On August 12, 2021 Brickell Biotech, Inc. ("Brickell" or the "Company") (Nasdaq: BBI), a clinical-stage pharmaceutical company focused on developing innovative and differentiated prescription therapeutics for the treatment of debilitating skin diseases, reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Vical, AUG 12, 2021, View Source [SID1234586430]).

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"We have made tremendous progress this year advancing our Phase 3 clinical development program for sofpironium bromide gel, 15% as a potential treatment option for primary axillary hyperhidrosis, or excessive underarm sweating. Since initiating the Phase 3 Cardigan I and II studies in late 2020, we have completed enrollment in both pivotal studies, and the last enrolled hyperhidrosis patient has completed the Cardigan I study," commented Robert Brown, Chief Executive Officer of Brickell. "We remain on track to announce topline data for both studies concurrently in the fourth quarter of 2021, and if these studies are successful, we expect to proceed towards an NDA submission to the U.S. FDA in mid-2022."

Mr. Brown continued, "Our development partner, Kaken Pharmaceutical, continues to ramp up its commercial launch of sofpironium bromide gel, 5% (ECCLOCK) in Japan. We are encouraged by Kaken’s early sales progress, as well as its continued investment in the commercialization of ECCLOCK, disease state awareness and lifecycle management activities. To this point, Kaken has recently initiated a Phase 1 clinical study to explore the pharmacokinetics (PK), safety and efficacy of sofpironium bromide gel in patients with primary palmoplantar hyperhidrosis, or excessive sweating from the palms and soles. We look forward to seeing the results of this study, which will help us determine next development steps, if any, in this new potential indication for sofpironium bromide gel."

Business and Recent Developments

Final patient has completed the Phase 3 Cardigan I study and enrollment completed in the Phase 3 Cardigan II study. Each pivotal clinical study is evaluating sofpironium bromide gel, 15% in approximately 350 subjects with primary axillary hyperhidrosis in the U.S.
Phase 1 clinical study assessing the PK, safety and efficacy of sofpironium bromide gel in patients with primary palmoplantar hyperhidrosis was initiated by Kaken in Japan. 5.3% and 2.8% of the population in Japan are estimated to be affected by primary palmar and plantar hyperhidrosis, respectively1.
Following the recent $8.1 million capital raise, the Company believes it has sufficient cash to fund its operations beyond the potential NDA submission to the U.S. FDA, which is anticipated in mid-2022.
Upcoming Milestones

Final patient expected to complete the Phase 3 Cardigan II study in the third quarter of 2021.
Expect to concurrently report topline results from the U.S. Cardigan I and II studies in the fourth quarter of 2021.
Potential NDA submission to the U.S. FDA anticipated in mid-2022, pending the outcome of the ongoing Phase 3 clinical program.
Kaken to continue ramping up commercialization efforts for ECCLOCK in Japan and evaluating additional hyperhidrosis indications for sofpironium bromide gel.
Financial Results

Second Quarter 2021 Financial Results

The Company reported cash and cash equivalents of $24.4 million as of June 30, 2021, compared to $30.1 million as of December 31, 2020.

Revenue was $0.2 million for the second quarter of 2021 and consisted of royalty revenue recognized from sales of ECCLOCK in Japan by Kaken, which increased from $17 thousand for the first quarter of 2021. Revenue was $0.6 million for the second quarter of 2020, which was driven by collaboration revenue recognized for research and development funding provided by Kaken to Brickell in 2018.

Research and development expenses were $8.8 million for the second quarter of 2021, compared to $2.7 million for the second quarter of 2020. This increase was primarily due to an increase in clinical costs related to the Phase 3 Cardigan studies, which were initiated in the fourth quarter of 2020.

General and administrative expenses were $2.9 million for the second quarter of 2021, compared to $3.0 million for the second quarter of 2020. The decrease was primarily due to lower costs for professional-related fees associated with capital raising activities that occurred in the second quarter of 2020.

