On May 10, 2021 Intra-Cellular Therapies, Inc. (Nasdaq: ITCI), a biopharmaceutical company focused on the development and commercialization of therapeutics for central nervous system (CNS) disorders, reported its financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Intra-Cellular Therapies, MAY 10, 2021, View Source [SID1234579594]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"The acceptance of our sNDAs by the FDA is an important step towards a potential new treatment option for patients living with bipolar depression, a condition with a limited number of approved treatments. While preparing for this significant opportunity, our team will continue to execute on our commercial objectives for CAPLYTA and advance our robust pipeline," said Dr. Sharon Mates, Chairman and CEO of Intra-Cellular Therapies.

First Quarter Financial Highlights:

Total revenues were $15.9 million for the first quarter of 2021, compared to $1.1 million of total revenues for the first quarter of 2020. Net product revenues of CAPLYTA were $15.6 million for the first quarter of 2021, compared to $0.9 million in net product revenues of CAPLYTA for the same period in 2020.
Cost of product sales were $1.5 million in the first quarter of 2021 compared to $0.1 million for the first quarter of 2020.
Research and development (R&D) expenses for the first quarter of 2021 were $15.1 million, compared to $16.0 million for the first quarter of 2020. This decrease is due primarily to a decrease in manufacturing expense and is partially offset by an increase of lumateperone clinical and non-clinical expenses.
Selling, general and administrative (SG&A) expenses were $52.6 million for the first quarter of 2021, compared to $34.1 million for the same period in 2020. This increase is primarily due to an increase in sales related labor costs and commercialization costs.
Net loss for the quarter ended March 31, 2021 was $52.7 million compared to a net loss of $47.4 million for the quarter ended March 31, 2020.
Cash, cash equivalents, restricted cash and investment securities totaled $613.4 million at March 31, 2021, compared to $658.8 million at December 31, 2020.
COMMERCIAL HIGHLIGHTS

Despite the continued impact of COVID-19 on the healthcare system during the first quarter, our commercial organization continued to effectively engage with our prescribing audience through a hybrid model of in-person and virtual interactions, enhanced by digital marketing initiatives.
First quarter CAPLYTA results reflect strong commercial execution delivering continued prescription growth, increasing total prescriptions 23% versus the fourth quarter.
CAPLYTA market access coverage is strong with greater than 95% of covered lives in both Medicare Part D and State Medicaid, the major payer channels in schizophrenia. Our LytaLink program continues to be highly competitive and effective in supporting prescribing physicians and eligible patients’ access to CAPLYTA.
CLINICAL HIGHLIGHTS

Lumateperone – Bipolar Depression Program:

The U.S. Food and Drug Administration (FDA) has accepted for review the sNDAs for lumateperone for the treatment of depressive episodes associated with bipolar I or II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate. The FDA has assigned a PDUFA target action date of December 17, 2021 for these applications.
At the American Psychiatric Association (APA) Annual Meeting held from May 1-3, 2021, we had several presentations describing lumateperone study results. These included results from Study ‘402, a global Phase 3 clinical trial evaluating lumateperone as adjunctive therapy to lithium or valproate in the treatment of major depressive episodes associated with bipolar I or bipolar II disorder and analyses from Study ‘404 highlighting the efficacy results of patients with bipolar depression who exhibit mixed features. Another presentation summarized the overall safety and tolerability profile of the bipolar depression monotherapy program.
Other Lumateperone Programs

Mixed Features program: Study ‘403 evaluating lumateperone 42 mg in patients with major depressive disorder (MDD) and in patients with bipolar depression who exhibit mixed features is ongoing.
Adjunctive MDD program: Clinical conduct in two studies, Studies ‘501 and ‘502, our Phase 3 clinical trials evaluating lumateperone 42 mg as an adjunctive therapy to antidepressants for the treatment of MDD is anticipated to begin later this year.
Lumateperone Long Acting Injectable (LLAI) formulation: Study ITI-007-025, a Phase 1 single ascending dose study of LLAI, a formulation designed to be administered subcutaneously and to maintain therapeutic levels of lumateperone for at least one month is ongoing. Initial results from this study are anticipated in the second half of 2021.
CAPLYTA- Schizophrenia

