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Salarius Pharmaceuticals Reports Business Highlights with Fourth Quarter and Full-Year 2021 Financial Results

On March 10, 2022 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing cancer therapies for patients in need of new treatment options, reported important corporate events and its financial results for the full year and the fourth quarter ended December 31, 2021 (Press release, Salarius Pharmaceuticals, MAR 10, 2022, View Source [SID1234609950]).

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We believe the progress made by Salarius Pharmaceuticals during 2021 and the early weeks of 2022 has once again been remarkable, including the Company’s recent expansion into the field of targeted protein degradation (TPD)," stated David Arthur, CEO of Salarius Pharmaceuticals. "TPD is a fast-growing field of cancer drug development with commercial market potential estimated in billions of dollars and enticing significant investment from some of the world’s largest pharmaceutical companies. TPD has the ability to develop drugs for historically undruggable cancer-promoting targets and our new TPD development program, built around the portfolio of assets acquired by Salarius in January 2022, is led by the product candidate SP-3164. Salarius now boasts a multipronged pipeline built around two exciting approaches to cancer drug development – protein inhibition and targeted protein degradation."

Financial Highlights:
•Cash and cash equivalents total $29.2 million on December 31, 2021, with an estimated runway into 2023
•For the three-month and 12-month periods ended December 31, 2021, net loss per common share, basic and diluted, of $0.09 and $0.31, respectively, compared to $0.10 and $0.50, respectively, for the same periods in 2020
Recent Business and Corporate Highlights:
•Acquisition of TPD assets from DeuteRx LLC in January 2022 expands Salarius’ oncology pipeline into a fast-growing area of drug research focused on pursuing historically undruggable cancer-promoting targets with estimated multi-billion-dollar commercial market potential
◦
◦Acquisition included the drug candidate SP-3164, additional targeted protein degrader programs, and a related intellectual property (IP) portfolio that includes issued composition of matter patents
◦SP-3164 is an orally administered cereblon-binding targeted protein degrader developed as a next-generation version of the widely studied molecular glue, avadomide (CC-122) with the potential for an improved efficacy and safety profile
SP-3164 leverages the data from 10 clinical trials investigating avadomide in more than 400 patients
Preclinical data suggests the potential for SP-3164 to be more efficacious than avadomide
Salarius will continue collaborating with DeuteRx scientists to complete Investigational New Drug (IND) enabling studies for SP-3164
First clinical trial expected in 2023
◦Consideration consists of cash and stock, with an upfront payment of $1.5 million in cash and 1 million shares of restricted stock; future payments linked to clinical, regulatory, and commercial milestones, and net sales royalties
•Salarius continues to advance clinical development of seclidemstat; data updates expected in 2022 from two Phase 1/2 clinical trials
◦Sarcoma trial investigating seclidemstat in combination with chemotherapy agents topotecan and cyclophosphamide (TC), in patients with relapsed/refractory Ewing sarcoma advanced to second lead-in safety dosing cohort treating patients with seclidemstat at 900 mg BID
◦Sarcoma trial investigating seclidemstat as a single-agent therapy in Myxoid Liposarcoma and other FET-rearranged sarcomas continues enrollment
◦Enrollment continues in investigator-initiated trial (IIT) at MD Anderson Cancer Center exploring seclidemstat in combination with azacytidine as a treatment for hematologic cancers; updates on biomarkers including immature cells (blasts) and drug activity measures expected during 2022
◦Research underway to identify new indications for seclidemstat in established large markets
Mr. Arthur concluded, "2022 is a year of significant optimism at Salarius given the potential value-building opportunities and milestones on the horizon. Salarius is moving to rapidly advance the development of SP-3164 with plans to file an IND and initiate our first clinical trial for that program in 2023. Building on our optimism around SP-3164 and based on the abundance of preclinical and clinical data collected around its predecessor, avadomide, it is our belief that SP-3164 will establish a superior safety profile with the potential to be more effective. Additionally, we continue advancing the clinical exploration of seclidemstat, our oral, reversible LSD1 inhibitor and Salarius’ most advanced investigational cancer drug candidate. We look forward to providing data updates in the coming months."

