On March 23, 2021 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported financial results and operational updates for the quarter and year ended December 31, 2020 (Press release, ORIC Pharmaceuticals, MAR 23, 2021, View Source [SID1234577023]).

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"2020 was a transformational year for ORIC during which we significantly grew and advanced our pipeline, expanded the team, and strengthened the balance sheet," said Jacob Chacko, M.D., president and chief executive officer. "The progress we made in 2020 has positioned us for a dynamic 2021, with our first data from two ongoing trials of our lead program ORIC-101 and three IND/CTAs for our other product candidates, a significant amount of development activity for a company at our stage."

Fourth Quarter 2020 and Other Recent Highlights

ORIC-101: Glucocorticoid Receptor (GR) Antagonist

ORIC-101 is a potent and selective GR antagonist, with two distinct mechanisms of action being evaluated in two Phase 1b trials in combination with: (1) Abraxane (nab-paclitaxel) in advanced or metastatic solid tumors and (2) Xtandi (enzalutamide) in metastatic prostate cancer.

Completed the Part I dose escalation portion of ORIC-101 in combination with nab-paclitaxel in solid tumors, selected the recommended Phase 2 dose (RP2D), and initiated the Part II dose expansion portion of the trial. The company expects to report interim safety, efficacy, and translational data from this trial in the first half of 2021.
Completed the Part I dose escalation portion of ORIC-101 in combination with enzalutamide in metastatic prostate cancer, selected the RP2D, and initiated the Part II dose expansion portion of the trial. The company expects to report interim safety, efficacy, and translational data from this trial in the second half of 2021.
Presented preclinical data at the 32nd EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium 2020 that demonstrated ORIC-101 reversed GR-mediated resistance to an androgen receptor (AR) degrader.
ORIC-533: CD73 Inhibitor
ORIC-533 is a highly potent, orally bioavailable CD73 inhibitor and has demonstrated more potent adenosine inhibition in preclinical studies compared to an antibody approach and other small molecule CD73 inhibitors.

Conducted a preclinical collaboration with an academic key opinion leader that generated compelling single agent activity in patient derived model systems in an undisclosed tumor type. The company plans to pursue a single agent clinical development path in this indication.
ORIC-533 continues to progress in Investigational New Drug (IND) enabling studies and the company expects to file an IND with the Food and Drug Administration (FDA) in the first half of 2021.
ORIC-944: PRC2 Inhibitor
ORIC-944 is a potent and selective allosteric inhibitor of polycomb repressive complex 2 (PRC2) that targets its regulatory embryonic ectoderm development (EED) subunit and has demonstrated single agent efficacy in multiple enzalutamide-resistant prostate cancer models in preclinical studies.

Licensed exclusive worldwide development and commercialization rights from Mirati Therapeutics, Inc. in August 2020.
ORIC-944 continues to progress in IND enabling studies and the company expects to file an IND with the FDA in the second half of 2021, with initial clinical development as a single agent in treatment-resistant prostate cancer.
ORIC-114: EGFR/HER2 Inhibitor
ORIC-114 is a brain penetrant, orally bioavailable, irreversible inhibitor designed to selectively target EGFR and HER2 with high potency against exon 20 insertion mutations.

Licensed exclusive development and commercialization rights worldwide excluding the People’s Republic of China, Hong Kong, Macau and Taiwan from Voronoi, Inc. in October 2020.
ORIC-114 continues to progress in IND enabling studies and the company expects to file a Clinical Trial Application (CTA) in South Korea in the second half of 2021.
Corporate:

The company completed a public offering of common stock in November 2020, raising gross proceeds of $133.3 million.
Anticipated Milestones

ORIC anticipates the following milestones in 2021:
ORIC-101: Report interim safety, efficacy, and translational data from ongoing combination trial with nab-paclitaxel in the first half of 2021
ORIC-101: Report interim safety, efficacy, and translational data from ongoing combination trial with enzalutamide in the second half of 2021
ORIC-533: File an IND in the first half of 2021
ORIC-944: File an IND in the second half of 2021
ORIC-114: File a CTA in the second half of 2021
Present additional preclinical and translational research data on ORIC-101, ORIC-533, ORIC-944, and ORIC-114 at scientific conferences in 2021
Fourth Quarter and Full year 2020 Financial Results

Cash, Cash Equivalents and Short-term Investments: Cash, cash equivalents and short-term investments totaled $293.6 million as of December 31, 2020, which included gross proceeds of $133.3 million from the follow-on financing completed in November 2020. The company expects its current cash, cash equivalents and short-term investments will be sufficient to fund its current operating plan into the second half of 2023.

