On May 12, 2021 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, reported that clinical results from its broad hematology program will be presented at the 26th European Hematology Association (EHA) (Free EHA Whitepaper) 2021 (EHA2021) Virtual Congress being held June 9 – 17, 2021 (Press release, BeiGene, MAY 12, 2021, View Source [SID1234579769]).

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"Following key head-to-head data from the positive ALPINE trial interim analysis and the previously announced ASPEN trial, we are thrilled to share additional updates from BRUKINSA’s broad global clinical program at EHA (Free EHA Whitepaper). In these trials, sustained responses with BRUKINSA treatment were observed consistently across multiple indications and patient subgroups, and it was well tolerated in patients, including those with previously treated B-cell malignancies who discontinued other BTK inhibitors due to intolerable adverse events," said Jane Huang, M.D., Chief Medical Officer, Hematology of BeiGene. "In addition to BRUKINSA, we are pleased to be presenting long-term efficacy results from our anti-PD-1 antibody tislelizumab in classical Hodgkin’s lymphoma, which is approved for use in China. We are also encouraged by the preliminary safety data of our novel investigational Bcl-2 inhibitor and look forward to further evaluating this recently advanced clinical candidate in combination with BRUKINSA for patients with hematologic malignancies."

To learn more about BeiGene’s research and development and activities around the EHA (Free EHA Whitepaper)2021 Virtual Congress, please visit View Source

Promising Head-to-Head and Long-Term Data Support BeiGene’s Aspiration to Improve Patient Outcomes for More Patients with BRUKINSA

Since its first-in-human study in 2014, a broad clinical program for BRUKINSA has provided a growing body of clinical evidence demonstrating its consistent efficacy and tolerability profile across B-cell malignancies, genotypes, and other patient characteristics. To further demonstrate the clinical profile of this molecule that was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity, BeiGene took a bold approach in the development of BRUKINSA, including two large-scale Phase 3 head-to-head trials against the first-generation BTK inhibitor ibrutinib:

ALPINE trial in relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) – in the recently announced positive interim results, BRUKINSA demonstrated superiority in objective response rate (ORR) per investigator assessment and non-inferiority in ORR per both investigator assessment and independent review committee (IRC). Data pertaining to progression-free survival (PFS), a secondary endpoint of the trial, were immature at the data cutoff for the interim analysis; however, the descriptive summaries of PFS showed an early trend favoring BRUKINSA. In addition, BRUKINSA demonstrated a statistically significant lower risk of atrial fibrillation or flutter compared to ibrutinib, and the overall safety profile of BRUKINSA was consistent with the previously seen profile in its clinical development program; and
ASPEN trial in Waldenström’s macroglobulinemia (WM) – in the results presented at last year’s ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) meetings, BRUKINSA demonstrated clinically meaningful improvements in safety and tolerability, including a lower risk of atrial fibrillation or flutter, and a favorable combined complete and very good partial response rate compared to ibrutinib.
In addition to the head-to-head trials demonstrating BRUKINSA’s clinical profile compared to ibrutinib, BRUKINSA is being evaluated in an ongoing Phase 2 trial in patients with previously treated B-cell malignancies who were intolerant to ibrutinib and/or acalabrutinib. Results from this trial at a prior data cutoff were presented in a poster at the 62nd ASH (Free ASH Whitepaper) Annual Meeting in December 2020, including that most intolerable adverse events patients experienced on other BTK inhibitors did not recur with BRUKINSA treatment, and that the vast majority of patients who were evaluable for response at the time of data cutoff maintained or improved their responses on BRUKINSA. Updated results from this trial will be presented at EHA (Free EHA Whitepaper)2021.

In June 2020, BRUKINSA received its first approvals in China in both R/R CLL and R/R mantle cell lymphoma (MCL). Long-term follow-up data from the clinical trials supporting these two approvals will be presented at EHA (Free EHA Whitepaper)2021. From results in the accepted abstracts, BRUKINSA demonstrated deep and durable responses across all subgroups in these trials, including high-risk patients, with a median follow-up over 33 months. No new safety signals were identified.

BeiGene will also share updates from the pivotal Phase 2 MAGNOLIA trial of zanubrutinib in patients with R/R marginal zone lymphoma at EHA (Free EHA Whitepaper)2021. Results from the MAGNOLIA trial were previously reported at ASH (Free ASH Whitepaper) 2020.

BeiGene to Present Long-Term Follow-up Data of Tislelizumab in Classical Hodgkin’s Lymphoma (cHL)

Immune checkpoint inhibitors have catalyzed a paradigm shift in cancer treatment since the initial clinical evaluation approximately 15 years ago. Based on preclinical evidence that binding to Fc gamma receptors (FcγR) on macrophages can compromise antitumor activity, tislelizumab was uniquely engineered with minimal FcγR-binding to abrogate a potential T-cell clearance and resistance mechanism and therefore potentially improve efficacy for patients.

