On November 10, 2022 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 quarter ended September 30, 2022 and provided a corporate update (Press release, Vincerx Pharma, NOV 10, 2022, View Source [SID1234623709]).

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"We continue progressing our lead clinical candidate, enitociclib, in prioritized indications in double-hit diffuse large B-cell lymphoma (DH-DLBCL), Richter syndrome and high-risk chronic lymphocytic leukemia (CLL). The recent Orphan Drug Designation for enitociclib further strengthens our program by offering important clinical development and commercialization benefits," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx.

"We are on track to submit our IND for our first-in-class small molecule drug conjugate (SMDC) VIP236 this year and continue to advance our first-in-class and potential best-in-class antibody-drug conjugate (ADC) VIP943 through IND-enabling studies and toward a targeted IND submission in the second half of 2023. Despite the challenging market environment, we are committed to executing across multiple programs and are motivated to strategically move towards achieving our goals of improving outcomes for patients while thoughtfully managing our cash resources."

RECENT CORPORATE HIGHLIGHTS

Orphan Drug Designation granted by the U.S. Food and Drug Administration (FDA) for the Company’s lead candidate, enitociclib (aka VIP152), a positive transcription elongation factor b (P-TEFb)/CDK9 inhibitor, for the treatment of high-grade B-cell lymphoma characterized by translocations of MYC and BCL2 (aka DH-DLBCL) Continued focus on prioritized enitociclib clinical studies:
Monotherapy (NCT02635672) and combination (NCT05371054) in patients with DH-DLBCL
Monotherapy (NCT04978779) in patients with high-risk CLL and Richter syndrome (RS)
Based on discussions with the FDA, combination with Bruton tyrosine kinase (BTK) inhibitor in patients with high-risk CLL (NCT04978779) will start after assessing the safety of enitociclib monotherapy in patients with CLL and RS. Anticipated start of the combination study is early 2023
Targeting initiation of Phase 2 studies in prioritized enitociclib indications in 2023
Enitociclib assigned by United States Adopted Name Council
Continue to implement cost reduction measures including focusing enitociclib studies on key U.S. sites with active patients
Advancement of first-in-class and potentially best-in-class bioconjugation assets continues to be prioritized:
IND submission for SMDC VIP236 on track by year end
IND filing for ADCs, VIP943 and VIP924, on track for 2H2023 and 1H2024, respectively
Upcoming poster presentations at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2022
Poster titled "VIP943 Is a Novel CD123 Antibody Drug Conjugate with In Vitro and In Vivo Efficacy in Acute Myeloid Leukemia (AML) Models"
Poster titled "Enitociclib (VIP152/formerly BAY1251152) Is a Selective and Active CDK9 Inhibitor: Preliminary Safety and Early Signs of Efficacy in Patients with Non-Hodgkin Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL)"
Poster titled "Preclinical Study of Enitociclib, a Selective CDK9 Inhibitor, in Combination with Bortezomib, Lenalidomide, Pomalidomide, or Venetoclax in the Treatment of Multiple Myeloma"
Received one-year extension of Small and Medium-Sized Enterprise (SME) status by the European Medicines Agency’s (EMA) Micro, Small and Medium-Sized Enterprise, enabling Vincerx to become eligible for EMA fee reductions and waiver and other financial incentives
THIRD QUARTER 2022 FINANCIAL RESULTS