Announced the publication of "Safety and tolerability of lumateperone 42 mg: An open-label antipsychotic switch study in outpatients with stable schizophrenia" (Correll et al. 2021) in the journal, Schizophrenia Research.
At APA we presented analyses from Study 303, our long-term safety schizophrenia study, evaluating the antidepressant effects of CAPLYTA in patients with schizophrenia with co-morbid depression, with and without concomitant antidepressant treatments.
Other Programs

ITI-1284 program: We introduced ITI-1284 ODT-SL, a deuterated form of lumateperone, a new molecular entity formulated as an orally disintegrating tablet for sublingual administration. We plan to initiate studies evaluating ITI-1284 ODT-SL for the treatment of behavioral disturbances in patients with dementia, the treatment of dementia-related psychosis and the treatment of certain depressive disorders in the elderly.
Phosphodiesterase type I inhibitor (PDE1) program: Our PDE1 inhibitor program is focused on diseases in which the PDE1 enzyme is over-expressed and/or abnormal immune cell function contributes to disease pathology. This suggests therapeutic potential across a variety of diseases, including neurological and cardiovascular diseases, as well as cancer.
We plan to advance our lead molecule, lenrispodun (ITI-214), into a Phase 2 clinical study in Parkinson’s disease in the second half of 2021.
At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting, we presented preclinical data supporting the potential of PDE1 inhibition to enhance the anti-tumor effects of immunotherapies by altering the tumor micro-environment. Our prior studies elucidating the neuroprotective and anti-inflammatory effects of PDE1 inhibitors in the brain by regulating microglia function (brain resident macrophage-like cells) led to our exploration of similar effects outside the brain. Therefore, we hypothesized PDE1 inhibition could result in antitumor effects by controlling similar functions of macrophages in the tumor micro-environment. Our experiments indicate PDE1 inhibition prevents the migration and accumulation of monocytes and macrophages in the tumor micro-environment and could represent a novel and broadly applicable approach to the treatment of immune responsive cancers.

ITI-333 program in opioid use disorder: Study ITI-333-001, a Phase 1 single ascending dose study evaluating the safety, tolerability and pharmacokinetics of ITI-333 in healthy volunteers is ongoing. Results from this study are anticipated in the second half of 2021.
Conference Call and Webcast Details

The Company will host a live conference call and webcast today at 8:30 AM Eastern Time to discuss the Company’s financial results and provide a corporate update. The live webcast and subsequent replay may be accessed by visiting the Company’s website at www.intracellulartherapies.com. Please connect to the Company’s website at least 5-10 minutes prior to the live webcast to ensure adequate time for any necessary software download. Alternatively, please call 1-(844) 835-6563 (U.S.) or 1-(970) 315-3916 (international) to listen to the live conference call. The conference ID number for the live call is 3244409. Please dial in approximately 10 minutes prior to the call.

CAPLYTA (lumateperone) is indicated for the treatment of schizophrenia in adults. CAPLYTA is available in 42 mg capsules.

Important Safety Information

Boxed Warning: Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death. CAPLYTA is not approved for the treatment of patients with dementia-related psychosis.

Contraindications: CAPLYTA is contraindicated in patients with known hypersensitivity to lumateperone or any components of CAPLYTA. Reactions have included pruritus, rash (e.g. allergic dermatitis, papular rash, and generalized rash), and urticaria.

