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]).

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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.

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]).

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"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.

On August 12, 2021 Onconova Therapeutics, Inc. (NASDAQ: ONTX) ("Onconova"), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported financial results for the three months ended June 30, 2021 and provided a business update (Press release, Onconova, AUG 12, 2021, View Source [SID1234586433]).

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Highlights for the second quarter of 2021 and subsequent weeks include:

Enrollment in the first cohort of the Phase 1 solid tumor study of ON 123300 in the United States is complete with no dose limiting toxicities (DLT’s) observed. The second cohort is currently open for enrollment.
The Phase 1 solid tumor study of ON 123300 in China is ongoing with no DLT’s observed to date. The study is currently enrolling the third dose cohort.
The investigator-initiated Phase 1/2 study evaluating rigosertib in combination with the checkpoint inhibitor nivolumab in KRAS mutated non-small cell lung cancer (NSCLC) continues to progress. Initial data from the trial provide preliminary evidence of the anti-cancer activity of rigosertib-nivolumab combination therapy in patients who had previously failed all standard of care treatment, including checkpoint inhibition, and show that the maximum tolerated dose of rigosertib in combination with nivolumab was not yet determined in the three cohorts of the trial’s dose-escalation phase.
The first patient was dosed in an investigator-initiated Phase 2 study designed to assess the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB)-associated locally advanced/metastatic squamous cell carcinoma (SCC), an ultra-rare and invariably fatal condition.
The Company strengthened its management team with the appointment of Mark Gelder, M.D., as Chief Medical Officer.
Preclinical data published in the peer-reviewed journal Molecular Cancer show that rigosertib synergistically enhanced the efficacy of immune checkpoint blockade in a murine melanoma model via the induction of immune-mediated cancer cell death, supporting the continued clinical evaluation of rigosertib in combination with checkpoint inhibitors.
Management Commentary

"During the second quarter we achieved key clinical and corporate milestones," said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. "In our lead ON 123300 program, we initiated our U.S. Phase 1 study and recently opened enrollment to the second cohort, and our partner, HanX Biopharmaceuticals, is currently enrolling to the third dose cohort of the complementary Phase 1 study underway in China. Through these trials, which are evaluating different dosing administration regimens, we aim to inform the design of a future Phase 2 basket trial evaluating ON 123300 in multiple high unmet need indications, including CDK 4/6 inhibitor refractory HR+ HER2- metastatic breast cancer. Given ON 123300’s ability in preclinical studies to overcome resistance to the most widely prescribed CDK 4/6 inhibitor, we believe this novel multi-kinase inhibitor has the potential to be a best-in-class therapy for this and other cancers."

Dr. Fruchtman continued, "Beyond our lead program, we also reported very encouraging preliminary results from the investigator-initiated study evaluating rigosertib plus nivolumab in advanced KRAS-mutated NSCLC. These results highlighted the doublet’s favorable safety profile and provided preliminary evidence of efficacy in an extremely challenging patient population. Additional preliminary data from the trial is expected to be presented at a RAS-focused medical meeting in September. Looking forward, we will continue to leverage investigator-initiated programs to further rigosertib’s clinical development, while maintaining our primary focus and resources on ON 123300. Milestones ahead for the remainder of the year include continued progress of our ON 123300 clinical trials, expansion of rigosertib investigator-initiated studies program, and potentially acquiring new assets to augment our pipeline. With a strong financial position and a talented management team that was recently bolstered by the appointment of Dr. Mark Gelder as CMO, we believe we are well positioned to take advantage of the product development opportunities presented."

Second Quarter Financial Results

Cash and cash equivalents as of June 30, 2021 were $43.7 million, compared with $19.0 million as of December 31, 2020. The Company believes that its cash and cash equivalents will be sufficient to fund ongoing clinical trials and business operations for more than eighteen months.

Research and development expenses were $1.9 million for the second quarter of 2021, compared with $4.8 million for the second quarter of 2020. The decrease was primarily related to higher clinical trial and consulting expenses in the 2020 period due to the INSPIRE study.

General and administrative expenses were $2.9 million for the second quarter of 2021, compared with $2.6 million for the second quarter of 2020. The increase was primarily due to expenses related to special meetings by proxy in the 2021 period.

Net loss for the second quarter of 2021 was $4.2 million, or $0.27 per share on 15.8 million weighted average shares outstanding, compared with a net loss for the second quarter of 2020 of $7.4 million, or $0.65 per share on 11.3 million weighted average shares outstanding.

Conference Call and Webcast

Onconova will host an investment community conference call today beginning at 4:30 p.m. Eastern Time, during which management will discuss financial results for the second quarter of 2021, provide a business update and answer questions. Interested parties can participate by dialing (855) 428-5741 (domestic callers) or (210) 229-8823 (international callers) and using conference ID 3876025.

A live webcast of the conference call will be available in the Investors & Media section of the Company’s website at www.onconova.com. A replay of the webcast will be available on the Onconova website for 90 days following the call.

On August 12, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Vincerx Pharma, AUG 12, 2021, View Source [SID1234586432]).