Vincerx Pharma had approximately $66.0 million in cash, cash equivalents and marketable securities as of September 30, 2022, as compared to approximately $111.5 million as of December 31, 2021. Based on its current business plans and assumptions, Vincerx believes its available cash will be sufficient to meet its operating requirements into late 2024.
Research and development expenses for the quarter ended September 30, 2022 were approximately $11.1 million, as compared to approximately $12.2 million for the same period in 2021. This decrease is the result of a decline in stock-based compensation expense of approximately $3.1 million. Excluding stock-based compensation expense, research and development expenses increased by approximately $2.0 million for the quarter ended September 30, 2022 compared to the quarter ended September 30, 2021, primarily due to an increase in manufacturing services associated with Vincerx’s ADC programs. Research and development expenses for the nine months ended September 30, 2022 were approximately $40.8 million, as compared to approximately $27.7 million for the same period in 2021. This increase for the nine months ended September 30, 2022 compared to the same period in 2021 primarily relates to increases in manufacturing services of approximately $6.6 million, including the initiation of manufacturing associated with Vincerx’s ADC programs, third party research and preclinical work of approximately $5.4 million, new employee salaries of approximately $2.6 million and clinical services of approximately $1.0 million, partially offset by a decline in stock-based compensation expense of approximately $4.5 million.
General and administrative expenses for the three and nine months ended September 30, 2022 were approximately $4.5 million and $14.9 million, respectively, as compared to approximately $5.7 million and $17.2 million for the same periods in 2021, respectively. These decreases were primarily the result of declines in stock-based compensation expense of approximately $1.2 million and $2.5 million for the three- and nine-month periods, respectively.
For the quarter ended September 30, 2022, Vincerx reported a net loss of approximately $16.9 million, or $0.80 per share, as compared to a net loss of approximately $24.5 million, or $1.39 per share, for the same period in 2021. The net loss for the quarter ended September 30, 2021 includes a loss from the change in fair value of our warrant liabilities of approximately $6.6 million, as compared to a loss of approximately $0.1 million for the same period in 2022. For the nine months ended September 30, 2022, Vincerx reported a net loss of approximately $51.7 million, or $2.46 per share, as compared to a net loss of approximately $32.8 million, or $2.06 per share, for the same period in 2021. The net loss for the nine months ended September 30, 2021 includes a gain from the change in fair value of our warrant liabilities of approximately $12.1 million, as compared to a gain of approximately $6.3 million for the same period in 2022.

On November 10, 2022 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported financial and operating results for the third quarter of 2022 (Press release, Lineage Cell Therapeutics, NOV 10, 2022, View Source [SID1234623708]). Lineage management will host a conference call and webcast today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its third quarter 2022 financial and operating results and to provide a business update.

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"A single administration of RG6501 (OpRegen), a proprietary retinal pigment epithelial cell transplant, across an area of atrophy in advanced AMD patients has shown the potential to slow, stop, or reverse the progression of GA in our phase 1/2a clinical trial. To our knowledge, this is the first intervention that has reported anatomical changes of this magnitude in the field of GA, so we are pleased with the continued progress on RG6501 and the efforts which have been made to initiate its next clinical trial," stated Brian M. Culley, Lineage CEO. "Looking ahead, our focus turns increasingly to planned regulatory interactions for OPC1 and VAC2, from which we expect to inform and enable their next phases of clinical development in spinal cord injury and oncology, respectively. In parallel, we are advancing our newly launched cell transplant programs in photoreceptors for vision disorders and auditory neurons for hearing loss, with initial preclinical studies from our photoreceptor program currently ongoing and the start of preclinical testing of our auditory neuron program anticipated prior to year-end. We believe that completing these efforts while maintaining our commitment to disciplined spending will help Lineage create shareholder value in the coming year."

Recent milestones and activities included:

– Announced appointment of Jill Howe as Chief Financial Officer effective November 14, 2022

Ms. Howe brings more than 20 years of significant strategic, financial, and operational experience to Lineage, with an emphasis on capital strategy, corporate finance, treasury management, global infrastructure, and operational excellence. Ms. Howe has successfully built biotechnology organizations and implemented operational infrastructures alongside the execution of over $1.66 billion of capital raising transactions and will bring extensive strategic experience to the role.
– Established new U.S. R&D facility and expanded current GMP manufacturing facility in Israel

New Carlsbad facility will allow us to broaden R&D capabilities in the U.S. and facilitate the advancement of current and future allogeneic cell transplant programs and partnerships; the expansion of the Israel-based facility is expected to increase infrastructure, including development and optimization of larger-scale clinical manufacturing processes, and continued execution under the ongoing collaboration with Roche and Genentech for RG6501 (OpRegen).
– Strengthened intellectual property portfolio

Company announced notice of allowance of two patents covering processes for manufacturing allogeneic oligodendrocyte progenitor and retinal pigmented epithelium cells.
– OPC1

Completed verification and validation and preclinical testing activities for the novel parenchymal spinal delivery (PSD) system to support an upcoming regulatory submission.
– VAC2

Pre-Investigational New Drug (IND) application briefing package submitted to the U.S. Food and Drug Administration (FDA) to support U.S. clinical development for immuno-oncology.
– ANP1 & PNC1

Continued process development and activities in support of ongoing and planned preclinical testing.
Some of the key upcoming milestones and activities anticipated by Lineage include:

– Planned Regenerative Medicine Advanced Therapy (RMAT) submission to FDA before year-end regarding an OPC1 IND amendment to enable clinical testing of a novel spinal cord delivery system.

