is a brain-penetrant HER2-selective inhibitor designed with the aim to address the combined medical need of treating tumors driven by HER2 mutations occurring through deletions, insertions, or duplications (collectively, known as HER2 Exon 20 Insertions, or HER2ex20), treating brain metastases, and avoiding treatment-limiting adverse events including due to off-target inhibition of wild-type EGFR. NVL-330 is advancing through IND-enabling studies.

Second Quarter 2023 Financial Results

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Cash Position & Operating Runway: Cash, cash equivalents and marketable securities were $431.2 million as of June 30, 2023. Nuvalent believes the existing cash, cash equivalents and marketable securities are expected to be sufficient to fund its current operating plan into the second half of 2025.
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R&D Expenses: Research and development (R&D) expenses were $25.9 million for the second quarter of 2023.
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G&A Expenses: General and administrative (G&A) expenses were $8.1 million for the second quarter of 2023.
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Net Loss: Net loss for the second quarter of 2023 was $29.1 million, or $0.51 per share.

On August 10, 2023 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies, reported financial results for the second quarter ended June 30, 2023 (Press release, Nkarta, AUG 10, 2023, View Source [SID1234634203]).

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"Nkarta remains well positioned to advance allogeneic cell therapy and lead the development of groundbreaking natural killer cell therapy candidates," said Paul J. Hastings, President and CEO of Nkarta. "We continue to learn, improve and explore potential opportunities for our best-in-class platform. In our June 2023 update on NKX101, we reported complete responses in unusually high-risk and heavily pre-treated patients with AML who received a disease-adapted lymphodepletion regimen. In our other co-lead program, NKX019, we continue to evaluate multiple strategies to treat patients with the most aggressive forms of lymphoma. We look forward to providing further updates as we advance both candidates."

Pipeline Updates
NKX101

In June 2023, Nkarta reported updated clinical data from its Phase 1 clinical trial evaluating NKX101 in patients with relapsed or refractory acute myeloid leukemia (AML).
As of data cut-off on June 10, 2023, in patients that received NKX101 after Flu/Ara-C lymphodepletion (LD), 4 of 6 achieved CR/CRi (67% CR/CRi rate) and 3 of 6 achieved a complete response with hematologic recovery (50% CR rate). Two of the 4 reported CR/CRi were MRD (measurable residual disease) negative.
As of data cut-off on June 10, 2023, in patients that received the highest doses of NKX101 (3 weekly doses at 1 billion or 1.5 billion cells per dose) after fludarabine/cyclophosphamide (Flu/Cy) LD, 4 of 18 achieved CR/CRi (22% CR/CRi rate) and 3 of 18 achieved a complete response with hematologic recovery CR (17% CR rate). There were no CRs at the lower doses of NKX101.
NKX101 was well tolerated across dose-levels and LD regimens. There were no dose-limiting toxicities observed across all cohorts.
Flu/Ara-C LD is expected to be the basis of NKX101 development moving forward.
As previously announced, Nkarta plans to present an update on the ongoing clinical trial of NKX101 in the first half of 2024. The update is expected to include additional patients treated with NKX101 at 1.5 billion cells/dose x 3 dose regimen following Flu/Ara-C LD, as well as longer-term follow up of patients who were in response as of the June 2023 data cut-off.
NKX019

In June 2023, Nkarta presented preliminary clinical data based on a November 2022 data cut-off from its Phase 1 dose escalation clinical trial of NKX019 in patients with relapsed or refractory non-Hodgkin lymphoma (NHL) at two scientific meetings: an oral presentation at the annual meeting of the European Hematology Association (EHA) (Free EHA Whitepaper) and an encore poster presentation at the International Conference on Malignant Lymphoma (17-ICML).
Nkarta is evaluating the potential for clinical development of NKX019 in non-malignant, B-cell mediated disease.
As previously announced, Nkarta plans to present updated results from its ongoing clinical trial of NKX019 in the second half of 2023. The update is expected to include patients enrolled in multiple dose expansion cohorts, as well as longer-term follow-up of patients who were in response as of the November 2022 data cut-off.
Other Corporate Highlights

