On March 9, 2023 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage precision oncology medicine company developing MasterKey therapies designed to overcome limitations of existing therapies by targeting families of oncogenic driver mutations in patients with genetically defined cancers, reported financial results for the fourth quarter and full year ended December 31, 2022, and provided a corporate update (Press release, Black Diamond Therapeutics, MAR 9, 2023, View Source [SID1234628409]).

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"We expect 2023 to be a catalyst rich year for Black Diamond following tremendous progress for our two novel MasterKey inhibitors, with BDTX-1535 continuing to advance through our Phase 1 clinical trial and BDTX-4933 meeting critical milestones to further progress toward the clinic. The clearance of the Investigational New Drug (IND) application for BDTX-4933 by the U.S. Food and Drug Administration (FDA) earlier this year marks our third IND allowance in three years and demonstrates the strength of our MAP drug discovery engine along with the expertise of our research and clinical teams. We are driven by the clear unmet need for next-generation precision medicines to treat cancers by overcoming resistance, minimizing on-target and wild-type mediated toxicities, and addressing brain disease. We’ve designed a robust pipeline of products with brain penetration properties to address these key challenges," said David Epstein, Ph.D., President and Chief Executive Officer of Black Diamond. "We anticipate a clinical update for BDTX-1535 in the second half of 2023. We also expect to initiate the Phase 1 clinical trial of BDTX-4933 in the first half of this year and to progress our FGFR program and another undisclosed program toward development candidate nominations in 2023. As we continue to mature our pipeline, we are acutely focused on delivering meaningful benefit to patients and we believe that our approach has the potential to strengthen the treatment landscape for genetically defined cancers."

Recent Developments & Upcoming Milestones:

BDTX-1535:

BDTX-1535, an epidermal growth factor receptor (EGFR) MasterKey inhibitor, is being developed to treat genetically defined cancer in patients whose tumors are positive for EGFR MasterKey mutations in glioblastoma multiforme (GBM), as well as in non-small cell lung cancer (NSCLC). BDTX-1535 is fourth generation EGFR inhibitor designed to be irreversible (covalent) and brain-penetrant, and is unique in that it targets a family of driver mutations in addition to acquired resistance mutations arising from the use of third generation EGFR inhibitors to treat EGFR-positive NSCLC.
In April 2022, Black Diamond dosed the first patient in its Phase 1 global clinical trial of BDTX-1535 for the treatment of NSCLC, including in patients with brain metastases, and GBM. The dose-escalation portion of the Phase 1 clinical trial is actively recruiting and enrolling patients, and the Company remains on track to provide a clinical update on BDTX-1535 in the second half of 2023.
In October 2022, Black Diamond presented two posters at the 34th European Organisation for Research and Treatment of Cancer—National Cancer Institute—American Association for Cancer Research (EORTC-NCI-AACR) (Free EORTC-NCI-AACR Whitepaper) Symposium in Barcelona, Spain, with new preclinical data. The Company detailed anti-tumor activity of BDTX-1535 in preclinical models and highlighted that BDTX-1535 is designed using Black Diamond’s proprietary MAP drug discovery engine to target EGFR mutations of both NSCLC and GBM. Black Diamond also showcased BDTX-1535’s ability to achieve potent anti-tumor activity against EGFR alterations and amplification across preclinical models of NSCLC classical driver, intrinsic resistance and acquired resistance EGFR mutations and GBM, including intercranial patient derived xenograft models.
BDTX-4933:

BDTX-4933 is designed as a brain-penetrant, small molecule MasterKey reversible oral inhibitor of oncogenic BRAF Class I, II and III active RAF dimers promoted by upstream oncogenic alterations expressed by human cancers, while also avoiding paradoxical activation.
In the first quarter of 2023, Black Diamond received allowance of its IND application from the FDA. The Company expects to initiate a Phase 1 clinical trial of BDTX-4933 in patients with tumors harboring all-class BRAF or RAS mutations in the first half of 2023.
In October 2022, Black Diamond presented a poster at the 34th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium highlighting preclinical data showing BDTX-4933 to be a brain-penetrant RAF MasterKey inhibitor active against tumors that are driven by a Class I, II, or III BRAF mutation, as well as by other oncogenic RAS pathway alterations that promote constitutive RAF dimer activation. BDTX-4933 demonstrated potent, on-target inhibition of the RAF-MEK-ERK signaling pathway and anti-tumor activity in multiple preclinical models, including intracranial tumor models.
Discovery-Stage Pipeline and MAP Drug Discovery Engine:

Black Diamond continues to leverage its MAP drug discovery engine to advance its discovery-stage pipeline to bring therapies to underserved patients. Black Diamond anticipates progressing its fibroblast growth factor receptor (FGFR) program towards development candidate nomination and nomination of a development candidate against an undisclosed target in 2023.
Corporate:

In June 2022, Black Diamond appointed Sergey Yurasov, M.D., Ph.D., as its Chief Medical Officer, bringing over 25 years of oncology drug development experience and regulatory expertise.
In December 2022, Black Diamond announced that existing board member, Mark Velleca, M.D., Ph.D., was appointed to succeed Robert (Bob) A. Ingram as the Chairman of the Board of Directors.
In December 2022, Black Diamond announced the spinout of Launchpad Therapeutics, Inc. (Launchpad), an antibody-focused precision oncology company incorporating Black Diamond’s undisclosed early discovery stage antibody programs enabled by the MAP drug discovery engine. In conjunction with the transaction, a $30 million Series A financing was co-led by Versant Ventures and New Enterprise Associates (NEA) to fund discovery and development activities of the new company. Black Diamond retains a minority equity interest in Launchpad to enable further value creation.
Financial Highlights

Cash Position: Black Diamond ended 2022 with approximately $122.8 million in cash, cash equivalents, and investments compared to $209.8 million as of December 31, 2021. Net cash used in operations was $85.1 million for the year ended December 31, 2022 compared to $100.1 million for the year ended December 31, 2021.
Research and Development Expenses: Research and development (R&D) expenses were $14.6 million for the fourth quarter of 2022, compared to $19.7 million for the same period in 2021. Research and development expenses were $64.4 million for the year ended December 31, 2022, compared to $96.8 million for the year ended December 31, 2021. The decrease in R&D expenses was primarily due to reduced clinical trial activities stemming from the discontinuation of the development of BDTX-189 to focus on upcoming milestones for our pipeline programs, BDTX-1535 and BDTX-4933.
General and Administrative Expenses: General and administrative (G&A) expenses were $7.2 million for the fourth quarter of 2022, compared to $6.4 million for the same period in 2021, and $28.4 million for the year ended December 31, 2022, compared to $30.0 million for the year ended December 31, 2021. The decrease in G&A expenses was primarily due to a decrease in legal and other professional fees.
Net Loss: Net loss for the fourth quarter of 2022 was $21.1 million, as compared to $25.9 million for the same period in 2021. Net loss for the year ended December 31, 2022 was $91.2 million compared to $125.6 million for the year ended December 31, 2021.
Financial Guidance

Black Diamond ended 2022 with approximately $122.8 million in cash, cash equivalents and investments, which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the third quarter of 2024.

On March 9, 2023 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the fourth quarter and full year ended Dec. 31, 2022 and provided an update on key strategic initiatives (Filing, 8-K, BioXcel Therapeutics, MAR 9, 2023, View Source [SID1234628408]).

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"Last year was a transformational period for the Company, highlighted by the launch of our first AI-discovered commercial product, IGALMI, in under four years since initiating human trials. A new treatment option is now available for patients suffering from agitation associated with schizophrenia or bipolar I or II disorder," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We are building on these achievements in 2023 and look to accelerate our growth through commercial execution of IGALMI. We are also on track for significant data readouts for our overall neuropsychiatric program that has potential to address an estimated 139 million agitation episodes in the U.S.1-3* We have two pivotal study readouts for BXCL501 expected in the second quarter of 2023. Lastly, we plan to advance our lead immuno-oncology program, BXCL701, into a Phase 2b registrational trial, pending further discussion with the FDA, in conjunction with exploring strategic options for our OnkosXcel subsidiary. These upcoming milestones, along with our strong financial foundation and late-stage programs, position BioXcel Therapeutics to deliver significant value to our shareholders while helping treat millions of patients."