In late 2019, tislelizumab received its first approval in China for patients with cHL who have received at least two prior therapies, based on clinical results from a pivotal Phase 2 trial conducted in China (NCT03209973). At the time of approval, with a median follow-up time of 14 months, the ORR as assessed by IRC was 76.9% (95% CI: 64.8, 86.5), including 61.5% of patients who achieved a CR, and the median PFS was not estimable (NE; 95% CI: 13.1, NE). Grade 3 and above adverse reactions occurring in ≥2% of patients included pneumonitis, weight increase, severe skin reactions and hypertension. At EHA (Free EHA Whitepaper)2021, long-term follow-up results from this trial will be available in an oral presentation.

BeiGene’s Growing Hematology Clinical Portfolio Now Includes Potent and Highly Selective BCL-2 Candidate BGB-11417

In addition to the established BRUKINSA and tislelizumab programs, BeiGene researchers are working to target other promising pathways to complement our existing medicines and drug candidates and expand our hematology portfolio for greater therapeutic potential, including BCL-2 – a protein known for its aberrant expression in many hematologic malignancies and promotion of cancer cell survival.

BGB-11417 is an investigational potent and highly selective BCL-2 inhibitor with a favorable pharmacokinetics profile. At EHA (Free EHA Whitepaper)2021, BeiGene will share preliminary safety data from an ongoing first-in-human Phase 1/1b study (NCT04277637) of BGB-11417 in patients with R/R B-cell malignancies. The Company also plans to evaluate the combination of BGB-11417 and BRUKINSA in patients with MCL and WM in the near future.

BeiGene’s Presentations at EHA (Free EHA Whitepaper)2021 Virtual Congress

Abstract #

Abstract Title

Session

Time

Lead Author

Oral Presentation

S207

Tislelizumab (BGB-A317) For Relapsed/Refractory Classical Hodgkin Lymphoma: Long-Term Follow-up Efficacy and Safety Results from A Phase 2 Study

Hodgkin lymphoma – Clinical

Presentation available on Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Live Q&A session on Sunday, June 13 at 10:00 a.m. ET (16:00 CEST)

Yuqin Song, M.D., Ph.D.

Beijing Cancer Hospital, China

Posters

EP783

Phase 2 Study of Zanubrutinib In Patients with Relapsed/Refractory Marginal Zone Lymphoma (MAGNOLIA Study)

Indolent and mantle-cell non-Hodgkin lymphoma – Clinical

Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Stephen Opat, MBBS, FRACP, FRCPA

Monash Health and Clinical Hematology Unit Monash University, Australia

EP64-2

Preliminary Results of the Phase 2 Study of Zanubrutinib in Patients with Previously Treated B-Cell Malignancies Intolerant to Ibrutinib and/or Acalabrutinib

Chronic lymphocytic leukemia and related disorders – Clinical

Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Mazyar Shadman, M.D.

Fred Hutchinson Cancer Research Center, University of Washington

EP789

Zanubrutinib In Patients with Relapsed/Refractory (R/R) Mantle Cell Lymphoma (MCL): Long-Term Efficacy and Safety Results from a Phase 2 Study

Indolent and mantle-cell non-Hodgkin lymphoma – Clinical

Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Yuqin Song, M.D., Ph.D.

Beijing Cancer Hospital, China

EP639

Zanubrutinib Monotherapy in Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia: 34-Month Follow-up Results

Chronic lymphocytic leukemia and related disorders – Clinical

Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Wei Xu, M.D., Ph.D.

The First Affiliated Hospital of Nanjing Medical University, China

EP525

Preliminary Safety Data from Patients with Relapsed/Refractory (R/R) B-Cell Malignancies Treated with the Novel B-Cell Lymphoma 2 (Bcl2) Inhibitor BGB-11417

Aggressive Non-Hodgkin lymphoma – Clinical

Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Chan Y. Cheah, MBBS, FRACP, FRCPA, DMedSc

Sir Charles Gairdner Hospital and Pathwest Laboratory Medicine, University of Western Australia Medical School, Linear Clinical Research, Australia

EP805

Efficacy and Safety of Zanubrutinib Versus Rituximab-Based Chemoimmunotherapy in Waldenström Macroglobulinemia: Matching-Adjusted Indirect Comparisons

Indolent and mantle-cell non-Hodgkin lymphoma – Clinical

Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Jorge J. Castillo, M.D.

Dana-Farber Cancer Institute

EP1174

Cost-Effectiveness of Zanubrutinib Versus Ibrutinib in Adult Patients with Waldenström Macroglobulinemia

Quality of life, palliative care, ethics, and health economics

Friday, June 11 at 3:00 a.m. ET (9:00 CEST)

Jorge J. Castillo, M.D.

