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Caladrius Biosciences Provides Corporate Update and Reports 2021 Third Quarter Financial Results

On November 4, 2021 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of innovative therapies designed to treat or reverse disease, reported a corporate update and financial results for the three and nine months ended September 30, 2021 (Press release, Caladrius Biosciences, NOV 4, 2021, View Source [SID1234594425]).

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"Caladrius continued to advance and optimize its development programs in the third quarter despite the ongoing challenges to clinical development posed by the global COVID-19 pandemic. A cash position of approximately $100 million, coupled with continued prudent cash management, enabled the Company to focus on refining and executing its development plans while identifying and evaluating attractive strategic corporate development opportunities," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "Additionally, Caladrius strengthened and further diversified its operations and management team with the addition of several highly qualified and experienced personnel, including the appointment of veteran drug developer, Kristen K. Buck, M.D., as Executive VP of R&D and Chief Medical Officer. Dr. Buck will play a key role in defining, optimizing, and implementing development strategies that maximize the probability of clinical and commercial success of existing programs. She will also contribute to one of our corporate priorities of adding promising new assets to our development portfolio by leading our technical evaluation efforts."

Product Development and Financing Highlights

HONEDRA (CLBS12) for the treatment of critical limb ischemia ("CLI")

With respect to HONEDRA, the Company’s SAKIGAKE-designated product candidate for the treatment of CLI and Buerger’s disease in Japan, the headwinds to enrollment in the ongoing registration-eligible trial, as discussed in prior quarters, have persisted. The multiple states of emergency declared by the Japanese government over the past 18 months due to the COVID-19 pandemic have made incremental enrollment exceedingly challenging, prompting Caladrius to consider alternate approaches to achieving development success. Since the trial continues to demonstrate positive trends in both safety and efficacy, the key criteria for consideration of conditional approval in Japan under the SAKIGAKE designation, the Company has decided to suspend enrollment activities in favor of focusing efforts in Japan on securing a partner to complete study enrollment with four remaining patients, if necessary, and/or to explore the possibility of submitting the existing data to the Japanese Regulatory Authorities under the SAKIGAKE designation. This decision is motivated by the Company’s desire to minimize additional operational and financial burden caused by enrollment delays and the lack of visibility on time to completion.

XOWNA (CLBS16) for the treatment of coronary microvascular dysfunction ("CMD")

XOWNA is an experimental regenerative therapy for the treatment of CMD. It was the subject of a recently reported positive Phase 2a study (the "ESCaPE-CMD trial") and is currently being evaluated in a U.S. Phase 2b study (the "FREEDOM Trial"). The FREEDOM Trial is a double-blind, randomized, placebo-controlled trial designed to assess the efficacy and safety of delivering autologous CD34+ cells (XOWNA) to subjects with CMD and without obstructive coronary artery disease. Early enrollment in the FREEDOM Trial proceeded as planned with the first patient treated in January 2021, however, the impact of the COVID-19 pandemic in the U.S. since then has contributed to a general slowing of enrollment. Caladrius has taken steps to accelerate enrollment by expanding the number of participating investigational sites and modifying the study protocol. These protocol amendments were implemented in the latter part of the quarter after agreement with the U.S. Food and Drug Administration, and we will assess their impact on enrollment in the coming months, at which time the Company expects to be in a better position to provide an informed estimate for enrollment feasibility and completion. Final data from the study are expected approximately 6 months after last patient/last visit in the study.

CLBS201 for the treatment of diabetic kidney disease ("DKD")

Progressive kidney failure is associated with attrition of the microcirculation of the kidney. Pre-clinical studies in kidney disease and injury models have demonstrated that protection or replenishment of the microcirculation results in improved kidney function. Based on these observations, the Company plans to initiate a Phase 1, open-label, proof-of-concept trial evaluating CLBS201, a CD34+ regenerative cell therapy investigational product for intra-renal artery administration, in patients with DKD. Although still pre-dialysis, these patients exhibit rapidly progressing stage 3b disease. The protocol, pending final approval from the Institutional Review Board, will be a staggered, sequentially dosed cohort of six patients overseen by an independent Data Safety Monitoring Board with the objective of determining the tolerance of intra-renal cell therapy injection in DKD patients and the ability of CLBS201 to regenerate kidney function. A key read-out of data will occur at the 6-month follow-up visit for all patients. The Company projects enrollment of this study to begin in the first quarter of 2022 with data from all subjects expected by the first quarter of 2023.