Company Highlights

Neuroscience Franchise

IGALMI (dexmedetomidine) sublingual film

IGALMI is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.4 Up to an estimated 16 million institutional episodes occur annually within these two patient populations in the U.S.1-3*

Commercial

· Enhanced Market Access: Continued to generate strong interest in IGALMI from key stakeholder groups to drive formulary access with initial 26-member field force.

o More than 600 hospital Pharmacy and Therapeutics (P&T) Committees decisions are scheduled, with over 65 total formulary wins to date.
o Targeting Integrated Delivery Network (IDN) universe of approximately 280,000 beds:
· More than 70,000 (~25%) target IDN beds are scheduled to vote; secured formulary approval for ~7,000 (2%) target beds.
o Three Group Purchasing Organization (GPO) contracts in place covering nearly 50% of target beds; in active discussions with other leading GPOs.

· Completed Deployment of IGALMI Institutional Sales Force: Integrated commercial team covers majority of U.S. agitation market.
o Expanded sales force to 70 representatives, extending coverage from 700 to more than 1,700 target hospitals.
o Current target reach is more than 1,100 hospitals and over 7,000 unique health care professionals to influence formulary access.

· Amplified Marketing Efforts: Increased IGALMI awareness and education planned.
o Launched new promotional campaign directed to health care providers (HCPs) through multiple channels.
o Established large-scale peer speaker program designed to educate thousands of target HCPs in 1H 2023.
o Planned promotional presence at leading national and regional conferences in 2023.

Medical Affairs

· Increased Field Team Engagement: Medical Science Liaison (MSL) and Medical Managed Care (MMC) teams continued to grow engagement with medical community and P&T committee members.
o Increased HCP interactions by 50% from Q3 to Q4 2022 and observed growing interest of medical community in obtaining clinical information.
o Established HCP relationships in top target academic medical centers.

· Clinical Data Dissemination:
o Published three manuscripts and two letters to the editor in 2022; the first publication from SERENITY II pivotal trial, published in the Journal of the American Medical Association (JAMA), has been viewed more than 32,000 times and cited by 15 other publications.
o Preparing four new manuscripts and 19 conference abstract submissions for 2023.

Development Pipeline

BXCL501, a proprietary, sublingual film formulation of dexmedetomidine, has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.

Alzheimer’s Disease-related Agitation: TRANQUILITY program is designed to evaluate BXCL501 for the acute treatment of Alzheimer’s-related agitation, where up to 100 million agitation episodes are estimated to occur in the U.S. annually.1*
o TRANQUILITY II: Trial is fully enrolled; nearing completion of three-month observation period in a few patients in assisted living facilities (ALFs) and residential settings.
· Data cleaning and verification in progress.
· Top-line data from pivotal trial expected in Q2 2023.
o TRANQUILITY III: Continuing enrollment of patients with moderate to severe dementia in nursing homes.

· Bipolar or Schizophrenia-related Agitation (At-Home Use): SERENITY III program is designed to evaluate BXCL501 for use at home, where up to 23 million agitation episodes are estimated to occur in the U.S. annually.1-3*

SERENITY III consists of two parts:

o Part 1: Assessing the efficacy and safety of 60mcg dose in acutely agitated patients with bipolar I or II disorder or schizophrenia in a supervised setting, similar to SERENITY I and II.
· More than 90% of patients enrolled and complete enrollment is imminent.
· Top-line efficacy data from pivotal trial expected in Q2 2023.
o Part 2: Evaluating the safety of self-administration of 60mcg dose at-home.
· Expect to initiate trial in Q2 2023.

