On August 10, 2021 Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genomically defined cancers, reported financial results for the second quarter of 2021 and highlighted recent corporate achievements (Press release, Day One, AUG 10, 2021, View Source [SID1234586230]).

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"Day One made significant progress across multiple clinical and corporate initiatives during the second quarter of 2021, including dosing the first patients in our ongoing FIREFLY-1 pivotal study of DAY101 in pediatric low-grade glioma," said Jeremy Bender, Ph.D., chief executive officer of Day One. "The success of our recent IPO reflects a strong commitment from our investors who, like all of us at Day One, recognize the therapeutic potential of DAY101. Entering the second half of 2021, we remain well positioned to advance our pipeline through key data readouts with the goal of fulfilling our mission of developing novel medicines to improve the lives of patients of all ages living with cancer." Program Highlights The Company announced first patients dosed in the FIREFLY-1 pivotal clinical trial of DAY101 in pediatric low-grade glioma (pLGG). FIREFLY-1 is being conducted in collaboration with the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and is designed to support the regulatory approval of DAY101. Initial data from FIREFLY-1 is expected in the first half of 2022. Day One has initiated a Phase 2 monotherapy trial of DAY101 in adult patients with recurrent, progressive, or refractory solid tumors harboring MAPK pathway aberrations. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to DAY101 for the treatment of low-grade gliomas harboring an activating RAF alteration that disproportionately affects children. If a New Drug Application in the United States for DAY101 is approved, Day One may be eligible to receive a Priority Review Voucher (PRV) from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be sold or transferred. The European Commission granted DAY101 Orphan Designation for the treatment of glioma based upon a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. Corporate Highlights The Company announced the successful closing of its upsized initial public offering, raising gross proceeds of $184.0 million, bringing total cash, cash equivalents and marketable securities to $310.0 million at the end of June 30, 2021. The company expects its current cash position to fund operations into the second half of 2023 and through key clinical milestones. Day One appointed Saira Ramasastry to its Board of Directors. Ms. Ramasastry currently serves as the Managing Partner of Life Sciences Advisory, LLC, and brings more than 20 years of experience to the Board as a life sciences-focused strategic consultant and investment banker. Second Quarter 2021 Financial Highlights Cash Position: Cash and cash equivalents and short-term investments totaled $310.0 million at June 30, 2021. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into the second half of 2023. R&D Expenses: Research and development expenses were $9.9 million for the second quarter 2021 and $1.4 million for the second quarter 2020. The increase was primarily due to additional employee compensation costs, clinical trial expenses, CMC activity and a milestone payment for DAY101. G&A Expenses: General and administrative expenses were $5.5 million for the second quarter 2021 and $0.9 million for the second quarter 2020. The increase was primarily due to additional employee compensation costs, legal, and professional expenses associated with being a public company. Net Loss: Net loss totaled $15.5 million and $2.4 million for the second quarter 2021 and 2020, respectively, with non-cash stock compensation expense of $2.5 million and $0.1 million for the second quarter of 2021 and 2020, respectively. Upcoming Events 12th Annual Wedbush PacGrow Healthcare Conference: Day One’s chief executive officer Jeremy Bender will be a participant on the Panel, "Bullseye – Targeted Oncology Part 2". The panel discussion will take place on Wednesday, August 11th at 10:20 am ET. Day One will also be available for one-on-one investor meetings during the conference. About DAY101 DAY101 is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. Studies have shown DAY101 has high brain distribution and exposure in comparison to other MAPK pathway inhibitors, thus potentially benefiting patients with primary brain tumors or brain metastases of solid tumors. DAY101 is a type II RAF inhibitor found to selectively inhibit both monomeric and dimeric RAF kinase, which may broaden its potential clinical application to treat an array of RAF-altered tumors. DAY101 has been studied in over 250 patients, and as a monotherapy demonstrated good tolerability and encouraging anti-tumor activity in pediatric and adult populations with specific MAPK pathway-alterations. In November 2020, Day One announced preliminary results from PNOC014, an ongoing Phase 1 Pacific Pediatric Neuro-Oncology Consortium (PNOC) network study with DAY101 sponsored by the Dana-Farber Cancer Institute. Preliminary results demonstrated that of the eight relapsed pLGG patients in the study with RAF fusions, two patients achieved a complete response by Response Assessment for Neuro-Oncology (RANO), three had a partial response, two achieved prolonged stable disease, and one experienced progressive disease. DAY101 also demonstrated a tolerable safety profile with the most common side effects being skin rash and hair color changes. DAY101 has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration who require systemic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options. The FDA has also granted Rare Pediatric Disease Designation to DAY101 for the treatment of low-grade gliomas harboring an activating RAF alteration that disproportionately affects children. In addition, DAY101 has received Orphan Drug designation from the FDA for the treatment of malignant glioma and orphan designation from the European Commission for the treatment of glioma. Day One is conducting a pivotal Phase 2 trial (FIREFLY-1) of DAY101 in pediatric, adolescent and young adult patients with pLGG. Day One also plans to study DAY101 alone or in combination with other agents that target key signaling nodes in the MAPK pathway, such as the Company’s MEK inhibitor pimasertib, in patient populations where various RAS and RAF alterations are believed to play an important role in driving disease.