Third Quarter 2021 Financial Summary

Research and development expenses were approximately $4.1 million for the three months ended September 30, 2021, compared to $3.0 million for the three months ended September 30, 2020, representing an increase of 36%. Research and development in both periods focused on the advancement of our ischemic repair platform and related to:

Expenses associated with efforts to continue execution and acceleration of enrollment of the FREEDOM Trial;

Expenses associated with the planning and preparation of an IND and Phase 1 proof-of-concept protocol for CLBS201 as a treatment for DKD; and

Ongoing expenses for HONEDRA in CLI and Buerger’s disease in Japan associated with maintenance of manufacturing facility and personnel qualification as well as Contract Research Organization engagement despite no subject treatment execution due to the COVID-19 imposed state of emergency in Japan.
General and administrative expenses were approximately $2.8 million for the three months ended September 30, 2021, compared to $2.3 million for the three months ended September 30, 2020, representing an increase of 22%. This increase was primarily due to an increase in directors and officers insurance premiums and strategic consulting expenses.

Overall, net losses were $6.9 million for the three months ended September 30, 2021, compared to $5.3 million for the three months ended September 30, 2020.

Balance Sheet Highlights

As of September 30, 2021, we had cash, cash equivalents and marketable securities of approximately $100.1 million. Based on existing programs and projections, the Company remains confident that its current cash balances will fund its operations for the next several years. Based on this favorable cash position, the Company continues its concerted efforts to identify and acquire additional development assets to diversify our portfolio of product candidates and to enhance the opportunity for near-term shareholder value creation.

Conference Call

Caladrius will hold a live conference call today, November 4, 2021, at 4:30 p.m. (ET) to discuss financial results, provide a business update and answer questions. To join the conference call, please refer to the dial-in information provided below. A live webcast of the call will also be available under the Investors & News section of the Caladrius website, View Source, and will be available for replay for 90 days after the conclusion of the call.

Please dial-in 10 minutes before the conference call starts.

For those unable to participate on the live conference call, an audio replay will be available that day starting at 7:30 p.m. (ET) until November 11, 2021, by dialing 855-859-2056 (U.S. Toll-Free) or 404-537-3406 (International) and by entering the replay passcode: 8378269.

Acceleron Reports Third Quarter 2021 Financial Results

On November 4, 2021 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the third quarter ended September 30, 2021 (Press release, Acceleron Pharma, NOV 4, 2021, View Source [SID1234594424]).

Corporate Highlights

On September 30, Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Acceleron Pharma Inc. announced that the companies entered into a definitive agreement under which Merck, through a subsidiary, will acquire Acceleron for $180 per share in cash for an approximate total equity value of $11.5 billion.
Program Highlights

Pulmonary

Sotatercept: Pulmonary Hypertension

Sotatercept acts as an investigational reverse-remodeling agent proposed to rebalance TGF-beta superfamily signaling. In preclinical models of pulmonary arterial hypertension (PAH), sotatercept reversed pulmonary arterial wall and right ventricular remodeling that are hallmarks of the disease.

Enrollment remains ongoing in the STELLAR Phase 3 trial of sotatercept in patients with PAH.
In August, Acceleron initiated the HYPERION Phase 3 trial of sotatercept in newly diagnosed patients with intermediate- and high-risk PAH.
The Company recently initiated the ZENITH Phase 3 trial of sotatercept in patients with WHO functional class III or IV PAH at high risk of mortality.
Study start up activities are underway for the CADENCE Phase 2 trial of sotatercept in patients with pulmonary hypertension due to left heart disease.
ACE-1334: Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD)

ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis.

