On November 12, 2021 Prelude Therapeutics Inc. (Nasdaq: PRLD), a clinical-stage precision oncology company, reported its financial results for the third quarter ended September 30, 2021 and provided an update on recent clinical and development pipeline progress (Press release, Prelude Therapeutics, NOV 12, 2021, View Source [SID1234595536]).

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"We continue to make significant progress advancing our novel pipeline of therapeutic candidates, most notably with the recent presentation of dose escalation data from the Phase 1 trials of our lead PRMT5 inhibitors, PRT543 and PRT811," said Kris Vaddi, PhD, Chief Executive Officer. "We were pleased by these initial data in unselected patients, which demonstrated key points of differentiation for our molecules, including good tolerability and potency, and a desirable therapeutic window. In addition, evidence of preliminary clinical activity was observed in multiple tumor types displaying preclinically validated genomic features. We look forward to leveraging learnings from these data as we execute on the dose escalation portion of the trials and evaluate PRT543 and PRT811 in biomarker-selected patient populations, with data readouts from these cohorts anticipated in 2022. Beyond our PRMT5 inhibitors, the balance of our pipeline continues to advance. During the quarter we received IND clearance from the FDA for PRT2527, our CDK9 inhibitor, positioning us to commence a Phase 1 study of this molecule before year-end."

Recent Highlights and Upcoming Milestones

PRT543

Phase 1 Dose Escalation Study Data Presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Annual Meeting; Data from Expansion Cohorts to be Presented in 2022: In October 2021, the Company presented data from the dose escalation portion of its Phase 1 trial of PRT543, which is designed to be a potent and selective inhibitor of PRMT5, in unselected patient populations. PRT543 demonstrated target engagement and inhibition of PRMT5 functional activity, as well as preliminary clinical activity. PRT543 was generally well tolerated. Patient enrollment is ongoing in specific biomarker-selected solid tumor and hematologic malignancy expansion cohorts. The Company expects to present data from the expansion cohorts in 2022.

Phase 1 Dose Escalation Data for PRT543 in Patients with Myeloid Malignancies to be Presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting: Data from the dose escalation portion of the ongoing Phase 1 clinical trial of PRT543 in patients with myelodysplastic syndrome (MDS) and myelofibrosis (MF), including safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity, will be featured during a poster session at the 63rd ASH (Free ASH Whitepaper) Annual Meeting and Exposition being held December 11-14, 2021.

PRT811

Phase 1 Dose Escalation Study Data Presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Annual Meeting; Dose Expansion Portion of Phase 1 Trial to Commence 4Q21: In October 2021, the Company presented data from the dose escalation portion of its Phase 1 trial of PRT811, which is designed to be a potent, selective, and brain penetrant PRMT5 inhibitor, in patients with unselected advanced solid tumors. PRT811 demonstrated dose dependent inhibition of PRMT5 activity and demonstrated signs of preliminary clinical activity. PRT811 was generally well-tolerated. Prelude will shortly commence the dose expansion portion of the Phase 1 trial in selected patients with central nervous system cancers (CNS) and non-CNS cancers. The Company expects to present data from the expansion cohorts in 2022.

PRT1419

Phase 1 Dose Escalation Portion of Oral and Intravenous (IV) PRT1419 Trial Ongoing: The dose escalation portion of the Company’s first-in-human Phase 1 study investigating both an oral and IV formulation of MCL-1 inhibitor, PRT1419, the Company’s third clinical candidate, in patients with relapsed/refractory hematologic malignancies, including acute myeloid leukemia and high-risk myelodysplastic syndromes, and solid tumors, remains ongoing. The Company expects to add dose expansion and combination cohorts to the Phase 1 clinical trial in the first half of 2022.

PRT2527

Dose Escalation Phase 1 Trial of PRT2527 on Track to Begin by Year-End: During the third quarter the Company received clearance for an Investigational New Drug (IND) application for PRT2527, which is designed to be a potent and selective CDK9 inhibitor. The Company anticipates beginning a Phase 1 trial of PRT2527 by year-end evaluating IV infusion monotherapy in patients with selected solid tumors.

Preclinical Data Presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Annual Meeting: In October 2021, the Company presented new preclinical data demonstrating that intermittent intravenous administration of PRT2527 demonstrated strong efficacy in hematological malignancies and solid tumor models with MYC dysregulation.