In July 2023, Nkarta announced the appointment of Alyssa Levin, CPA, CA, as Chief Financial and Business Officer. Ms. Levin will be responsible for leading Nkarta’s corporate finance, business development, information technology, and human resources functions.
Second Quarter 2023 and Recent Financial Highlights

As of June 30, 2023, Nkarta had cash, cash equivalents, restricted cash, and investments of $302.2 million.
Research and development (R&D) expenses were $25.1 million for the second quarter of 2023. Non-cash stock-based compensation expense included in R&D expense was $2.1 million for the second quarter of 2023.
General and administrative (G&A) expenses were $11.7 million for the second quarter of 2023. Non-cash stock-based compensation expense included in G&A expense was $2.5 million for the second quarter of 2023.
Net loss was $33.3 million, or $0.68 per basic and diluted share, for the second quarter of 2023. This net loss includes non-cash charges of $9.2 million that consisted primarily of share-based compensation of $4.6 million and an impairment charge of $4.1 million against right-of-use assets that Nkarta plans to sublease.
Financial Guidance

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into 2025.
About NKX101
NKX101 is an allogeneic, cryopreserved, off-the-shelf cancer immunotherapy candidate that uses natural killer (NK) cells derived from the peripheral blood of healthy adult donors. It is engineered with a chimeric antigen receptor (CAR) targeting NKG2D ligands on tumor cells. NKG2D, a key activating receptor found on naturally occurring NK cells, induces a cell-killing immune response through the detection of stress ligands that are widely expressed on cancer cells. NKX101 is also engineered with a proprietary membrane-bound form of interleukin-15 (IL-15) for greater persistence and activity without exogenous cytokine support. To learn more about the NKX101 clinical trial in adults with AML, please visit ClinicalTrials.gov.

About NKX019
NKX019 is an allogeneic, cryopreserved, off-the-shelf cancer immunotherapy candidate that uses natural killer (NK) cells derived from the peripheral blood of healthy adult donors. It is engineered with a humanized CD19-directed chimeric antigen receptor (CAR) for enhanced tumor cell targeting and a proprietary, membrane-bound form of interleukin-15 (IL-15) for greater persistence and activity without exogenous cytokine support. CD19 is a biomarker for normal and malignant B cells, and it is a validated target for B cell cancer therapies. To learn more about the NKX019 clinical trial in adults with advanced B cell malignancies, please visit ClinicalTrials.gov.

On August 10, 2023 NexImmune, Inc. (Nasdaq: NEXI), a biotechnology company developing a novel approach to immunotherapy designed to orchestrate a targeted immune response by directing the function of antigen-specific T cells in oncology, autoimmune and infectious diseases, reported financial results for the second quarter of 2023 (Press release, NexImmune, AUG 10, 2023, View Source [SID1234634202]).

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"Our previously announced strategic prioritization of our injectable modality (AIM INJ) underscores our commitment to delivering on the significant potential of our artificial antigen-presenting cell (aAPC) nanoparticles to drive antigen-specific T cell function beyond cell therapy approaches and oncology. In our clinical trials with our adoptive cell therapy, we have demonstrated the ability to expand AML tumor-specific T cells with combined memory and effector functions and have observed clinical responses. Furthermore, our collaborations have generated preclinical data that highlights the potential to drive response in solid tumor indications, as well as non-malignant immunology diseases such as MS and SLE." said Kristi Jones, NexImmune’s CEO.

"Our cells exhibit potential superior potency and durability in preclinical models, combining a low dose of AIM multi-tumor expanded T cells with low doses of T-cell engaging bispecific agents, and showing synergistic tumor killing. We believe that these data support the importance of combining a "fit" multi-tumor specific T cell approach with other T cell immuno-oncology mechanisms and represent a unique opportunity to transform treatment paradigms, especially in solid tumors."

"We remain excited about advancing the injectable programs to the clinic and we are encouraged by initial dialogue with the FDA. We believe that the ability to bypass the host dendritic cell and directly engage T cells in the body provides the potential for greater specific T cell responses. Preclinical data to date has been consistent across modalities in the ability to selectively activate and expand important tumor specific T cells with cancer cell killing capacity. In autoimmune disease models, our injectable nanoparticles have been shown to selectively reduce and suppress the disease-causing T cells that reside in the lymph node and target organs in diseases such as T1D. Several manufacturing components have already been produced and process transfer plans are underway. We also continue to advance our Class II approach designed to address diseases like rheumatoid arthritis (RA) and celiac disease."
"We remain confident in the potential therapeutic benefit of our AIM platform-based product candidates and their ability to significantly impact the emerging, rapidly moving field of antigen specific immunotherapies and novel combinations and look forward to providing updates in the future."