Three-Month and 12-Month Financial Results:
For the three-month period ended December 31, 2021, Salarius’ reported net loss was $4.1 million, or $0.09 per basic and diluted share, compared to a net loss of $1.8 million, or $0.10 per basic and diluted share for the same period in 2020. The loss for the three-month period ended December 31, 2021, increased by $2.3 million compared to the same time span last year, primarily because the Company had higher overall costs and no grant revenue in the current period.
For the 12-month period ended December 31, 2021, Salarius’ reported net loss was $12.8 million, or $0.31 per basic and diluted share, compared to a net loss of $7.4 million, or $0.50 per basic and diluted share for the same period in 2020. The loss for the 12-month period ended December 31, 2021, increased by $5.4 million compared to the loss from operations for the same period last year, resulting from increased charges related to R&D personnel and higher clinical trial costs more than offsetting lower drug development costs.
Net cash used for operating activities during the 12-month period ended December 31, 2021, totaled $10.2 million, essentially the same as approximately $10.3 million in the prior year. During 2021, the Company collected approximately $4.1 million on its outstanding grant receivable compared to $0.8 million in the prior year.
As of December 31, 2021, total cash, cash equivalents, and restricted cash were $29.2 million, compared to $11.1 million at year-end 2020. The increase in cash was primarily driven by the sale of equity securities during the first quarter of 2021. Current cash and cash equivalents are expected to fund our current planned operations into 2023.
Conference Call Information:
Salarius Pharmaceuticals will host a conference call and live audio webcast on Thursday, March 10, 2022, at 5:00 p.m. ET, to discuss its corporate and financial results for the fourth quarter and full-year 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 6:00 p.m. ET on March 10, 2022.

Jubilant Therapeutics Inc. to Present Data on Isoform Selective PAD4 inhibitors in the Treatment of Cancer at the American Association of Cancer Research Annual (AACR) Meeting 2022

On March 10, 2022 Jubilant Therapeutics Inc. (‘Jubilant’), a biopharmaceutical company advancing small molecule precision therapeutics to address unmet medical needs in oncology and autoimmune diseases, reported that it will present data on inhibitors of peptidyl arginine deiminase type-4 (PAD4), a potential therapeutic target to treat cancer and autoimmune diseases (Press release, Jubilant Therapeutics, MAR 10, 2022, View Source [SID1234609940]). The meeting will be held at the American Association of Cancer Research Annual (AACR) (Free AACR Whitepaper) in New Orleans between April 8 to 13, 2022, both in person and virtually.

Jubilant Therapeutics Inc. to Present Data on Isoform Selective PAD4 inhibitors in the Treatment of Cancer at the American Association of Cancer Research Annual (AACR) (Free AACR Whitepaper) Meeting 2022
Jubilant Therapeutics Inc. to Present Data on Isoform Selective PAD4 inhibitors in the Treatment of Cancer at the American Association of Cancer Research Annual (AACR) (Free AACR Whitepaper) Meeting 2022
Details of the presentation are as follows:

Title: Novel, isoform selective PAD4 inhibitors for the treatment of Cancer
Presentation Type: Poster
Presenters: Luca Rastelli, Chief Scientific Officer
Dhanalakshmi Sivanandhan, Vice President – Discovery Biology
Session: Novel Targets and Pathways
Time and Date: 12th April 2022, 9:00 AM – 12:30 PM
Link to Abstract: View Source!/10517/presentation/15071

About PAD4

Peptidyl Arginine Deiminase type-4 (PAD4) is an enzyme that converts protein arginine or mono-methylarginine to citrulline. The PAD4-mediated hypercitrullination reaction in neutrophils causes the release of nuclear chromatin to form a chromatin network termed Neutrophil Extracellular Traps (NETs). NETs have been shown to be associated with several pathological processes including cancer metastasis, fibrosis, ischemic stroke, preeclampsia, thrombosis and other autoimmune diseases. Jubliant Therapeutics Inc. is targeting PAD4 inhibition as a novel strategy to treat cancer progression & metastasis and autoimmune diseases without inducing immunosuppression as seen with most other agents approved for autoimmune diseases.

Eiger BioPharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Update

On March 10, 2022 Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), a commercial-stage biopharmaceutical company focused on the development of innovative therapies to treat and cure Hepatitis Delta Virus (HDV) and other serious diseases, reported financial results for fourth quarter and full year 2021 and provided a business update (Press release, Eiger Biopharmaceuticals, MAR 10, 2022, View Source [SID1234609938]).

"We expect 2022 to be a transformational year for Eiger with topline data planned from two Phase 3 studies," said David Cory, President and CEO of Eiger. "The Phase 3 TOGETHER study of Peginterferon Lambda for COVID-19 is expected to readout later this month and the Phase 3 D-LIVR study of Lonafarnib for HDV is planned to readout by end of year. We look forward to reporting results from these potentially registration enabling studies as well as progress across our other late-stage pipeline programs with multiple catalysts this year."

