On May 12, 2022 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported its financial results for the three-month period ended March 31, 2022, and other recent developments (Press release, NeuBase Therapeutics, MAY 12, 2022, View Source [SID1234614371]).

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"We are pleased with the progress being made across our development pipeline of therapeutic programs to treat DM1, HD, and KRAS-driven cancers. The new data we’ve announced to date this year have further validated the use of our PATrOL platform to design novel genetic medicines that target and rescue gene dysfunctions, with the potential for clinically impactful outcomes in both rare and common diseases," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. "We continue to execute the development strategy for our DM1 program, which includes a series of IND-enabling studies scheduled to report data throughout CY2022. Last quarter, we presented pharmacodynamic data that illustrated a single intravenous (IV) dose or multiple subcutaneous (SC) doses of our DM1 development candidate resolves the genetic defect and myotonia in skeletal muscle of the gold-standard mouse model of the disease. Building off these results, we plan on announcing at ASGCT (Free ASGCT Whitepaper) additional pharmacokinetic (PK) data, which will illustrate the exposure levels of our development candidate when administered via systemic administration in skeletal muscles, heart, and brain, tissues that are affected in DM1. We expect these data to support further advancement of our lead candidate for DM1 and validate a differentiated whole-body solution for this disease. Considering this progress, we believe the submission of an IND application to the FDA is on track for the fourth quarter of CY2022."

Second Quarter of Fiscal Year 2022 and Recent Operating Highlights

Myotonic Dystrophy Type 1 (DM1) Program: NeuBase is making steady progress advancing IND-enabling studies for its development candidate in the DM1 program, which includes PK, absorption, distribution, metabolism, and excretion (ADME), and bioavailability via IV and SC routes of administration, exploratory and IND-enabling Good Laboratory Practice (GLP) toxicology, and mechanism of action studies. In addition, Good Manufacturing Practice (GMP) of NeuBase’s development candidate to support Phase 1/2 clinical trials has been successfully implemented via contract manufacturing organizations.
In March 2022, the Company presented a robust data package through posters and presentations at the 2022 MDA Clinical & Scientific Conference demonstrating that systemic administration of the Company’s lead DM1 candidate, NT-0231.F, in the HSALR model achieves clinically relevant molecular and functional rescue, including genetic target engagement, displacement of sequestered MBNL1, resolution of nuclear aggregates, rescue of the spliceopathy, and reversal of myotonia (delayed muscle relaxation after contraction). In PK studies of NT-0231.F in wild-type BALB/C mice, a single IV or SC dose showed high volume of distribution, suggesting wide tissue distribution.
The Company announced that two abstracts have been accepted for presentation at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting. The presentations will include new preclinical data on the biodistribution in key tissues of NT-0231.F.
With continued positive results from in vitro and in vivo preclinical studies, the Company expects to file an IND application for NT-0231.F in the fourth quarter of CY2022.
Huntington’s Disease (HD) Program: The HD program is currently in preclinical development. In CY2022, NeuBase expects to present new preclinical data describing the pharmacology of a candidate compound in the brain after systemic administration, nominate a development candidate and initiate scale-up and toxicology activities.
KRAS Oncology Program: The Company is conducting in vitro mechanistic studies and in vivo pharmacology studies for the KRAS program (KRAS G12V and G12D mutations). Existing in vivo data show activity illustrating allele-selective engagement of mutant KRAS at the DNA and RNA levels, with abrogation of downstream hyperactive signaling through multiple RAS pathway members, resulting in anti-tumor activity.
Financial Results for the Second Fiscal Quarter Ended March 31, 2022

As of March 31, 2022, the Company had cash and cash equivalents of approximately $39.0 million, compared with approximately $52.9 million as of September 30, 2021.
For the fiscal quarter ended March 31, 2022, the Company reported a net loss of approximately $9.9 million, or a net loss of $0.30 per share, compared with a net loss of approximately $5.5 million, or a net loss of $0.24 per share, for the same period last year.
For the fiscal quarter ended March 31, 2022, total operating expenses were approximately $9.9 million, consisting of approximately $3.1 million in general and administrative expenses and $6.8 million of research and development expenses. This compares with total operating expenses of approximately $5.9 million for the same period last year, consisting of approximately $2.7 million in general and administrative expenses and $3.2 million in research and development expenses.
Financial Results for the Six-Month Period Ended March 31, 2022