In October, Acceleron initiated a Phase 1b study to evaluate the activity of ACE-1334 in patients with SSc-ILD.
Hematology

REBLOZYL (luspatercept-aamt):

REBLOZYL is the first and only erythroid maturation agent approved in the United States, Europe, and Canada designed to promote late-stage red blood cell (RBC) production. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

Acceleron recognized approximately $32.0 million in royalty revenue from approximately $160 million in net sales of REBLOZYL in the third quarter of 2021. This compares with approximately $25.6 million in royalty revenue from approximately $128 million in net sales of REBLOZYL in the second quarter of 2021.
Net sales of REBLOZYL for the third quarter include approximately $20 million to $25 million of sales from an inventory build due to the transition to wholesaler distribution and approximately $13.5 million of net sales outside of the United States.
In September, the United States Food and Drug Administration granted luspatercept Orphan Drug designation for the treatment of anemia in patients with alpha-thalassemia.
Enrollment remains ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS.
Enrollment remains ongoing in the INDEPENDENCE Phase 3 trial in patients with anemia-associated with myelofibrosis.
Three clinical abstracts on REBLOZYL have been accepted for presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition scheduled for December 11 to 14, 2021.
Financial Results

Cash Position – Cash, cash equivalents and investments as of September 30, 2021 were $652.5 million, compared with $857.5 million as of December 31, 2020. Based on Acceleron’s current operating plan and projections, the Company believes that its current cash, cash equivalents and investments, along with the expected royalty revenue from REBLOZYL sales, will be sufficient to fund the Company’s projected operating requirements for the foreseeable future.
Revenue – Revenue for the third quarter of 2021 was $34.2 million, which includes $2.2 million of cost share revenue and $32.0 million of royalty revenue from net sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
R&D Expenses – GAAP R&D expenses were $59.7 million for the third quarter of 2021. Non-GAAP R&D expenses were $53.1 million for the third quarter of 2021, excluding $5.6 million and $1.0 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
SG&A Expenses – GAAP SG&A expenses were $45.1 million for the third quarter of 2021. Non-GAAP SG&A expenses were $39.1 million for the third quarter of 2021, excluding $5.8 million and $0.1 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Net Loss – The Company’s GAAP net loss for the third quarter of 2021 was $70.5 million, or $1.16 per share. Non-GAAP adjusted net loss for the third quarter was $57.9 million, or $0.95 per share, excluding $11.4 million and $1.2 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Non-GAAP Financial Measures

Acceleron supplements its results of operations prepared in accordance with U.S. generally accepted accounting principles, or GAAP, with certain non-GAAP financial measures, including non-GAAP R&D expense, non-GAAP SG&A expense, adjusted net loss and adjusted net loss per share, that exclude stock-based compensation expense and depreciation and amortization expense. These results should not be viewed as a substitute for the Company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures provide investors with additional insight into underlying trends of the Company’s ongoing business, and are important in comparing current results with prior period results. Investors are cautioned that there are material limitations associated with the use of non-GAAP financial measures. In addition, other companies may report similarly titled non-GAAP measures, but calculate them differently, which reduces their usefulness as a comparative measure. In the reconciliation tables below, Acceleron presents these non-GAAP financial measures reconciled to their comparable GAAP financial measures.

Conference Call and Webcast

The Company will not be holding a quarterly earnings call or webcast.

Aligos Therapeutics Reports Recent Business Progress and Third Quarter 2021 Financial Results

On November 4, 2021 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in viral and liver diseases, reported recent business progress and financial results for the third quarter ended, September 30, 2021 (Press release, Aligos Therapeutics, NOV 4, 2021, View Source [SID1234594423]).

"The last quarter has been busy and productive for Aligos," said Lawrence Blatt, PhD, MBA, Chairman and CEO of Aligos. "We now have three of our four drug candidates targeting chronic hepatitis B, each of which has a distinct additive or synergistic mechanism of action, dosing in the clinic. Additionally, enrollment of CHB patients has improved, and our planned safety and viral kinetic data readout for the first three cohorts of STOPS remains on track for the first half of 2022. In parallel to this effort, the Aligos team was able to successfully file the CTA for our first drug candidate for the treatment of NASH, ALG-055009. The progress made by our team over the last quarter positions us well to be able to deliver key data for multiple programs in 2022."