Discovery Programs

The Company continues to expect to initiate IND-enabling studies for PRT-SCA2, which is designed to be a SMARCA2 protein degrader, by year-end. The Company also continues to make progress in the PRT-K4 discovery program and expects to initiate IND-enabling studies by year-end.

Corporate Update

On November 5, 2021, Brian Piper, our Chief Financial Officer, notified the Company that he will be resigning from the Company, effective November 19, 2021, to pursue other opportunities.

The Company reported the appointment of Laurent Chardonnet as its new Chief Financial Officer starting November 29, 2021. Mr. Chardonnet joins from Axcella Health where, since 2019, he served as Senior Vice President, CFO. Prior to Axcella, he spent 15 years at Incyte Corporation where he held roles of increasing responsibility including Vice President Finance, Treasurer and Principal Accounting Officer, Head of Finance and Administration for the company’s European division, and Vice President of Alliances and Global Strategy. Mr. Chardonnet received his Master of Business Administration from Vanderbilt University and his initial business degree from the Institut Supérieur de Gestion in Paris

The Company and Dr. David Mauro, the Company’s Chief Medical Officer, mutually agreed that Dr. Mauro would depart from the Company to pursue other opportunities. Dr. Mauro’s last day with the Company was on November 9, 2021. The Company has an ongoing search for a successor. Dr. Victor Sandor, former Chief Medical Officer of Array Biopharma and current board member and chair of the Science and Technology Committee, will provide strategic and operational oversight of clinical development during this time.

Third Quarter 2021 Financial Results

Cash, Cash Equivalents, and Marketable Securities: Cash, cash equivalents, and marketable securities as of September 30, 2021 were $320.9 million.

Research and Development (R&D) Expenses: For the third quarter of 2021, R&D expense increased by $7.4 million to $22.7 million for the three months ended September 30, 2021 from $15.3 million for the three months ended September 30, 2020. The increase was mainly due to increased clinical research costs to support the advancement of our clinical programs as well as an increase in discovery-stage program expenses. Our chemistry, manufacturing and other costs for the clinical trials also increased.

General and Administrative (G&A) Expenses: For the third quarter of 2021, G&A expense increased by $5.2 million to $8.1 million for the three months ended September 30, 2021 from $2.9 million for the three months ended September 30, 2020. The increase was primarily due to an increase in personnel related expense due to an increase in employee headcount and an increase in our professional fees as we expanded our operations to support our research and development efforts and incurred additional costs to operate as a public company.

Net Loss: For the third quarter of 2021, net loss was $30.7 million, or $0.66 per share, compared with a net loss of $16.8 million, or $5.25 per share, for the same period in 2020.

Financial Guidance: The Company believes that its current cash, cash equivalents and marketable securities will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2023.

On November 12, 2021 Celularity Inc. (Nasdaq: CELU) ("Celularity"), a clinical-stage biotechnology company developing placental-derived allogeneic cell therapies, reported financial results for the third quarter ended September 30, 2021, and provided a corporate update (Press release, Celularity, NOV 12, 2021, View Source [SID1234595535]).

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"We have continued to make significant progress over the past months, including the establishment of multiple strategic collaborations to extend further the potential uses of our technology platform, with the goal of bringing potentially transformative therapeutics to patients who require them most," said Robert J. Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity. "Moreover, we have advanced multiple preclinical and clinical programs targeting significant unmet medical needs, including acute myeloid leukemia and glioblastoma. We believe we are well-positioned to continue executing our business strategy and developing cellular therapies for cancer, infectious and degenerative diseases that leverage our proprietary placental-based platform technology."

Third Quarter Corporate Highlights

Appointed Andrew L. Pecora, M.D., F.A.C.P., C.P.E., as President.
Entered into a research collaboration with Oncternal Therapeutics (Nasdaq: ONCT) to evaluate placental derived-cellular therapies targeting receptor-tyrosine kinase-like Orphan Receptor 1 (ROR1) in preclinical studies. The collaboration will initially explore the use of Oncternal’s ROR1-targeted chimeric antigen receptor (CAR) gene modification as part of Celularity’s CYNK natural killer cell and CyCART platforms. The partnership will also explore the use of monoclonal antibody, cirmtuzumab, in combination with Celularity’s CYNK natural killer cells.
Third Quarter 2021 Financial Results