Warnings & Precautions: Antipsychotic drugs have been reported to cause:

Cerebrovascular Adverse Reactions in Elderly Patients with Dementia-Related Psychosis, including stroke and transient ischemic attack. See Boxed Warning above.
Neuroleptic Malignant Syndrome (NMS), which is a potentially fatal reaction. Signs and symptoms include: high fever, stiff muscles, confusion, changes in breathing, heart rate, and blood pressure, elevated creatinine phosphokinase, myoglobinuria (and/or rhabdomyolysis), and acute renal failure. Patients who experience signs and symptoms of NMS should immediately contact their doctor or go to the emergency room.
Tardive Dyskinesia, a syndrome of uncontrolled body movements in the face, tongue, or other body parts, which may increase with duration of treatment and total cumulative dose. TD may not go away, even if CAPLYTA is discontinued. It can also occur after CAPLYTA is discontinued.
Metabolic Changes, including hyperglycemia, diabetes mellitus, dyslipidemia, and weight gain. Hyperglycemia, in some cases extreme and associated with ketoacidosis, hyperosmolar coma or death, has been reported in patients treated with antipsychotics. Measure weight and assess fasting plasma glucose and lipids when initiating CAPLYTA and monitor periodically during long-term treatment.
Leukopenia, Neutropenia, and Agranulocytosis (including fatal cases). Complete blood counts should be performed in patients with pre-existing low white blood cell count (WBC) or history of leukopenia or neutropenia. CAPLYTA should be discontinued if clinically significant decline in WBC occurs in absence of other causative factors.
Decreased Blood Pressure & Dizziness. Patients may feel lightheaded, dizzy or faint when they rise too quickly from a sitting or lying position (orthostatic hypotension). Heart rate and blood pressure should be monitored and patients should be warned with known cardiovascular or cerebrovascular disease. Orthostatic vital signs should be monitored in patients who are vulnerable to hypotension.
Falls. CAPLYTA may cause sleepiness or dizziness and can slow thinking and motor skills, which may lead to falls and, consequently, fractures and other injuries. Patients should be assessed for risk when using CAPLYTA.
Seizures. CAPLYTA should be used cautiously in patients with a history of seizures or with conditions that lower seizure threshold.
Sleepiness and Trouble Concentrating. Patients should use caution when operating machinery or motor vehicles until they know how CAPLYTA affects them.
Body Temperature Dysregulation. CAPLYTA should be used with caution in patients who may experience conditions that may increase core body temperature such as strenuous exercise, extreme heat, dehydration, or concomitant anticholinergics.
Dysphagia. CAPLYTA should be used with caution in patients at risk for aspiration.
Drug Interactions: CAPLYTA should not be used with CYP3A4 inducers, moderate or strong CYP3A4 inhibitors and UGT inhibitors.

Special Populations: Newborn infants exposed to antipsychotic drugs during the third trimester of pregnancy are at risk for extrapyramidal and/or withdrawal symptoms following delivery. Breastfeeding is not recommended. Use of CAPLYTA should be avoided in patients with moderate or severe liver problems.

Adverse Reactions: The most common adverse reactions in clinical trials with CAPLYTA vs. placebo were somnolence/sedation (24% vs. 10%) and dry mouth (6% vs. 2%).

Please click here to see full Prescribing Information including Boxed Warning.

About CAPLYTA (lumateperone)

CAPLYTA 42mg/day is an oral, once daily atypical antipsychotic approved for the treatment of schizophrenia of adults. While the mechanism of action of CAPLYTA in the treatment of schizophrenia is unknown, the efficacy of CAPLYTA could be mediated through a combination of antagonist activity at central serotonin 5-HT2A receptors and postsynaptic antagonist activity at central dopamine D2 receptors.

Lumateperone is being investigated for the treatment of bipolar depression, depression and other neuropsychiatric and neurological disorders. CAPLYTA is not FDA approved for these disorders.