Recent Business Progress
Aligos’ portfolio of drug candidates has continued to advance during the quarter with the following accomplishments achieved:

S-Antigen Transport-Inhibiting Oligonucleotide Polymers (STOPS; ALG-010133)
Our Phase 1b dose range finding trial (NCT04485663) evaluating subjects with CHB is ongoing. Enrollment in the second cohort, evaluating the 200 mg dose level, was completed and enrollment in the third cohort, evaluating a 400 mg dose level, is now underway. We remain on track to unblind and share topline safety and viral kinetic data, including Hepatitis B S-antigen (HBsAg) data, from the first three cohorts in the first half of 2022.
Capsid Assembly Modulator (CAM; ALG-000184)
Our Phase 1b dose range finding trial (NCT04536337) evaluating CHB subjects is ongoing. Enrollment of hepatitis B E-antigen (HBeAg) negative subjects into 50 and 100 mg dose cohorts is now complete. Enrollment of a 10 mg dose cohort is underway to determine the minimum efficacious dose. A cohort of HBeAg-positive CHB patients receiving a 100 mg dose for 28 days has also been fully enrolled in China.
A poster describing the safety, PK, and antiviral activity of the 50 and 100 mg doses of ALG-000184 given for 28 days in HBeAg-negative CHB subjects will be presented at The Liver Meeting (American Association for the Study of Liver Disease) later this month.
Antisense Oligonucleotide (ASO; ALG-020572)
Our Phase 1a/b multi-part study (NCT05001022) evaluating ALG-020572 in healthy volunteers and CHB subjects has recently commenced. Enrollment of healthy volunteers in the single ascending dose (SAD) portion of the study was initiated in October 2021.
Nonalcoholic Steatohepatitis (NASH; ALG-055009, a thyroid hormone receptor beta (THR-B) agonist)
The CTA for ALG-055009 was filed in France to enable a SAD and multiple ascending dose (MAD) study in healthy volunteers and hyperlipidemic subjects, respectively.
Study startup activities remain on track with dosing in healthy volunteers expected to begin in the fourth quarter of 2021.
Aligos has a broad CHB portfolio that targets different clinically validated mechanisms of action in the hepatitis B virus life cycle. The portfolio includes a STOPS molecule, ALG-000184, a class II CAM, ALG-010133, ALG-020572, an ASO, and ALG-020755, an siRNA drug candidate. The properties of these candidates indicate that their use in combination could yield potentially best-in-class treatment regimens that may achieve higher rates of functional cure than current standard of care. For each of these drug candidates, Aligos plans to initially establish proof of concept as monotherapy in Phase 1 dose range finding trials before evaluating them in combination in subsequent trials.

Financial Results for the Third Quarter 2021
Net losses for the three months ended September 30, 2021 were $33.1 million or basic and diluted net loss per common share of $(0.78) compared to net losses of $33.3 million or basic and diluted net loss per common share of $(11.00) for the three months ended September 30, 2020.

Research and development (R&D) expenses for the three months ended September 30, 2021, were $28.1 million compared with $17.3 million for the same period of 2020. The increase in R&D expenses for this comparative period is primarily attributable to increased expenses related to the Company’s continued development and manufacturing of ALG-010133, ALG-000184 and ALG-020572 clinical trial activities, as well as increases in salaries and employee-related expenses and preclinical programs. Total R&D stock-based compensation expense incurred for the three months ended September 30, 2021, was $1.9 million compared with $0.3 million for the same period for 2020.

General and administrative (G&A) expenses for the three months ended September 30, 2021, were $6.5 million compared with $4.2 million for the same period of 2020. The increase in G&A expenses for this comparative period is primarily attributable to higher employee-related costs associated with the growth of the Company’s operations and additional professional, legal and consulting services related to being a public company. Total G&A stock-based compensation expense incurred for the three months ended September 30, 2021, was $1.7 million compared with $0.7 million for the same period for 2020.

Cash, cash equivalents and investments totaled $242.7 million as of September 30, 2021 compared with $243.5 million as of December 31, 2020.

Bellicum Reports Third Quarter 2021 Financial Results and Provides Operational Update

On November 4, 2021 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the third quarter 2021 and provided an operational update (Press release, Bellicum Pharmaceuticals, NOV 4, 2021, View Source [SID1234594422]).

"In the third quarter, Bellicum broadened the potential impact of its CaspaCIDe technology through an additional licensing agreement with MD Anderson," said Rick Fair, President and Chief Executive Officer. "We remain focused on the clinical studies of our next generation GoCAR-T cell therapies, and plan to provide future updates later this year and in the first quarter of 2022 on our progress for both programs. We are also thrilled to announce the appointment of Charity Scripture as Chief Development Officer to lead our clinical and regulatory efforts."