Dana-Farber Cancer Institute

BeiGene Oncology

BeiGene is committed to advancing best and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D team of approximately 2,300 colleagues dedicated to advancing more than 80 clinical trials involving more than 13,000 patients. Our expansive portfolio is directed by a predominantly internalized clinical development team supporting trials in more than 40 countries. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company with both mono- and combination therapies prioritized in our research and development. The Company currently markets three medicines discovered and developed in our labs: BTK inhibitor BRUKINSA in the United States, China, Canada, and additional international markets; and non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab and PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen and Bristol Myers Squibb. We also plan to address greater areas of unmet need globally through our collaborations including with Amgen, EUSA Pharma, Bio-Thera, Seagen, Mirati Therapeutics, and Zymeworks. BeiGene has also entered into a collaboration with Novartis Pharma AG granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

On May 12, 2021 Inventiva (Euronext Paris and Nasdaq: IVA), a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS) and other diseases with significant unmet medical need, reported its cash position as of March 31, 2021 and its revenues for the first quarter of 2021, and provided an update on its collaboration with AbbVie in auto-immune diseases (Press release, Inventiva Pharma, MAY 12, 2021, View Source [SID1234579786]).

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Cash Position

As of March 31, 2021, Inventiva’s cash and cash equivalents stood at €107.8 million compared to €113.0 million as of December 31, 2020.

Net cash used in operating activities amounted to €7.8 million in the first quarter of 2021 compared to €3.6 million for the same period in 2020. R&D expenses for the first quarter, mainly driven by the development of lanifibranor in NASH, were up 22% compared to the first quarter of 2020. This increase in cash used is due to the preparation for the initiation of NATIV3, a Phase III clinical trial evaluating lanifibranor in NASH, while the first quarter of 2020 had been positively impacted by the receipt of a €4.2 million non-recurrent late payment of the 2018 research tax credit.

Net cash from investing activities for the first quarter of 2021 amounted to €1.1 million, as compared to no net cash from investing activities generated in the first quarter of 2020.

No net cash from financing activities was generated over the first quarter of 2021 while Inventiva recorded €14.6 million of net cash from financing activities for the same period in 2020, notably related to the issuance of €15 million (gross proceeds) of ordinary shares in February 2020.

Over the first quarter of 2021, the Company recorded a positive exchange rate effect on cash and cash equivalent of €3.7 million.

Considering its current R&D and clinical development programs, and excluding additional financial resources, Inventiva has adjusted its projected cash runway by one quarter, allowing the Company to finance its operating activities through the third quarter of 2022 compared to the fourth quarter of 2022 as previously communicated.

Revenues

The Company’s revenues for the first quarter of 2021 amounted to €0.1 million, similar to the amounts received in the first quarter of 2020.

Update on the collaboration with AbbVie in auto-immune diseases2

Cedirogant, a clinical stage RORγ inverse agonist co-discovered by Inventiva with potential in several auto-immune diseases, demonstrated promising activity as an oral psoriasis agent during a Phase Ib clinical trial3 led by AbbVie. Following these results, AbbVie has decided to move the drug candidate into a Phase IIb dose-ranging study, planned to be initiated in the second half of 2021.

As part of this collaboration, Inventiva is eligible to receive development, regulatory and commercial milestone payments as well as royalty payments. As such, the Company expects to receive another milestone payment upon the initiation by AbbVie of the Phase IIb clinical trial with cedirogant.

Next key milestones expected

Initiation of NATIV3 Phase III clinical trial evaluating lanifibranor in NASH – planned for the first half of 2021
Initiation by AbbVie of a Phase IIb clinical trial with cedirogant – expected in the second half of 2021
Strategy update on the development of odiparcil – planned for 2021
Publication of the results of the Phase II clinical trial evaluating lanifibranor for the treatment of Non-Alcoholic Fatty Liver Disease (NAFLD) in patients with type 2 diabetes (T2DM) – planned for the first half of 2022

Upcoming investor conference participation

Jefferies Virtual Healthcare Conference, June 1-4, 2021
SVB Leerink CybeRx Series: Liver Disease Day, June 17, 2021
Citi’s 16th Annual BioPharma Conference 2021, September 8-9, 2021
H.C. Wainwright 23rd Annual Global Investment Conference, September 13-15, 2021
Portzampac Health Biotech Seminar 2021, October 6, 2021
Stifel Healthcare Conference 2021, November 16-17, 2021
Jefferies 2021 London Healthcare Conference, November 16-18, 2021

Upcoming scientific conference participation

International Liver Congress 2021, June 23-26, 2021
Paris NASH Meeting, October 22-23, 2021
AASLD The Liver Meeting, November 12-15, 2021

Next financial results publication

Revenues and cash position for the first half of 2021: Wednesday, July 28, 2021 (after U.S. market close)

On May 12, 2021 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing potential new medicines for patients with pediatric cancers, solid tumors, and other cancers, reported important corporate events and its financial results for the first quarter ended March 31, 2021 (Press release, Salarius Pharmaceuticals, MAY 12, 2021, View Source [SID1234579802]).