R&D Expenses: Research and development (R&D) expenses were $12.1 million for the three months ended December 31, 2020, compared to $7.0 million for the three months ended December 31, 2019, an increase of $5.1 million. For the year ended December 31, 2020, R&D expenses were $35.9 million compared to $22.8 million for the same period of 2019, an increase of $13.1 million. The increases for the 2020 periods were primarily driven by an increase in external expenses related to the advancement of ORIC-101, ORIC-533 and exploratory research programs, as well as higher personnel costs, including additional non-cash stock-based compensation of $0.4 million and $1.5 million for the three and twelve months ended December 31, 2020, as compared to the same periods in 2019, respectively.

IPR&D Expenses: In-process research and development (IPR&D) expenses of $11.9 million and $24.8 million for the three and twelve months ended December 31, 2020 related to charges the company recorded for the cash payment and fair value of common stock shares issued to Mirati and Voronoi for the development and commercialization rights to ORIC-944 and ORIC-114. There were no similar costs incurred in 2019.

G&A Expenses: General and administrative (G&A) expenses were $4.3 million for the three months ended December 31, 2020, compared to $1.8 million for the three months ended December 31, 2019, an increase of $2.5 million. For the year ended December 31, 2020, G&A expenses were $13.4 million compared to $5.7 million for the same period in 2019, an increase of $7.7 million. These increases were primarily due to higher personnel costs, including additional non-cash stock-based compensation of $0.9 million and $2.7 million for the three and twelve months ended December 31, 2020, as compared to the same periods in 2019, respectively, higher professional services and related costs to operate as a public company.

On March 23, 2021 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated agents that target the underlying mechanisms of cancer, reported financial results for the year and three months ended December 31, 2020 and a corporate update (Press release, Aptose Biosciences, MAR 23, 2021, View Source [SID1234577022]).

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The net loss for the quarter ended December 31, 2020 was $14.7 million ($0.17 per share) compared with $7.7 million ($0.13 per share) for the quarter ended December 31, 2019. The net loss for the year ended December 31, 2020 was $55.2 million ($0.67 per share), compared with $26.3 million ($0.52 per share) for the year ended December 31, 2019. Total cash and cash equivalents and investments as of December 31, 2020 were $122.4 million. Based on current operations, Aptose expects that cash on hand and available capital provide the Company with sufficient resources to fund all planned Company operations including research and development into the first half of 2023.

"During 2020, Aptose executed on our three clinical trials which are now all well under way: two studies with our cluster selective kinase inhibitor luxeptinib (CG-806) and one with our MYC repressor APTO-253," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "Dose escalation continues in each of these trials, and we and our investigators are encouraged by indicators of luxeptinib’s anti-cancer activity and safety profile. With APTO-253, we are observing consistent MYC repression, an indicator of activity that suggests future potential for broad oncology application. We look forward to providing the next complete data update at the 2021 EHA (Free EHA Whitepaper) Virtual Congress."

Separately, Aptose reported that Gregory Chow, Executive Vice President and Chief Financial Officer, is resigning to pursue an opportunity at a private biopharma company. Dr. Rice will serve as Chief Accounting Officer and Jotin Marango, M.D., Ph.D., Chief Business Officer will assume Chief Financial Officer duties until a permanent replacement is announced. In addition, Aptose and Mr. Chow intend to enter into a consulting agreement that would become effective upon his departure on March 26, 2021. "It has been an honor and a pleasure to work with Greg," said Dr. Rice. "Greg has been a trusted business partner and an integral part of the development of Aptose, and Greg departs Aptose on the best of terms and we are certain he will be successful in this next adventure."

