On March 9, 2023 Onconova Therapeutics, Inc. (NASDAQ: ONTX), ("Onconova"), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported that the Company intends to release its fourth quarter and full year 2022 financial results on Thursday, March 16, 2023 (Press release, Onconova Therapeutics, MAR 9, 2023, View Source [SID1234628472]). Management plans to host a conference call and live webcast at 4:30 p.m. ET on the same day to discuss these results and provide an update on its pipeline programs.

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Conference Call and Webcast Information

Interested parties who wish to participate in the conference call may do so by dialing (800) 715-9871 for domestic and (646) 307-1963 for international callers and using conference ID 3097517.

Those interested in listening to the conference call via the internet may do so by visiting the investors and media page on the Company’s website at www.onconova.com and clicking on the webcast link. In addition to the live webcast, a replay will be available on the Onconova website for 90 days following the call.

On March 9, 2023 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported updates and financial results for the fourth quarter and full year ended December 31, 2022 (Press release, Poseida Therapeutics, MAR 9, 2023, View Source [SID1234628471]).

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"For Poseida, 2022 was a year of execution, progress and validation for our proprietary technology and programs in both cell and gene therapy. In cell therapy, we advanced two fully allogeneic CAR-T programs into the clinic and presented early clinical data for both programs at ESMO (Free ESMO Whitepaper) I-O in December 2022. We also established a strategic partnership with Roche, a global leader in oncology, focused on our allogeneic CAR-T platform in hematological indications," said Mark Gergen, Chief Executive Officer of the Company. "In gene therapy, our Takeda-partnered in vivo gene therapy program in Hemophilia A continued to demonstrate promise as a potential functional cure with proof of principle data presented at ASH (Free ASH Whitepaper). Along with the rest of the industry, we continue to manage through headwinds as we deal with macro-economic issues and conditions that can impact multiple aspects of our business. We remain focused on advancing our business and continuing to drive innovation as reflected at our recent R&D Day."

Program Updates

CAR-T Programs
In cell therapy, the Company is focused on three allogeneic CAR-T programs with two programs currently progressing in Phase 1 clinical trials and one expected IND during the year:

MUC1-C Program
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate targeting solid tumors derived from epithelial cells, including breast and ovarian cancers. The Company is currently evaluating P-MUC1C-ALLO1 in a Phase 1 clinical trial and presented early clinical data in December 2022 at ESMO (Free ESMO Whitepaper) I-O in Geneva, Switzerland. Data highlighted at the meeting showed that P-MUC1C-ALLO1 was well tolerated, with no dose-limiting toxicities (DLTs), cytokine release syndrome (CRS), graft vs host disease (GVHD) or immune effector cell-associated neurotoxicity syndrome (ICANS). In addition, P-MUC1C-ALLO1 showed encouraging clinical activity, including an objective partial response in a breast cancer patient at the lowest dose. The Company expects to present further clinical data updates for the program at a medical meeting in 2023.

BCMA Program
P-BCMA-ALLO1 is an allogeneic CAR-T product candidate being developed to target relapsed/refractory multiple myeloma (R/R MM) in partnership with Roche. The Company is currently evaluating P-BCMA-ALLO1 in a Phase 1 clinical trial and shared early clinical data from the program at ESMO (Free ESMO Whitepaper) I-O in December 2022. P-BCMA-ALLO1 was well tolerated, with no DLTs, CRS, GVHD or ICANS. P-BCMA-ALLO1 demonstrated responses in heavily pre-treated patients with R/R MM at the lowest dose, including in patients who had previously received BCMA-targeted therapy and patients with high-risk disease. In the fourth quarter of 2022, the Company also successfully added the ability to manufacture P-BCMA-ALLO1 at its internal GMP pilot plant in San Diego. The Company expects to present further clinical updates for the program at a medical meeting in 2023, subject to clearance with Roche.

CD19CD20 Program
P-CD19CD20-ALL01 is a preclinical allogeneic CAR-T product being developed to target B-cell malignancies in partnership with Roche. P-CD19CD20-ALL01 is the Company’s first dual CAR program and contains two fully functional CAR molecules to target cells that express either CD19 or CD20. The Company believes that by targeting both CD19 and CD20, there is potential to overcome some of the issues of earlier generation CD19 CAR-T products where antigen escape has been observed. The Company expects to file an IND for P-CD19CD20-ALLO1 in mid-2023.

Gene Therapy Programs
The Company is advancing multiple preclinical gene therapy programs in liver-directed diseases:

OTC Program
P-OTC-101 is an in vivo program for the treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene. The Company is developing the P-OTC-101 program utilizing a hybrid delivery system and working on an updated timeline for the program. The Company presented data at its R&D Day in February 2023, highlighting continued advancements in preclinical models leading towards a potential functional cure of OTC Deficiency.