On May 10, 2021 TYME Technologies, Inc. (Nasdaq: TYME) (the "Company" or "Tyme"), an emerging biotechnology company developing cancer metabolism-based therapies (CMBTs), reported that its abstract featuring data from the Company’s investigational therapy, SM-88, has been selected for publication at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held virtually June 4-8, 2021 (Press release, TYME, MAY 10, 2021, View Source [SID1234579613]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Details about the abstract selected for publication can be found below:

Title: Phase II Study of SM-88 in Ewing’s and Other Sarcomas
Abstract Number: e23505
Authors: Giuseppe Del Priore, MD, MPH, Victoria S. Chua, Kitty Zheng, Ted Kim, Semmie Kim, Sant P. Chawla, MD
Session: Publication Only – Sarcoma

The Publication Only abstracts are under embargo until 5:00 p.m. ET on May 19, 2021. For more information about the Annual Meeting, please visit: asco.org.

About SM-88

SM-88 is an oral investigational modified proprietary tyrosine derivative that is believed to interrupt the metabolic processes of cancer cells by breaking down the cells’ key defenses and leading to cell death through oxidative stress and exposure to the body’s natural immune system. Clinical trial data have shown that SM-88 has demonstrated encouraging tumor responses across 15 different cancers, including pancreatic, lung, breast, prostate and sarcoma cancers with minimal serious grade 3 or higher adverse events. SM-88 is an investigational therapy that is not approved for any indication in any disease.

On May 10, 2021 Yale University, in partnership with Boehringer Ingelheim, reported the launch of a Biomedical Data Science Fellowship program designed to attract and support some of the brightest and most innovative minds in data science from around the world (Press release, Boehringer Ingelheim, MAY 10, 2021, View Source [SID1234579522]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Post-doctoral researchers awarded a three-year fellowship will have access to Yale’s robust computational resources, biomedical data repositories and faculty expertise. In addition, they will benefit from access to Boehringer Ingelheim’s corporate labs, scientists and executives. Applicants are invited to submit research proposals for consideration by June 15. If approved for a fellowship, they will be jointly mentored throughout the research process by industry experts and scientists from Boehringer Ingelheim — one of the world’s leading pharmaceutical companies — as well as Yale’s world-class researchers and scholars. Fellowship training begins Sept. 1.

"This collaboration with Boehringer Ingelheim creates a world-class data science fellowship program that will drive development of novel methods and tools to analyze and interpret the many large and complex biomedical datasets that have been created in recent years," said Yale School of Public Health Professor of Biostatistics, Genetics, Statistics, and Data Science Hongyu Zhao, PhD, principal investigator for the project.

The program will be based at the Yale Center for Biomedical Data Science (CBDS) in New Haven, Connecticut. The center is an essential part of a growing data science hub at Yale University, which has identified integrated data science as one of its primary investment areas over the next decade. CBDS is located within Yale School of Medicine and currently supports more than 100 faculty members and researchers representing such disciplines as bioinformatics, modeling, statistics, computer science, artificial intelligence, mathematics, biology, precision medicine and public health.

"In partnering with a top-tier academic and research institution like Yale, we aim to recruit and train a new generation of highly skilled data scientists to help us accelerate development of novel treatments and therapies for human disease and improve health outcomes for our patients," said Jan Nygaard Jensen, PhD, Global Head of Computational Biology and Digital Sciences at Boehringer Ingelheim.

The partnership reflects a mutual vision between Boehringer Ingelheim and Yale University. It is part of a comprehensive strategic program at Boehringer Ingelheim which will harness the power of data science to transform drug discovery and development. The aim is to catalyze the next breakthrough therapies that change lives by accelerating timelines, improving scientific and clinical success and further elevating patient centricity.

"Boehringer Ingelheim is pleased to build upon our successful relationships with Yale to foster the next generation of scientists and harness the power of data science to bring our vision of making new and better medicines for patients in need," said Clive R. Wood, PhD, Senior Corporate Senior Vice President, Global Head, Discovery Research, Boehringer Ingelheim. "We believe our shared ambition and outlook will build a world-class data science community to attract outstanding researchers and work to achieve breakthroughs that patients need."