Program Highlights and Current Updates

BPX-601 GoCAR-T

Enrollment in the Phase 1/2 dose escalation clinical trial in patients with previously treated metastatic castration-resistant prostate cancer (mCRPC) is ongoing. Bellicum expects to announce the first interim data in mCRPC in the first quarter of 2022.
BPX-603 GoCAR-T

Enrollment is ongoing in the Phase 1/2 clinical trial for BPX-603 in patients with solid tumors that express human epidermal growth factor 2 (HER2), including breast, endometrial, ovarian, gastric, and colorectal cancers. BPX-603 is the company’s first dual-switch GoCAR-T product candidate, which incorporates Bellicum’s iMC activation and CaspaCIDe safety switch technologies. The company expects to announce initial Phase 1 data from this trial in the fourth quarter of 2021.
CaspaCIDe License Agreements

In September, Bellicum entered into an additional license agreement with The University of Texas MD Anderson Cancer Center covering certain intellectual property and technology rights regarding the company’s CaspaCIDe (inducible caspase-9, or iC9) safety switch and related technologies, and the use of rimiducid. Under this agreement, MD Anderson will have the option to incorporate CaspaCIDe into certain cellular therapy programs. Upon exercise of each option – typically expected to be upon out-license of an MD Anderson program that incorporates iC9 – Bellicum will receive an upfront payment and will be entitled to a percentage of certain consideration paid to MD Anderson by the third party. Bellicum also will receive a single-digit-percent royalty on global sales of the product. Concurrent with the execution of the agreement, Bellicum granted a license to CaspaCIDe for two programs and received an upfront fee of $5 million.
Charity Scripture Named Chief Development Officer Effective December 1, 2021

Dr. Scripture rejoins Bellicum in a full-time capacity after spending the last year as VP, Business and Development Operations at ACELYRIN, a private biopharmaceutical company. Previously, Dr. Scripture was Vice President, Clinical & Medical Affairs at Bellicum. Prior to joining Bellicum, Dr. Scripture held clinical development leadership positions at AbbVie/Stemcentrx and Pharmacyclics, and spent almost a decade with Amgen in oncology clinical development and medical affairs. Prior to joining industry Dr. Scripture worked in clinical practice at Dartmouth-Hitchcock Medical Center. Dr. Scripture holds a Bachelor of Science degree from Hamilton College, a Master’s Degree in Pharmacology and Toxicology from Dartmouth Medical School, and a Doctorate of Pharmacy degree from University of North Carolina at Chapel Hill, and completed a Clinical Pharmacology Drug Development fellowship at the National Cancer Institute.
Update on Nasdaq Compliance

On November 2, 2021, Nasdaq notified Bellicum that it had not regained compliance with the Market Value Rule by November 1, 2021, and unless the company requests a hearing before the Nasdaq Hearings Panel by November 9, 2021, the company’s securities will be delisted from Nasdaq. Bellicum intends to timely request a hearing before the Panel to appeal this determination, which the company expects will stay any further action by Nasdaq until the conclusion of the hearing process.
Financial Results for the Third Quarter and Nine Months Ended September 30, 2021

Revenues: Bellicum reported revenue of $5.0 million and $5.7 million for the three and nine months ended September 30, 2021, respectively, compared to $0.0 million during the nine months ended September 30, 2020. The increase in revenues in the nine months ended September 30, 2021 was primarily due to a license fee of $5.0 million received from MD Anderson for certain option and license rights to CaspaCIDe and related technologies, and $0.7 million earned from a supply agreement with Takeda Development for the supply of rimiducid for potential use in clinical trials of TAK-007 (CD19 CAR-NK cell therapy).

R&D Expenses: Research and development expenses were $6.3 million and $19.5 million for the three and nine months ended September 30, 2021, respectively, compared to $8.1 million and $30.3 million for the three and nine months ended September 30, 2020, respectively. The decrease in expenses in the third quarter of 2021 resulted primarily from reduced rivo-cel commercialization activities and the corporate restructuring implemented during the fourth quarter of 2020, which resulted in a reduction in force.