Total other income, net was $0.4 million for the second quarter of 2021, compared to $7 thousand for the second quarter of 2020. The increase was primarily due to a gain on extinguishment of debt of approximately $0.4 million that resulted from the forgiveness of the Paycheck Protection Program Loan in June 2021.

Brickell’s net loss was $11.1 million for the second quarter of 2021 compared to $5.1 million for the second quarter of 2020.

Conference Call and Webcast Information

Brickell’s management will host a conference call today at 4:30 p.m. ET to discuss the financial results and recent corporate developments. The dial-in number for the conference call is 1-877-705-6003 for domestic participants and 1-201-493-6725 for international participants, with Conference ID #13720599. A live webcast of the conference call can be accessed at View Source or through the "Investors" tab on the Brickell Biotech website at View Source A replay will be available on this website shortly after conclusion of the event for 90 days.

About Sofpironium Bromide

Sofpironium bromide is Brickell’s lead investigational product candidate and is a new chemical entity that belongs to a class of medications called anticholinergics. Anticholinergics block the action of acetylcholine, a chemical that transmits signals within the nervous system that are responsible for a range of bodily functions, including activation of the sweat glands. Sofpironium bromide was retrometabolically designed. Retrometabolic drugs are intended to exert their action locally and are potentially rapidly metabolized into a less active metabolite once absorbed into the blood. Sofpironium bromide gel, 15% is currently being evaluated in a U.S. pivotal Phase 3 clinical program for the treatment of primary axillary hyperhidrosis, and sofpironium bromide gel, 5% is approved in Japan for the same indication under the brand name ECCLOCK. Sofpironium bromide was discovered at Bodor Laboratories, Inc. by Dr. Nicholas Bodor D.Sc., d.h.c. (multi), HoF, Graduate Research Professor Emeritus, University of Florida.

About Hyperhidrosis

Hyperhidrosis is a debilitating, life-altering medical condition where a person sweats beyond what is physiologically required for thermoregulation of the body. More than 15 million people, or 4.8% of the population of the United States, and 12.76% of the population in Japan, are believed to suffer from hyperhidrosis1,2. Primary axillary (underarm) hyperhidrosis is the targeted first indication for sofpironium bromide and is the most common site of occurrence of hyperhidrosis, affecting an estimated 65% of patients with hyperhidrosis in the United States. Additional information can be found on the International Hyperhidrosis Society website: View Source

On August 12, 2021 Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported financial results for the second quarter ended June 30, 2021 (Press release, Nkarta, AUG 12, 2021, View Source [SID1234586429]).

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"Nkarta continues to set the pace for NK cell therapy as we build on the strengths of our next generation platform and advance our two co-lead clinical programs," said Paul J. Hastings, President and Chief Executive Officer of Nkarta. "During the period, we initiated collaboration activities with CRISPR Therapeutics, added new platform capabilities for rapid innovation, and expanded our manufacturing footprint – all designed to stay ahead of the technology curve and transform the scientific insights of cell therapy into meaningful medicines for cancer patients. Nkarta remains on track to report initial clinical data from our Phase 1 study of NKX101 by the end of this year."

RECENT ACCOMPLISHMENTS AND FUTURE MILESTONES

NKX101

Nkarta aims to present initial clinical data from its ongoing clinical trial of NKX101 by year end 2021. In the Phase 1 study, patients receive multiple doses of NKX101 during a 28-day treatment cycle and are eligible to receive subsequent cycles of treatment upon evidence of tolerability and disease response.
NKX019

Nkarta expects patient dosing in a Phase 1 clinical trial of NKX019 to initiate in the second half of 2021 and has begun manufacturing of clinical supply of NKX019 at its in-house cGMP clinical manufacturing facility in South San Francisco, California.
Manufacturing

Nkarta entered a lease agreement to establish a combined manufacturing facility and company headquarters. The manufacturing facility will produce materials for potential pivotal trials and commercial launch of multiple engineered NK cell therapy products. The expanded manufacturing footprint, centered in South San Francisco, California, builds upon Nkarta’s existing 2,700 square foot cGMP clinical manufacturing facility.
Pipeline and Platform

In May 2021, Nkarta and CRISPR Therapeutics announced a research and development collaboration to co-develop and co-commercialize two chimeric antigen receptor (CAR) NK cell product candidates, one targeting CD70, and one combining NK and T cells (NK+T), each enhanced with genome engineering. The collaboration also gives Nkarta a license to CRISPR/Cas9 gene editing technology for use in its own engineered NK cell therapy products.