Initially the program will award as many as three competitive fellowships per year, up to a total of nine over the first five years. In addition to receiving research funding and mentorship, program fellows will be invited to participate in campus and corporate visits, networking events and annual symposia.

A joint selection committee comprising representatives of Yale and Boehringer Ingelheim will set annual data-driven research themes for the program. These themes may include such topics as genomic analysis, biomarkers, data-driven therapeutic research, medical image informatics, precision medicine and translational medicine. The selection committee will consider proposed research projects’ alignment with prioritized themes in judging submissions and post-doctoral applicants.

Yale’s data science ecosystem is supported by a host of cutting-edge research institutions working collaboratively. In addition to CBDS, they include Yale’s Systems Biology Institute, Center for Mendelian Genomics, Center for RNA Science and Medicine, and Center for Medical Informatics. Yale’s biobanks and technology core include: the Yale BioBank GENERATIONS, VA Million Veteran Program, Center for Research Computing, Center for Genomic Health, and Center for Genomic Analysis, which houses the ninth largest genomic library in the world.

Xinxin (Katie) Zhu, MD, PhD, executive director of the Yale Center for Biomedical Data Science, said the fellowship program offers an exciting opportunity for the development of innovative data-driven approaches for different medical conditions that can be translated from the lab to the patient’s bedside. It is an especially opportune time, she said.

"The vast amount of biomedical data being generated today has created a tremendous need for highly skilled data scientists who can use this information to advance care," said Zhu.

Specialists in biomedical data science and health informatics can identify statistical associations and patterns of disease. They also can develop complex machine learning models and simulations of molecular, cellular and organismic systems to increase the probability of clinical success through precision medicine and other methods.

"This helps clinicians and pharmaceutical companies such as Boehringer Ingelheim identify potential new pathways for treatment and eradication of disease," Zhu said.

On May 10, 2021 Allergan Aesthetics, an AbbVie company (NYSE: ABBV) and Soliton (NASDAQ: SOLY) reported a definitive agreement under which Allergan Aesthetics will acquire Soliton and RESONICTM, its Rapid Acoustic Pulse device which recently received U.S. Food and Drug Administration (FDA) 510(k) clearance and is a non-invasive treatment for the short-term improvement in the appearance of cellulite (Press release, AbbVie, MAY 10, 2021, View Source [SID1234580065]). The acquisition of Soliton expands and complements Allergan Aesthetics’ Body Contouring treatment portfolio which includes CoolSculpting Elite.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The novel platform technology uses non-invasive rapid, high-frequency sound waves to disrupt targeted cellular structures and connective tissue, physically impacting the fibrous septae beneath the skin that contribute to the dimpled appearance of cellulite. In clinical trial data submitted to the FDA, after a single treatment session RESONICTM demonstrated significant improvement and strong patient satisfaction with 92.9 percent of subjects agreeing or strongly agreeing their cellulite appeared improved.

"There is a huge unmet need to address cellulite and effective treatments have been elusive and frustrating for consumers," said Carrie Strom, President, Global Allergan Aesthetics and Senior Vice President, AbbVie. "Soliton’s technology offers a new, completely non-invasive approach with clinically-proven results to reduce the appearance of cellulite with no patient downtime. The addition of this technology complements Allergan Aesthetics’ portfolio of body contouring treatments. Health care providers will now have another option to address consumers’ aesthetic concerns."

"Allergan Aesthetics’ brand recognition, global footprint, track record and commitment to developing best-in-class aesthetic treatments makes the Company ideally suited to maximize the commercial potential of the RESONICTM rapid acoustic pulse technology," said Walter Klemp, Executive Chairman, Soliton. "I am proud of the passion and accomplishments of the Soliton team and thankful for the ongoing support of our investors which have culminated in this transaction. We look forward to working with Allergan Aesthetics to ensure a successful completion of this transaction."