– Response to a pre-IND regulatory submission which should provide clarity on a VAC2 CMC, nonclinical, and clinical information package to inform future U.S. clinical development, expected around year-end.

– Completion of an R&D manufacturing process sufficient to support initiation of preclinical testing and the initiation of such testing with ANP1 for the treatment of hearing loss, anticipated prior to year-end.

– An additional OPC1 manuscript from a Phase 1/2a clinical study in subacute cervical spinal cord injury.

– Submission of a grant application to California Institute for Regenerative Medicine (CIRM) for the continued support of the clinical development of OPC1.

– Clinical data update from the ongoing VAC2 Phase 1 non-small cell lung cancer (NSCLC) study, pending release from Cancer Research UK (CRUK).

– Evaluation of new partnership opportunities and/or expansion of existing collaborations.

– Continued participation in investor and partnering meetings and medical and industry conferences to broaden awareness of our mission, programs, and accomplishments.

Balance Sheet Highlights

Cash, cash equivalents, and marketable securities totaled $66.4 million as of September 30, 2022, which is expected to support planned operations into Q3 2024.

Third Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from licensing fees, royalties, collaboration revenues, and research grants. Total revenues for the three months ended September 30, 2022 were approximately $3.0 million, a net increase of $0.7 million as compared to $2.3 million for the same period in 2021. The increase was driven by collaboration and licensing revenue recognized from deferred revenues from the Roche Agreement, partially offset by less royalty revenues.

Operating Expenses: Operating expenses are comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended September 30, 2022 were $8.0 million, a decrease of $0.1 million as compared to $8.1 million for the same period in 2021.

R&D Expenses: R&D expenses for the three months ended September 30, 2022 were $3.6 million, a net increase of $0.8 million as compared to $2.8 million for the same period in 2021. The net increase was primarily driven by higher OpRegen related expenses to support the Roche collaboration.

G&A Expenses: G&A expenses for the three months ended September 30, 2022 were $4.4 million, a net decrease of approximately $0.9 million as compared to $5.3 million for the same period in 2021. The decrease was primarily driven by $1.1 million in lower litigation and legal expenses and $0.3 million in lower investor relations expense, partially offset by a $0.5 million increase in payroll and related benefits expense.

Loss from Operations: Loss from operations for the three months ended September 30, 2022 was $5.2 million, a decrease of $1.6 million as compared to $6.8 million for the same period in 2021.

Other Expenses, Net: Other expenses, net for the three months ended September 30, 2022 reflected other expense, net of $0.3 million, compared to other expense, net of $2.0 million for the same period in 2021. The net change was primarily driven by a decrease in the value of marketable equity securities and exchange rate fluctuations related to Lineage’s international subsidiaries.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended September 30, 2022 was $6.1 million, or $0.04 per share (basic and diluted), compared to a net loss attributable to Lineage of $7.8 million, or $0.05 per share (basic and diluted), for the same period in 2021.

Conference Call and Webcast

Interested parties may access today’s conference call by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call" or provide conference ID number 5262180. A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through November 17, 2022, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 5262180.

On November 10, 2022 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported that financial results for the three-month period ended September 30, 2022 and provided a corporate update (Press release, Spectrum Pharmaceuticals, NOV 10, 2022, View Source [SID1234623707]).