· Adjunctive Treatment for Major Depressive Disorder (MDD) for At-Home Use: Phase 1b Multiple Ascending Dose (MAD) trial is designed to test safety and tolerability of daily dosing of BXCL501 to inform proof-of-concept (POC) trial dose selection in combination with selective serotonin – or serotonin-norepinephrine reuptake inhibitors (SSRIs or SNRIs, respectively) in MDD patients. Over 300 million antidepressant prescriptions are filled annually in the U.S.,5* and current treatments are limited by slow onset of action and incomplete responses.
o Enrollment has been completed; cohorts have received either daily or twice-daily dosing regimens for seven days, including a final cohort in combination with an SNRI.
o Data cleaning and verification in progress.
o Top-line results are expected in Q2 2023.

Research and Development Pipeline

BXCL502: Novel serotonergic receptor antagonist being developed for chronic treatment of agitation in patients with dementia and other related neuro-psychiatric conditions.

· Protype formulation has been completed.
· Undergoing Investigational New Drug application-enabling studies.
· Development path leverages efficacy and safety data from prior Phase 3 pivotal studies conducted by other pharma companies.

Emerging Pipeline: Leveraging our proprietary AI platform and deep multidisciplinary neuroscience and development expertise to identify novel pipeline candidates for neuropsychiatric disorders and neuro-rare diseases.

· Large language models, knowledge graphs, deep learning and machine learning methodologies centered around neuro-circuitry and receptor targets that modulate them.
· This strategy has enabled discovery of numerous promising product concepts that have been rank-ordered using an index for transformative care and commercial opportunity.
· Company is prioritizing these opportunities to select development candidates.

OnkosXcel Therapeutics

OnkosXcel Therapeutics is a subsidiary of BioXcel Therapeutics focused on the sustained growth of the Company’s immuno-oncology (I-O) franchise, including BXCL701, its most advanced I-O program. BXCL701 is an investigational, oral innate immune activator in development for the treatment of aggressive forms of prostate cancer and other solid and liquid tumors.

· Small Cell Neuroendocrine Metastatic Castration-Resistant Prostate Cancer (SCNC) Program: Presented positive Phase 2a efficacy data for BXCL701 in combination with KEYTRUDA (pembrolizumab) at the 2023 ASCO (Free ASCO Whitepaper) GU Cancers Symposium, demonstrating clinical proof-of-concept. In 2023, there will be an estimated 288,3006 new prostate cancer patients in the United States, with approximately 11,000 patients7 progressing to SCNC.
o BXCL701 has demonstrated activity in two prostate cancer subtypes ("cold" tumors).
· Data from the SCNC cohort showed response rate, duration of response, and safety data consistent with previously presented data from the adenocarcinoma cohort.
o BXCL701 in combination with KEYTRUDA demonstrated manageable adverse events (AEs) with no evidence of potentiation of immune-related AEs.
o Planned Phase 2b potential pivotal study for BXCL701 monotherapy and in combination with KEYTRUDA in SCNC expected to initiate in 2H 2023, subject to further discussions with FDA.
o Expect to initiate Phase 1b/2 trial in small cell lung cancer (SCLC) in 2H 2023.

· Predictive Biomarker for BXCL701: DPP9 overexpression was identified as a potential actionable biomarker for BXCL701 response.

· Hosted BXCL701 Key Opinion Leader (KOL) Day: Esteemed oncology experts highlighted the development landscape, challenges with current immunotherapy in prostate cancer, BXCL701 mechanism of action, and positive results from Phase 2 trial of BXCL701 in rare forms of prostate cancer.
o Planning to pursue additional indications in partnership with Georgetown Lombardi Cancer Center and Dana-Farber Cancer Institute in pancreatic cancer and AML, respectively.

Strategic Advancements: Actively evaluating strategic options for OnkosXcel Therapeutics, including potential partnering or third-party investments.

Corporate Updates

Patent Portfolio: The company is developing a broad global intellectual property portfolio, with over 100 patent applications in prosecution and multiple patents issued as of January 31, 2023.

