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Viracta Therapeutics Reports Second Quarter 2021 Financial Results and Provides Clinical and Corporate Updates

On August 12, 2021 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported financial results for the second quarter of 2021 and provided clinical and corporate updates (Press release, Sunesis, AUG 12, 2021, View Source [SID1234586438]).

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"We entered the second half of the year with strong momentum thanks to the completion of key milestones across our pipeline," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta. "We believe Viracta is strongly-positioned to advance our novel therapy for the treatment of EBV-positive cancers forward, with our pivotal NAVAL-1 trial now open for enrollment and the recent clearance of our IND for EBV-positive solid tumors paving the way for a multicenter Phase 1b/2 trial. These two trials are targeting patient populations with a significant unmet medical need, and our expansion into solid tumors could meaningfully broaden our addressable patient market."

Second Quarter 2021 and Recent Highlights

Clinical

NAVAL-1, a global pivotal trial in Epstein-Barr virus (EBV)-positive (EBV+) relapsed/refractory (R/R) lymphoma, now open for enrollment. NAVAL-1 (Nanatinostat in Combination with Valganciclovir) is a global, multicenter, open-label Phase 2 basket trial. The trial, which will include multiple subtypes of R/R EBV-positive (EBV+) lymphoma patients, is designed to evaluate the anti-tumor activity of the combination treatment of nanatinostat with valganciclovir and is anticipated to enroll up to approximately 140 patients. The primary endpoint of the trial is objective tumor response rate as assessed by an independent review committee. If successful, Viracta believes this trial could support multiple new drug application (NDA) filings across various EBV+ lymphoma subtypes.
Announced FDA clearance of IND application for a Phase 1b/2 trial in EBV+ solid tumors. The multicenter Phase 1b/2 trial is designed to evaluate the safety and preliminary efficacy of Viracta’s all-oral combination regimen in patients with advanced EBV+ solid tumors, and in combination with the PD-1 inhibitor pembrolizumab in recurrent or metastatic nasopharyngeal carcinoma. Initiation of the trial is expected in the second half of 2021.
Corporate

Strengthened company leadership with the appointment of Ayman Elguindy, Ph.D., as Chief Scientific Officer, and additional key additions to management team. Dr. Elguindy has over 23 years of experience studying the role of viruses in cancer and spent the last decade as a faculty member at the Yale University School of Medicine, most recently as an Associate Professor in the Department of Pediatrics, Section of Infectious Disease, and Department of Pathology. Viracta also recently appointed Patric Nelson, MBA, as Senior Vice President, Business Development and Corporate Strategy and Biljana Nadjsombati, Pharm.D., as Vice President, Pharmaceutical Development.
Added as a member of the Russell 2000 Index and other FTSE Russell indexes. In June 2021, Viracta was included as a member of the small-cap Russell 2000 Index, the all-cap Russell 3000 Index, and the Russell Microcap Index.
Hosted key opinion leader webinar. The webinar featured presentations by key opinion leaders (KOLs) Pierluigi Porcu, M.D. (Thomas Jefferson University) and Kristen Cunanan, Ph.D. (Stanford Medicine Quantitative Sciences Unit) who discussed the current treatment landscape and unmet medical need in R/R EBV+ lymphoma, and the design of NAVAL-1. For a replay of the webinar, click here.
Anticipated 2021 Milestones

Initiation of a global Phase 1b/2 clinical trial in EBV+ solid tumors: H2 2021
Updated data from Phase 1b/2 trial in R/R EBV+ lymphoma (VT3996-201): H2 2021
Second Quarter 2021 Financial Results