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Financial Highlights:

Total cash and cash equivalents of $36.6 million as of March 31, 2021 resulting from financing activities including $23.0 million gross proceeds in an underwritten public offering closed March 8, 2021; Cash position sufficient to fund the current seclidemstat clinical trials through completion

Three-month period ended March 31, 2021 net loss per common share – basic and diluted – of $0.06, compared to $0.22 for the same period ended March 31, 2020
"The first quarter of 2021 and recent months were a period of significant activity for Salarius, with the company accomplishing several key milestones, including strengthening our capital position with more than $30 million gross proceeds raised during the quarter, the completion of the dose-escalation stage of the Phase 1/2 trial of seclidemstat in Ewing sarcoma, and the initiation of the dose-expansion stage of the trial, which now also includes FET-rearranged sarcomas, also known as Ewing-related sarcomas which share biology similar to Ewing sarcoma," said David Arthur, President and CEO of Salarius. "As a result, Salarius is operating from a position of strength as we work to advance our clinical trials exploring seclidemstat as a potential treatment for Ewing sarcoma, select FET-rearranged sarcomas, advanced solid tumors and hematologic cancers. By mid-year 2021, we expect up to three total active clinical trials in up to five patient populations evaluating single-agent seclidemstat and up to three combination therapies."

Recent Business and Corporate Events:

Completion of dose escalation in Phase 1/2 Ewing sarcoma clinical trial determined seclidemstat’s safety profile and established recommended Phase 2 dose (RP2D)
A refractory Ewing sarcoma patient treated with single-agent seclidemstat for 168 days (six 28-day cycles) demonstrated preliminary signal of drug activity
Full findings from dose escalation to be disclosed in poster presentation session followed by a poster discussion during the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting; one of three abstracts accepted for poster presentation during the ASCO (Free ASCO Whitepaper) Annual Meeting
Preliminary efficacy from an ongoing Phase 1 dose-escalation trial of seclidemstat in patients with advanced solid tumors (AST) supports inclusion of FET-rearranged sarcomas in Ewing sarcoma trial
Subset of FET-rearranged sarcoma patients treated with seclidemstat in the AST clinical trial demonstrated encouraging signs of drug activity
Preliminary efficacy findings from the dose-escalation stage of AST trial to be reported in a poster session during 2021 ASCO (Free ASCO Whitepaper) Annual Meeting
Dose expansion initiated; actively recruiting three patient groups across Ewing sarcoma and FET-rearranged sarcomas; data expected in 2022
Ewing sarcoma patients to be treated with seclidemstat in combination with topotecan and cyclophosphamide as a second- or third-line therapy
FET-rearranged sarcoma patients, including myxoid liposarcoma, to be treated with single-agent seclidemstat as second-, third- or fourth-line therapy
Mr. Arthur continued, "With initiation of the dose-expansion stage of the trial in Ewing and FET-rearranged sarcomas, we have positioned seclidemstat as a second- and third-line Ewing sarcoma therapy in combination with a common chemotherapy treatment. This increases the number of available patients and should make it easier for physicians to treat patients earlier in the continuum of patient care. Expanding the trial to include myxoid liposarcoma and other FET-rearranged sarcoma patients provides a potential new therapy for patients who are relapsed or refractory to standard of care treatment. Our goal is to make a difference in the lives of patients fighting cancer, and we believe making seclidemstat available to these patients is a step forward in this journey. In the coming months, we look forward to announcing new clinical trials studying seclidemstat in additional patient populations."

Three-Month Financial Results:
For the three-month period ended March 31, 2021, Salarius’ reported net loss was $1.9 million, or $0.06 per basic and diluted share, compared to a net loss of $2.1 million, or $0.22 per basic and diluted share for the same period in 2020. The loss before other income for the three-month period ended March 31, 2021 decreased by $0.6 million compared to the loss before other income for the same time span last year, primarily due to lower general and administrative costs that more than offset an increase in research and development costs.

Net cash used for operating activities during the three-month period ended March 31, 2021 totaled $2.7 million, a decline of approximately $1.0 million compared to the same span last year due to a $0.9 million payment received under the Company’s contract with the Cancer Prevention and Research Institute of Texas (CPRIT).