· Neuroscience Franchise (BXCL501 and pipeline): Company’s patent portfolio, includes five issued U.S. patents, with four listed in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the Orange Book), as well as three patents from Japan and four patents from other countries. Additionally, the portfolio has 13 utility patent applications and four provisional applications in the U.S. and 87 utility patent applications in other countries, as well as four Patent Cooperation Treaty (PCT) applications not yet in the national phase.

· Immuno-oncology Franchise (BXCL701 and pipeline): Company’s patent portfolio, includes one issued patent in the U.S., one in Japan, and eight in other countries, as well as five provisional applications and seven utility patent applications in the U.S., including one with a Notice of Allowance, and 35 utility patent applications in other countries.

Fourth Quarter and Full Year 2022 Financial Results

Net Revenue: Net revenue was approximately $238,000 for the quarter and $375,000 for the full year 2022, which resulted from early product trials and reflects limited market access. Due to the Company’s direct shipping model to hospitals, wholesaler stocking was neither expected nor occurred.

Research and Development (R&D) Expenses: R&D expenses were $32.5 million for the fourth quarter of 2022, compared to $12.5 million for the same period in 2021.

R&D expenses were $91.2 million for the full year 2022, compared to $52.7 million for the same period in 2021. The increased expenses for both the fourth quarter and the full year were primarily attributable to an increase in clinical trial costs related to multiple major pivotal BXCL501 clinical programs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $20.7 million for the fourth quarter of 2022, as compared to $13.6 million for the same period in 2021.

SG&A expenses were $68.8 million for the full year 2022, as compared to $54.2 million for 2021. The increased costs for both the fourth quarter and full year were primarily due to personnel and costs related to the launch of IGALMI in the U.S.

Net Loss: BioXcel Therapeutics had a net loss of $54.8 million for the fourth quarter of 2022, compared to a net loss of $26.1 million for the same period in 2021. For the full year, BioXcel Therapeutics reported a net loss of $165.8 million, compared to a net loss of $106.9 million for the same period in 2021. The loss for the year includes approximately $17.3 million in non-cash stock-based compensation. Total cash expenditures for 2022 totaled approximately $135.3 million.

Cash and cash equivalents totaled $193.7 million at December 31, 2022, compared to $233.0 million at December 31, 2021. To date in the first quarter of 2023, the Company utilized its existing at-the-market equity facility and received net proceeds of approximately $24 million.

The Company believes that full execution of our strategic financing with Oaktree and Qatar Investment Authority would result in a cash runway into 2025 for the Company.

Anticipated Milestones

· Clinical Trial Readouts
o Top-line data from pivotal TRANQUILITY II trial in Q2 2023
o Top-line efficacy data from pivotal SERENITY III trial in Q2 2023
o Top-line results from Phase 1b MAD trial for MDD program in Q2 2023
· Clinical Trial Initiations
o Initiate SERENITY III Part 2 in Q2 2023
o Initiate Phase 2b potential pivotal study of BXCL701 in SCNC in 2H 2023

Conference Call

BioXcel Therapeutics will host a conference call and webcast at 8:30 a.m. ET on March 9 to discuss its fourth quarter and full-year 2022 financial results and provide an update on recent operational highlights. To access the call, please dial 877-407-5795 (domestic) and 201-689-8722 (international). A live webcast will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a replay will be available through June 9, 2023.

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News/Events menu of the Investors & Media section of its website.

About IGALMI (dexmedetomidine) sublingual film

INDICATION

IGALMI is indicated for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. Limitations of Use: The safety and effectiveness of IGALMI have not been established beyond 24 hours from the first dose.

IMPORTANT SAFETY INFORMATION

IGALMI can cause serious side effects, including:

· Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.

Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.
· Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.
· Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.

The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.

These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.

Patients should tell their healthcare provider about their medical history, including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.

Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201-1088 or [email protected].

On March 9, 2023 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for severe unmet medical needs, reported the first patient was dosed in a Phase 1b/2a trial evaluating bemcentinib in combination with the current standard of care, immune checkpoint inhibitor pembrolizumab and doublet chemotherapy, for the treatment of 1st line (1L) Non-Small Cell Lung Cancer (NSCLC) patients harboring STK11 mutations (STK11m) (Press release, BerGenBio, MAR 9, 2023, View Source [SID1234628407]).