Cash position – Cash and cash equivalents totaled approximately $122.7 million as of June 30, 2021. Viracta expects to end 2021 with greater than $100 million in cash and cash equivalents, which it expects will be sufficient to fund its operations into 2024.
Research and development expenses – Research and development expenses were $5.4 million and $9.5 million for the three and six-months ending June 30, 2021, respectively, compared to $3.4 million and $6.8 million for the same periods in 2020. This increase was primarily due to incremental costs associated with the initiation of the NAVAL-1 trial.
General and administrative expenses – General and administrative expenses were $3.9 million and $7.7 million for the three and six-months ending June 30, 2021, respectively, compared to $0.9 million and $1.9 million for the same periods in 2020. This increase was primarily due to expenses associated with operating as a public company and non-recurring, costs related to the merger with Sunesis Pharmaceuticals, as well as non-cash share-based compensation.
Acquired in-process research and development – Non-recurring, non-cash operating expenses of $0 and $84.5 million associated with the write-off of in-process research and development acquired in the merger were recorded for the three and six-months ending June 30, 2021.
Gain on Royalty Purchase Agreement – Gain on Royalty Purchase Agreement for the six-months ending June 30, 2021 was associated with upfront proceeds of $13.5 million received in connection with the multi-license milestone and royalty monetization transaction with XOMA Corporation in March 2021.
Adjusted loss from operations – Adjusted loss from operations for the six-months ended June 30, 2021, excluding the non-recurring and non-cash operating expenses associated with the write-off of in-process research and development acquired in the merger (a non-GAAP measure) was $3.7 million, compared to an unadjusted loss from operations of $88.2 million. There is not a comparative adjustment to loss from operations for the same period in 2020.
Net loss – Net loss was $9.2 million, or $0.25 per share (basic and diluted) for the quarter ended June 30, 2021, compared to a net loss of $4.2 million, or $15.52 per share (basic and diluted), for the same period in 2020. Net loss was $88.4 million, or $3.37 per share (basic and diluted) for the six months ended June 30, 2021, compared to a net loss of $8.7 million, or $32.13 per share (basic and diluted), for the same period in 2020.
About Nanatinostat

Nanatinostat (VRx-3996) is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which is key to inducing latent viral genes which are epigenetically silenced in EBV-associated malignancies. The nanatinostat and valganciclovir combination is being investigated in various subtypes of relapsed/refractory EBV+ lymphoma in multiple clinical trials, including a registration-enabling global, multicenter, open-label basket trial.

CymaBay Reports Second Quarter and Six Months Ended June 30, 2021 Financial Results and Provides Corporate Update

On August 12, 2021 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the second quarter ended June 30, 2021 (Press release, CymaBay Therapeutics, AUG 12, 2021, View Source [SID1234586437]).

In July 2021, CymaBay announced a development funding agreement with Abingworth, pursuant to which CymaBay will receive up to $100 million of funding for seladelpar development costs, of which $75 million will be received in three installments over approximately six months. CymaBay has an option to receive an additional $25 million within approximately two months of the completion of enrollment of CymaBay’s Phase 3 RESPONSE clinical trial. In exchange, CymaBay will make fixed payments spread over a six-year period based on regulatory approval in the U.S. or the E.U. after the first such regulatory approval is obtained, as well as pay fixed and capped sales milestones based on U.S. product sales. CymaBay has the ability to accelerate payment at a reduced amount upon regulatory approval and in the event of a change of control of CymaBay. CymaBay retains upside potential for seladelpar in the U.S. along with full worldwide commercial rights.

CymaBay also continued to make progress conducting the development program for seladelpar in primary biliary cholangitis (PBC). Additional clinical sites were activated in North America and Europe during the second quarter in RESPONSE, a global Phase 3 registrational study evaluating seladelpar in patients with PBC, and in ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional safety data to support registration. Enrollment of patients in both of these studies continued through the second quarter and is expected to increase as more sites are activated through the rest of 2021.

Sujal Shah, President and CEO of CymaBay, stated, "In addition to adding experienced talent across the functional areas vital to getting seladelpar to patients, we were thrilled to have secured the additional funding needed to complete Phase 3 development of seladelpar in PBC through a non-dilutive, risk-sharing funding agreement with Abingworth. Abingworth has had a long history of funding innovative companies in life sciences and shares our belief that seladelpar has the opportunity to meaningfully improve the care of patients with PBC. This strategic funding agreement further strengthens our balance sheet and allows us to maintain our dedicated focus on completing development of seladelpar to deliver value to patients and shareholders."

