On December 5, 2022 Veru Inc. (NASDAQ: VERU), a biopharmaceutical company focused on developing novel medicines for COVID-19 and other viral ARDS-related diseases and for oncology, reported financial results for its fiscal 2022 fourth quarter and full year ended September 30, 2022 and provided a business update (Press release, Veru, DEC 5, 2022, View Source [SID1234624805]).

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"This has been a transformational year for Veru. We reported positive Phase 3 results demonstrating that sabizabulin treatment resulted in a statistically and clinically significant reduction in death in hospitalized moderate to severe COVID-19 patients at high risk for ARDS and death, which was published in the NEJM Evidence," said Mitchell Steiner, M.D., Chairman, President, and Chief Executive Officer of Veru. "As sabizabulin is being reviewed in the U.S. and internationally for potential emergency use authorization, we are preparing for commercialization and will be ready to deliver this treatment to patients, if authorized."

Dr. Steiner added, "While FC2 revenue decreased this past year due to business challenges experienced by our largest telemedicine customers, we are working diligently to regenerate FC2 product sales, and our recently launched telemedicine platform has shown steady market uptake to date."

Infectious Disease Program Highlights

Sabizabulin: A Novel Oral, First-in-Class, Microtubule Disruptor for the Treatment of Hospitalized Moderate to Severe COVID-19 Patients at High Risk for Acute Respiratory Distress Syndrome (ARDS)

In November 2022, the U.S. FDA’s Pulmonary-Allergy Drugs Advisory Committee met with the Company to review its request for Emergency Use Authorization (EUA) of sabizabulin. The Advisory Committee voted 8-5 that the known and potential benefits of sabizabulin when used for the treatment of adult patients hospitalized with COVID-19 at high risk of ARDS do not outweigh the known and potential risks of sabizabulin. The FDA considers the Advisory Committee’s input as part of their review, but the FDA makes the final decision on issuing an EUA.

In October 2022, the Company presented data from the Phase 3 trial of sabizabulin in a late-breaker oral presentation at IDWeek (Infectious Disease Week) 2022.

In August 2022, Australia’s Therapeutic Goods Administration (TGA) granted the Company an expedited provisional registration regulatory pathway for sabizabulin treatment in hospitalized COVID-19 patients at high risk for ARDS.

In August 2022, the Company presented the Phase 3 trial results of sabizabulin at the 11th International Conference on Emerging Infectious Diseases (ICEID).

In July 2022, European Medicines Agency’s (EMA) Emergency Task Force (ETF) initiated the review of sabizabulin for emergency use in the EU member states.

In July 2022, United Kingdom’s (UK’s) Medicines and Healthcare Products Regulatory Agency (MHRA) supported an expedited review of the marketing authorization application for sabizabulin treatment in hospitalized COVID-19 patients at high risk for ARDS.

On July 6, 2022, The New England Journal of Medicine Evidence published results from the Phase 3 trial evaluating the efficacy and safety of oral sabizabulin in hospitalized COVID-19 patients.

Breast Cancer Program Highlights

Enobosarm, a Novel Oral Selective Androgen Receptor Targeting Agonist, for the 3rd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer with sufficient AR Expression

We are enrolling patients in the Phase 3 multicenter, international, open label, randomized (1:1) ARTEST registrational trial to evaluate enobosarm versus either exemestane ± everolimus or a selective estrogen receptor modulator (SERM) as the active comparator for the treatment of AR+ER+HER2- metastatic breast cancer in approximately 210 patients with sufficient AR expression in their breast cancer tissue who had previously received a nonsteroidal aromatase inhibitor, fulvestrant, and a CDK4/6 inhibitor. In January 2022, the FDA granted Fast Track designation to the ARTEST Phase 3 registrational program.

Enobosarm and Abemaciclib, CDK 4/6 Inhibitor, Combination Therapy for the 2nd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer with sufficient AR Expression

We are enrolling patients in the Phase 3 multicenter, open label, randomized (1:1), active control ENABLAR-2 trial to evaluate enobosarm and abemaciclib combination versus an alternative estrogen blocking agent (fulvestrant or an aromatase inhibitor) in subjects with AR+ER+HER2- metastatic breast cancer who have failed first line palbociclib (a CDK 4/6 inhibitor) plus an estrogen blocking agent (non-steroidal aromatase inhibitor or fulvestrant) and who have sufficient AR expression in their breast cancer tissue in approximately 186 subjects. We have a collaboration and supply agreement with Eli Lilly for this trial.

Sabizabulin for the 3rd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer with sufficient AR Expression

We intend to conduct a Phase 2b open label, multicenter, randomized (1:1) trial evaluating sabizabulin 32mg versus active comparator (exemestane ± everolimus or a SERM, physician’s choice) for the treatment of AR+ER+HER2- metastatic breast cancer in approximately 200 patients with sufficient AR expression in their breast cancer tissue who have previously received a nonsteroidal aromatase inhibitor, fulvestrant, and a CDK4/6 inhibitor.