Select Second Quarter 2023 Clinical and Business Highlights
Clinical and Preclinical Updates
AIM INJ, an injectable "Off-the-shelf" Antigen-Specific Immunotherapy, and Other Preclinical Research
•Initiated multiple preclinical studies to evaluate NEXI-100 as a monotherapy and in combination with other cancer immunotherapies to support the Company’s oncology program.
•2023 FOCIS Annual Meeting poster presentation reported AIM nanoparticle expansion of EBV-specific T-cell from healthy donors and patients with MS revealed a functional defect in the EBV response of MS patients to select antigen peptide targets, which may be a contributing factor in the pathogenesis of MS as well as other EBV related non-malignant diseases such as SLE.
•In partnership with Yale University Professor Kevan Herold and the Juvenile Diabetes Research Fund (JDRF), evaluation of AIM injectable nanoparticles in type 1 diabetes (T1D) confirmed their ability to drive T cell function in vivo as evidenced by a significant delay in the onset of spontaneous T1D that is associated with a reduction in T1D inducing memory T cells in pancreatic lymph nodes and the pancreas. Future experiments will be conducted to test the addition of suppressive and apoptotic 2nd signals to the AIM nanoparticles in addition to therapeutic combination approaches with anti-CD3 agents.
•Continued to advance neo-antigen our melanoma research collaboration with NYU Langone’s Perlmutter Cancer Center.
•Continued to work in other areas of autoimmune diseases, including vitiligo, MS, HTLV-1-associated myelopathy and others.
NEXI-001 Relapsed Refractory AML Post Allo-HSCT
•A marked increase in antigen-specific cells was observed with increasing dose levels, consistent with a dose response observation. The persistence of antigen-specific T-cells with phenotypes associated with anti-tumor effect and immunologic memory was consistently observed in blood and bone marrow.
•Phase 1 data reported at ASCO (Free ASCO Whitepaper) 2023 shows NEXI-001 is well tolerated with a favorable safety profile. An immunologic and clinical dose responses were observed. Since ASCO (Free ASCO Whitepaper), the clinical response observed in the Cohort 3 patient with poor prognosis extramedullary disease who received the highest cell dose tested (200M x 3, Cycle 1 only) has now been maintained up to nine months. As of the most recent follow-up, the patient remains asymptomatic with no evidence of disease and continues to be monitored.
Manufacturing and Regulatory:
•Transitioning the IND engine capability: Established ACT cell therapy and nanoparticle platform manufacturing to enable IND engine in blood and solid tumor indications. The Company is proactively transitioning this approach to the injectable program.
•Preparing to manufacture clinical material for the planned injectable IND: Completed clinical protein production, selected polymer vendor and the transfer activities to our CDMO are underway to support the planned submission of an IND for NEXI-101 (injectable) in 2H2024.
Select Second Quarter 2023 Financial Highlights
Cash and cash equivalents for the Company as of June 30, 2023 were $16.3 million compared to $34.6 million at December 31, 2022. Based upon current operating plans, NexImmune expects that its existing cash and cash equivalents will enable the Company to fund its operating and capital expenditure requirements into the fourth quarter of 2023.

Research and development expenses were $4.9 million in the second quarter of 2023, compared to $11.8 million for the same period in the prior year. The decrease of $6.9 million was due primarily to the completion of preclinical manufacturing work, pausing of clinical trials, and reduction in personnel-related expenses resulting from terminations.

General and administrative expenses were $2.9 million, compared to $4.1 million for the same period in the prior year. The decrease was primarily due to decreases in legal and other administrative fees expenses and in personnel-related expenses.

Net loss, according to U.S. generally accepted accounting principles in the, or GAAP, was $7.6 million for the quarter, or a basic and diluted GAAP net loss per share of $0.29. This compares to a net loss of $15.9 million, or a basic and diluted GAAP net loss per share of $0.69, for the same period in the prior year.