Nkarta continues to integrate important scientific insights, processes and breakthroughs into its next generation platform. Platform capabilities include:

Multiplexed CRISPR/Cas9 genome engineering
"Armored" cells with membrane-bound IL-15 for persistence
Enhanced expansion, persistence and activity against tumor microenvironment inhibition via CISH deletion
Cytokine activation using IL-12, -15 and -18 to enhance anti-tumor activity persistence and memory-like properties
No requirement for cytokine support
Multi-dose and multi-cycle clinical trial designs
SECOND QUARTER 2021 FINANCIAL HIGHLIGHTS

Cash and Cash Equivalents: As of June 30, 2021, Nkarta had cash, cash equivalents, restricted cash and short-term investments of $280.3 million.

R&D Expenses: Research and development expenses were $16.0 million for the second quarter of 2021. Non-cash stock-based compensation expense included in R&D expense was $1.7 million for the second quarter of 2021.

G&A Expenses: General and administrative expenses were $5.7 million for the second quarter of 2021. Non-cash stock-based compensation expense included in G&A expense was $1.9 million for the second quarter of 2021.

Net Loss. Net loss was $21.5 million, or $0.66 per basic and diluted share, for the second quarter of 2021.
FINANCIAL GUIDANCE

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into at least the second half of 2023.
About NKX101
NKX101 is an investigational, off-the-shelf cancer immunotherapy that uses natural killer (NK) cells derived from the peripheral blood of healthy donors and engineered with membrane-bound IL15 and 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. By engineering NKX101 with the proprietary NKG2D-based CAR, the ability of NK cells to recognize and kill tumor cells in pre-clinical models is increased significantly compared to non-engineered NK cells. The addition of membrane-bound IL15, a proprietary version of a cytokine for activating NK cell growth, has been shown in pre-clinical models to enhance the proliferation, persistence and sustained activity of NK cells. A multi-center Phase 1 clinical trial of NKX101 in patients with relapsed/refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndromes (MDS) is currently enrolling. Additional information about the clinical trial is available on ClinicalTrials.gov, identifier NCT04623944.

About NKX019
NKX019 is an investigational, off-the-shelf cancer immunotherapy that uses natural killer (NK) cells derived from the peripheral blood of healthy donors and engineered with a CD19-directed chimeric antigen receptor (CAR) and a proprietary, membrane-bound form of interleukin 15 (IL-15). CD19 is a biomarker for normal and malignant B cells, and it is a validated target for B cell cancer therapies. Via its CAR, NKX019 targets and binds to CD19 and eliminates CD19-expressing cells via a robust immune response in preclinical studies. Preclinical models also demonstrate enhanced proliferation, persistence and activity of NK cells with the membrane-bound IL-15, an important cytokine for NK cell survival. Initiation of a Phase 1 clinical trial of NKX019 in patients with relapsed/refractory B cell malignancies in multiple centers in the United States and Australia is planned for the second half of 2021.

About Nkarta’s Platform and Natural Starting Materials
Nkarta’s engineering platform utilizes healthy adult donors as the source for NK cells. By enlisting this natural source of NK cells, Nkarta starts with bona fide NK cells endowed with inherent tumor-recognizing ability and potent cytotoxic function. Healthy donor-derived NK cells are also available in abundance, providing a large quantity of cells with which to begin the efficient two-week manufacturing process. Finally, healthy donor-derived adult cells consist of a diverse repertoire of NK cells, providing Nkarta with the potential to capitalize on the inherent diversity of the innate immune system in selecting donors or NK cell populations with optimal characteristics.