Recent Corporate Highlights

Executed a non-dilutive financing transaction with Abingworth for the development of seladelpar in PBC. Under the terms of the agreement, CymaBay will receive up to $100 million of funding for seladelpar development costs, of which $75 million will be received in three installments over approximately six months. CymaBay has an option to receive an additional $25 million within approximately two months of the completion of enrollment of CymaBay’s Phase 3 RESPONSE clinical trial.
Conducted enrollment activities for RESPONSE, a 52-week, placebo-controlled, randomized, global, Phase 3 registrational study evaluating the safety and efficacy of seladelpar in patients with PBC. This study is targeting enrollment of 180 patients who have an inadequate response to, or intolerance to, ursodeoxycholic acid, in a 2:1 randomization to oral, once daily seladelpar 10 mg or placebo. The primary outcome measure is the responder rate at 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level < 1.67 times the upper limit of normal with at least a 15% decrease from baseline and has a normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the level of pruritus at 6-months for patients with moderate to severe pruritus at baseline assessed by a numerical rating scale recorded with an electronic diary.
Conducted enrollment activities for ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety data to support registration.
Initiated enrollment in a Phase 2a proof-of-pharmacology study to evaluate the potential for MBX-2982, a GPR119 agonist, to prevent hypoglycemia in patients with type 1 diabetes (T1D). The study is being conducted by the AdventHealth Translational Research Institute in Orlando, Florida and fully funded by The Leona M. and Harry B. Helmsley Charitable Trust with CymaBay retaining full rights to MBX-2982.
Completed a Phase 1 single ascending dose and multiple asending dose study of CB-0406, a non-agonist ligand of PPARg that attenuates the expression of inflammatory genes. While the study showed CB-0406 had improved pharmacokinetics versus those historically achieved with the pro-drug arhalofenate, CB-0406’s safety profile did not support continued development as a result of the occurrence of a small number of reversible cases of thrombocytopenia at higher doses.

Held $106.1 million in cash, cash equivalents and short-term investments as of June 30, 2021. We believe that cash and investments, along with the initial $75 million funding raised through the development funding agreement with Abingworth in July 2021 are sufficient to fund CymaBay’s current operating plan into 2023.

Due to the ongoing effects of the global coronavirus pandemic, CymaBay continues to conduct its operations remotely for all employees, which has allowed business activities to continue as seamlessly as possible. The recent emergence of the Delta variant has led to uncertainty regarding the duration and effects that the pandemic will have on future operating milestones. CymaBay continues to closely monitor pandemic developments and their associated risks to the business, including the conduct of its clinical development of seladelpar, and will continue to take actions to mitigate them where possible. Further, all CymaBay’s actions will continue to be guided by a commitment to ensuring the health and safety of its employees as well as patients enrolled in its clinical studies.
Second Quarter and Six Months Ended June 30, 2021 Financial Results

Research and development expenses for the three months ended June 30, 2021 and 2020 were $16.7 million and $7.9 million, respectively. Research and development expenses for the six months ended June 30, 2021 and 2020 were $29.1 million and $17.5 million, respectively. Research and development expenses in the three and six months ended June 30, 2021 were higher than the corresponding periods in 2020 primarily due to an increase in clinical trial activities following our resumption of clinical development of seladelpar in PBC in late 2020. In particular, cost increases were primarily driven by the enrollment activities associated with RESPONSE and ASSURE, our two active global late-stage clinical trials in PBC. In the three and six months ended June 30, 2020, costs incurred were primarily associated with the termination and shutdown of our Phase 3 PBC, Phase 2b NASH, and Phase 2 PSC clinical trials, and other studies, after the seladelpar development program was placed on hold from November 2019 through July 2020.

General and administrative expenses for the three months ended June 30, 2021 and 2020 were $6.5 million and $3.2 million, respectively. General and administrative expenses for the six months ended June 30, 2021 and 2020 were $11.8 million and $7.6 million, respectively. General and administrative expenses in the three and six months ended June 30, 2021 were higher than the corresponding periods in 2020 due to higher employee compensation associated with the hiring of additional personnel and an increase in consulting and other expenses upon resumption of development of seladelpar in the second half of 2020.