G&A Expenses: General and administrative expenses were $1.7 million and $5.5 million for the three and nine months ended September 30, 3021, respectively, compared to $4.2 million and $12.1 million for the three and nine months ended September 30, 2020, respectively. The decrease in expenses in the third quarter of 2021 was primarily due to the reduction in force.

Loss from Operations: Bellicum reported a loss from operations of $3.0 million and $19.8 million for the three and nine months ended September 30, 2021, respectively, compared to a loss from operations of $12.3 and $38.7 million for the three and nine months ended September 30, 2020, respectively. The results for the nine months ended September 30, 2021 include a net loss on dispositions of $0.5 million relating to the early termination of the South San Francisco office space during the first quarter of 2021. The results for the nine months ended September 30, 2020 included a net gain on dispositions of $3.8 million due to the sale of the Houston manufacturing facility in the second quarter of 2020. Cash used in operating activities was $17.1 million for the nine months ended September 30, 2021, compared to cash used in operating activities of $43.3 million for the nine months ended September 30, 2020.

Net Income/Loss: Bellicum reported net income of $1.2 million for the three months ended September 30, 2021 and a net loss of $12.2 million for the nine months ended September 30, 2021, compared to a net loss of $0.9 million and $26.5 million for the three and nine months ended September 30, 2020, respectively. The results in the third quarter of 2021 included revenue of $5.0 million from the license agreement with MD Anderson and a non-cash gain of $4.3 million recognized from the change in the derivative warrant and private placement option fair value liability.

Shares Outstanding: As of October 27, 2021, Bellicum had 8,397,803 shares of common stock and 452,000 shares of preferred stock outstanding. Each share of preferred stock is convertible into 10 shares of common stock.

Cash Position and Guidance: Bellicum reported cash and cash equivalents and restricted cash totaling $20.8 million as of September 30, 2021, compared to $37.0 million as of December 31, 2020. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second quarter of 2022.

Oncternal Provides Business Update and Announces Third Quarter 2021 Financial Results

On November 4, 2021 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported financial results for the third quarter of 2021. Oncternal management will host a webcast today at 5:00 p.m. ET to provide a business update and discuss its third quarter of 2021 financial results (Press release, Oncternal Therapeutics, NOV 4, 2021, View Source [SID1234594421]).

"We are excited about the progress of our pipeline during this quarter, especially as we continue to advance towards a registration study for our lead asset, zilovertamab, which we believe can play a key role in the treatment paradigm of patients with mantle cell lymphoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We are also very pleased to introduce ONCT-534, a pre-clinical first-in-class dual-action androgen inhibitor, as part of our pipeline. Tumor-resistance mechanisms related to the expression of splice variants such as AR-V7 represents an important unmet need for prostate cancer patients, and we are committed to developing new treatment options for them. Finally, our cell therapy program targeting ROR1 continues to move forward, with progress on both our lead candidate ONCT-808, an autologous CAR-T, and potential next-generation therapies based on NK cells."

Recent Highlights

Zilovertamab (ROR1 antibody, formerly cirmtuzumab or UC-961):

Interactions with FDA continue on potential registration pathways.
An abstract titled Phase 1b/2 Study of Cirmtuzumab and Ibrutinib in Mantle Cell Lymphoma (MCL) or Chronic Lymphocytic Leukemia (CLL) (Abstract 3534) (NCT0308887) has been accepted for a poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta on December 13, 2021. Abstracts are available on the ASH (Free ASH Whitepaper) website at www.hematology.org.
This abstract reflects early data from June 2021, shortly after our interim data was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting. The results remain encouraging and consistent with previous data releases and will be further updated with additional patients and follow-up at the ASH (Free ASH Whitepaper) meeting.
The investigator-sponsored study of zilovertamab and paclitaxel for metastatic or locally advanced, unresectable breast cancer at UC San Diego (NCT02776917) has completed enrollment and the results are expected to be presented at a scientific conference or in a publication.
The investigator-sponsored study of zilovertamab for treatment of patients with detectable CLL on venetoclax at UC San Diego (NCT04501939) is actively enrolling patients.
ONCT-216 (ETS inhibitor, formerly TK216):