FVIII Program
The Company is advancing its P-FVIII-101 preclinical program partnered with Takeda, which is in development for the in vivo treatment of Hemophilia A. P-FVIII-101 utilizes piggyBac gene modification delivered via lipid nanoparticle that has demonstrated stable and sustained Factor VIII expression in animal models. The Company presented preclinical data from this program at the ASH (Free ASH Whitepaper) annual meeting in New Orleans in December 2022, which showed that P-FVIII-101 achieved and sustained normalized (>50%) human Factor VIII activity following a single dose and delivered therapeutic Factor VIII activity in mice following single and repeat doses, indicating the potential for dose titration. The data support that, with the Company’s piggyBac delivery system, the therapeutic transgene expression cassette can be stably integrated into the genome of liver cells and provide consistent and durable therapeutic activity.

PAH Program
Announced at the Company’s R&D Day in February 2023, P-PAH-101 is a liver-directed gene therapy to treat Phenylketonuria (PKU), an inherited genetic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene resulting in buildup of phenylalanine in the body. If left untreated, PKU can affect a person’s cognitive development. P-PAH-101 utilizes piggyBac technology combined with its hybrid adeno-associated virus (AAV) and nanoparticle delivery system. The Company’s preclinical data has demonstrated the potential to resolve phenylalanine to normal levels following a single treatment in juvenile and adult mice. P-PAH-101 is partnered with Takeda and currently in preclinical development.

Expansion of Gene Therapy Scientific Advisory Board
In 2022 the Company announced the formation of its Gene Therapy Scientific Advisory Board (SAB), chaired by George M. Church, Ph.D. In March 2023, the Company announced the appointment of five new members to its Gene Therapy SAB to provide counsel on the Company’s gene therapy programs in development.

Financial Results for the Fourth Quarter and Full Year 2022
Revenues
Revenues were $10.1 million and $130.5 million for the fourth quarter and the full year ended December 31, 2022, compared to $31.2 million for both the fourth quarter and the full year ended December 31, 2021. The increase of revenues was primarily due to revenues earned from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022, and the Takeda collaboration and license agreement, which became effective in the fourth quarter of 2021.

Research and Development Expenses
Research and development expenses were $33.9 million for the fourth quarter ended December 31, 2022, compared to $39.1 million for the same period in 2021. The decrease was primarily driven by the wind-down of the Company’s P-BCMA-101 and P-PSMA-101 autologous programs as the Company transitioned to an allogeneic platform, offset by an increase in personnel expenses as a result of increased headcount, which included an increase in stock-based compensation expense.

For the full year ended December 31, 2022, research and development expenses were $152.9 million, compared to $136.7 million for the same period in 2021. The increase was primarily due to an increase in personnel expenses as a result of increased headcount, which included an increase in stock-based compensation expense, and an increase in overall active clinical programs offset by the wind-down of the Company’s P-BCMA-101 autologous program as the Company transitioned to the allogeneic program.

General and Administrative Expenses
General and administrative expenses for the fourth quarter ended December 31, 2022 and 2021, were $9.4 million and $9.6 million, respectively. The decrease was primarily related to a decrease in insurance expense, offset by an increase in personnel expenses due to an increase in headcount.

For the full year ended December 31, 2022 and 2021, general and administrative expenses were $37.5 million and $35.9 million, respectively. The increase was primarily related to an increase in personnel expenses due to an increase in headcount, which included an increase in stock-based compensation expense.

Net Income (Loss)
Net loss was $33.3 million for the fourth quarter ended December 31, 2022, compared to a net income of $1.5 million for the same period of 2021. Net loss for the full year ended December 31, 2022 and 2021, were $64.0 million and $125.0 million, respectively.

Cash Position
As of December 31, 2022, the Company’s cash, cash equivalents and short-term investments balance was $282.5 million. The Company expects that its cash, cash equivalents and short-term investments together with the remaining near-term milestones and other payments from Roche will be sufficient to fund operations into at least mid-2024.

On March 9, 2023 MorphoSys AG (FSE: MOR; NASDAQ: MOR) reported thgat it will publish its results for the financial year 2022 on March 15, 2023 at 09:00 pm CET (08:00 pm GMT; 04:00 pm EDT) (Press release, MorphoSys, MAR 9, 2023, View Source;messagePartId=0.1 [SID1234628469]).