Conference Call Information:
Salarius Pharmaceuticals will host a conference call and live audio webcast on Wednesday, May 12, 2021, at 5:00 p.m. ET, to discuss its corporate and financial results for the first quarter 2021. Interested participants and investors may access the conference call by dialing either:

An audio webcast will be accessible via the Investors Events and Presentations section of the Company’s website View Source An archive of the webcast will remain available for 90 days beginning at approximately 5:30 p.m. ET, on May 12, 2021.

On May 12, 2021 Xilio Therapeutics, a biotechnology company developing tumor-selective immuno-oncology therapies for people living with cancer, reported the presentation of data from preclinical studies of XTX101, its tumor-selective anti-CTLA-4 antibody, demonstrating combination potential with anti-PD-1 therapy, as well as enhanced preclinical activity and improved tolerability compared to ipilimumab, an anti-CTLA-4 antibody therapeutic approved by the U.S. Food and Drug Administration (Press release, Xilio Therapeutics, MAY 12, 2021, View Source [SID1234579820]). The findings will be reported today in a poster presentation at The New York Academy of Sciences’ Frontiers in Cancer Immunotherapy 2021 Virtual Symposium.

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Xilio is leveraging its proprietary platform to engineer novel molecules that are designed to be activated in the tumor microenvironment and have the potential to result in localized clinical activity without dose-limiting toxicities. XTX101 is specifically designed to target the anti-CTLA-4 effect geographically within the tumor and to minimize off-tumor peripheral effects. XTX101 is activated in a protease-dependent manner with high binding affinity to CTLA-4, potentially enabling it to overcome CTLA-4 inhibition of T cell activation and freeing T cells to attack cancer.

"The broad clinical benefit of CTLA-4 blockade, as with ipilimumab, for the treatment of cancer is well-established; however, challenging toxicities arising from systemic immune activation have limited use of these agents as both monotherapy and in combination, including with anti-PD-1 agents," said Rónán O’Hagan, Ph.D., chief scientific officer of Xilio. "XTX101 has been engineered to overcome the tolerability and potency limitations associated with other anti-CTLA-4 antibodies by applying our proprietary masking technology to the antibody and engineering enhanced binding to target receptors. We believe these data validate our approach, and we observed that XTX101 induces tumor-selective biological activity and robust tumor growth inhibition, with favorable tolerability, in preclinical studies. We look forward to advancing XTX101 into a planned Phase 1 clinical trial in the second half of 2021."

Data reported in a poster entitled, "Tumor-Activated Anti-CTLA-4 Monoclonal Antibody, XTX101, Demonstrates Monotherapy and Anti-PD-1 Combination Benefit in Preclinical Models," include:

In a colon cancer model, the combination of XTX101 with an anti-PD-1 antibody showed robust tumor growth inhibition, including two complete responses (CRs) (n=8), where treatment with XTX101 or the anti-PD-1 agent as a monotherapy achieved only modest tumor growth inhibition and no CRs.
No significant body weight loss was observed in animals treated with either XTX101 or anti-PD-1 as a monotherapy or the combination regimen, suggesting that XTX101 can be effectively combined with anti-PD-1 without enhanced toxicity.
In a bladder cancer model, XTX101 monotherapy demonstrated tumor growth inhibition superior to ipilimumab, while a dose of 3 mg/kg of ipilimumab was required to achieve similar activity of XTX101 at 0.3 mg/kg, suggesting XTX101 has 10-fold higher potency than ipilimumab.
XTX101 as a monotherapy induced an increase in CD8+ T cells within the tumor, and a decrease in T regulatory cells in the tumor compared to ipilimumab. In addition, XTX101 achieved CRs without increasing CD4+ T cells in the blood.

On May 12, 2021 Veru Inc. (NASDAQ: VERU), an oncology biopharmaceutical company with a focus on developing novel medicines for the management of prostate and breast cancer, reported that net revenues increased 34% and gross profit rose 47% for its fiscal 2021 second quarter ended March 31, 2021, attributable to record high quarterly FC2 US prescription net revenues (Press release, Veru, MAY 12, 2021, View Source [SID1234579883]).