Key Corporate Highlights

"Luxeptinib" adopted as generic name for CG-806 – The United States Adopted Name (USAN) Council recently adopted "luxeptinib" as the generic name for Aptose’s lead drug candidate CG-806, an oral cluster-selective kinase inhibitor. Aptose will use "luxeptinib" for all future references of the drug, including in scientific publications and corporate materials. After April 1, 2021, the USAN information on luxeptinib will be posted on the USAN Web site (www.ama-assn.org/go/usan).

Luxeptinib Phase 1 a/b Clinical Study in AML – In October 2020, Aptose announced dosing of the first patient with R/R acute myeloid leukemia (AML) in a Phase 1 a/b clinical study with luxeptinib, the only known clinical agent that potently inhibits both FLT3 and BTK, in addition to other driver kinases, giving it broad therapeutic potential across the spectrum of lymphoid and myeloid hematologic malignancies. Encouraging anti-leukemic activity has been observed at the first dose level of 450mg bid, including a patient with a complete response who remains on study after multiple cycles with no apparent safety signals. Aptose completed the 450 mg bid dose cohort, and has escalated to the 600 mg bid dose. Complete updated data will be presented at EHA (Free EHA Whitepaper) in June. Currently, 6 U.S. sites are open for screening and enrolling patients for the study, and more information is available at www.clinicaltrials.gov (NCT04477291).

Luxeptinib Phase 1 a/b Clinical Study in B-cell Malignancies – Aptose is treating patients at the fifth dose level of 750 mg BID in its Phase 1 a/b dose escalation study with luxeptinib in patients with B-cell malignancies, including chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphomas (NHL), who have failed or are intolerant to current therapies. To date, Aptose has observed on-target activity, including inhibition of multiple oncogenic driver kinases, lymphocytosis and tumor reductions. As previously reported at the 2020 ASH (Free ASH Whitepaper) Annual Meeting, encouraging benefit has been observed in a patient with follicular lymphoma who has been on luxeptinib for more than one year and remains on drug at this time. At the 750 mg dose, steady state plasma levels exceed 2 micromolar and to date we have observed no safety trends that we believe would prevent further dose escalation. Updated data will be presented at EHA (Free EHA Whitepaper) in June. Currently, 30 U.S. sites are open for screening and enrolling patients for the study, and more information is available at www.clinicaltrials.gov (NCT03893682).

APTO-253 Phase 1 a/b Clinical Study in AML and MDS – APTO-253 is the only known clinical-stage molecule that can directly target and inhibit expression of the MYC oncogene, shown to reprogram survival signaling pathways and contribute to drug resistance in many malignancies, including hematologic malignancies. In the ongoing Phase 1 a/b clinical study of APTO-253 in patients with relapsed or refractory AML or high-risk MDS, Aptose has escalated dosing to the fifth dose level of 150 mg/ m2. APTO-253 treatment has demonstrated MYC repression, an indicator of activity that suggests future potential for broad oncology application. The investigational drug continues to be well tolerated with no evidence of drug-related adverse events, including no observed myelosuppression. More information is available at www.clinicaltrials.gov (NCT02267863).

Aptose Expansion of Senior Leadership Team – Earlier this month, Aptose announced the appointment of George Melko, Pharm.D., as Vice President, Regulatory Affairs; and Robert Killion Jr., Ph.D. as Vice President, CMC. Dr. Melko brings more than 20 years of senior regulatory experience to Aptose, with a strong focus on oncology. Most recently he served as Vice President of Regulatory Affairs for biotechnology companies Tmunity Therapeutics and Tessa Therapeutics, which included developing regulatory strategy operations, policy and procedure design, serving as an FDA liaison and document preparation/submission. Dr. Killion has been named to the newly established position of Vice President of Chemistry, Manufacture and Control (CMC). Dr. Killion’s more than 20 years of CMC experience span roles in Relypsa, Gilead, Genentech, Roche and Syntex. The full press release can be accessed here.
RESULTS OF OPERATIONS