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Third Quarter 2022 and Recent Business Update

Launched ROLVEDON (eflapegrastim-xnst) injection for adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia into an estimated approximately $2 billion market.
Closed on debt financing with SLR Capital Partners, LLC (SLR) for a term loan facility of up to $65 million, of which $30 million was funded at closing.
As of September 30, 2022, the Company had $100.3 million of cash, cash equivalents, and marketable securities available to optimize the ROLVEDON commercial launch and extends the Company’s cash runway through 2024.
The U.S. Food and Drug Administration’s ("FDA") Oncologic Drugs Advisory Committee ("ODAC") reviewed poziotinib for the treatment of patients with previously treated locally advanced or metastatic non-small cell lung cancer ("NSCLC") harboring HER2 exon 20 insertion mutations and voted 9-4 that the current benefits of poziotinib do not outweigh its risks. The PDUFA date for poziotinib is scheduled for November 24, 2022.
"The approval of ROLVEDON marks a significant accomplishment for Spectrum and our partner Hanmi Pharmaceutical. We are proud that the product became commercially available in October and are excited to be competing in an estimated $2 billion market opportunity. Importantly, we have been preparing for this milestone and have the financial runway to support ROLVEDON in the marketplace," said Tom Riga, President and Chief Executive Officer of Spectrum Pharmaceuticals. "While we are disappointed by the ODAC vote on poziotinib, we are awaiting the final response from the FDA and will continue to act in the best interest of the Company, our shareholders, and the patients who need our medicines."

Three-Month Period Ended September 30, 2022 (All numbers are from Continuing Operations)

Total research and development expenses were $13.3 million during the quarter, compared to $20.9 million in the same period in 2021. The $7.5 million decrease is primarily attributable to completed program spend associated with ROLVEDON, as well as a decrease in personnel-related expenses of $3.0 million related to the strategic restructuring that began in January 2022.

Selling, General and Administrative expenses were $8.3 million during the quarter, compared to $12.2 million in the same period in 2021. The $4.0 million decrease is primarily due to lower costs associated with employee compensation, benefits, and other headcount related expenses related to the reduction of workforce announced in January, as well as decreases in stock-based compensation.

Spectrum recorded a net loss of $21.9 million, or a $0.12 loss per basic and diluted share, in the three-month period ended September 30, 2022, compared to a net loss of $33.1 million, or a $0.21 loss per basic and diluted share, in the comparable period in 2021.

Cash Position and Guidance

The Company had a total cash, cash equivalents, and marketable securities balance of approximately $100.3 million at September 30, 2022.

The webcast will be archived under the "Events and Presentations" section of the Company’s investor relations website.

On November 10, 2022 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported financial results for the third quarter ended September 30, 2022, and provided a clinical and corporate update (FilingPress release, Milestone Pharmaceuticals, NOV 10, 2022, View Source [SID1234623706]).

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"The positive feedback we received from both clinicians and patient advocacy groups following the presentation of further RAPID data at the American Heart Association (AHA) meeting earlier this week strengthen our conviction that etripamil has the potential to serve as a promising option for patients with paroxysmal supraventricular tachycardia (PSVT)," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We are keenly focused on preparations for our planned New Drug Application (NDA), which we expect to submit to the U.S. Food and Drug Administration (FDA) in mid-2023 pending agency feedback."

Recent Updates
Announcement of Positive Topline Efficacy and Safety Results from Phase 3 RAPID Clinical Trial of Etripamil Nasal Spray in Patients with PSVT. In October 2022, the Company announced that the Phase 3 RAPID clinical trial of etripamil, a new chemical entity and Milestone’s lead investigational product, in patients with PSVT met its primary endpoint, with 64.3% of patients self-administering etripamil converting to sinus rhythm within 30 minutes compared to 31.2% on placebo (HR = 2.62, p<0.001). Safety and tolerability data from the RAPID trial continue to support the potential for at-home use of etripamil, with findings consistent with those observed in prior trials and now including data with an optional repeat dose. The most common randomized treatment emergent adverse events (RTEAEs), adverse events (AEs) which occurred within 24 hours of study drug administration, were related to the nasal administration site. The Company believes that results from the RAPID trial together with data from the already completed NODE-301 trial could fulfill the efficacy requirement for an NDA submission for etripamil in patients with PSVT. Milestone plans to submit an NDA to the U.S. FDA in mid-2023 pending agency feedback.