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Second Quarter Financial Highlights: Fiscal 2021 vs Fiscal 2020

Net revenues increased 34% to $13.3 million from $9.9 million
FC2 prescription net revenues climbed 48% to $10.3 million from $7.0 million
Gross profit rose 47% to $10.9 million from $7.4 million
Gross margin increased to 82% of net revenues from 75% of net revenues
Operating loss was $1.5 million versus $0.3 million
Net loss was $2.8 million, or $0.04 per share, compared with $0.8 million, or $0.01 per share.
Year-to-Date Financial Highlights: Fiscal 2021 vs Fiscal 2020

Net revenues increased 36% to $28.0 million from $20.5 million, a record high for the six-month period ended March 31, 2021
FC2 prescription net revenues climbed 49% to $19.4 million from $13.0 million
Gross profit rose 48% to $21.7 million from $14.7 million
Gross margin increased to 78% of net revenues from 72% of net revenues
Operating income was $17.7 million, which includes an $18.4 million gain on the December 2020 sale of the PREBOOST business. Adjusted operating loss, which excludes the gain on the sale of the PREBOOST business, was $0.7 million versus $2.1 million
Net income, which includes the gain on the sale of the PREBOOST business, was $14.4 million and diluted EPS was $0.18. Adjusted net loss, which excludes the gain on the sale of the PREBOOST business, was $4.0 million compared with $4.1 million and adjusted diluted loss per share was $0.06, which remained consistent with fiscal 2020.
Balance Sheet Information

Cash and cash equivalents were $136.7 million as of March 31, 2021 versus $13.6 million as of September 30, 2020
Net accounts receivable were $5.1 million as of March 31, 2021 versus $5.2 million as of September 30, 2020.
"We reported another great quarter largely based on all-time record high quarterly FC2 net revenues from the U.S. prescription channel," said Mitchell Steiner, M.D., Chairman, President and Chief Executive Officer of Veru Inc. "We also will be enrolling our first patient in our Phase 3 clinical trial of sabizabulin in high risk hospitalized COVID-19 patients this week. Because of sabizabulin’s anti-inflammatory and anti-viral properties and its favorable safety profile, we think sabizabulin could be that desperately needed oral therapeutic to prevent deaths in hospitalized patients with moderate to severe COVID-19 disease who are at risk for Acute Respiratory Distress Syndrome (ARDS). COVID-19 remains a serious threat worldwide and effective treatments are desperately needed."

Dr. Steiner noted: "We are advancing our novel oral drug candidates for the treatment of prostate and breast advanced cancers. We plan this month to enroll our first patient in the Phase 3 VERACITY clinical trial of sabizabulin for metastatic castration and androgen receptor targeting agent resistant prostate cancer. We plan to also enroll this month our first patient in the Phase 2 clinical trial of VERU-100, a novel long-acting GnRH antagonist injection formulation for androgen deprivation therapy. Next month, the Phase 3 ARTEST enobosarm for 3rd line AR+ER+ metastatic breast cancer is also expected to start enrolling. We are now a solid late clinical stage oncology biopharmaceutical company with novel drug candidates in development."

Pharmaceutical Pipeline Highlights:

Sabizabulin (VERU-111) a Novel Oral Agent for the Treatment of Hospitalized COVID-19 Patients at High Risk for Acute Respiratory Distress Syndrome (ARDS)- Phase 3 Clinical Study.

We expect to enroll our first patient within a few days in our Phase 3 clinical trial of sabizabulin, a novel once a day orally dosed small molecule that has both broad anti-viral and anti-inflammatory activities which may serve a two-pronged approach to the treatment of COVID-19 virus infection and the subsequent debilitating inflammatory effects that lead to ARDS and death, in high risk hospitalized COVID-19 patients. The Phase 3 clinical trial is a double-blind, multicenter, multinational, randomized (2:1), placebo-controlled trial evaluating daily oral doses of 9 mg sabizabulin for up to 21 days versus placebo in 300 hospitalized patients (200 subjects will be treated with sabizabulin and 100 subjects will receive placebo/standard of care) who tested positive for the SARS-CoV-2 virus and who are at high risk for ARDS. Because of better oral bioavailability, the systemic blood levels from the 9 mg sabizabulin dosage are similar to the 18 mg sabizabulin formulation used in the Phase 2 clinical study. Subjects in the sabizabulin and placebo arms will also be allowed to receive standard of care. The primary efficacy endpoint will be proportion of patients that die on study up to Day 60. Secondary endpoints will include the proportion of patients without respiratory failure, days in ICU, WHO Ordinal Scale for Clinical Improvement change from baseline, days on mechanical ventilations, days in the hospital, and viral load. The study will be conducted in the United States, Brazil, Argentina, Mexico, and Colombia. Enrollment is targeted to be completed by calendar year-end.

In February of this year, the Company announced positive clinical results from the Phase 2 trial evaluating sabizabulin for the treatment of hospitalized patients with COVID-19 who were at high risk for ARDS. We conducted a double-blind, randomized, placebo-controlled Phase 2 clinical trial evaluating daily oral once a day dosing of sabizabulin 18 mg versus placebo in approximately 40 hospitalized COVID-19 patients who were at high risk for ARDS. This trial was conducted in 5 sites across the United States. Patients that were hospitalized with documented evidence of COVID-19 infection with symptoms and who were at high risk for ARDS were enrolled. Subjects received either sabizabulin 18 mg or placebo as well as standard of care for 21 days or until released from hospital. The primary efficacy endpoint was the proportion of patients that were alive without respiratory failure at Day 29. For the primary endpoint in the modified intent to treat population, sabizabulin compared to placebo had a statistically significant and clinically meaningful 81% relative reduction in death or respiratory failure at Day 29. With respect to secondary endpoints, sabizabulin had a statistically significant 82% relative reduction in patient mortality and statistically significant reduction in days in ICU; there was also a decrease in days on mechanical ventilation versus placebo. Sabizabulin was well tolerated with a good safety profile.