On August 10, 2021 Tesis Labs, a leader in targeted genetic sequencing, and Personal Genome Diagnostics Inc. (PGDx), a leader in cancer genomics, reported a new collaboration to maximize the power of genetic sequencing and bioinformatics (Press release, Personal Genome Diagnostics, AUG 10, 2021, View Source [SID1234586269]). Through this collaboration, the companies intend to combine resources and expertise to create new genomics solutions that could combat cancer and improve outcomes for patients, and advance market access initiatives to accelerate adoption in the market.

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The National Institutes of Health estimates that by 2040, there will be 29.5 million new cancer cases and nearly 16.4 million deaths worldwide per year. This new strategic collaboration brings together highly specialized clinical laboratory and biotechnology teams, enabling their expertise, creativity, and passion for delivering the latest genetic and bioinformatic insights to advance cancer predisposition, profiling, and treatment.

"We are excited to partner with PGDx and leverage our aligned vision and collective expertise to enable new and creative clinical opportunities," stated Ron King, Tesis Labs CEO. "Genetic sequencing and genetic biomarkers are revealing new opportunities for medicine. Our clinical expertise and approaches to unlocking the power of genetic sequencing are bringing new insights to cancer research, profiling and treatment."

"We are thrilled to partner with Tesis Labs, a company that shares our passion for expanding the reach of genetic sequencing through decentralized testing," said Megan Bailey, Chief Executive Officer of PGDx. "We look forward to working with Tesis Labs to further progress advancements in genomic insights and patient-centered oncology care."

Tesis uses a genetically integrated medical platform for targeted genetic sequencing and comprehensive genetic data collection to support many medical specialties. The company’s existing labs are in Denver, Lafayette, Colo., and Houston.

PGDx currently offers three pan-cancer NGS kitted solutions – elio tissue complete, an FDA cleared kit, elio plasma complete, a comprehensive liquid biopsy solution, and elio plasma resolve, which has received FDA breakthrough device designation – that provide researchers and clinicians with the ability to identify biomarkers and profile tumors through advanced genomic sequencing within their own hospital systems and laboratories.

On August 10, 2021 Verrica Pharmaceuticals Inc. ("Verrica") (Nasdaq: VRCA), a dermatology therapeutics company developing medications for skin diseases requiring medical interventions, reported financial results for the second quarter ended June 30, 2021 (Press release, Verrica Pharmaceuticals, AUG 10, 2021, View Source [SID1234586203]).

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"This quarter, we continued to ramp up commercial preparations for the potential FDA approval of VP-102, our lead product candidate for the treatment of molluscum contagiosum, including strengthening our senior leadership team and ensuring patient access to VP-102 through productive dialogue with medical providers and payors," said Ted White, Verrica’s President and Chief Executive Officer. "With a strong financial position, and a PDUFA goal date of September 23, 2021, we continue to invest in our commercial capabilities and, if approved, we look forward to the opportunity to launch VP-102 in the fourth quarter of 2021."