Additional Data from the RAPID Trial Featured During Late-Breaking Session at the American Heart Association (AHA) Scientific Sessions 2022. More extensive data from the Phase 3 RAPID trial were presented earlier this week at the AHA Scientific Sessions. Data showed statistically significant and clinically meaningful conversion rates in favor of etripamil over placebo at timepoints even beyond the 30-minute mark, including etripamil conversion rates reaching approximately 74% and 80% by 60 and 90 minutes, respectively. Data were presented documenting the faster median time to conversion, 17.2 minutes after administration of etripamil compared to more than 3-fold longer with placebo. In addition, the presentation included detailed pooled analyses of the Phase 3 RAPID and NODE-301 studies that showed significant reductions in emergency department visits and lower use of additional medical interventions in favor of etripamil. A complete evaluation of adjudicated ambulatory electrocardiogram (ECG) measures was also presented, revealing no occurrences of second-degree or greater atrioventricular (AV) block in either arm.

Recruitment Continues in the ReVeRA Phase 2 Proof-of-Concept Trial in Patients Experiencing AFib-RVR. Enrollment continues in ReVeRA, Milestone’s Phase 2 double-blind, placebo-controlled, proof-of-concept trial of etripamil nasal spray in emergency-department patients experiencing AFib-RVR (atrial fibrillation with a rapid ventricular rate). The trial, in which patients are randomized 1:1 to receive either 70 mg of etripamil or placebo, is designed to assess the safety and efficacy of etripamil nasal spray to reduce elevated ventricular rates in patients with symptomatic AFib-RVR. The trial is being conducted in Canada in collaboration with the Montreal Heart Institute and other research centers and is imminently planned to expand into other regions. The primary endpoint will assess reduction in ventricular rate, with key secondary endpoints including the time to achieve the maximum reduction in rate and duration of the effect. Additionally, based on results from the etripamil program in PSVT, Milestone has started planning a second Phase 2 trial to evaluate at-home self-administered etripamil in patients with AFib-RVR.

Successful Completion of RAPID Phase 3 Clinical Trial Triggers Milestone Payment from Ji Xing Pharmaceuticals Limited. The positive results of the RAPID Phase 3 clinical trial have triggered a $3.5 million payment from Milestone’s partner, Ji Xing Pharmaceuticals Limited (Ji Xing). In May 2021, Milestone and Ji Xing entered into an exclusive license agreement to develop and commercialize etripamil for PSVT in Greater China. Under the terms of the agreement, Milestone granted Ji Xing an exclusive license to develop and, if regulatory approval is obtained, commercialize etripamil in patients with PSVT in Greater China. Milestone is eligible to receive up to $107.5 million in milestone payments and royalties on future sales of etripamil in Greater China. Under the terms of the agreement, Milestone will supply etripamil and delivery devices to Ji Xing. Ji Xing will be responsible for development and commercialization costs in Greater China
Third Quarter 2022 Financial Results
As of September 30, 2022, Milestone had cash, cash equivalents, and short-term investments of $77.2 million and 30.4 million common shares and 12.3 million common shares issuable upon exercise of pre-funded warrants outstanding. We expect our current cash, cash equivalents, and short-term investments to fund operations through 2023.

We generated revenue of $1.5 million from milestone payments under the License Agreement for the three months and nine months ended September 30, 2022 compared to no revenue in the three months ended September 30, 2021 and revenue of $15 million from upfront payments under the License Agreement during the nine months ended September 30, 2021.

Research and development (R&D) expense for the third quarter of 2022 was $9.8 million, which was consistent with $9.7 million for the same period in the prior year. For the nine months ended September 30, 2022, R&D expense was $29.3 million compared with $27.8 million for the prior year period. The $1.5 million increase in R&D expense in the nine months ended September 30, 2022 is the result of clinical personnel related costs and clinical consulting fees. Regulatory costs increased due to personnel related costs.

General and administrative (G&A) expense for the third quarter of 2022 was $4.0 million, compared with $3.0 million for the prior year period. The difference is primarily the result of increase in personnel-related costs and consulting fees for general and administrative expenses. For the nine months ended September 30, 2022, G&A expense was $11.6 million compared with $8.6 million for the prior year period. The $3.0 million increase in G&A expense in the nine months ended September 30, 2022 is primarily the result of increased personnel related costs and consulting fees for general and administrative expenses.