For the six-month period ended March 31, 2022, the Company reported a net loss of approximately $17.7 million, or a net loss of $0.54 per share, compared with a net loss of approximately $9.6 million, or a net loss of $0.41 per share, for the same period last year.
For the six-month period ended March 31, 2022, total operating expenses were approximately $17.2 million, consisting of approximately $6.0 million in general and administrative expenses and $11.2 million of research and development expenses. This compares with total operating expenses of approximately $10.6 million for the same period last year, consisting of approximately $5.4 million in general and administrative expenses and $5.2 million in research and development expenses.

On May 12, 2022 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the first quarter 2022 and provided an operational update (Press release, Bellicum Pharmaceuticals, MAY 12, 2022, View Source [SID1234614370]).

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"I am pleased with the growing momentum in patient recruitment and enrollment after recent COVID-19-related delays at our sites," said Rick Fair, President and Chief Executive Officer. "We remain on track to report results from our ongoing BPX-601 and BPX-603 trials early next year. I appreciate our team’s and investigators’ hard work and commitment to execute to plan in 2022."
Program Highlights and Current Updates

BPX-601 GoCAR-T
•Enrollment in the Phase 1/2 dose escalation clinical trial in patients with previously treated metastatic castration-resistant prostate cancer (mCRPC) is ongoing. The company expects to present a data update on BPX-601 in the first quarter of 2023.

BPX-603 GoCAR-T
•Enrollment is ongoing in the Phase 1/2 clinical trial for BPX-603 in patients with solid tumors that express human epidermal growth factor 2 (HER2), including breast, endometrial, ovarian, gastric, and colorectal cancers. The company expects to present a data update on BPX-603 in the first half of 2023.

Financial Results for the First Quarter 2022

R&D Expenses: Research and development expenses were $4.5 million for the first quarter 2022, compared to $6.5 million for the first quarter 2021. The decrease in R&D expenses for the first quarter 2022 was primarily due to reduced expenses related to rivo-cel activities, a discontinued development program.

G&A Expenses: General and administrative expenses were $1.5 million in the first quarter 2022 compared to $2.0 million for the comparable period in 2021. The decrease in G&A expenses for the first quarter 2022 compared to the first quarter 2021 was primarily due to reduced share-based compensation expense.

Loss from Operations: Bellicum reported a loss from operations of $5.9 million for the first quarter 2022, compared to $8.9 for the comparable period in 2021.

Net Income/Loss: Bellicum reported a net loss of $7.6 million for the first quarter 2022, compared to a net loss of $11.3 million for the first quarter 2021. The results included a loss from the change in fair value of warrant derivative liabilities of $1.6 million for the first quarter of 2022, which was primarily driven by an increase in our stock price over the first quarter of 2022.

Shares Outstanding: As of May 9, 2022, Bellicum had 8,609,661 shares of common stock and 452,000 shares of preferred stock outstanding. Each share of preferred stock is convertible into 10 shares of common stock.

Cash Position and Guidance: Bellicum reported cash and cash equivalents and restricted cash totaling
$41.3 million as of March 31, 2022, compared to $47.7 million as of December 31, 2021.

On May 12, 2022 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company developing the next generation of therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported its financial results for the quarter ended March 31, 2022 and provided a business update (Press release, Phio Pharmaceuticals, MAY 12, 2022, View Source [SID1234614369]).

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"We are pleased enrollment is open for our first-in-human clinical trial for PH-762 at the Gustave Roussy Institute, one of the largest cancer centers in Europe. This study will evaluate the safety, tolerability, pharmacokinetics and checkpoint anti-tumor activity of PH-762 in a neoadjuvant setting in subjects with advanced melanoma. The clinical study will feature a dose escalation of PH-762 with top-line data from the first group of patients expected in the first quarter of 2023," said Dr. Geert Cauwenbergh, Principal Executive Officer of Phio. "In addition, the new preclinical data we recently presented at the AACR (Free AACR Whitepaper) Annual Meeting shows the potential of PH-894 to be used in treating patients who do not respond to anti-PD-1 therapy, or patients who progress after initially responding to treatment with checkpoint inhibitors. The reaction of the investment community when this data was presented, reinforces the relevance of a compound like PH-894, an INTASYL compound specifically targeting BRD4, in patients who may not respond or relapse after PD-1 therapy. We expect to finalize IND-enabling studies for PH-894 in the second half of 2022," concluded Dr. Cauwenbergh.