Under the terms of the transaction, Allergan Aesthetics will pay $22.60 per share in cash for each outstanding share of Soliton. Soliton’s enterprise value for the transaction is approximately $550 million and was approved by the Boards of Directors of both companies. The transaction is subject to customary closing conditions, including clearance by the U.S. antitrust authorities under the Hart-Scott-Rodino Act and approval of Soliton’s shareholders. Guggenheim Securities served as financial advisor to Soliton and Hogan Lovells served as legal counsel to Soliton.

RESONICTM has also received FDA 510(k) clearance for use in conjunction with laser for tattoo removal and has demonstrated clinical results in fibrotic scars.

On May 10, 2021 Celcuity Inc. (NASDAQ:CELC), a clinical-stage biotechnology company pursuing an integrated companion diagnostic (CDx) and therapeutic strategy for treating patients with cancer, reported financial results for the first quarter ended March 31, 2021 and summarized recent business progress (Press release, Celcuity, MAY 10, 2021, View Source [SID1234580661]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"Celcuity took a transformational strategic step in April when we entered into a global licensing agreement with Pfizer to obtain exclusive rights to develop and commercialize gedatolisib, a pan-PI3K/mTOR inhibitor, in clinical development to treat patients with ER+/HER2-negative advanced or metastatic breast cancer," said Brian Sullivan, CEO and co-founder of Celcuity. "Celcuity is planning to initiate, subject to feedback from the FDA, a Phase 2/3 clinical trial evaluating gedatolisib in combination with palbociclib and an endocrine therapy in the first half of 2022. We have a highly experienced drug development team and the financial resources in place to advance the gedatolisib program and are excited by the opportunity to utilize our CELsignia cellular analysis platform to support the development of a potential first-in-class targeted cancer therapy like gedatolisib."

First Quarter 2021 Business Highlights and Other Recent Developments

In January, Celcuity entered a collaboration with Sarah Cannon Research Institute and Pfizer Inc. to conduct an open-label Phase 2 clinical trial. This trial will evaluate the efficacy and safety of two Pfizer targeted therapies, VIZIMPRO, a pan-HER inhibitor, and XALKORI, a c-Met inhibitor, in patients with previously treated metastatic HER2-negative breast cancer selected with Celcuity’s CELsignia Multi-Pathway Activity Test. Celcuity believes there is significant clinical interest in finding new diagnostic tests and targeted therapies for patients with metastatic HER2-negative breast cancer whose disease progressed on prior therapies. Patient enrollment is expected to begin in the second or third quarter of 2021 with interim results in the second half of 2022.
Celcuity raised approximately $43.0 million of gross proceeds from financings in the first quarter of 2021 and April 2021.
In late February, Celcuity completed a successful follow-on public offering that raised gross proceeds of approximately $27.6 million.
In early April, Celcuity entered into a debt financing agreement with Innovatus Life Sciences Lending Fund I, LP to provide up to $25.0 million in term loans with the first tranche of $15.0 million funded at closing. Celcuity will be able to draw on two additional tranches of $5.0 million each upon the achievement of certain clinical trial and financing milestones.
In March, Celcuity entered into a clinical trial collaboration with MD Anderson, Novartis, and Puma Biotechnology to evaluate the efficacy and safety of Novartis’ targeted therapy TABRECTA and Puma’s NERLYNX in patients with metastatic HER2-negative breast cancer selected by Celcuity’s CELsignia Multi-Pathway Activity Test. This is Celcuity’s second clinical trial to treat patients diagnosed with hyperactive HER2 and c-Met signaling breast cancers with matching targeted therapies and Celcuity now has five clinical trial collaborations in place.
In April, Celcuity entered a worldwide licensing agreement with Pfizer for the exclusive right to develop and commercialize gedatolisib. Gedatolisib is in Phase 1b clinical development for the treatment of patients with ER+/HER2-negative advanced or metastatic breast cancer. Celcuity announced preliminary data for the 103 patients enrolled in the expansion portion of the ongoing Phase 1b clinical trial evaluating gedatolisib, plus Ibrance and endocrine therapy. As of the January 11, 2021 data cut-off, 53 of the 88 evaluable patients (60%) had an objective response. Gedatolisib was also generally well tolerated, with the majority of treatment-related adverse events (TRAE) being Grade 1 or 2. The most common Grade 3 or 4 TRAEs related to gedatolisib were stomatitis and rash. Celcuity plans to meet with the FDA later this year to discuss its clinical development plans for gedatolisib.
In April, Celcuity presented results of studies evaluating gedatolisib, inavolisib (a PI3K-α inhibitor), and navitoclax (a BCL inhibitor) in breast and ovarian patient tumors in two posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The results showed that gedatolisib inhibited nine times more signaling test activity in tumors with hyperactive RAS network signaling, on average, than inavolisib, when evaluated at equal concentrations with the CELsignia test. Gedatolisib at one-fifth the concentration of inavolisib (30 nM vs. 150 nM), inhibited five times more signaling activity as quantified by the CELsignia test. Data also showed that synergistic cooperation between PI3K/mTOR and BCL signaling was detected, suggesting potential patient benefit of combining gedatolisib with a BCL inhibitor.
First Quarter 2021 Financial Results
Unless otherwise stated, all comparisons are for the first quarter ended March 31, 2021, compared to the first quarter ended March 31, 2020.