Net loss for the three months ended June 30, 2021 and 2020 was $23.2 million and $10.7 million, or ($0.34) and ($0.16) per diluted share, respectively. Net loss for the six months ended June 30, 2021 and 2020 was $40.8 million and $23.8 million, or ($0.59) and ($0.35) per diluted share, respectively. Net loss was higher largely due to increases in clinical operating expenses, which were incurred following the resumption of our clinical development of seladelpar in PBC during the second half of 2020. We expect our operating expenses to increase in 2021 as we continue to execute on our clinical development plans.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss second quarter 2021 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13720877. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

Kezar Life Sciences Reports Second Quarter Financial Results and Provides Business Updates

On August 12, 2021 Kezar Life Sciences, Inc., (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, reported its second quarter 2021 financial results and corporate highlights (Press release, Kezar Life Sciences, AUG 12, 2021, View Source [SID1234586436]).

"We were pleased this quarter to share the final data update from our completed Phase 1b portion of the MISSION study, building on the positive safety, tolerability and early efficacy profile of KZR-616. I commend the entire team at Kezar on another quarter of outstanding execution across both of our programs and look forward to providing meaningful updates from both before the end of the year," said John Fowler, Kezar’s Co-founder and Chief Executive Officer. "We are also pleased to announce that Kezar recently submitted an IND application to the FDA for KZR-261, our first-in-class protein secretion inhibitor. Like the platform potential we see for KZR-616 in autoimmunity, we believe KZR-261 could have broad potential across the oncology landscape. We look forward to sharing more about our planned Phase 1 trial design and the potential of our protein secretion drug discovery platform in the coming months."

Clinical Highlights & Updates

KZR-616: Selective Immunoproteasome Inhibitor

MISSION – Phase 2 clinical trial in patients with lupus nephritis (LN) (NCT03393013)

At the European Congress of Rheumatology (EULAR 2021) in June, Kezar presented final clinical data from the completed Phase 1b dose escalation portion of the MISSION study in 47 patients with systemic lupus erythematous, including two patients with active proliferative LN. KZR-616 demonstrated improvement across all exploratory efficacy measures and was well-tolerated up to 75 mg subcutaneously once weekly for 13 weeks. In the two patients with LN, improvements in renal function correlated with reductions in a key biomarker of kidney inflammation, uCD163.

The safety and tolerability profile observed with KZR-616 was consistent with previously reported data and supports treatment for chronic use. No new safety or tolerability signals were observed from previously reported data.
These data support the development of KZR-616 in multiple immune-mediated diseases.
Doses being investigated in the Phase 2 clinical trials are 60 mg (MISSION Phase 2 in LN) and 45 mg (PRESIDIO Phase 2 in dermatomyositis and polymyositis) with weekly subcutaneous dosing.
The amended Phase 2 open-label portion of the MISSION trial in patients with active, proliferative LN opened for enrollment in August 2020 and is actively recruiting. The primary efficacy endpoint for the trial is the proportion of patients achieving a renal response measured by a 50% or greater reduction in urine protein to creatinine ratio (UPCR) at six months.
Kezar reiterates prior guidance and expects interim data to be reported in Q4 2021, and topline data are expected in the first half of 2022.
PRESIDIO – Phase 2 clinical trial in patients with dermatomyositis (DM) and polymyositis (PM) (NCT04033926)

The PRESIDIO Phase 2, placebo controlled cross-over trial of KZR-616 in DM and PM is actively enrolling. Additionally, a 12-month open-label extension study is open to patients completing the 32-week placebo-controlled trial (NCT04628936).
Topline data are expected in the first half of 2022.
KZR-261: Protein Secretion Inhibitor

KZR-261 is a first-in-class protein secretion inhibitor that targets the Sec61 translocon and has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies.
Kezar submitted an investigational new drug (IND) application for KZR-261 to the U.S. Food and Drug Administration (FDA) in August 2021. The company plans to initiate a Phase 1 clinical trial of KZR-261, which will assess safety, tolerability and preliminary tumor activity of KZR-261 in solid tumors.
At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2021 Virtual Annual Meeting in April, Kezar presented preclinical data on its novel small molecule inhibitors of the Sec61 translocon. These data support the therapeutic potential of inhibiting Sec61 and the protein secretion pathway as a way to generate novel therapies to treat multiple tumor indications.
Corporate Updates