Business Highlights and Recent Developments

In May 2021, the Company announced that the U.S. Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) goal date for the New Drug Application (NDA) for VP-102 (cantharidin 0.7% Topical Solution) for the treatment of molluscum contagiosum by three months to September 23, 2021 to allow the Agency additional time to review information requested and submitted regarding the Company’s training program and distribution model.
The Company continued to expand its U.S. commercial operations during the quarter in preparation for the potential FDA approval of VP-102, and has made key hires in marketing, sales and payor functions to support product launch and commercialization. The Company will be focusing its sales efforts in Dermatology, Pediatric Dermatology and key academic centers and health systems.
The Company strengthened its management team in anticipation of the potential commercial launch of VP-102 with the appointment of Terry Kohler as Chief Financial Officer, effective July 16, 2021. Mr. Kohler is a strategic and operational finance leader with over 20 years of commercial business experience, most recently at a global pharmaceutical company with annual revenues over $2 billion.
The Company continues to prepare to submit an Investigational New Drug Application for LTX-315 in the second half of 2021 for use in all malignant and pre-malignant dermatological indications, other than metastatic melanoma and metastatic merkel cell carcinoma.
Financial Results

Second Quarter 2021 Financial Results

Research and development expenses were $3.4 million in the second quarter of 2021, compared to $3.5 million for the same period in 2020. The decrease was primarily attributable to lower clinical costs related to Verrica’s development of VP-102 for external genital warts and common warts.
General and administrative expenses were $7.3 million in the second quarter of 2021, compared to $5.1 million for the same period in 2020. The increase was primarily driven by increased headcount and other expenses related to pre-commercial activities for VP-102, as well as an increase in insurance, professional fees and other operating expenses.
For the second quarter of 2021, net loss on a GAAP basis was $11.8 million, or $0.43 per share, compared to a net loss of $9.4 million, or $0.38 per share, for the same period in 2020.
For the second quarter of 2021, non-GAAP net loss was $9.6 million, or $0.35 per share, compared to a non-GAAP net loss of $7.9 million, or $0.32 per share, for the same period in 2020.
Year-to-Date June 2021 Financial Results

Verrica recognized license revenues of $12.0 million for the six months ended June 30, 2021 related to the Collaboration and License Agreement (the "Torii Agreement") with Torii Pharmaceutical Co., Ltd ("Torii"). There were no license revenues recognized in 2020.
Research and development expenses were $8.8 million for the six months ended June 30, 2021, compared to $8.4 million for the same period in 2020. The increase was primarily attributable to a one-time $2.3 million milestone payment to Lytix Biopharma AS upon the achievement of a regulatory milestone for LTX-315, partially offset by decreased Chemistry, Manufacturing and Controls ("CMC") and clinical costs related to Verrica’s development of VP-102 for molluscum contagiosum, external genital warts, and common warts.
General and administrative expenses were $13.9 million for the six months ended June 30, 2021, compared to $10.1 million for the same period in 2020. The increase was primarily driven by increased headcount and other expenses related to pre-commercial activities for VP-102, as well as an increase in insurance, professional fees and other operating expenses.
For six months ended June 30, 2021, net loss on a GAAP basis was $12.7 million, or $0.46 per share, compared to a net loss of $19.2 million, or $0.77 per share, for the same period in 2020.
For the six months ended June 30, 2021, non-GAAP net loss was $8.8 million, or $0.32 per share, compared to a non-GAAP net loss of $16.7 million, or $0.67 per share, for the same period in 2020.
As of June 30, 2021, Verrica had aggregate cash, cash equivalents, and marketable securities of $90.1 million. The Company believes that its existing cash, cash equivalents, and marketable securities as of June 30, 2021 will be sufficient to support planned operations at least into the first quarter of 2023.
Non-GAAP Financial Measures

In evaluating the operating performance of its business, Verrica’s management considers non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude stock-based compensation charges and non-cash interest expense that are required by GAAP. Verrica believes that non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share provides useful information to both management and investors by excluding the effect of certain non-cash expenses and items that Verrica believes may not be indicative of its operating performance, because either they are unusual and Verrica does not expect them to recur in the ordinary course of its business, or they are unrelated to the ongoing operation of the business in the ordinary course. non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share should be considered in addition to results prepared in accordance with GAAP, but should not be considered a substitute for, or superior to, GAAP results. Non-GAAP loss from operations, non-GAAP net loss and non-GAAP net loss per share have been reconciled to the nearest GAAP measure in the tables following the financial statements in this press release.