Revenues for the three months ended September 30, 2021, increased $7.3 million compared to the prior year period. This was primarily due to an increase in license, royalty and other revenues driven by (i) recognition of previously deferred revenue as a result of the termination of the Sanuwave license agreement of $6.8 million, and (ii) revenues from supply and distribution agreements recognized during the third quarter of 2021 of $1.4 million, partially offset by a decrease in product sales and rentals revenues of $0.6 million resulting from the sale of the MIST/UltraMIST assets in August 2020.
Research and development expenses for the three months ended September 30, 2021, increased $12.5 million compared to the prior year period. The increase in research and development expenses was primarily due to higher stock-based compensation expense resulting from awards granted in connection with the consummation of the business combination as well as higher clinical trial costs related to Celularity’s CYNK-001 clinical trial and higher personnel costs.
Selling, general and administrative expenses for the three months ended September 30, 2021, increased $13.3 million compared to the prior year period, primarily due to higher stock-based compensation expense resulting from awards granted in connection with the consummation of the business combination, and a $5.3 million charge related to an estimated legal settlement and higher personnel costs.
Net income for the third quarter of 2021 was $49.9 million, or $0.47 per share (basic) and $0.40 per share (diluted).
Year to Date Financial Results

Revenue for the nine months ended September 30, 2021, increased $5.4 million compared to the prior year period. The increase was due to an increase of $9.1 million in license, royalty and other revenues primarily driven by (i) recognition of previously deferred revenue as a result of the termination of the Sanuwave license agreement of $6.8 million, and (ii) revenues from supply and distribution agreements recognized during the third quarter of 2021 of $1.4 million, partially offset by a decrease in product sales and rentals revenues of $3.6 million resulting from the sale of the MIST/UltraMIST assets in August 2020.
Research and development expenses for the nine months ended September 30, 2021, increased $24.9 million compared to the prior year period. The increase in research and development expenses was primarily due to higher stock-based compensation resulting from awards granted in connection with the consummation of the business combination, higher cell therapy process development and research expenses related to the CyCART-19 program, and higher personnel costs.
Selling, general and administrative expenses for the nine months ended September 30, 2021, increased $32.8 million compared to the prior year period, primarily due to higher stock-based compensation resulting from awards granted in connection with the consummation of the business combination , a charge related to an estimated legal settlement and higher personnel costs.
Net loss for the nine months ended September 30, 2021, was $96.1 million, or $2.00 per share (basic and diluted).

On November 12, 2021 Kenjockety Biotechnology, Inc., an early-stage biotechnology company focused on discovery and development of novel therapeutics for the treatment of patients with drug-resistant cancers, reported new data for their lead BsAb program targeting Pgp and CD47 (Press release, Kenjockety Biotechnology, NOV 12, 2021, View Source [SID1234595527]). These data will be shared in a poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting in Washington, DC.

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"These results demonstrate proof-of-concept for our lead bispecific antibody which is now ready for IND enabling studies," said Robert Arathoon, PhD, Founder and CEO of Kenjockety Biotechnology. "With this novel approach we engineer bispecific antibodies to precisely target tumor but not normal cells, enabling enhanced efficacy while reducing toxicities seen with small molecules or monoclonal antibodies."

The presentation includes preclinical results demonstrating in vivo efficacy for a Pgp X CD47 BsAb, both as monotherapy and in combination with paclitaxel in multiple tumor models. Kenjockety’s unique approach enables a multi-modal mechanism of action. This is achieved by antagonizing functional aspects of both targets: inhibiting Pgp to reduce drug resistance while targeting CD47 to enhance immune attack. Additionally, Kenjockety’s results illustrate tumor-specific targeting by engineering BsAbs to have strong binding only in the presence of both targets.

"Our proprietary BASE Platform facilitates the design and engineering of new therapeutics that antagonize Efflux Pumps, like Pgp, together with immuno-oncology targets, such as CD47 or other TAAs," said Dr. Arathoon. "These therapeutics have the potential to provide significant clinical benefits for patients with a wide array of cancers."

Presentation Information

Title: Bispecific Antibodies (BsAbs) Targeting ABCB1/P-Glycoprotein (Pgp) and CD47 Provide a Multimodal, Tumor-Specific Approach to Combat Drug-Resistant Cancers

Abstract Number: 282

Date/Time: Saturday, November 13th, 7:00 a.m. – 8:30 p.m.