Kezar formed a Clinical Advisory Committee in June, comprised of world-renowned thought leaders in immunology, rheumatology, neurology and nephrology. Appointments to the committee include: Rohit Aggarwal, MD, MS, Professor of Medicine, University of Pittsburgh; Prof. Olivier Benveniste, MD, PhD, Professor of Internal Medicine & Immunology, Sorbonne Université, Pitie Salpetriere Hospital; Mazen Dimachkie, MD, Professor of Neurology, University of Kansas Medical Center; Ingrid Lundberg, MD, PhD, Professor of Medicine, Karolinska Institute; Samir V. Parikh, MD, Assistant Professor of Medicine, Ohio State University Wexner Medical Center; and Onno Teng, MD, PhD, Nephrology Clinical Scientist, Leiden University Medical Center.
Micki Klearman, MD, rheumatologist and internist, was appointed to Kezar’s Board of Directors, bringing over two decades of biopharmaceutical experience with her significant contributions to the fields of immunology and rheumatology.
Second Quarter 2021 Financial Results

Cash, cash equivalents and marketable securities totaled $129 million as of June 30, 2021, compared to $140 million as of December 31, 2020. The decrease in cash, cash equivalents and marketable securities was primarily attributable to cash used by the company in operations to advance its clinical-stage programs and preclinical research and development, offset by the net proceeds from the issuance of common stock in February 2021, under the "at-the-market" Sales Agreement with Cowen and Company, LLC.
Research and development expenses for the second quarter of 2021 increased by $2.2 million to $9.3 million compared to $7.1 million in the second quarter of 2020. This increase was primarily related to advancing the KZR-616 clinical program in multiple indications and the protein secretion preclinical program.
General and administrative expenses for the second quarter of 2021 increased by $1.0 million to $3.7 million compared to $2.7 million in the second quarter of 2020. The increase was primarily due to an increase in stock-based compensation and personnel and recruiting expenses as a result of an increase in headcount and salaries and an increase in the cost of directors’ and officers’ liability insurance.
Net loss for the second quarter of 2021 was $13.0 million, or $0.25 per basic and diluted common share, compared to a net loss of $9.5 million, or $0.22 per basic and diluted common share, for the second quarter of 2020.
Total shares of common stock outstanding were 48.1 million shares as of June 30, 2021. Additionally, there were outstanding pre-funded warrants to purchase 3.8 million shares of common stock at an exercise price of $0.001 per share and outstanding options to purchase 7.3 million shares of common stock at a weighted-average exercise price of $5.86 per share as of June 30, 2021.
About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b clinical trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from Kezar’s research and discovery efforts targeting protein secretion pathway. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered by Kezar through a robust medicinal chemistry campaign in which several scaffolds were progressed through the company’s proprietary platform evaluating Sec61 modulation. As a result, Kezar has established a broad library of protein secretion inhibitors. KZR-261 has demonstrated several encouraging properties that lead to its potential to be an anti-cancer agent for the treatment of solid and hematologic malignancies. An IND submission in solid tumors was filed in August 2021 and a Phase 1 trial is expected to commence once the IND goes into effect.

About Lupus Nephritis

Lupus nephritis (LN) is one of the most serious complications of systemic lupus erythematosus. LN is a disease comprising a spectrum of vascular, glomerular and tubulointerstitial lesions and develops in approximately 50% of SLE patients within 10 years of their initial diagnosis. LN is associated with considerable morbidity, including an increased risk of end-stage renal disease requiring dialysis or renal transplantation and an increased risk of death. There are limited approved therapies for the treatment of LN. Management typically consists of induction therapy to achieve remission and long-term maintenance therapy to prevent relapse.

About Dermatomyositis and Polymyositis

Dermatomyositis (DM) and Polymyositis (PM) are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Approximately 30,000 to 120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM.

Achieve Reports Financial Results for Second Quarter 2021 and Provides Corporate Update

On August 12, 2021 Achieve Life Sciences, Inc. (Nasdaq:ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine addiction, reported second quarter 2021 financial results and provided an update on the cytisinicline clinical development program (Press release, OncoGenex Pharmaceuticals, AUG 12, 2021, View Source [SID1234586435]).