About Nkarta’s NK Cell Technologies
Nkarta has pioneered a novel discovery and development platform for the engineering and efficient production of allogeneic, off-the-shelf natural killer (NK) cell therapy candidates. The approach harnesses the innate ability of NK cells to recognize and kill tumor cells. To enhance the inherent biological activity of NK cells, Nkarta genetically engineers the cells with a targeting receptor designed to recognize and bind to specific proteins on the surface of cancerous cells. This receptor is fused to co-stimulatory and signaling domains to amplify cell signaling and NK cell cytotoxicity. Upon binding the target, NK cells become activated and release cytokines that enhance the immune response and cytotoxic granules that lead to killing of the target cell. All of Nkarta’s NK current cell therapy candidates are also engineered with a membrane-bound IL15, a proprietary version of a cytokine known for activating NK cell growth, to enhance the persistence and activity of the NK cells.

Nkarta’s manufacturing process generates an abundant supply of NK cells that, at commercial scale, is expected to be significantly lower in cost than other current allogeneic and autologous cell therapies. Key to this efficiency is the rapid expansion of donor-derived NK cells using a proprietary NKSTIM cell line, leading to the production of hundreds of individual doses from a single manufacturing run. The platform also features the ability to freeze and store CAR NK cells for an extended period of time and is designed to enable immediate, off-the-shelf administration to patients at the point of care.

On August 12, 2021 Elevation Oncology, Inc. (Nasdaq: ELEV), a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, reported financial results for the quarter ended June 30, 2021 (Press release, Elevation Oncology, AUG 12, 2021, View Source [SID1234586428]).

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"The second quarter marked a pivotal period for Elevation Oncology, with our debut in the public markets raising over $100 million which extends our cash runway into Q2 2023 and positions the Company to execute on our lead program, seribantumab, and build an industry leading precision oncology pipeline," said Shawn M. Leland, PharmD, RPh, Founder and Chief Executive Officer of Elevation Oncology. "Looking ahead, we anticipate completing enrollment of the first 20 patients in Cohort 1 of the tumor-agnostic Phase 2 CRESTONE study of seribantumab for patients with tumors harboring an NRG1 fusion later this year or in early 2022, and presenting the clinical data from the interim analysis in mid-2022 at a major medical conference. Through our recent coloration with Caris Life Sciences, we are identifying oncogenic fusions and driver mutations to enable pipeline expansion opportunities within genomically defined patient populations as part of our commitment to expand the potential of precision medicine."

Recent Business Highlights

Entered into a collaboration with Caris Life Sciences. In June 2021, Elevation Oncology and Caris announced a strategic collaboration to jointly discover and develop therapeutics targeted against oncogenic fusions and driver alterations. The two Companies will leverage genomic data from Caris’s leading WTS and WES molecular diagnostics platform, prioritize targets that are likely to be actionable driver alterations, and jointly discover and develop therapeutics to target them.

Completed a successful initial public offering (IPO). The Company’s common stock commenced trading on The NASDAQ Global Market under the ticker symbol "ELEV" on June 25, 2021. The IPO raised $106.5 million in gross proceeds, before deducting underwriting discounts and commissions and estimated offering expenses.

Strengthened corporate leadership. During the second quarter, the Company appointed Joseph Ferra as Chief Financial Officer, bringing to Elevation two decades of biopharma industry leadership including in investment banking and, most recently, as a public-company CFO. Further, Elevation Oncology appointed Michael Carruthers, an experienced biotech executive, to the Board of Directors and Chair of the Audit Committee.
Clinical Development and Pre-Clinical Data

Opened additional clinical trial sites in CRESTONE. There are now 26 trial sites that are open and enrolling across the US. Through the "just-in-time" clinical site model in partnership with Caris Life Sciences, Tempus, and US Oncology, there are over 400 available sites that can be activated within CRESTONE.