Interim clinical data update for ONCT-216 in patients with relapsed or refractory Ewing sarcoma was accepted for oral presentation at the 2021 Connective Tissue Oncology Society (CTOS) Virtual Annual Meeting on November 13, 2021.
The Phase 2 expansion cohort targeting up to 21 Ewing sarcoma patients is active and enrolling patients, designed to evaluate clinical responses to single agent ONCT-216 using an optimized dosing regimen, treating for 28 days per cycle, to intensify the amount of ONCT-216 administered over time.
ROR1 Cell-Therapy Program:

Selected ONCT-808 as our lead autologous CAR-T targeting ROR1 candidate and made progress on key IND-enabling activities.
Initiated a collaboration with Celularity Inc. to evaluate placental derived T and NK-cell therapies targeting ROR1.
Formed a cell therapy scientific advisory board (SAB) with cross-disciplinary industry and academic experts to support the advancement of our ROR1 cell therapy pipeline.
Joined Karolinska Institutet’s NextGenNK Competence Center for the development of next generation NK cell-based cancer immunotherapies.
Dual-Action Androgen Inhibitor (DAARI) Program:

Selected ONCT-534 (formerly GTx-534) as our lead DAARI preclinical product candidate, based on significant new preclinical data and assessments. We believe ONCT-534 will address unmet medical needs related to tumor resistance to currently approved products for patients with advanced prostate cancer.
Presented preclinical in-vitro and in-vivo data as a virtual poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October. The data presented showed that ONCT-534 binds to the AR-AF-1, or N-terminal domain of the androgen receptor (AR) and induces degradation of both AR full length proteins and AR-SV proteins, including the AR-V7 splice variant. ONCT-534 demonstrated potent anti-tumor activity in AR-V7 expressing tumor xenografts in an animal model.
Corporate:

Appointed Steven Hamburger, Ph.D. as Senior Vice President, Regulatory Affairs and Quality Assurance.
Expected Upcoming Milestones

Zilovertamab (ROR1 antibody) program
Clinical data update for patients with MCL and CLL treated with zilovertamab plus ibrutinib in the ongoing Phase 1b/2 study to be presented at the American Society for Hematology (ASH) (Free ASH Whitepaper) 2021 Annual Meeting in Atlanta in December 2021
Ongoing FDA interactions regarding potential registration pathways for zilovertamab in patients with MCL
ONCT-216 (ETS inhibitor) programs
Clinical data update for patients with relapsed or refractory Ewing sarcoma to be shared in an oral presentation at the Connective Tissue Oncology Society (CTOS) 2021 Virtual Annual Meeting in November 2021
ROR1 CAR-T program
IND submission in the first half of 2022
The Company will host an R&D Day on January 25, 2022 to provide a comprehensive update of its pipeline of product candidates and key development priorities, which will include key opinion leaders in the fields of MCL therapies, cellular immunotherapies, and androgen receptor resistance in prostate cancer
Third Quarter 2021 Financial Results

Our grant revenue was $2.1 million for the third quarter ended September 30, 2021. Our grant revenue is derived from a sub-award under a grant from the California Institute for Regenerative Medicine (CIRM) to UC San Diego, which was awarded to advance our Phase 1b/2 clinical trial evaluating zilovertamab in combination with ibrutinib for the treatment of patients with MCL or CLL, and from two grant awards received from the National Institutes of Health, or NIH, during the third quarter ended September 30, 2021 for up to $2.2 million to support pre-clinical and other research activities for our ONCT-216 and ONCT-534 programs, including $0.7 million payable to subawardees.

Our total operating expenses for the third quarter ended September 30, 2021 were $11.8 million, including $1.5 million in non-cash stock-based compensation. Research and development expenses for the quarter totaled $9.0 million, and general and administrative expenses for the quarter totaled $2.8 million. Net loss for the third quarter was $9.6 million, or a loss of $0.19 per share, basic and diluted.

As of September 30, 2021, we had $97.4 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into 2023. As of September 30, 2021, we had approximately 49.4 million shares of common stock outstanding.

Management Webcast

As previously announced, Oncternal will host a webcast today, November 4, 2021, at 5:00 p.m. ET (2:00 p.m. PT). The live webcast will be available online at investor.oncternal.com. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.