Quarter in Review and Recent Corporate Updates

Enrollment open for the Company’s Phase 1b clinical study to evaluate the safety, tolerability, pharmacokinetics and anti-tumor activity of PH-762 in a neoadjuvant setting in subjects with advanced melanoma.
Presented new in vivo data at the AACR (Free AACR Whitepaper) Annual Meeting 2022 that provide a strong rationale for the clinical use of PH-894, a BRD4-targeting, self-delivering RNAi, as a monotherapy, as well as in combination with systemic PD-1 therapy.
Continue to develop PH-3861, a dual-targeting INTASYL towards PD-1 and BRD4. Last year this program reported data that show PH-3861 elicited complete cure of tumors in an in vivo hepatoma model and outperformed the efficacy of the small molecule and antibody control treatments toward the same targets. In addition, local INTASYL therapy was shown to induce a systemic anti-tumor response with clearance of untreated distal tumors.
Upcoming Pipeline Milestones

Expect to finalize IND-enabling studies for PH-894 in the second half of 2022.
On track to report top-line data from the first group of patients with advanced melanoma in the clinical trial for PH-762 in the first quarter of 2023.
Additional data publications on the Company’s pipeline programs.
Financial Results

Cash Position

At March 31, 2022, the Company had cash of $20.5 million as compared with $24.1 million at December 31, 2021. The Company expects its current cash will be sufficient to fund currently planned operations to the second quarter of 2023.

Research and Development Expenses

Research and development expenses were approximately $1.6 million for the quarter ended March 31, 2022, compared with approximately $2.4 million for the quarter ended March 31, 2021. The decrease was primarily due to the preclinical studies and manufacturing costs to support the Company’s clinical trial with PH-762, which were completed in the prior year period, offset by increases in CRO costs in preparation for the start of the Company’s clinical trial with PH-762 and personnel-related expenses due to an increase in headcount as compared to the prior year period.

General and Administrative Expenses

General and administrative expenses were approximately $1.1 million for the quarter ended March 31, 2022, compared with approximately $1.2 million for the quarter ended March 31, 2021. The decrease was primarily due to decreases in legal and patent fees offset by an increase in stock-based compensation expense.

Net Loss

Net loss was $2.6 million, or $0.19 per share, for the quarter ended March 31, 2022, compared with $3.4 million, or $0.32 per share, for the quarter ended March 31, 2021. The decrease in net loss was primarily attributable to the decrease in research and development expenses as described above.

On May 12, 2022 Checkmate Pharmaceuticals, Inc. (Nasdaq: CMPI) ("Checkmate"), a clinical stage biopharmaceutical company focused on developing its proprietary technology to harness the power of the immune system to combat cancer, reported first quarter 2022 financial results and provided a business update (Press release, Checkmate Pharmaceuticals, MAY 12, 2022, View Source [SID1234614368]).

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"We remain focused on the execution of the clinical program for vidutolimod and are delighted with the opportunity to become part of Regeneron, who will help to accelerate the development of vidutolimod as a potential novel treatment for multiple tumor types," said Alan Bash, President and Chief Executive Officer of Checkmate. "This is an exciting opportunity for all stakeholders as we bring this innovative medicine forward and work to make a meaningful difference for patients with difficult to treat cancers."