About VP-102

Verrica’s lead product candidate, VP-102, is a proprietary drug-device combination product that contains a GMP-controlled formulation of cantharidin (0.7% w/v) delivered via a single-use applicator that allows for precise topical dosing and targeted administration. VP-102 is currently under U.S. Food and Drug Administration (FDA) review, with a PDUFA goal date of September 23, 2021, and could potentially be the first product approved by the FDA to treat molluscum contagiosum — a common, highly contagious skin disease that affects an estimated six million people in the United States, primarily children. If approved, VP-102 will be marketed in the United States under the conditionally accepted brand name YCANTH. In addition, Verrica has successfully completed a Phase 2 study of VP-102 for the treatment of common warts and a Phase 2 study of VP-102 for the treatment of external genital warts.

About Molluscum Contagiosum (Molluscum)

There are currently no FDA-approved treatments for molluscum, a highly contagious viral skin disease that affects approximately six million people — primarily children — in the United States. Molluscum is caused by a pox virus that produces distinctive raised, skin-toned-to-pink-colored lesions that can cause pain, inflammation, itching and bacterial infection. It is easily transmitted through direct skin-to-skin contact or through fomites (objects that carry the disease like toys, towels or wet surfaces) and can spread to other parts of the body or to other people, including siblings. The lesions can be found on most areas of the body and may carry substantial social stigma. Without treatment, molluscum can last for an average of 13 months, and in some cases, up to several years.

On August 10, 2021 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology," Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted therapies for women’s cancers, reported second quarter financial results for the period ended June 30, 2021 (Press release, Olema Oncology, AUG 10, 2021, View Source [SID1234586231]).

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"We made important progress in the second quarter of 2021 as we advanced the clinical development of our lead candidate, OP-1250, an investigational complete estrogen receptor (ER) antagonist (CERAN), and strengthened our corporate foundation to ensure that we have the talent and resources in place to support our future success," said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. "We have seen robust enrollment in the ongoing Phase 1/2 clinical trial of OP-1250 and look forward to sharing interim dose-escalation data at a medical meeting in the fourth quarter of this year."

Corporate Highlights and Anticipated Milestones

Significant progress advancing the Phase 1/2 clinical trial of OP-1250 in patients with metastatic, ER+ / HER2- breast cancer. As of June 4, 2021, 28 patients have been enrolled across five dose-escalation cohorts. OP-1250 has demonstrated oral bioavailability and a dose-proportional pharmacokinetic profile consistent with predictions from Olema nonclinical models. A maximum tolerated dose has not been identified. The Company plans to present interim safety, tolerability, pharmacokinetic and initial efficacy data at a medical meeting in the fourth quarter of 2021, pending abstract acceptance.
Advance into monotherapy dose expansion in the second half of 2021.
Initiate a Phase 1b clinical trial of OP-1250 in combination with a CDK4/6 inhibitor in the first quarter of 2022.
Second Quarter 2021 Financial Highlights

Cash, cash equivalents and marketable securities as of June 30, 2021 were $318.1 million. Olema anticipates that this cash balance will be sufficient to fund operations through the end of 2023.
Research and development (R&D) expenses were $11.9 million for the quarter ended June 30, 2021, compared to $1.9 million for the same period of the prior year. The increase in R&D expenses was primarily due to increased expenditures to advance the Phase 1/2 clinical trial of OP-1250, the increase in nonclinical development activities, higher personnel-related expenses as headcount grew to support the advancement of the clinical and nonclinical programs, and higher non-cash stock-based compensation expenses.
General and administrative (G&A) expenses were $4.6 million for the quarter ended June 30, 2021, compared to $0.5 million for the same period of the prior year. The increase in G&A expenses was primarily due to higher personnel-related expenses associated with increases in the number of G&A personnel supporting the growth of the organization, public company-related expenses and other corporate costs, and non-cash share-based compensation expenses.
Net loss for the quarter ended June 30, 2021 was $16.4 million, compared to $2.5 million for the same period of the prior year.