Prostate Cancer Program Highlights

Sabizabulin for the Treatment of Metastatic Castration and Androgen Receptor Targeting Agent Resistant Prostate Cancer

We are enrolling patients in the Phase 3 VERACITY open label, randomized (2:1), multicenter trial evaluating sabizabulin 32mg versus an alternative androgen receptor targeting agent for the treatment of chemotherapy naïve men with metastatic castration resistant prostate cancer who have tumor progression after previously receiving at least one androgen receptor targeting agent. The primary endpoint is radiographic progression free survival in approximately 245 patients.

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

We are enrolling patients in the Phase 2 clinical dose finding trial of VERU-100 for androgen deprivation therapy of advanced prostate cancer. The trial design for a future Phase 3 registrational trial of approximately 100 patients has been agreed upon by the FDA.

Urev – Sexual Health Program Highlights

ENTADFI (tadalafil and finasteride) capsule, a new Treatment for Benign Prostatic Hyperplasia (BPH)

The Company recently initiated the U.S. commercial launch and availability of ENTADFI– an FDA-approved oral, once daily product for benign prostatic hyperplasia (BPH) that is approved for men with an enlarged prostate that are experiencing the signs and symptoms of BPH for up to 26 weeks.

FC2 Female Condom/Internal Condom

The Company markets and sells the FC2 Female Condom, an FDA-approved product for dual protection against unplanned pregnancy and the transmission of sexually transmitted infections.

Full Year Financial Summary: Fiscal 2022 vs Fiscal 2021

Total net revenues decreased to $39.4 million from $61.3 million

Gross profit decreased to $30.6 million from $47.9 million

Gross margin remained consistent at 78% of net revenues

Research and development expenses increased to $70.6 million from $32.7 million

Operating loss was $83.2 million compared with operating income of $13.0 million, which included an $18.4 million gain on the December 2020 sale of the PREBOOST business

Net loss was $83.8 million, or $1.05 per diluted share, compared with net income, which included the gain on sale of the PREBOOST business, of $7.4 million, or $0.09 per diluted share

Balance Sheet Information

Cash and cash equivalents were $80.2 million as of September 30, 2022 versus $122.4 million as of September 30, 2021

Net accounts receivable were $3.6 million as of September 30, 2022 versus $8.8 million as of September 30, 2021

Event Details

The audio webcast will be accessible under "Investor Kit" in the Investors page of the Company’s website at www.verupharma.com. To join the conference call via telephone, please dial 1-800-341-1602 (domestic) or 1-412-902-6706 (international) and ask to join the Veru Inc. call. An archived version of the audio webcast will be available for replay on the Company’s website for approximately three months. A telephonic replay will be available on December 5, 2022 at approximately 12:00 p.m. ET by dialing 1-877-344-7529 (domestic) or 1-412-317-0088 passcode 4646397 (international) for one week.

On December 5, 2022 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will host a virtual event to discuss initial data from the safety lead-in portion of the ongoing Phase 2 SELECT-AML-1 clinical trial and to review the unmet need in newly diagnosed, unfit acute myeloid leukemia (AML) (Press release, Syros Pharmaceuticals, DEC 5, 2022, View Source [SID1234624804]). The event will be held on Saturday, December 10, 2022 beginning at 12:00 p.m. ET.

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The event will feature presentations by Syros management, who will review the initial SELECT-AML-1 data and plans for the randomized portion of the Phase 2 trial, as well as Daniel Pollyea, M.D., M.S., Associate Professor of Medicine, Clinical Director of Leukemia Services, University of Colorado School of Medicine, who will provide his perspective on the evolving treatment landscape.

Participants can register for the event here. In addition, a live webcast of the presentation will be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

Initial data from the SELECT-AML-1 trial will also be presented on Saturday, December 10, 2022 in a poster session at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

On December 5, 2022 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for revumenib for the treatment of adult and pediatric patients with relapsed or refractory (R/R) acute leukemia harboring a KMT2A rearrangement (KMT2Ar) (Press release, Syndax, DEC 5, 2022, View Source [SID1234624803]). Revumenib is the Company’s highly selective, oral menin inhibitor.

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"The Breakthrough Therapy Designation underscores revumenib’s potential as a first- and best-in-class therapy to meaningfully change the treatment paradigm for patients with R/R KMT2Ar acute leukemia, whether it presents clinically as acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL), in adults or children," said Michael A. Metzger, Chief Executive Officer. "Revumenib has the potential, if approved, to be the first drug to address the significant unmet need in KMT2Ar leukemia believed to occur in up to 10% of all acute leukemias, including in approximately 80% of infant acute leukemias. Syndax is committed to bringing revumenib to these patients as quickly as possible and we look forward to working collaboratively with the FDA to expedite a potential approval of revumenib."