Commercial expense for the third quarter of 2022 was $2.7 million, compared with $1.6 million for the prior year period. The difference is primarily the result of an increase in costs associated with consulting and marketing analytics. For the nine months ended September 30, 2022, commercial expense was $6.5 million compared with $4.8 million for the prior year period. The $1.7 million increase in commercial expense for the nine months ended September 30, 2022 over the same period in the prior year is primarily the result of personnel related costs and consulting fees.

For the third quarter of 2022, operating loss was $14.6 million, compared to operating loss of $14.2 million for the prior year period. For the nine months ended September 30, 2022, Milestone’s operating loss was $45.2 million, compared to $26.0 million in the prior year period.
About Paroxysmal Supraventricular Tachycardia
Paroxysmal supraventricular tachycardia (PSVT) is a condition affecting approximately two million Americans that is characterized by intermittent episodes of a rapid heartbeat that starts and stops suddenly. Episodes of supraventricular tachycardia (SVT) are often associated with symptoms such as palpitations, sweating, chest pressure or pain, shortness of breath, sudden onset of fatigue, lightheadedness or dizziness, fainting, and anxiety. Certain intravenous medications, including adenosine, beta-blockers and calcium channel blockers, have long been used for the acute treatment of PSVT. However, these medications must be administered under medical supervision, usually in an emergency department or other acute care setting.

About Atrial Fibrillation with Rapid Ventricular Rate
Atrial fibrillation (AFib) is a common arrhythmia marked by an irregular and often rapid heartbeat. AFib is estimated to affect five million patients in the United States, a prevalence projected by the Centers for Disease Control to increase to twelve million patients within the next 10 years. Atrial fibrillation with rapid ventricular rate (AFib-RVR) is a condition that some patients with AFib experience and includes episodes of abnormally high heart rate, often with symptoms such as palpitations, shortness of breath, dizziness and weakness. Oral calcium channel blockers and/or beta blockers are commonly used to reduce the heart rate in this condition. When AFib-RVR occurs, symptoms often cause patients to seek acute care in the emergency department, where standard-of-care procedures include intravenous administration of calcium channel blockers or beta blockers under medical supervision. Milestone’s market research indicates that 30-40% of patients with AFib experience one or more symptomatic episodes of RVR per year that require treatment, suggesting a target addressable market of approximately three to four million patients in 2030 for etripamil in patients with AFib.

About Etripamil
Etripamil, a new chemical entity, is Milestone’s lead investigational product. It is a novel calcium channel blocker designed to be a rapid-response therapy for episodic cardiovascular conditions. As a nasal spray that is self-administered by the patient, etripamil has the potential to shift the current treatment experience for many patients from the emergency department to a medically unsupervised setting. Milestone is conducting a comprehensive development program for etripamil, with Phase 3 trials ongoing in paroxysmal supraventricular tachycardia (PSVT) and a Phase 2 proof-of-concept trial that is underway in patients with atrial fibrillation with rapid ventricular rate (AFib-RVR).

On November 10, 2022 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company focused on development of targeted therapies for a number of different cancers, reported financial results for the third quarter ended September 30, 2022 and other recent business developments (Press release, Celcuity, NOV 10, 2022, View Source [SID1234623705]).

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"We are encouraged by the significant interest from clinical sites across the world to participate in our Phase 3 VIKTORIA-1 study for gedatolisib," said Brian Sullivan, CEO and Co-Founder of Celcuity. "We completed selection of sites for the study in the third quarter, and we are now focused on activating these selected sites. We are pleased that updated data from our Phase 1b study in advanced breast cancer will be reviewed during a Spotlight Poster Discussion Session at the 2022 San Antonio Breast Cancer Symposium in December 2022. Finally, I am excited that Dr. Polly Murphy has joined our Board of Directors. Her business development and commercial experience will provide a very helpful perspective to our board."