Session: Poster and Exhibit Hall

Presenter: Dr. Robert Arathoon, Founder and CEO, Kenjockety Biotechnology, Inc.

On November 12, 2021 Verrica Pharmaceuticals Inc. ("Verrica") (Nasdaq: VRCA), a dermatology therapeutics company developing medications for skin diseases requiring medical interventions, reported financial results for the third quarter ended September 30, 2021 (Press release, Verrica Pharmaceuticals, NOV 12, 2021, View Source [SID1234595518]).

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"We are pleased that the issues identified at the CMO unrelated to VP-102 have been successfully resolved, enabling us to move toward approval," said Ted White, Verrica’s President and Chief Executive Officer. "We remain confident in VP-102’s commercial potential. There is a high unmet medical need for molluscum treatments—the viral skin disease affects approximately 6 million people a year in the U.S., mostly children, and there are no FDA-approved treatments. We look forward to continuing our dialogue with the FDA on the appropriate path forward for approval of VP-102."

Mr. White continued: "In addition, we are excited to continue the expansion of our portfolio into dermatologic oncology by advancing LTX-315, an oncolytic peptide, into clinical development for the treatment of basal cell carcinoma. Skin cancer is the most common cancer in the U.S., with 5 million diagnoses of basal and squamous cell carcinomas each year. We recently submitted an IND for LTX-315 and look forward to initiating our Phase 2 trial in basal cell carcinoma in the first quarter of 2022."

Business Highlights and Recent Developments

On September 20, 2021, Verrica announced that the U.S. Food and Drug Administration ("FDA") issued a Complete Response Letter ("CRL") regarding its New Drug Application ("NDA") for VP-102 (cantharidin 0.7% Topical Solution) for the treatment of molluscum contagiosum ("molluscum"). According to the CRL, the FDA identified deficiencies at a facility of a contract manufacturing organization ("CMO"), which were not specifically related to the manufacturing of VP-102 but instead raised general quality issues at the facility. The FDA did not identify any clinical, safety or product specific Chemistry, Manufacturing, and Controls ("CMC") deficiencies related to VP-102. Following the CRL, on September 22, 2021 Verrica received a General Advice Letter from the FDA with recommendations to improve YCANTH’s user interface.
On November 5, 2021, Verrica was notified that the inspection of the CMO has been classified as "voluntary action indicated" ("VAI"), is now closed and that the VAI classification will not directly negatively impact FDA’s assessment of the Company’s NDA regarding this CMO. With the satisfactory resolution of the facility inspection, Verrica has engaged the FDA to determine the next steps toward the potential approval of VP-102 for the treatment of molluscum.
In October 2021, the Company submitted an Investigational New Drug Application ("IND") for LTX-315, a first-in-class oncolytic peptide, for use in basal cell carcinoma. The Company expects to initiate our Phase 2 trial in basal cell carcinoma in the first quarter of 2022.
Financial Results

Third Quarter 2021 Financial Results

Research and development expenses were $3.8 million in the third quarter of 2021, compared to $5.0 million for the same period in 2020. The decrease was primarily attributable to lower CMC (Chemistry, Manufacturing, and Controls) and clinical costs related to Verrica’s development of VP-102 for external genital warts and common warts, partially offset by increased compensation costs.
General and administrative expenses were $8.0 million in the third quarter of 2021, compared to $4.6 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 third quarter of 2021, net loss on a GAAP basis was $12.8 million, or $0.47 per share, compared to a net loss of $10.5 million, or $0.42 per share, for the same period in 2020.
For the third quarter of 2021, non-GAAP net loss was $11.0 million, or $0.40 per share, compared to a non-GAAP net loss of $9.0 million, or $0.36 per share, for the same period in 2020.
Year-to-Date September 2021 Financial Results