The BTD is supported by Phase 1 data from the AUGMENT-101 trial. Ten of 37 patients (27%) with age and phenotype agnostic KMT2Ar acute leukemia treated at doses meeting the protocol defined criteria for the recommended Phase 2 dose (RP2D) and evaluable for efficacy as of the March 2022 data cutoff achieved a complete remission as measured by a CR/CRh. Included in this analysis were patients treated in Arm A (226 and 276 mg q12 hours not receiving a strong CYP3A4 inhibitor) and Arm B (113 and 163 mg q12 hours receiving a strong CYP3A4 inhibitor).

As previously announced, additional data from the Phase 1 portion of the AUGMENT-101 trial will be presented during two oral sessions at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 10, 2022. The abstracts (Abstracts #63 and #376) describe data on the 60 patients with R/R mutant NPM1 (n=14) or KMT2A rearranged (MLLr; n=46) acute leukemia that were evaluable for efficacy as of the March 2022 data cutoff date. Additional analyses from the trial that will be presented at the ASH (Free ASH Whitepaper) Annual Meeting indicate a 27% CR/CRh rate at doses meeting the protocol defined criteria for the RP2D in all efficacy evaluable patients (13/48) and in patients with an NPM1 mutation (3/11). There were no discontinuations due to treatment-related adverse events.

The FDA grants BTD to expedite the development and regulatory review of drugs that are intended for serious or life-threatening conditions. The designation is based on preliminary clinical evidence indicating that a drug may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy. BTD affords all of the benefits of the fast track program, eligibility for rolling review and potentially priority review, and additional engagement to facilitate an expedited development plan and regulatory review.

About Revumenib

Revumenib is a potent, selective, small molecule inhibitor of the menin-MLL binding interaction that is being developed for the treatment of KMT2A rearranged, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), and NPM1 mutant AML. Revumenib is currently being evaluated in several clinical trials, including the Company’s pivotal AUGMENT-101 Phase 1/2 open-label clinical trial for the treatment of relapsed/refractory (R/R) acute leukemias. Robust clinical activity with durable responses have been reported in the Phase 1 portion of AUGMENT-101. Revumenib was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Commission for the treatment of patients with AML, and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation.

About KMT2A (MLL) Rearranged Acute Leukemia

Rearrangements of the KMT2A (mixed lineage leukemia or MLL) gene give rise to KMT2Ar acute leukemia known to have a poor prognosis, with less than 25% of adult patients surviving past five years. KMT2A genes produce fusion proteins that require interaction with the protein called menin to drive leukemic cancer growth. Disruption of the menin-KMT2Ar interaction has been shown to halt the growth of KMT2Ar leukemic cells.

KMT2Ar acute leukemia can phenotypically appear as AML, ALL, or mixed phenotype acute leukemia (MPAL) and is routinely diagnosed through currently available cytogenetic or molecular diagnostic techniques. The median overall survival (OS) after standard of care first-line treatment, including intensive chemotherapy and transplant, is less than 1 year and the majority of patients suffer relapse within 5 years. Most R/R patients treated with second-line therapy relapse within the first year. With third line treatment or beyond, only a small percentage of patients achieve complete remission (CR), and the median OS is less than 3 months.

About AUGMENT-101

AUGMENT-101 is a Phase 1/2 open-label trial designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of orally administered revumenib. The Phase 1 dose escalation portion of AUGMENT-101 was separated into two cohorts based on concomitant treatment with a strong CYP3A4 inhibitor. Arm A enrolled patients not receiving a strong CYP3A4 inhibitor, while Arm B enrolled patients receiving a strong CYP3A4 inhibitor. The Phase 2 pivotal portion of AUGMENT-101 is currently underway. Patients will be enrolled across each of the following trial populations: patients with NPM1 mutant AML, patients with KMT2Ar (MLLr) AML, and patients with KMT2Ar (MLLr) ALL. Discussions with the FDA have confirmed that AUGMENT-101 may potentially serve as the basis for regulatory filings in each patient population. The primary endpoint for each of the trials will be efficacy as measured by complete remission rate (CR + CRh), with key secondary endpoints including duration of response (DOR) and overall survival (OS).

Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX) and Incyte (Nasdaq: INCY) reported that results from the Phase 1/2 trial of axatilimab, Syndax’s anti-CSF-1R antibody, in patients with recurrent or refractory chronic graft-versus-host disease (cGVHD) following two or more prior lines of therapy, were published in the Journal of Clinical Oncology (Press release, Syndax, DEC 5, 2022, View Source [SID1234624802]).