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"Approximately 20% of non-squamous NSCLC patients harbor STK11m and do not currently have effective treatment options," said Martin Olin, Chief Executive Officer of BerGenBio. "One specific attribute of this group is that they almost all demonstrate high levels of AXL activation. We are elated to have dosed the first patient in our trial and to continue our evaluation of bemcentinib and its ability to inhibit AXL to revive STK11m NSCLC patients’ response to checkpoint inhibitors and chemotherapy."

The trial’s lead investigator, Rajwanth Veluswamy, M.D., MSCR, Assistant Professor of Medicine, Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai (New York, NY), commented, "Today, STK11 mutations are correlated with a very poor prognosis for patients suffering from NSCLC. These mutations are widely recognized for their ability to impede the activity of anti-PD-1/L1 therapy. My colleagues and I are driven to find a better outcome for this large patient population and are eager to assess bemcentinib’s potential in achieving this goal."

The global, open-label Phase 1b/2a trial is designed to determine the safety, tolerability and efficacy of bemcentinib with standard of care treatments in untreated advanced/metastatic non-squamous NSCLC patients with STK11 mutations and no actionable mutations. The Phase 1b portion of the study will evaluate the safety and feasibility of bemcentinib in combination with pembrolizumab and doublet chemotherapy in 1L advanced/metastatic non-squamous NSCLC patients, regardless of STK11 status. The Phase 2a expansion part will assess the efficacy of bemcentinib in the same treatment combination in 1L advanced/metastatic non-squamous NSCLC patients with STK11 mutations.

On March 9, 2023 Aptose Biosciences Inc. (Nasdaq: APTO; TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, reported financial results for the fourth quarter and full year ended December 31, 2022, on Thursday, March 23, 2023, after the close of the market, and provide a corporate update (Press release, Aptose Biosciences, MAR 9, 2023, View Source [SID1234628406]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Conference Call & Webcast:

Date: Thursday, March 23, 2023
Time: 5:00 PM ET
Audio Webcast Only: link
Q&A Participant Registration Link*:

here

(https://register.vevent.com/register/BI9394078d0ea14714aca591ffe06992f1)
*Analysts interested in participating in the question-and-answer session will pre-register for the event from the participant registration link above to receive the dial-in numbers and a personal PIN, which are required to access the conference call. They also will have the option to take advantage of a Call Me button and the system will automatically dial out to connect to the Q&A session.

The audio webcast also can be accessed through a link on the Investor Relations section of Aptose’s website here. A replay of the webcast will be available on the company’s website for 30 days.

The press release, the financial statements and the management’s discussion and analysis for the quarter and year ended December 31, 2022 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

On March 9, 2023 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, reported recent business progress and financial results for the fourth quarter and full year 2022 (Press release, Aligos Therapeutics, MAR 9, 2023, View Source [SID1234628405]).

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"Last year was very productive for our team," said Lawrence Blatt, PhD, MBA, Chairman & CEO of Aligos. "We made great progress advancing our NASH, COVID-19, and CHB drug candidates. In particular, our drug candidate for NASH, ALG-055009, continued to generate positive data in the clinic that differentiates it from other THR-beta agonists in development. Additionally, important nonclinical advances were made for ALG-097558, our protease inhibitor for COVID-19, which is anticipated to enter the clinic in the first half of this year. Finally, our CAM-E, ALG-000184, continues to generate potentially best-in-class antiviral activity data, including HBsAg reductions that are significantly more substantial than competitor CAM-E drugs, suggesting that it may be an important component in future treatments for CHB. Taken together, we expect these programs to have multiple important data readouts as we continue to advance them throughout 2023. These data have the potential to be important drivers of shareholder value and we look forward to sharing them as they emerge."