Verrica recognized license revenues of $12.0 million for the nine months ended September 30, 2021 related to the Collaboration and License Agreement with Torii Pharmaceutical Col, Ltd. There were no license revenues recognized in 2020.
Research and development expenses were $12.6 million for the nine months ended September 30, 2021, compared to $13.4 million for the same period in 2020. The decrease was primarily attributable to decreased CMC and clinical costs related to Verrica’s development of VP-102 for molluscum contagiosum, external genital warts, and common warts, partially offset by a one-time $2.3 million milestone payment to Lytix Biopharma AS upon the achievement of a regulatory milestone for LTX-315.
General and administrative expenses were $21.9 million for the nine months ended September 30, 2021, compared to $14.7 million for the same period in 2020. The increase was primarily a result of expenses related to increased headcount, an increase in insurance, professional fees and other operating costs, and an increase in expenses related to pre-commercial activities for VP-102.
For the nine months ended September 30, 2021, net loss on a GAAP basis was $25.5 million, or $0.95 per share, compared to a net loss of $29.7 million, or $1.19 per share, for the same period in 2020.
For the nine months ended September 30, 2021, non-GAAP net loss was $19.8 million, or $0.73 per share, compared to a non-GAAP net loss of $25.6 million, or $1.03 per share, for the same period in 2020.
As of September 30, 2021, Verrica had aggregate cash, cash equivalents, and marketable securities of $79.5 million. The Company believes that its existing cash, cash equivalents, and marketable securities as of September 30, 2021 will be sufficient to support planned operations into the third quarter of 2022.
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. If approved, VP-102 would 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. VP-102 would 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 November 12, 2021 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the third quarter ended September 30, 2021 (Press release, Applied Therapeutics, NOV 12, 2021, View Source [SID1234595517]).

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"This quarter we announced positive data in both the Galactosemia pediatric study as well as our pilot SORD Deficiency study, highlighting our execution across programs and expansion into additional indications with AT-007," said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. "We are excited to launch our registrational study in SORD later this year and remain focused on preparations for our anticipated commercial launch in Galactosemia in 2022."

Recent Highlights

Reported biomarker data from pilot trial of AT-007 in SORD deficiency. In October 2021, the Company highlighted results from a pilot open-label study in 8 SORD Deficiency patients. In the study, AT-007 reduced blood sorbitol levels by approximately 66% from baseline through 30 days of treatment with a range of reduction from baseline of 54%-75%. AT-007 was safe and well tolerated in all patients. The Company plans to initiate a registrational study by the end of 2021.

Reported pediatric biomarker data from ACTION-Galactosemia Kids. In October 2021, the Company reported pediatric biomarker data from the ACTION-Galactosemia Kids study. The results demonstrated a substantial reduction in plasma galactitol of approximately 40%, which was statistically significant (p<0.001) vs. placebo. Additionally, analysis of the 47 children in the ACTION-Galactosemia Kids study demonstrated a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels displayed greater disease severity vs. children with lower plasma galactitol levels at baseline. This data is the first demonstration of correlation of a biochemical biomarker with severity of disease in Galactosemia patients. This data will be presented as a late-breaking abstract at the 14th International Congress on Inborn Errors of Metabolism (ICIEM) November 21-24, 2021.
Financial Results

Cash and cash equivalents and short-term investments totaled $108.8 million as of September 30, 2021, compared with $125.6 million at June 30, 2021.

Research and development expenses for the three months ended September 30, 2021 were $17.6 million, compared to $19.9 million for the three months ended September 30, 2020. The decrease of $2.3 million was related to a decrease in drug manufacturing and formulation costs of $5.2 million primarily related to the completion and release of AT-001 and AT-007 drug product batches in the three months ended March 31, 2021; an increase in clinical and pre-clinical expense of $2.1 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension study and the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in personnel expenses of $0.6 million due to the increase in headcount in support of our clinical program pipeline; and an increase in regulatory and other expenses of $0.2 million.

General and administrative expenses were $10.8 million for the three months ended September 30, 2021, compared to $10.0 million for the three months ended September 30, 2020. The increase of $0.8 million was primarily related to an increase in commercial expenses of $0.8 million related to the expansion of the commercial department; an increase in other expenses of $0.3 million relating to increased costs of rent and other office expenses; an increase in stock-based compensation of $0.2 million; an increase in insurance expenses of $0.1 million related to increased insurance costs; and a decrease in legal and professional fees $0.6 million due to lower external legal fees.

Net loss for the third quarter of 2021 was $28.4 million, or $1.09 per basic and diluted common share, compared to a net loss of $29.8 million, or $1.33 per basic and diluted common share, for the third quarter 2020.