A summary of the results of operations for the years ended December 31, 2020 and 2019 is presented below:

Year ended December 31,
(in thousands except per Common Share data) 2020 2019

Revenues $ — $ —
Research and development expenses 29,288 16,835
General and administrative expenses 26,480 10,022
Net finance income 530 580
Net loss $ (55,238 ) $ (26,277 )
Unrealized gain/(loss) on securities available-for-sale (18 ) 18
Total comprehensive loss $ (55,256 ) $ (26,259 )
Basic and diluted loss per Common Share $ (0.67 ) $ (0.52 )

Net loss of $55.2 million for the year ended December 31, 2020 increased by approximately $28.9 million as compared with $26.3 million for the year ended December 31, 2019, primarily as a result of an increase of $19.1 million in stock-based compensation in the current period, a combined increase in program costs and related labor costs of approximately $9.8 million on our luxeptinib development program and higher cash-based general and administrative expenses of approximately $549 thousand. These expenses were partially offset by lower costs of $545 thousand on our APTO-253 development programs.

Research and Development Expenses

The research and development ("R&D") expenses for the years ended December 31, 2020 and 2019 were as follows:

R&D expenses increased by $12.5 million to $29.3 million for the year ended December 31, 2020 as compared with $16.8 million for the comparative period in 2019. Changes to the components of our R&D expenses presented in the table above are primarily as a result of the following activities:

Program costs for luxeptinib increased by approximately $7.9 million, mostly as a result of higher manufacturing costs, including costs to scale up manufacturing and research costs associated with optimizing the formulation, higher costs associated with the luxeptinib Phase 1a/b trial and the costs associated with the luxeptinib AML trial.
Program costs for APTO-253 decreased by approximately $545 thousand, mostly as a result of lower manufacturing costs and lower clinical trial costs related to the APTO-253 Phase 1a/b trial.
Personnel-related expenses increased by $1.9 million, mostly related to new positions hired since the second quarter of 2019 to support the luxeptinib Phase 1a/b and APTO-253 Phase 1a/b clinical trials and the luxeptinib AML Phase 1a/b clinical trial.
Stock-based compensation increased by approximately $3.2 million in the year ended December 31, 2020, compared with the year ended December 31, 2019, mostly related to an increase in the number of options granted during the year ended December 31, 2020 and a higher grant date fair value of options as compared with the year ended December 31, 2019, and a higher rate of forfeitures in the comparative period in 2019.
General and Administrative Expenses

The general and administrative expenses for the years ended December 31, 2020 and 2019 are as follows:

General and administrative expenses for the year ended December 31, 2020 were approximately $26.5 million as compared with $10.0 million for the comparative period in 2019, an increase of approximately $16.5 million. The increase was primarily as a result of the following:

General and administrative expenses, other than stock-based compensation and depreciation of equipment, increased by approximately $549 thousand in the year ended December 31, 2020 primarily as a result of higher personnel related costs, higher insurance costs and higher office administrative costs offset by lower financing costs and lower travel expenses.
Stock-based compensation increased by approximately $15.9 million in the year ended December 31, 2020, compared with the year ended December 31, 2019 mostly related to an increase in the number of restricted share units and options granted during the year ended December 31, 2020, and a higher grant date fair value of options as compared with December 30, 2019.
COVID-19 did not have a significant impact on our results of operations for the year ended December 31, 2020. We have not experienced and do not foresee material delays to the enrollment of patients or timelines for the luxeptinib Phase 1a/b trial due to the variety of clinical sites that we have actively recruited for this trial. Similarly, we do not expect our enrollment of the luxeptinib AML trial to be negatively impacted by COVID-19 as we plan to use a variety of clinical sites for this trial as well. APTO-253, which is administered intravenously, requires the need for hospital / clinical site resources to assist and monitor patients during each infusion and, based on the current conditions caused by COVID-19, future enrollment of patients on this trial is likely to be negatively impacted. As of the date of this report, we have not experienced material delays in the manufacturing of luxeptinib or APTO-253 related to COVID-19. Should our manufacturers be required to shut down their facilities due to COVID-19 for an extended period of time, our trials may be negatively impacted.