Recent Events & Highlights

Announced completion of enrollment in the Phase 3 ORCA-2 clinical trial evaluating the efficacy and safety of 3.0 mg cytisinicline dosed 3 times daily (TID) compared to placebo in adult smokers
Awarded grant from the National Institute on Drug Abuse (NIDA) of the National Institutes of Health (NIH) for the evaluation of cytisinicline in cessation of nicotine e-cigarette use
Issued two new patents from the United States Patent and Trademark Office covering the novel 3.0 mg TID cytisinicline dosing regimen
Closed financing with gross proceeds of $23 million, prior to deducting underwriting discounts and offering expenses
"In the second quarter, we continued to demonstrate our commitment to stakeholders by delivering on key milestones that advance our clinical program, and ultimately, cytisinicline’s potential ability to help millions of people who struggle with nicotine addiction to live healthier lives," commented John Bencich, Chief Executive Officer of Achieve. "We will remain committed to the execution of the combustible cigarette cessation program, while in parallel preparing for our anticipated expansion into the e-cigarette cessation indication in partnership with the NIH."

Phase 3 ORCA-2 Trial Fully Enrolled

The Phase 3 ORCA-2 trial completed enrollment of 810 adult smokers at 17 clinical sites in the United States. The participants have been randomized to one of three study arms to determine the efficacy and safety of cytisinicline administered for either six or twelve weeks, compared to placebo. The primary endpoint is biochemically verified continuous abstinence during the last four weeks of treatment in the six and twelve-week cytisinicline treatment arms compared to placebo. Each treatment arm will be compared independently to the placebo arm and the trial will be determined to be successful if either or both of the cytisinicline treatment arms show a statistical benefit compared to placebo. Topline ORCA-2 data results are expected to be reported within the first half of 2022.

Awarded Grant from NIH for Nicotine e-cigarette Cessation

Achieve announced that it has been awarded a grant from the NIH to evaluate the use of cytisinicline as a treatment for cessation of nicotine e-cigarette use. The initial grant will be used to complete key clinical and regulatory activities enabling the next stage of the grant award and initiation of the Phase 2 ORCA-V1 clinical study evaluating cytisinicline in approximately 150 adult nicotine e-cigarette users in the United States.

Patents Issued for 3.0 mg TID Dosing Regimen

Achieve announced that the United States Patent and Trademark Office has issued U.S. Patent No. 11,083,715 and U.S. Patent No. 11,083,716 covering the novel 3.0 mg TID cytisinicline dosing regimen. Not including any patent term extensions to which Achieve may be entitled, the newly issued patents will expire in the third quarter of 2040. Upon approval of cytisinicline by the U.S. Food and Drug Administration (FDA), Achieve anticipates these patents would be included in the FDA’s Orange Book, which lists approved drugs and related patent and exclusivity information.

Completed $23 Million Financing

In May 2021, Achieve announced the closing of an underwritten public offering of 3,285,714 shares of its common stock at a public offering price of $7.00 per share, which includes the exercise in full by the underwriters of their overallotment option to purchase additional shares of common stock. The gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses, were $23.0 million.

Financial Results

As of June 30, 2021, the company’s cash, cash equivalents, and restricted cash was $42.1 million. Total operating expenses for the three and six months ended June 30, 2021 were $11.3 million and $19.3 million, respectively. Total net loss for the three and six months ended June 30, 2021 was $11.3 million and $19.3 million, respectively.

As of August 12, 2021, Achieve had 9,452,223 shares outstanding.

Conference Call Details

Achieve will host a conference call at 4:30pm Eastern time today, Thursday, August 12, 2021. To access the webcast, log on to the investor relations page of the Achieve website at View Source Alternatively, access to the live conference call is available by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 8694398. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.

Bellicum Reports Second Quarter 2021 Financial Results and Provides Operational Update

On August 12, 2021 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the second quarter 2021 and provided an operational update (Press release, Bellicum Pharmaceuticals, AUG 12, 2021, View Source [SID1234586434]).