Established additional diagnostic partnerships. With the addition of Genomic Testing Collaborative, PathGroup, and Exactis, there are now a total of nine partnerships in place to support the identification and enrollment of patients with tumors harboring an NRG1 fusion in CRESTONE, including pre-existing partnerships with Ashion Analytics (now Exact Sciences), NeoGenomics, Caris Life Sciences, Strata Oncology, Tempus, and US Oncology.

Presented new preclinical data on additional tumor models harboring an NRG1 fusion. Along with its collaborators in the Marc Ladanyi laboratory at Memorial Sloan Kettering (MSK), the Company presented data at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting 2021. The preclinical data was in pancreatic and cholangiocarcinoma PDX models on the specific inhibition of HER3 with seribantumab to block NRG1 fusion signaling. These results further support the investigation of seribantumab for the treatment of any solid tumor harboring an NRG1 fusion regardless of fusion partner in the ongoing Phase 2 CRESTONE study.

Published a preclinical manuscript on the effect of seribantumab in NRG1 fusion models. A publication in Clinical Cancer Research highlights the specific inhibition of HER3 by seribantumab in preclinical NRG1 fusion in vitro and in vivo PDX models of lung and ovarian cancer. These results showed that seribantumab efficiently inhibited ligand-dependent activation of HER3 by NRG1 fusions, destabilizes the entire ERBB family signaling pathway including the activation of EGFR, HER2, and HER4, and established a predicted biologically effective dose range of seribantumab for tumors driven by an NRG1 fusion that provides confidence in the optimized clinical dose and schedule of 3g weekly being studied in the CRESTONE study.

Published a clinical manuscript. The Phase 1 dose escalation and expansion study for seribantumab monotherapy in patients with advanced solid tumors was published in Investigational New Drugs. The study was designed to evaluate the safety and tolerability of seribantumab monotherapy in patients with any solid tumor, not tumors harboring an NRG1 fusion. Seribantumab monotherapy was well tolerated across all dose levels and a maximum tolerated dose was not reached. Safety and PK data from this study support the 3g weekly dosing of seribantumab in the CRESTONE study which is the first study of seribnatumab in patients with tumors harboring an NRG1 fusion.
Upcoming Milestones

Complete enrollment of the first 20 patients in Cohort 1 of the Phase 2 CRESTONE study and conduct an interim analysis (Q4 2021 – Q1 2022)

Meet with the U.S. Food & Drug Administration to discuss the Phase 2 CRESTONE study (H1 2022)

Present clinical data from CRESTONE interim analysis at a major medical meeting (mid-2022)
Second Quarter 2021 Financial Results

As of June 30, 2021, the Company had cash and cash equivalents totaling $158.0 million, which is expected to fund current operations into the second quarter of 2023.

Research and development expenses for the second quarter 2021 were $3.9 million, compared to $3.0 million for the second quarter 2020. The increase in R&D expense was primarily related to an increase in clinical trial expenses associated with the CRESTONE study.

General and administrative expenses for the second quarter 2021 were $1.1 million, compared to $0.4 million for the second quarter 2020. The increase in G&A expense was primarily related to personnel costs, professional services and consulting, and other administrative costs.

Net loss for the second quarter 2021 was $5.1 million, compared to $3.4 million for the second quarter 2020.

About Seribantumab and NRG1 Gene Fusions

Seribantumab is a fully human IgG2 monoclonal antibody that binds to human epidermal growth factor receptor 3 (HER3). HER3 is traditionally activated through binding of its primary ligand, neuregulin-1 (NRG1). The NRG1 gene fusion is a rare genomic alteration that combines NRG1 with another partner protein to create chimeric NRG1 "fusion proteins". The NRG1 fusion protein is often also able to activate the HER3 pathway, leading to unregulated cell growth and proliferation. Importantly, NRG1 gene fusions are predominantly mutually exclusive with other known genomic driver mutations and are considered a unique oncogenic driver event associated with tumor cell survival.