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MorphoSys’ Management team will host a conference call and webcast on March 16, 2023 at 01:00 pm CET (12:00 pm GMT; 8:00 am EDT) to present the results for the financial year 2022 and provide an outlook for 2023.

The conference call will start with a presentation by the Management team followed by a Q&A session.

A live webcast and slides will be made available at the Investors section on MorphoSys’ website, www.morphosys.com.

To join the conference call via phone, participants may pre-register and will receive dedicated dial-in details to easily and quickly access the call via the following website:

View Source 8&linkSecurityString=469447192

On March 9, 2023 aTyr Pharma, Inc. (Nasdaq: LIFE) ("aTyr" or the "Company"), a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported fourth quarter and full year 2022 results and provided a corporate update (Press release, aTyr Pharma, MAR 9, 2023, View Source [SID1234628467]).

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"2022 was an important year for aTyr as we advanced our lead therapeutic candidate, efzofitimod, to a global pivotal Phase 3 study in patients with pulmonary sarcoidosis, the most prevalent form of interstitial lung disease (ILD)," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "This study, which is known as EFZO-FIT, is expected to be the largest interventional study in sarcoidosis to date and is currently enrolling patients at multiple centers in the U.S., Europe and Japan."

"We are also expanding the clinical development program for our novel immunomodulator efzofitimod with plans to initiate a Phase 2 proof-of-concept study in patients with systemic sclerosis (SSc, or scleroderma)-associated ILD (SSc-ILD) this year, which increases the potential market opportunity in these forms of ILD with high unmet medical need. As a result of program prioritization and proceeds from a recent public offering, we project that we will have sufficient cash to fund both of our efzofitimod clinical trials and the company’s operations into 2026. We are very proud of our progress and look forward to the year ahead as we continue our pursuit to develop a new class of medicines."

Fourth Quarter 2022 and Subsequent Period Highlights

Continued enrollment in EFZO-FIT, a global pivotal Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously once a month for a total of 12 doses. The study intends to enroll up to 264 subjects with pulmonary sarcoidosis and is currently enrolling at multiple centers in the U.S., Europe and Japan.

Kyorin Pharmaceutical Co., Ltd. (Kyorin), aTyr’s partner for the development and commercialization of efzofitimod for ILD in Japan, dosed the first patient in Japan in EFZO-FIT. This achievement triggered a $10.0 million milestone payment to aTyr in the fourth quarter. Under the collaboration and license agreement with Kyorin, aTyr has received $20.0 million in upfront and milestone payments to date and is eligible to receive up to an additional $155.0 million in the aggregate upon the achievement of certain development, regulatory and sales milestones, as well as tiered royalties on any net sales in Japan. Kyorin has the exclusive rights to develop and commercialize efzofitimod in Japan for all forms of ILD.

Announced U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for a Phase 2 randomized, double-blind, placebo-controlled, proof-of-concept study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with SSc-ILD. This is expected to be a 28-week study with three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo dosed intravenously monthly for a total of 6 doses. It is expected that the study will enroll 25 patients with progressive disease who are currently receiving background mycophenolate therapy at multiple centers in the U.S. The primary objective of the study will be to evaluate the efficacy of multiple doses of intravenous efzofitimod on pulmonary, cutaneous and systemic manifestations in patients with SSc-ILD. The Company plans to initiate this study in 2023.

Received European Union (EU) orphan drug designation for efzofitimod for the treatment of sarcoidosis from the European Commission based on the opinion of the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for EMA orphan status, a product must, among other things, be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating for which either no satisfactory method of diagnosis, prevention or treatment exists, or if such method exists, the medicine is of significant benefit to those affected by such condition. EMA orphan drug designation provides certain benefits, including the potential for 10 years of market exclusivity following market authorization, potential reduction in regulatory fees and a centralized EU market authorization process.

Announced that the Company will present data on the mechanism of action and an exposure-efficacy analysis for efzofitimod at the upcoming American Thoracic Society (ATS) 2023 International Conference, which is scheduled to take place May 19 – 24 in Washington, DC.

Received a Notice of Allowance from the European Patent Office for a patent covering the use of efzofitimod in combination with the anti-fibrotic pirfenidone for the treatment of lung inflammation or fibrosis.

Announced latent transforming growth factor beta binding protein 1 (LTBP1) as the target of a domain of aspartyl-tRNA synthetase (DARS). LTBP1 is a key regulator of transforming growth factor beta (TGF-β), a central player in the pathogenesis of fibrotic diseases. The Company expects to present additional findings around the interaction between LTBP1 and this domain of DARS at an upcoming scientific conference.