"In the second quarter, Bellicum expanded the potential impact of its CaspaCIDe technology through a licensing agreement with two leading oncology research and treatment centers of excellence for use in four of their cell therapy constructs," said Rick Fair, President and Chief Executive Officer. "The agreement provides potential future revenue as the projects progress to the market. We remain focused on the clinical studies of our next generation GoCAR-T cell therapies and our trials of BPX-601 in prostate cancer and BPX-603 in HER2+ solid tumors are on track. We look forward to providing future updates on our clinical progress for both programs."

Program Highlights and Current Updates

BPX-601 GoCAR-T
•Patient enrollment is ongoing in the Phase 1/2 dose-escalation clinical trial evaluating BPX-601 and rimiducid in patients with previously treated metastatic castration-resistant prostate cancer (mCRPC) and pancreatic cancer.
•Bellicum expects to provide a Phase 1 data update on BPX-601 and rimiducid in patients with mCRPC in the first quarter of 2022.

BPX-603 GoCAR-T
•Bellicum is conducting its 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. BPX-603 is the company’s first dual-switch GoCAR-T product candidate, which incorporates Bellicum’s iMC activation and CaspaCIDe safety switch technologies. The company expects to present initial Phase 1 data from this trial in the fourth quarter of 2021.

CaspaCIDe License Agreements
• In June, Bellicum entered into a license agreement with the University of North Carolina Lineberger Comprehensive Cancer Center (UNC Lineberger) and Massachusetts General Hospital covering certain intellectual property and technology rights regarding the company’s CaspaCIDe (inducible caspase-9, or iC9) safety switch and related technologies, and the use of rimiducid. This agreement covers four CAR-T programs currently in development that incorporate Bellicum’s technology, two of which are owned by UNC Lineberger and two of which are co-owned by both institutions.
Financial Results for the Second Quarter and Six Months Ended June 30, 2021

R&D Expenses: Research and development expenses were $6.7 million and $13.2 million for the three and six months ended June 30, 2021, respectively, compared to $11.8 million and $22.2 million for the three and six months ended June 30, 2020, respectively. The decrease in expenses in the second quarter of 2021 and the first half of the year resulted primarily from reduced rivo-cel commercialization activities and the corporate restructuring implemented during the fourth quarter of 2020, which resulted in a reduction in force.

G&A Expenses: General and administrative expenses were $1.8 million and $3.8 million for the three and six months ended June 30, 3021, respectively, compared to $3.8 million and $7.9 million for the three and six months ended June 30, 2020, respectively. The decrease in expenses in the second quarter of 2021 and the first half of the year was primarily due to the reduction in rivo-cel activities and reduction in force.

Loss from Operations: Bellicum reported a loss from operations of $7.9 million and $16.7 million for the three and six months ended June 30, 2021, respectively, compared to a loss from operations of $11.8 and $26.4 million for the three and six months ended June 30, 2020, respectively. The results for the six months ended June 30, 2021 include a net loss on dispositions of $0.5 million relating to the early termination in the first quarter of 2021 of the San Francisco office space. The results for the six months ended June 30, 2020 included a net gain on dispositions of $3.8 million due to the sale of the Houston manufacturing facility in the second quarter of 2020. Cash used in operating activities was $16.1 million for the six months ended June 30, 2021, compared to cash used in operating activities of $30.5 million for the six months ended June 30, 2020.

Net Loss: Bellicum reported net loss of $2.2 million and $13.5 million for the three and six months ended June 30, 2021, respectively, compared to a net loss of $43.2 million and $25.6 million for the three and six months ended June 30, 2020, respectively. The results in 2021 included a non-cash gain of $5.6 million and $3.2 million recognized from the change in the derivative warrant and private placement option fair value liability for the three and six months ended June 30, 2021, respectively. The results in 2020 included a non-cash loss of $30.7 million and a non- cash gain of $2.1 million related to the change in fair value of the warrant and private placement option liability for the three and six months ended June 30, 2020, respectively.

Shares Outstanding: As of August 4, 2021, Bellicum had 8,397,803 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 $21.8 million as of June 30, 2021, compared to $37.0 million as of December 31, 2020. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second quarter of 2022.