NRG1 fusions have been identified in a variety of solid tumors, including lung, pancreatic, gallbladder, breast, ovarian, colorectal, neuroendocrine, cholangiocarcinomas, and sarcomas. In preclinical experiments, seribantumab prevented the activation of HER3 signaling in cells that harbor an NRG1 gene fusion and destabilized the entire ERBB family signaling pathway including the activation of HER2, EGFR, and HER4. In addition to extensive nonclinical characterization and testing, seribantumab has been administered to over 800 patients across twelve Phase 1 and 2 studies, both as a monotherapy and in combination with various anti-cancer therapies. Seribantumab is currently being evaluated in the Phase 2 CRESTONE study for patients with solid tumors of any origin that have an NRG1 fusion.

About the CRESTONE Study

Clinical Study of Response to Seribantumab in Tumors with Neuregulin-1 (NRG1) Fusions. CRESTONE is a Phase 2 tumor-agnostic "basket trial" of seribantumab in patients with solid tumors that harbor an NRG1 fusion and have progressed after at least one prior line of standard therapy. The primary objective of the study is to describe the anti-tumor activity and safety of seribantumab as a monotherapy specifically in patients whose solid tumor is uniquely driven by an NRG1 gene fusion. CRESTONE offers a clinical trial opportunity for patients with advanced solid tumors who have not responded or are no longer responding to treatment. Patients are encouraged to talk to their doctor about genomic testing of their tumor. CRESTONE is open and enrolling today in the United States. For more information visit www.NRG1fusion.com.

On August 12, 2021 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 second quarter 2021 financial results and provided a business update (Press release, Checkmate Pharmaceuticals, AUG 12, 2021, View Source [SID1234586427]).

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"We remain excited about the prospects for vidutolimod in melanoma, based upon compelling clinical data to date, as well as our expansion into new tumor types. We are focused squarely on execution of our clinical trials to deliver upon these opportunities, and we anticipate multiple clinical data readouts in 2022," said Barry Labinger, President and Chief Executive Officer of Checkmate.

Second Quarter Business Update

Advancing patient recruitment activities and enrollment across our ongoing clinical trials evaluating vidutolimod, including:
A Phase 2 trial of vidutolimod in combination with nivolumab in anti-PD-1 refractory advanced melanoma, supported by a clinical collaboration with Bristol Myers Squibb.
A randomized Phase 2/3 trial of vidutolimod in combination with nivolumab vs. nivolumab monotherapy in first-line metastatic or unresectable melanoma, also supported by the clinical collaboration with Bristol Myers Squibb.
A Phase 2 trial of vidutolimod in combination with pembrolizumab in recurrent or metastatic squamous cell head and neck cancer. Interim data readouts anticipated beginning 1H 2022 and maturing throughout 2022.
Ongoing start-up activities for the planned expansion of the development program for vidutolimod into cutaneous squamous cell carcinoma and Merkel cell carcinoma, supported by a clinical collaboration with Regeneron to evaluate the combination of vidutolimod and Libtayo (cemiplimab).
Second Quarter 2021 Financial Results

Research and development expenses (R&D): R&D expenses for the three months ending June 30, 2021 were $14.9 million, compared to $6.5 million for the same period in the prior year. This increase reflected a milestone payment of $4.0 million in the second quarter of 2021 triggered by initiation of patient dosing in our refractory melanoma trial, as well as increases in personnel and operating expense for the planning and execution of additional clinical trials with vidutolimod.
General and administration expenses (G&A): G&A expenses for the three months ending June 30, 2021 were $4.1 million, compared to $1.8 million for the same period in the prior year. This increase was primarily attributable to increases in personnel and operating expense incurred in connection with Checkmate operating as a publicly traded company.
Cash, cash equivalents and investments: Cash, cash equivalents and available-for-sale investments were $95.6 million as of June 30, 2021.

On August 12, 2021 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage oncology company developing potentially first-in-class therapeutics that combine both targeted and immune-mediated mechanisms, reported financial results and provided a corporate update for the second quarter ended June 30, 2021 (Press release, Tempest Therapeutics, AUG 12, 2021, View Source [SID1234586426]).