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"During the past quarter, we achieved an important milestone in dosing the first patient in our Phase 1b study of VIP152, our selective PTEFb/CDK9 inhibitor, in MYC-driven indications, building upon the compelling signals of monotherapy activity observed in the dose-escalation study and exploratory cohort in double-hit lymphoma," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx. "Looking ahead, we intend to continue our strong execution in the clinic with the initiation of our Phase 1 dose escalation study in CLL relapsed/refractory to venetoclax and BTK inhibitors in the second half of this year. Our balance sheet provides us with a solid foundation to continue to execute on our clinical and regulatory goals."

Recent Highlights

Announced first patient dosed in Phase 1b study of VIP152, a potent and selective inhibitor of CDK9, in MYC-driven relapsed or refractory aggressive lymphomas and advanced solid tumors
Ongoing Phase 1b expansion, first-in-human (FIH) study is in patients with advanced cancer and consists of two expansion arms. Arm 1 will enroll up to 30 patients with relapsed/refractory aggressive lymphoma, including DLBCL, transformed follicular lymphoma, and blastoid mantle cell lymphoma. Arm 2 will enroll up to 40 patients with advanced solid tumors, including patients with ovarian cancer, triple negative breast cancer, castration-resistant neuroendocrine prostate cancer, and any other solid tumor with MYC aberration. All patients must have confirmed MYC overexpression or translocation.

Presented clinical data in a poster presentation entitled "Safety and efficacy of VIP152, a PTEFb / CDK9 inhibitor, in patients with double-hit lymphoma" at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting

Published an article entitled "Changing for the Better: Discovery of the Highly Potent and Selective CDK9 Inhibitor VIP152 Suitable for Once Weekly Intravenous Dosing for the Treatment of Cancer," in the Journal of Medicinal Chemistry

Announced inclusion in the Russell 3000 and Microcap Indexes
Second Quarter 2021 Financial Results

Vincerx Pharma ended the second quarter with $85.6 million in cash and cash equivalents, which includes the proceeds from the recent public warrant redemption, compared to $61.8 million at December 31, 2020.

Net loss for the second quarter ended June 30, 2021 was $2.0 million, or $0.12 per share, basic and diluted.

Research and development (R&D) expenses were $10.7 million for the quarter ended June 30, 2021, consisting primarily of $3.0 million in headcount related costs, $3.3 million of outside services in preparation for and support of our clinical trials and $4.4 million in stock-based compensation expense.

General and administrative (G&A) expenses were $6.7 million for the quarter ended June 30, 2021, consisting primarily of $1.2 million in headcount related costs, $3.3 million of outside services in support of our operations as a public company and $2.2 million in stock-based compensation expense.

On August 12, 2021 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period ended June 30, 2021 and provided a corporate update (Press release, Spectrum Pharmaceuticals, AUG 12, 2021, View Source [SID1234586431]).

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"Momentum continues to build with poziotinib and the submission of the NDA later this year is our top corporate priority," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "We are also seeking clarification on the recent CRL for ROLONTIS and are planning to have a Type A meeting with the FDA as soon as possible."

Pipeline Updates

Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations

Preparation is continuing for the poziotinib new drug application (NDA) seeking an indication for the use of poziotinib in patients with previously treated locally advanced or metastatic NSCLC with HER2 exon 20 insertion mutations. Submission of the NDA, based on the positive results of Cohort 2 from the ZENITH20 clinical trial, is planned for later this year.
Clinically meaningful data for poziotinib was presented in June at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting that showed its CNS activity in patients with NSCLC with EGFR or HER2 exon 20 mutations. These data were based on Cohorts 1-3 and included three patients achieving intracranial complete responses.
Enrollment for Cohort 4 of first-line patients with NSCLC HER2 exon 20 mutations is continuing in the ZENITH20 clinical trial. Poziotinib is currently being administered at a dose of 8mg BID in first line treatment.
Patient enrollment is also continuing in Cohort 5 which is now dosing exclusively at 8mg BID.
ROLONTIS (eflapegrastim), a novel long-acting G-CSF

Received a complete response letter (CRL) from the U.S. Food and Drug Administration (FDA) in connection with the biologics license application (BLA) for ROLONTIS. The CRL cited deficiencies related to manufacturing and a reinspection will be necessary. The company is seeking further clarification from the FDA and plans to meet with the agency as soon as possible.
Three-Month Period Ended June 30, 2021 (All numbers are from Continuing Operations and are approximate)

GAAP Results

Spectrum recorded a net loss of $49.9 million, or $0.32 loss per basic and diluted share, in the three-month period ended June 30, 2021, compared to a net loss of $32.2 million, or $0.29 loss per basic and diluted share, in the comparable period in 2020. Total research and development expenses were $29.1 million in the quarter, as compared to $21.7 million in the same period in 2020. Selling, general and administrative expenses were $15.0 million in the quarter, compared to $14.7 million in the same period in 2020.

The company ended the quarter with cash, cash equivalents, and marketable securities of $158.8 million.

Non-GAAP Results

Spectrum recorded a non-GAAP net loss of $39.3 million, or $0.25 loss per basic and diluted share, in the three-month period ended June 30, 2021, compared to a non-GAAP net loss of $31.8 million, or $0.28 loss per basic and diluted share, in the comparable period in 2020. Non-GAAP research and development expenses were $27.8 million, as compared to $20.6 million in the same period of 2020. Non-GAAP selling, general and administrative expenses were $11.9 million, as compared to $11.8 million in the same period in 2020.

Conference Call

This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website View Source on August 12, 2021 at 4:30 p.m. Eastern/1:30 p.m. Pacific.