Recent Business Progress

Aligos Portfolio of Drug Candidates

NASH Program

ALG-055009 – Evaluation of single and multiple ascending doses (SAD, MAD) in healthy volunteers and subjects with hyperlipidemia was completed and MAD data were presented in November at The Liver Meeting (AASLD). Data from this Phase 1 study continue to be favorable and are positively differentiated compared to competitor drugs in the same class. We remain on track to submit a Phase 2 clinical trial filing by the end of 2023.
COVID-19

ALG-097558 – First-in-human-enabling nonclinical studies are ongoing. We anticipate initiating the clinical evaluation of single and multiple ascending doses of this drug candidate in healthy volunteers in Q2 2023, with top line safety and PK data available by Q4 2023.
HBV Programs

ALG-000184 (CAM-E) – Evaluation of a range of doses given over 28 days to both HBeAg positive and HBeAg negative CHB subjects was completed. Additionally, new interim data from cohorts evaluating dosing durations of up to 48 weeks in HBeAg positive subjects were presented by Professor Hou, Nanfang Medical University, China at the Asian Pacific Association for the Study of the Liver meeting (APASL) in February 2023. This presentation demonstrated favorable safety and antiviral activity for up to 12 weeks of dosing, including notable reductions in HBsAg levels. We anticipate sharing additional safety and antiviral activity data for up to 48 weeks of dosing from these and other ongoing cohorts at scientific conferences throughout 2023.

ALG-125755 (siRNA) – The SAD evaluation of ALG-125755 in healthy volunteers was completed and doses up to 200 mg were found to have a favorable safety and PK profile (Gane et al., APASL 2023). As a result, SAD cohorts in CHB subjects are now being evaluated. We anticipate sharing emerging data from these cohorts at scientific conferences throughout 2023.
Financial Results for the Fourth Quarter and Full Year 2022

Cash, cash equivalents and investments totaled $125.8 million as of December 31, 2022, compared with $205.8 million as of December 31, 2021. We continue to believe our cash balance provides sufficient cash to fund planned operations through the end of 2024.

Net losses for the three months ended December 31, 2022, were $21.9 million or basic and diluted net loss per common share of $(0.51), compared to net losses of $37.7 million or basic and diluted net loss per common share of $(0.89) for the three months ended December 31, 2021.

Net losses for the year ended December 31, 2022, were $96.0 million or basic and diluted net loss per common share of $(2.25), compared to net losses of $128.3 million or basic and diluted net loss per common share of $(3.22) for the year ended December 31, 2021.

Research and development (R&D) expenses for the three months ended December 31, 2022, were $19.1 million compared with $28.6 million for the same period of 2021. The decrease in R&D expenses for this comparative period is primarily attributable to a decrease in third-party expenses due to our continued wind down related to the discontinuation of our STOPS and ASO programs, and the manufacturing of drug supply in advance of our clinical and nonclinical activities. Total R&D stock-based compensation expense incurred for the three months ended December 31, 2022, was $1.9 million compared with $1.9 million for the same period of 2021.

R&D expenses for the year ended December 31, 2022 were $85.1 million, compared with $104.2 million for the same period of 2021. The decrease in R&D expenses for this comparative period is primarily attributable to a decrease in third-party expenses due to our continued wind down related to the discontinuation of our STOPS and ASO programs, and the manufacturing of drug supply in advance of our clinical and nonclinical activities. Total R&D stock-based compensation expense incurred in the year ended December 31, 2022, was $8.0 million, compared with $7.6 million for the same period of 2021.

General and administrative (G&A) expenses for the three months ended December 31, 2022, were $7.1 million compared with $9.7 million for the same period of 2021. The decrease in G&A expenses for this comparative period is primarily attributable to facility costs and outside services costs. Total G&A stock-based compensation expense incurred for the three months ended December 31, 2022, was $1.6 million compared with $1.7 million for the same period of 2021.

General and administrative (G&A) expenses for the year ended December 31, 2022, were $26.4 million compared with $28.5 million for the same period of 2021. The decrease in G&A expenses for this comparative period is primarily attributable to facility costs and outside services costs. Total G&A stock-based compensation expense incurred for the year ended December 31, 2022, was $6.7 million compared with $5.9 million for the same period of 2021.