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"The second quarter of 2021 was an exciting period as Tempest became a public company and the team drove progress in all three of our novel programs," said Steve Brady, chief executive officer of Tempest. "We look forward to the planned opening of the TPST-1120 randomized study in first line hepatocellular carcinoma in collaboration with Roche and the first combination study of TPST-1495, and remain focused on delivering potentially value-creating milestones over the next year and beyond."

Recent Highlights

Public Company Transition: successfully closed merger and concurrent PIPE financing, allowing Tempest to become a public company listed on the Nasdaq Capital Market, and extending runway into 2023 through multiple potential catalysts.
TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist): continued enrollment in monotherapy dose optimization towards recommended Phase 2 dose ("RP2D").
TPST-1120 (clinical PPARα antagonist): (i) completed monotherapy dose escalation and selected 600mg BID as RP2D; (ii) observed stable disease ("SD") in 50% of the monotherapy-treated patients, including prolonged SD in patients with refractory cholangiocarcinoma; and (iii) observed a deep, confirmed partial response in a patient with checkpoint inhibitor-refractory fourth line renal cell carcinoma in the combination study with nivolumab (->60% by RECIST 1.1, durable through 4 scans and ongoing).
TREX-1 Inhibitor (preclinical, tumor-selective STING pathway activator): (i) progressed lead series to picomolar IC50 potency in biochemical assays; and (ii) demonstrated significant proof of concept in a mouse tumor model with systemic delivery of a lead series molecule.
Board of Directors: Christine Pellizzari, J.D., Geoff Nichol, M.B., Ch.B., M.B.A., and Ronit Simantov, M.D., joined the Board of Directors, bringing deeper financial, legal, and clinical development expertise to Tempest.
Planned Near-Term Milestones

TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist): (i) selection of monotherapy RP2D expected in the first half of 2022; (ii) commencement of a combination study with an anti-PD-1 checkpoint inhibitor expected prior to the end of 2021; and (iii) commencement of monotherapy expansion in targeted indications and biomarker-selected patient populations expected in the first half of 2022.
TPST-1120 (clinical PPARα antagonist): (i) identification of RP2D of TPST-1120 in combination with nivolumab expected prior to the end of 2021; and (ii) commencement of first line randomized Phase 1b/2 study in hepatocellular carcinoma patients, under a collaboration with F. Hoffman La Roche, expected within the third quarter.
TREX-1 Inhibitor (preclinical tumor-selective STING pathway activator): planned selection of development candidate in the first half of 2022.
Financial Results

Second Quarter

Tempest ended the second quarter of 2021 with $68.5 million in cash and cash equivalents and short-term restricted cash, compared to $18.8 million in December 31, 2020. The increase was primarily due to the merger and concurrent PIPE, which closed in June 2021.
Net loss and net loss per share for the second quarter of 2021 were $7.1 million and $7.63, respectively, compared to $5.2 million and $11.42, respectively, for the second quarter of 2020. The increase was primarily due to an increase in compensation expense and professional fees associated with the merger.
Research and development expenses for the second quarter of 2021 were $4.2 million, compared to $4.1 million for the same period in 2020. The $0.1 million increase was primarily attributable to increased compensation expenses.
For the three months ended June 30, 2021, general and administrative expenses were $2.6 million compared to $1.1 million for the same period in 2020. The increase was primarily due to growth in compensation expense and professional fees associated with the merger.
Year-to-Date

Net cash used in operations for the six months ended June 30, 2021 was $6.2 million.
Net loss and net loss per share for the six months ended June 30, 2021 were $12.4 million and $17.30, respectively, compared to $9.5 million and $21.28, respectively, for the same period in 2020.
Research and development expenses for the six months ended June 30, 2021 were $7.8 million compared to $7.1 million for the same period in 2020. The $0.7 million increase was primarily due to increased compensation expenses and consulting services.
For the six months ended June 30, 2021, general and administrative expenses were $4.1 million compared to $2.4 million for the same period in 2020.