Conference Call and Webcast

Aptose will host a conference call to discuss results for the year and quarter ended December 31, 2020 today, Tuesday, March 23, 2021 at 5:00 PM ET. Participants can access the conference call by dialing 1-844-882-7834 (North American toll-free number) and 1-574-990-9707 (international/toll number) and using conference ID # 9179487. The conference call can be accessed here and will also be available through a link on the Investor Relations section of Aptose’s website at View Source An archived version of the webcast along with a transcript will be available on the Company’s website for 30 days. An audio replay of the webcast will be available approximately two hours after the conclusion of the call for seven days by dialing 1-855-859-2056 (toll free number) and 1-404-537-3406 (international/toll number), using the conference ID # 9179487.

The press release, the financial statements and the management’s discussion and analysis for the year and quarter ended December 31, 2020 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

On March 23, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported that it will release fourth quarter and year-end 2020 financial results Tuesday, March 30, 2021 (Press release, Forma Therapeutics, MAR 23, 2021, View Source [SID1234577021]). Forma management will host an investment community conference call at 8 a.m. Eastern Time, on March 30, 2021, to discuss these financial results and provide a business update.

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Investors may participate by dialing (833) 301-1146 in the U.S. or Canada, or (914) 987-7386 internationally, and by referring to Conference ID 5893542. A live webcast of the conference call will be available in the "News & Investors" section of Forma’s website at www.formatherapeutics.com.

On March 23, 2021 Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products, reported that Kenneth A. Berlin, President and Chief Executive Officer of Advaxis, will participate in the Benzinga Biotech Small Cap Conference to be held March 24-25, 2021 (Press release, Advaxis, MAR 23, 2021, https://ir.advaxis.com/news-releases/news-release-details/advaxis-present-corporate-overview-benzinga-biotech-small-cap [SID1234577019]). Mr. Berlin will present on March 24th, 2021 at 2:30 pm ET.

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Investors can view the presentation and request a 1×1 meeting once they register for the conference here.

On March 23, 2021 Lantern Pharma Inc. (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform oncology drug discovery and development, reported that it will present new data on LP-184, a next-generation, targeted small molecule in the acylfulvene drug class, showcasing its potency across various 2D and 3D models of prostate cancer, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting, taking place from April 10-15, 2021 (Press release, Lantern Pharma, MAR 23, 2021, View Source;utm_medium=rss&utm_campaign=new-data-supporting-lp-184-advancement [SID1234577018]).

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Lantern’s poster will highlight the spectrum of DNA damage repair defects, occurring in 25-30% of metastatic castration resistant prostate cancer (mCRPC) patients, against which LP-184 shows nanomolar potency. "In vitro studies of LP-184 continue to show potentially best in class efficacy in metastatic prostate cancer with a novel mechanism of action," stated Panna Sharma, CEO and President of Lantern Pharma. "Our ongoing work of LP-184 in metastatic prostate cancer in collaboration with Georgetown University has shown effectiveness in tumor models with mutations in DNA damage repair pathway components including in genes involved in Homologous Recombination and Transcription Coupled-Nucleotide Excision Repair. We look forward to sharing this new data at AACR (Free AACR Whitepaper) and developing this compound towards the clinic and for the benefit of prostate cancer patients."

Details of the abstract are as follows:

Title: LP184, a novel alkylating agent, is efficacious in prostate cancer models with DNA damage repair defects
Session Category: Experimental and Molecular Therapeutics
Session Title: Novel Antitumor Agents
Permanent Abstract Number: 1249
The full text of the abstract is now available on the AACR (Free AACR Whitepaper) Annual Meeting 2021 website.
The accompanying poster will be available to view to registered participants during the conference via the AACR (Free AACR Whitepaper) e-poster website on April 10, 2021, and published on the company website after the conference.