Total operating expenses were $2.79 million for the first quarter of 2021, compared to $2.31 million for the first quarter of 2020.

Research and development (R&D) expenses were $2.24 million for the first quarter of 2021, compared to $1.85 million for the first quarter of 2020. The approximately $0.39 million increase during the first three months of fiscal year 2021, compared to the first three months of fiscal year 2020, resulted from a $0.06 million increase in compensation related expenses, which included a decrease of approximately $0.04 million of non-cash stock-based compensation expense. In addition, other research and development expenses increased $0.33 million due to clinical validation and laboratory studies, and operational and business development activities.

General and administrative (G&A) expenses were $0.56 million for the first quarter of 2021, compared to $0.46 million for the first quarter of 2020. The approximately $0.09 million increase during the first three months of fiscal year 2021, compared to the first three months of fiscal year 2020, resulted primarily from a $0.08 million increase in professional fees associated with being a public company and director and officer insurance.

Net loss for the first quarter of 2021 was $2.79 million, or $0.25 per share, compared to a net loss of $2.25 million, or $0.22 per share, for the first quarter of 2020. Non-GAAP adjusted net loss for the first quarter of 2021 was $2.34 million, or $0.21 per share, compared to non-GAAP adjusted net loss of $1.78 million, or $0.17 per share, for the first quarter of 2020. Non-GAAP adjusted net loss excludes stock-based compensation expense. Because this item has 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 first quarter of 2021 was $2.52 million, compared to $1.83 million for the first quarter of 2020.

At March 31, 2021, Celcuity had cash and cash equivalents of $34.9 million, compared to cash and cash equivalents of $11.6 million at December 31, 2020. On April 8, 2021, Celcuity paid an upfront license fee of $5.0 million in conjunction with the Pfizer gedatolisib license agreement and received $14.5 million of net proceeds from a debt financing agreement. Taking into account these two events subsequent to the end of the first quarter, Celcuity has approximately $44.0 million of cash-on-hand.

Anticipated Milestones
Celcuity expects to do the following over the next twelve months:

Announce additional clinical trial collaborations in the first half of 2021 utilizing the CELsignia platform.
Initiate Phase 2/3 clinical trial for gedatolisib in breast cancer in the first half of 2022 pending discussions with the FDA regarding the clinical development pathway.
Provide interim results from the FACT-1 and FACT-2 trials in late 2021 or early 2022.
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 first quarter financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-877-407-8035 and international callers should dial 201-689-8035. A live webcast presentation can also be accessed using this weblink: View Source A replay of the webcast will be available on the Celcuity website following the live event.