Recent Business Updates

On April 19, 2022, Regeneron Pharmaceuticals, Inc. and Checkmate Pharmaceuticals, Inc. announced a definitive agreement for the acquisition of Checkmate by Regeneron at an all-cash price of $10.50 per share of Checkmate common stock. The proposed acquisition values Checkmate at a total equity value of approximately $250 million. On May 2, 2022, Scandinavian Acquisition Sub, Inc., a wholly owned subsidiary of Regeneron, commenced a tender offer for all of the outstanding shares of Checkmate common stock. The transaction is expected to close in mid-2022.
First Quarter 2022 Financial Results

Research and development expenses (R&D): R&D expenses for the first quarter of 2022 were $11.6 million, compared to $10.4 million for the same period in the prior year. This increase was primarily driven by higher third-party contract research organization, internal personnel and consulting costs related to our ongoing clinical trials. First quarter 2021 expenses included the impact of a $2.0 million milestone payment to Kuros in March 2021, which became payable upon initiating dosing of the first patient in a Phase 2 clinical trial. There was no corresponding expense in Q1 2022.
General and administration expenses (G&A): G&A expenses for the first quarter of 2022 were $4.2 million, compared to $3.8 million for the same period in the prior year. This increase was primarily attributable to professional fees incurred in Q1 2022 in connection with the proposed acquisition by Regeneron and recruiting costs associated with hiring a chief executive officer in March 2022.
Cash, cash equivalents and investments: Cash, cash equivalents and available-for-sale investments were $60.1 million as of March 31, 2022.

On May 12, 2022 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the first quarter ended March 31, 2022 and recent corporate highlights (Press release, Checkpoint Therapeutics, MAY 12, 2022, View Source [SID1234614367]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "The positive topline results generated in the first quarter from our ongoing registrational trial of cosibelimab in metastatic cutaneous squamous cell carcinoma ("cSCC") laid the foundation for a potentially transformational year for our lead immunotherapy product candidate and for our company as a whole. We remain on track to submit our U.S. Biologics License Application ("BLA") for cosibelimab later this year and continue to evaluate partnership opportunities for the potential commercialization of cosibelimab in Europe and other key territories worldwide." Mr. Oliviero continued, "Our focus remains on advancing our pipeline of potentially life-saving novel oncology therapies with the goal of expanding patient access globally through a disruptive pricing strategy, beginning with the $30 billion and growing PD-(L)1 market."

Recent Corporate Highlights:

In January 2022, Checkpoint announced positive topline results from the ongoing registration-enabling clinical trial evaluating the safety and efficacy of its anti-PD-L1 antibody, cosibelimab, administered as a fixed dose of 800 mg every two weeks in patients with metastatic cSCC. The study met its primary endpoint, with cosibelimab demonstrating a confirmed objective response rate of 47.4% (95% CI: 36.0, 59.1) based on independent central review of 78 patients enrolled in the metastatic cSCC cohort using Response Evaluation Criteria in Solid Tumors version 1.1 criteria. Checkpoint intends to submit a BLA for cosibelimab in late 2022, followed by a Marketing Authorization Application submission in Europe and other territories worldwide. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, Checkpoint believes cosibelimab has the potential to be a market disruptive product in the $30 billion and growing PD-(L)1 class.
In April 2022, Checkpoint announced that the results of its pivotal trial of cosibelimab in cSCC were selected for poster presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held at McCormick Place, in Chicago, June 3-7, 2022.
Financial Results:

Cash Position: As of March 31, 2022, Checkpoint’s cash and cash equivalents totaled $41.5 million, compared to $54.7 million at December 31, 2021, a decrease of $13.2 million.
R&D Expenses: Research and development expenses for the first quarter of 2022 were $14.7 million, compared to $4.2 million for the first quarter of 2021, an increase of $10.5 million. Research and development expenses for the first quarters of 2022 and 2021 each included $0.2 million of non-cash stock expenses.
G&A Expenses: General and administrative expenses for the first quarter of 2022 were $2.2 million, compared to $2.4 million for the first quarter of 2021, a decrease of $0.2 million. General and administrative expenses for the first quarter of 2022 included $0.7 million of non-cash stock expenses, compared to $1.2 million for the first quarter of 2021.
Net Loss: Net loss attributable to common stockholders for the first quarter of 2022 was $16.8 million, or $0.20 per share, compared to a net loss of $6.5 million, or $0.09 per share, in the first quarter of 2021. Net loss for the first quarter of 2022 included $0.9 million of non-cash stock expenses, compared to $1.4 million for the first quarter of 2021.