Program Highlights

Lonafarnib: Oral Prenylation Inhibitor for HDV

D-LIVR Phase 3 multi-center, global study
Two Lonafarnib-based regimens, both with potential for registration
Lonafarnib oral and Lonafarnib / peginterferon alfa combination
Fully enrolled N=407, with topline data planned by end of 2022
D-LIVR study design includes conservative powering assumptions, modeling response rates well below what was demonstrated in Phase 2
Ukraine and Russia Update
Ukraine:
Eiger continues to closely assess this evolving situation, prioritizing patient care and patient monitoring to ensure continuity in the study
Eiger believes the study remains more than adequately powered to demonstrate statistical significance over placebo even if these patients discontinue from study
Russia:
To date, no interruption to patient visits, safety monitoring, or drug supply; contingency plans in place to ensure continuity of drug supply, sample storage and analysis to preserve integrity of results
Peginterferon Lambda: Well-tolerated Interferon for HDV

LIMT-2 Phase 3 multi-center, global study
Peginterferon Lambda monotherapy for registration
Enrolling patients, targeting N=150
Peginterferon Lambda and Lonafarnib Combination for HDV

LIFT-2 Phase 2 study at National Institutes of Health
Initiating 1H22, targeting N=30
Potential to be interferon of choice in HDV combination therapies
Peginterferon Lambda for Non-hospitalized COVID-19 Infection

Novel mechanism of action, agnostic to variants and mutations
TOGETHER Phase 3 study fully enrolled, N>1,800
Includes unvaccinated and vaccinated patients across multiple variants
Topline data planned in March 2022
Avexitide for Rare Metabolic Disorders

Granted Breakthrough Therapy Designation for Congenital Hyperinsulinism (HI)
Granted Rare Pediatric Disease Designation for HI; PRV eligible
Phase 3 ready in 2022
Zokinvy for Progeria and Processing-Deficient Progeroid Laminopathies

Successful U.S. commercial launch
Approximately 80% of identified U.S. patients converted to commercial supply
EMA review of MAA
CHMP opinion expected in first half of 2022
Corporate

Cash, cash equivalents and investments of $106.1 million to begin 2022 expected to fund planned operations through Q3 2023
Fourth Quarter and Full Year 2021 Financial Results

Cash, cash equivalents, and total investments as of December 31, 2021 totaled $106.1 million compared to $128.8 million on December 31, 2020.

Net product sales of Zokinvy were $3.4 million for fourth quarter 2021, as compared to $3.0 million for third quarter 2021. The increase reflects shipment of product under a reimbursed early access program approved in France, partially offset by fewer shipments to the U.S. specialty pharmacy due to timing of patient refills. The Company commercially launched Zokinvy in the U.S. in January 2021 and reported full year 2021 net sales of $12.1 million.

Cost of Sales was $0.1 million and $0.7 million for fourth quarter and full year 2021, respectively, and related to certain manufacturing, shipping, and distribution costs associated with Zokinvy that were incurred after FDA approval.

Research and Development expenses were $18.2 million and $64.4 million for fourth quarter and full year 2021, respectively, as compared to $12.5 million and $41.6 million for the same periods in 2020. The increases in fourth quarter and full year 2021 expenses were primarily due to contract manufacturing and clinical trial related expenses for increased clinical development activities and personnel related expenses, including stock-based compensation, due to an increase in headcount.

Selling, General and Administrative expenses were $6.0 million and $23.9 million for fourth quarter and full year 2021, respectively, as compared to $5.4 million and $20.6 million for the same periods in 2020. The increases in fourth quarter and full year 2021 were primarily due to an increase in personnel related expenses attributed to an increase in headcount and an increase in outside services, including consulting and advisory services to support the growth of the Company.

Total operating expenses include non-cash expenses of $2.8 million and $10.7 million for fourth quarter and full year 2021, respectively, as compared to $2.1 million and $7.8 million for the same periods in 2020.

The Company reported net losses of $21.8 million, or $0.64 per share, and $33.9 million, or $1.00 per share, for fourth quarter and full year 2021, respectively, as compared to $18.8 million, or $0.58 per share, and $65.1 million, or $2.31 per share, for the same periods in 2020.

As of December 31, 2021, the company had 34.6 million of common shares outstanding.

CONFERENCE CALL
At 4:30 PM Eastern Time today, March 10, 2022, Eiger will host a conference call to discuss its financial results and provide a business update. The live and replayed webcast of the call will be available through the company’s website at www.eigerbio.com. To participate in the live call by phone, dial (844) 743-2495 (U.S.) or (661) 378-9529 (International) and enter conference ID 4338659. The webcast will be archived and available for replay for at least 90 days after the event.

AcelRx Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial Results

On March 10, 2022 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, reported its fourth quarter and full year 2021 financial results (Press release, AcelRx Pharmaceuticals, MAR 10, 2022, View Source [SID1234609937]).

"We are executing on our strategy to expand and diversify our product portfolio by acquiring commercial-ready, or late-stage development-ready assets that address what we consider as unmet market needs for medically supervised settings. In particular, we are currently focused on the development and ultimate approval of our newly secured pre-filled syringes and Niyad (nafamostat) product candidates which could potentially provide multiple value creating catalysts for our shareholders in the near-term," said Vince Angotti, Chief Executive Officer of AcelRx. Mr. Angotti continued, "In addition, we have taken deliberate actions to adapt to the evolving healthcare environment as many medical procedures resulting in moderate-to-severe pain that were previously performed in surgical centers or hospitals have now been shifted to procedural suites, allowing us the opportunity to introduce DSUVIA into these settings. We have thus shifted our commercial resources, beginning in the third quarter, to focus on this new customer base, resulting in solid growth in DSUVIA unit sales in the fourth quarter 2021."