On August 10, 2021 aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotherapeutics company, reported that the company has expanded its research collaboration with The Ohio State University (OSU) to deepen the understanding of the immune mechanisms of sarcoid granuloma formation and identify potential biomarkers of efficacy for the company’s lead therapeutic candidate, ATYR1923, which is currently in clinical development for the treatment of pulmonary sarcoidosis (Press release, aTyr Pharma, AUG 10, 2021, View Source [SID1234586253]). The research will be conducted in the laboratory of Elliott Crouser, M.D., Professor of Pulmonology, Critical Care and Sleep Medicine at OSU. Dr. Crouser specializes in sarcoidosis research and treatment and will serve as the principal investigator.

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The collaboration, which expands upon a successful pilot proof-of-concept study, will assess the effect of ATYR1923 on sarcoid granuloma formation in vitro in blood samples taken from sarcoidosis patients. The study will focus on identifying the relevant immune mechanisms triggered in granuloma formation and analyze promising biomarkers predictive of strong granuloma formation in order to assess whether they could be used as predictive biomarkers for treatment selection or treatment response to ATYR1923.

"We look forward to working with aTyr on this important initiative to expand the current understanding of the underlying mechanisms involved in pulmonary sarcoidosis, particularly the formation of granulomas. This work has the potential to identify promising biomarkers that may be used to predict treatment response, including to ATYR1923. Current treatment options for pulmonary sarcoidosis are limited, and the ability to determine a patient population that may benefit from a potential treatment such as ATYR1923 presents the opportunity to take a much-needed step forward in managing this disease," said Dr. Crouser.

"We are very pleased to expand this research collaboration with OSU and Dr. Crouser. This collaboration will build upon the successful findings from research conducted with Dr. Crouser that were recently accepted to be presented at the upcoming European Respiratory Society International Congress in September, which demonstrate the ability of a splice variant of histidyl-tRNA synthetase, the active portion of ATYR1923, to disrupt sarcoid granuloma formation in vitro — a hallmark of this debilitating disease," said Sanjay Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "The research generated from this collaboration may help direct us to biomarkers indicative of a population that may be sensitive to treatment with ATYR1923, which could lead to improved patient outcomes."

Sarcoidosis is an inflammatory disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body. Sarcoidosis in the lungs is called pulmonary sarcoidosis and occurs in more than 90% of all sarcoidosis patients. Approximately 150,000 to 200,000 Americans live with pulmonary sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, permanent loss of lung function and death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with serious side-effects when used long-term that many patients cannot tolerate.

Dr. Crouser received his medical degree from the Medical College of Ohio at Toledo, OH. He completed an internship, residency and fellowship at The Ohio State University Wexner Medical Center in Columbus, OH. He is board certified in Internal Medicine with subspecialty certifications in Pulmonary Disease and Critical Care. He is a leader in sarcoidosis research and treatment and currently serves as the Chair of the Foundation for Sarcoidosis Research’s Scientific Advisory Board. In 25 years, his laboratory has contributed to the publication of more than 100 peer-reviewed manuscripts, including the first clinical practice guidelines for sarcoidosis, which were endorsed by the American Thoracic Society in 2020. Ohio State’s Sarcoidosis Specialty Clinic was named a Center of Excellence in 2020 by the World Association of Sarcoidosis and Other Granulomatous Disorders.

About ATYR1923

aTyr is developing ATYR1923 as a potential therapeutic for patients with severe inflammatory lung diseases. ATYR1923, a fusion protein comprised of the immuno-modulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective modulator of neuropilin-2 that downregulates the innate and adaptive immune response in inflammatory disease states. aTyr has completed enrollment in a proof-of-concept Phase 1b/2a trial evaluating ATYR1923 in patients with pulmonary sarcoidosis. This Phase 1b/2a study is a multi-ascending dose, placebo-controlled, first-in-patient study of ATYR1923 that has been designed to evaluate the safety, tolerability, steroid sparing effect, immunogenicity and pharmacokinetic profile of multiple doses of ATYR1923. Proof-of-mechanism for ATYR1923 was established in a Phase 2 clinical trial in COVID-19 patients with severe respiratory complications, which demonstrated that ATYR1923 reduced inflammatory cytokine levels in patients consistent with preclinical models, including cytokines that are implicated in sarcoidosis and other forms of interstitial lung disease.