Raised gross proceeds of approximately $52.0 million through the issuance of 23,125,000 shares of common stock in February 2023 from a public offering, which included the partial exercise of the underwriters’ option to purchase additional shares.
Year Ended 2022 Financial Highlights and Cash Position

Cash & Investment Position: Cash, restricted cash, cash equivalents and investments as of December 31, 2022, were $69.3 million. Subsequent to the end of the year, the Company received a $10.0 million milestone payment in connection with the Kyorin Agreement and generated gross proceeds of approximately $52.0 million from the public offering of common stock. Based on the Company’s current operational plans and existing cash, aTyr believes the Company’s cash runway will extend into 2026.

R&D Expenses: Research and development expenses were $42.8 million for the year ended 2022, which consisted of clinical trial costs for the Phase 3 EFZO-FIT study, manufacturing costs for the efzofitimod and ATYR2810 programs, and research and development for the efzofitimod and discovery programs.

G&A Expenses: General and administrative expenses were $14.0 million for the year ended 2022.
Conference Call and Webcast Details

aTyr will host a conference call and webcast today at 5:00 p.m. EST / 2:00 p.m. PST to discuss its financial results and provide a corporate update. Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information. Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: View Source An audio replay will be available for at least 90 days following the event.

About Efzofitimod

aTyr is developing efzofitimod as a potential therapeutic for patients with fibrotic lung disease. Efzofitimod, a fusion protein comprised of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective modulator of neuropilin-2 that downregulates innate immune responses in inflammatory disease states. aTyr’s lead indication for efzofitimod is pulmonary sarcoidosis, a major form of interstitial lung disease. Clinical proof-of-concept for efzofitimod was recently established in a Phase 1b/2a multiple-ascending dose, placebo-controlled study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated safety and a consistent dose response and trends of benefit of efzofitimod compared to placebo on key efficacy endpoints, including steroid reduction, lung function, clinical symptoms and inflammatory biomarkers. aTyr is currently conducting EFZO-FIT, a Phase 3 study of efzofitimod in pulmonary sarcoidosis patients.

On March 9, 2023 Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a clinical stage, specialty pharmaceutical company focused on rare and orphan diseases, reported a business update and announces financial results for the fourth quarter and full year of 2022 (Press release, Quoin Pharmaceuticals, MAR 9, 2023, View Source [SID1234628466]).

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Quoin CEO, Dr. Michael Myers, said, "2022 as a whole was a year of significant progress for Quoin and the fourth quarter continued that trend of operational execution across a number of important metrics. We were very pleased to announce the dosing of the first patient in our ongoing double blinded, placebo controlled study in Netherton Syndrome patients. This study has continued to recruit and dose patients since then and overall interest in participation in the study remains very high.

We were also excited to announce in October our plans to initiate a second clinical trial in Netherton Syndrome patients which we believe further establishes the company’s leadership position related to this disease. Since then, Quoin has been cleared by the Food and Drug Administration (FDA) to initiate this study under the Company’s currently open Investigational New Drug (IND). This second study is investigating the safety and efficacy of QRX003 in Netherton Syndrome patients currently receiving off-label systemic therapy, primarily biologic therapy. Both of our clinical studies will be run concurrently with each other and utilize the same investigators and clinical sites, resulting in high levels of operational synergies and cost savings. As this second trial is a single arm, open label study, Quoin will have access to the data, which we look forward to sharing in the coming months. We believe Quoin is the only pharmaceutical company currently conducting two clinical trials in Netherton Syndrome patients under an open IND application.

We also continued to work closely with our 8 partners covering the 60 countries under our current commercial arrangements to determine requirements to advance QRX003 into Compassionate Use or Early Access Programs in the various local markets, in advance of formal regulatory approval. With the anticipated generation of clinical data this year, we are hopeful that this will provide a major step forward towards that goal.

Both of our dermatological rare disease research programs with Queensland University of Technology for treatment of Scleroderma and Netherton Syndrome continue to make progress under the expert scientific guidance of the program coordinators and we are working towards the initiation of clinical testing in Australia for at least one of these programs in 2H 2023.

Finally, last month we completed a public offering of our shares with aggregate net proceeds of approximately $6 million, which further extends our cash runway into 2H 2024."

Financial Highlights

Quoin ended the 2022 year with approximately $12.9 million in cash and marketable securities.
Net loss for the quarter ended December 31, 2022 was approximately $2.0 million. Net loss for the year ended December 31, 2022 was approximately $9.4 million.
Investors are encouraged to read the Company’s Annual Report on Form 10-K when it is filed with the Securities and Exchange Commission (the "SEC"), which will contain additional details about Quoin’s financial results as of December 31, 2022, and for the full year ended December 31, 2022.