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A total of 40 patients with refractory disease who received a median of four prior systemic therapies, some of which included ibrutinib, ruxolitinib and belumosudil, were treated in the Phase 1/2 dose escalation trial. Thirty-nine patients were evaluable for response as of the data cutoff. All study participants were treated for a median duration of 29 weeks, with all responding patients treated for a median duration of 38 weeks at the time of data cut-off. Results showed:

Overall response rate by cycle 7 day 1 was 82% (18/22) in the Phase 2 cohort (1mg/kg every two weeks) and 67% (26/39) among all evaluable patients treated in the dose escalation study. Best ORR (complete response + partial response) observed at any point during the study in all patients treated was 69% (27/39).
Median duration of response for Phase 2 responding patients was not reached; 33% of patients experiencing sustained response lasting 20 weeks or longer.
A decrease in glucocorticoids doses was observed in 52% of responding patients.
Responses were observed across a range of organ systems with difficult to treat manifestations such as lung (5/16), skin (5/35), and joints and fascia (19/31).
A clinically meaningful disease improvement using the Lee Symptom Score of at least 7 points was seen in 58% (21/36) of all evaluable patients.
Axatilimab was well tolerated with a favorable safety profile in this refractory population. The most common adverse events were consistent with on-target effects of CSF-1R inhibition. In the Phase 1 cohort, two dose limiting toxicities were reported, both at the 3 mg/kg every two weeks dose. There were no ≥Grade 3 on-target toxicities of CSF-1R blockade seen in the Phase 2 cohort. There was no incidence of cytomegalovirus or other viral reactivation, and no apparent increases in risk for infection. Serious adverse events occurred in 40% (16/40) of patients, with seven (18%) patients discontinuing the study intervention due to adverse events, four (10%) of which were deemed treatment-related.

"We believe axatilimab is well positioned to potentially be a first- and best-in-class anti-CSF-1R antibody for cGVHD," said Kate Madigan, M.D., Chief Medical Officer of Syndax. "The robust, durable responses and multi-organ clinical benefit observed in this Phase 1/2 trial in refractory patients support the potential for axatilimab to serve as an effective intervention in this underserved population."

"The results of this Phase 1/2 dose finding study are very encouraging regarding the ability of axatilimab to provide meaningful clinical responses in chronic GVHD patients that have been refractory to multiple prior therapies" said Carrie Kitko, M.D., Medical Director of the Pediatric Stem Cell Transplant Program at the Vanderbilt-Ingram Cancer Center, and the corresponding author of the Phase 1/2 publication. "Combining both anti-inflammatory and anti-fibrotic effects through the targeting of disease associated macrophages would be particularly exciting for this patient population that would benefit from a reduction in sclerotic and fibrotic manifestations of chronic GVHD. In the past simply stopping further progression of those manifestations was considered a "win", but some of these patients are actually having improvements in joint range of motion and skin tightening."

The article, titled "Axatilimab for chronic graft-versus-host disease after failure of at least two prior systemic therapies: results of a Phase 1/2 study," is available online.

About Chronic Graft-Versus-Host Disease
Chronic graft-versus-host disease, an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation which can last for years. Chronic GVHD is estimated to develop in approximately 40% of transplant recipients, and affects approximately 14,000 patients in the U.S.1,2 Chronic GVHD typically manifests across multiple organ systems, with skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.3

About Axatilimab
Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as cGVHD and IPF. Phase 1/2 data of Axatilimab in cGVHD demonstrating its broad activity and tolerability was last presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.

Enrollment in the Company’s global pivotal Phase 2 AGAVE-201 Phase 2 study evaluating the efficacy, safety, and tolerability of axatilimab in patients with recurrent or refractory active cGVHD who have received at least two prior lines of systemic therapy is complete, and topline data is expected mid-2023. Additionally, a Phase 1 combination trial of ruxolitinib and axatilimab, led by Incyte, is in preparation and expected to initiate by end of the first quarter of 2023, and a Phase 2b trial of axatilimab in patients with idiopathic pulmonary fibrosis led by Syndax is expected to begin in the fourth quarter of 2022.

For more information about AGAVE-201, visit View Source

On December 5, 2022 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported that on December 5, 2022, Spectrum’s Board of Directors granted an aggregate of 736,988 inducement restricted stock units ("RSUs") to commercial non-executive employees who were hired specifically for the launch of ROLVEDON (Press release, Spectrum Pharmaceuticals, DEC 5, 2022, View Source [SID1234624799]). The awards were granted under Spectrum’s 2022 Employment Inducement Incentive Award Plan ("Inducement Plan") as employment inducement awards pursuant to Nasdaq Listing Rule 5635(c)(4). The Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously employees of Spectrum, or following a bona fide period of non-employment, as an inducement material to such individuals’ entering into employment with Spectrum, pursuant to Nasdaq Listing Rule 5635(c)(4).

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The RSUs will vest over three years on the first three anniversaries of the grant date, subject to continued service through each applicable vesting date.