Third Quarter 2022 Business Highlights and Other Recent Developments

●In July, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Celcuity’s lead drug product candidate gedatolisib, an investigational pan-PI3K/mTOR inhibitor, for the treatment of HR+/HER2- locally advanced, inoperable or metastatic breast cancer that has progressed after treatment with a CDK4/6 inhibitor in combination with a nonsteroidal aromatase inhibitor. This designation allows for more intensive guidance from the FDA and a potentially accelerated review time if relevant criteria are met. Gedatolisib previously received Fast Track designation from the FDA in January 2022.

●In August, Celcuity amended its existing debt facility with an affiliate of Innovatus Capital Partners, LLC to provide Celcuity with up to $75 million in term loans, a $50 million increase from the original debt facility. Among other changes, the amended facility provides Celcuity with the ability to draw on a $20 million tranche following the closing of the $100 million private placement announced in May 2022. The closing of the private placement is expected to occur shortly after the first patient in VIKTORIA-1 receives their first dose at a clinical site located in the United States, provided that such dose occurs on or before December 31, 2022.

●In September, Celcuity appointed Polly Murphy, D.V.M., Ph.D., to its Board of Directors. Dr. Murphy has over 20 years of business development and commercial strategy experience at leading global pharmaceutical companies, including 12 years in leadership positions with increasing responsibility at Pfizer.

●In the third quarter, Celcuity completed selection of 200 clinical trial sites for the Phase 3 VIKTORIA-1 clinical trial. The first clinical trial site was also activated. VIKTORIA-1 is evaluating the safety and efficacy of gedatolisib in combination with fulvestrant with or without palbociclib in adults with HR+/HER2- advanced breast cancer whose disease progressed while receiving prior CDK4/6 therapy.

●An abstract with updates to results from Celcuity’s Phase 1b study was accepted for a Spotlight Poster Discussion presentation at the 2022 San Antonio Breast Cancer Symposium. Ongoing enrollment in the FACT-1 and FACT-2 trials for CELsignia selected patients who have early-stage HR+/HER2- breast cancer with interim results expected in mid-2023.

Third Quarter 2022 Financial Results

Unless otherwise stated, all comparisons are for the third quarter ended September 30, 2022, compared to the third quarter ended September 30, 2021.

The following discussion should be read in conjunction with Celcuity’s unaudited condensed consolidated financial statements and related notes on Form 10-Q for the third quarter ended September 30, 2022. Total operating expenses were $10.6 million for the third quarter of 2022, compared to $5.6 million for the third quarter of 2021.

Research and development (R&D) expenses were $9.6 million for the third quarter of 2022, compared to $5.0 million for the third quarter of 2021. Of the approximately $4.7 million increase in R&D expenses in the third quarter of 2022, compared to the third quarter of 2021, $1.2 million was related to employee related expenses, including consulting fees. The remaining $3.5 million increase in R&D expenses is primarily related to activities supporting the initiation of the VIKTORIA-1 pivotal trial and costs for existing clinical trials.

General and administrative (G&A) expenses were $1.0 million for the third quarter of 2022, compared to $0.6 million for the third quarter of 2021. The approximately $0.4 million increase in G&A expenses during the third quarter of 2022, compared to the third quarter of 2021, arose primarily from approximately $0.3 million in non-cash stock-based compensation.

Net loss for the third quarter of 2022 was $10.9 million, or $0.75 loss per share, compared to a net loss of $6.0 million, or $0.41 loss per share, for the third quarter of 2021. For purposes of calculating net loss per share, the reported net loss was increased by an additional $.02 loss per share, related to the approximately $0.3 million deemed dividend resulting from a warrant modification. Non-GAAP adjusted net loss for the third quarter of 2022 was $9.5 million, or $0.63 loss per share, compared to non-GAAP adjusted net loss of $5.1 million, or $0.35 loss per share, for the third quarter of 2021. Non-GAAP adjusted net loss excludes stock-based compensation expense, issuance of common stock and non-cash interest. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the third quarter of 2022 was $9.3 million, compared to $4.0 million for the third quarter of 2021. At September 30, 2022, Celcuity had cash and cash equivalents of $57.5 million.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the third quarter 2022 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-888-999-6281 and international callers should dial 1-848-280-6550 and reference Celcuity. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=f9185283fd. A replay of the webcast will be available on the Celcuity website following the live event.