Sabizabulin a Novel, Oral, Androgen Receptor Transport Disruptor for the Treatment of Metastatic Castration and Androgen Receptor Targeting Agent Resistant Prostate Cancer – Phase 3 VERACITY Clinical Study.

Sabizabulin is a novel, oral, new chemical entity that targets microtubules in the cytoskeleton to disrupt androgen receptor transport. We anticipate enrolling patients this month into the open label, randomized (2:1), multicenter Phase 3 VERACITY clinical trial of sabizabulin 32 mg versus an alternative androgen receptor targeting agent for the treatment of chemotherapy naïve men with metastatic castration resistant prostate cancer who have failed at least one androgen receptor targeting agent. Based on the recently conducted Phase 2 PK study, the blood levels of the Phase 3 clinical trial sabizabulin 32 mg drug dose formulation are similar to the Phase 1b/2 VERU-111 63 mg dosage formulation. The primary endpoint is median radiographic progression free survival. The Phase 3 VERACITY clinical trial is expected to enroll approximately 245 patients.

In the Phase 1b /Phase 2 clinical trial in metastatic castration and androgen receptor targeting agent resistant prostate cancer, chronic daily administration of sabizabulin was well tolerated with a good safety profile. There was also evidence of efficacy including PSA declines and objective and durable tumor responses (partial and complete responses).

VERU-100, a Novel, Proprietary Long-Acting Gonadotropin-Releasing Hormone (GnRH) Antagonist Peptide, 3-Month Subcutaneous Depot Formulation, for Androgen Deprivation Therapy of Advanced Prostate Cancer – Phase 2 Clinical Study.

We anticipate initiation of the Phase 2 clinical trial of VERU-100 androgen deprivation therapy for hormone sensitive advanced prostate cancer this month. The Phase 2 VERU-100 clinical trial is expected to enroll approximately 35 patients. VERU-100 formulation is designed to address the current limitations of commercially available ADT. Androgen deprivation therapy is currently the mainstay of advanced prostate cancer treatment and is used as a foundation of treatment throughout the course of the disease even as other endocrine, chemotherapy, or radiation treatments are added or stopped. Specifically, VERU-100 is a chronic, long-acting GnRH antagonist peptide administered as a small volume, three-month depot subcutaneous injection without a loading dose. VERU-100 immediately suppresses testosterone with no testosterone surge upon initial or repeated administration, a problem that occurs with currently approved luteinizing hormone-releasing hormone (LHRH) agonists used for ADT. There are no GnRH antagonist depot injectable formulations commercially approved beyond a one-month injection. A Phase 3 registration clinical trial in approximately 100 men is anticipated to begin in the second half of calendar year 2021.

Enobosarm a Novel Oral Selective Androgen Receptor Targeted Agonist, for the Treatment of Androgen Receptor Positive (AR+), Estrogen Receptor Positive (ER+) and Human Epidermal Growth Factor Receptor 2 Negative (HER2-) Metastatic Breast Cancer – Phase 3 ARTEST Clinical Study and Phase 2 Enobosarm Combination Study.

We expect to commence our pivotal enobosarm Phase 3 ARTEST clinical study in the second quarter of calendar year 2021. Enobosarm is the first new class of targeting endocrine therapy in advanced breast cancer in decades. Enobosarm is an oral, new chemical entity, selective androgen receptor agonist that targets and activates the androgen receptor (AR), a tumor suppressor, in AR+ER+HER2- metastatic breast cancer without the unwanted masculinizing side effects. Enobosarm has extensive nonclinical and clinical experience having been evaluated in 25 separate clinical studies in approximately 1,450 treated subjects, including three Phase 2 clinical studies in advanced breast cancer involving more than 250 patients. In the two Phase 2 clinical studies conducted in women with AR+ER+HER2- metastatic breast cancer, enobosarm demonstrated significant antitumor efficacy in heavily pretreated cohorts that failed estrogen receptor targeting agents, chemotherapy, and/or CDK 4/6 inhibitors and was well tolerated with a favorable safety profile. In the fourth quarter of calendar 2020, the FDA agreed to the Phase 3 multicenter, international, open label, and randomized (1:1) ARTEST registration clinical trial design to evaluate the efficacy and safety of enobosarm monotherapy versus physician’s choice of either exemestane or a SERM as an active comparator for the treatment of metastatic AR+ER+HER2- breast cancer in approximately 210 patients who have failed a nonsteroidal aromatase inhibitor, fulvestrant and a CDK4/6 inhibitor (3rd line treatment in a metastatic setting) The primary endpoint is median radiographic progression-free survival. In a separate clinical development program, enobosarm in combination with abemaciclib, CDK4/6 inhibitor, will be evaluated in a 2nd line metastatic setting in AR+ER+ metastatic breast cancer. A Phase 2 study to evaluate the efficacy and safety of enobosarm in combination with CDK 4/6 inhibitor (abemaciclib) compared to estrogen receptor blocking agent (Active Control ) for the treatment of AR+ER+HER2- metastatic breast cancer in patients that have failed an estrogen receptor blocking agent plus a CDK 4/6 inhibitor (palbociclib) is expected to commence in calendar Q3 2021.

Sabizabulin a Novel, Oral, Cytoskeleton Disruptor Agent for the Treatment of Systemic Chemotherapy including Taxane Resistant Metastatic Triple Negative Breast Cancer – Phase 2b Clinical Study.

Sabizabulin is also being evaluated for the treatment of taxane chemotherapy resistant metastatic triple negative breast cancer in a planned Phase 2b clinical study in approximately 200 women expected to begin in calendar Q3 2021. Metastatic triple negative breast cancer is an aggressive form of breast cancer that occurs in approximately 15% of all breast cancers. This form of breast cancer does not express ER, progesterone receptor (PR), or HER2 and is resistant to endocrine therapies. The first line of treatment usually includes combination chemotherapy which includes IV taxane chemotherapy. Almost all women will eventually develop taxane resistance. Sabizabulin is an oral, first-in-class, new chemical entity that targets and inhibits microtubules to disrupt the cytoskeleton. Sabizabulin is not a substrate for P-glycoprotein drug resistance protein. Over expression of P-glycoprotein is a common mechanism that results in taxane resistance in triple negative breast cancer. Preclinical studies in human triple negative breast cancer grown in animal models demonstrate that sabizabulin significantly inhibits cancer proliferation, migration, metastases, and invasion of triple negative breast cancer cells and tumors that have become resistant to paclitaxel (taxane). Using the safety information from the Phase 1b and Phase 2 sabizabulin prostate cancer clinical studies, the Company plans to meet with the FDA and to commence a three cohort Phase 2b clinical study in calendar Q3 2021 to evaluate oral daily dosing of sabizabulin monotherapy, TRODELVY monotherapy, and sabizabulin + TRODELVY combination therapy in approximately 200 women with metastatic triple negative breast cancer that have become resistant to at least 2 systemic chemotherapies including a taxane.

TADFIN (Tadalafil 5mg and Finasteride 5mg Combination Capsule) for the Treatment of Lower Urinary Tract Symptoms Caused by Benign Prostatic Hyperplasia (BPH) – NDA Filed by FDA; PDUFA Date December 2021.

TADFIN (tadalafil 5mg and finasteride 5mg combination capsule) was developed to treat urinary tract symptoms caused by BPH. Tadalafil (CIALIS) is currently approved for treatment of BPH and erectile dysfunction and finasteride is currently approved for treatment of BPH (finasteride 5mg PROSCAR) and male pattern hair loss (finasteride 1mg PROPECIA). The co-administration of tadalafil and finasteride has been shown to be more effective for the treatment of BPH than finasteride alone with the additional benefit of ameliorating erectile dysfunction. An NDA for TADFIN has been accepted for review by FDA with a PDUFA date in December 2021. If approved, TADFIN is expected to be marketed and distributed by telemedicine and telepharmacy groups.

Legacy Female Health Business
As previously announced, the Company continues to explore the full range of strategic alternatives for its legacy Female Health Company Business, which markets the FC2 Female Condom (Internal Condom), including continuing to operate the business.

Non-GAAP Financial Information
Certain financial results for fiscal years 2021 and 2020 are presented on both a reported and a non-GAAP, adjusted basis. Reported results were prepared in accordance with U.S. GAAP and include all revenue and expenses recognized during the period. The non-GAAP results are adjusted to exclude the one-time gain on sale of PREBOOST in the first quarter of fiscal year 2021. Management believes non-GAAP financial measures provide useful information to investors regarding the Company’s results of operations and assist management, analysts, and investors in evaluating the performance of the Company’s business. Non-GAAP financial measures should be considered in addition to, and not as a substitute for, measures of financial performance prepared in accordance with GAAP. The Company has reconciled these non-GAAP financial measures to the nearest reported GAAP measures in the reconciliation table below.

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