FY 2021 and Recent Highlights

AcelRx entered into a license agreement with Laboratoire Aguettant (Aguettant) providing AcelRx with two innovative pre-filled syringe product candidates for the U.S. The expected market opportunity for these two product candidates exceeds $100 million, and AcelRx currently plans to file New Drug Applications for both in 2022.

A second license transaction with Aguettant was completed establishing Aguettant as the commercial partner for DZUVEO in Europe with an expected launch in the third quarter of 2022. AcelRx is entitled to receive up to approximately $55 million in combined up-front and sales-based milestone payments.

AcelRx announced the closing of its acquisition of Lowell Therapeutics, Inc. (Lowell) in January 2022 in a transaction for consideration of approximately $32.5 million plus net cash acquired and certain other adjustments, and which includes up to approximately $26.0 million of contingent consideration payable in cash or stock at AcelRx’s option, upon the achievement of regulatory and sales-based milestones. Niyad (nafamostat) is the lead product, with a targeted indication of anticoagulation of the extracorporeal circuit, and which has received Breakthrough Device Designation from the FDA, as well as an ICD-10 procedural code from CMS which allows for reimbursement. Annual peak sales potential for Niyad is expected to exceed $200 million.

Since January 2021, six articles on DSUVIA were published reporting the benefits of administering DSUVIA in place of IV opioids, including reducing perioperative opioid use, rapid recovery times, efficacy and safety among a wide range of demographics and the overall advantages of sublingual delivery. Of note, one of these articles was a commentary published in Military Medicine, identifying DSUVIA as the next evolution in battlefield pain management. An additional study of DSUVIA for painful cosmetic procedures has been accepted for publication by the American Journal of Cosmetic Surgery and another study on the use of DSUVIA during general anesthesia for lengthy plastic surgery procedures has been submitted for publication.

As of December 31, 2021, AcelRx has achieved 725 approvals compared to our initial target of 615. As of February 28, 2022, AcelRx has achieved 813 formulary approvals for DSUVIA.

In February 2022, AcelRx was notified that it had met all requirements set by the U.S. Food and Drug Administration (FDA) with regards to the FDA Warning Letter regarding certain DSUVIA promotional materials, dated February 11, 2021, and a Closeout Letter is expected in Q1 2022.
Financial Information

The cash, cash equivalents and short-term investments balance was $51.6 million as of December 31, 2021.

8,960 units of DSUVIA were sold in the fourth quarter of 2021, compared to 3,710 units in the third quarter of 2021; however, the Company has recognized only $2 thousand in net revenues in the fourth quarter 2021 as a result of a $0.3 million reserve for potential returns related to a certain wholesale customer that purchased product for the Department of Defense (DoD) in 2020. The DoD has purchased exclusively from a secondary wholesale customer instead of their primary wholesaler, and therefore the Company has recorded a reserve in the event this product is not ultimately sold to the DoD.

Unit sales growth in the first two months of Q1 2022, compared to the first two months of Q4 2021 is 63%.

Combined R&D and SG&A expenses for the fourth quarter of 2021 totaled $6.9 million compared to $8.7 million for the fourth quarter of 2020. Excluding non-cash depreciation and stock-based compensation expense, these amounts were $5.6 million for the fourth quarter of 2021, compared to $7.5 million for the fourth quarter of 2020. R&D and SG&A expenses for the year ended December 31, 2021 totaled $35.0 million compared $40.3 million for the year ended December 31, 2020. Excluding non-cash depreciation and stock-based compensation expense, these figures were $29.7 million for the year ended December 31, 2021, compared to $35.4 million for the year ended December 31, 2020. The decrease in combined R&D and SG&A expenses in the fourth quarter and year ended 2021 was primarily due to reductions in personnel-related costs, including travel expense, partially offset by increased Catalent manufacturing-related DSUVIA development expenses.

Net loss for the fourth quarter of 2021 was $7.9 million, or $0.06 per basic and diluted share, compared to $8.9 million, or $0.10 per basic and diluted share, for the fourth quarter of 2020. Net loss for the year ended December 31, 2021 was $35.1 million, or $0.29 per basic and diluted share, compared to $40.4 million, or $0.47 per basic and diluted share, for the year ended December 31, 2020.
2022 Guidance

The Company’s 2022 year-end goals include the submission of two NDAs for its pre-filled syringe product candidates, pending outcome of FDA feedback that is expected in the second quarter, and the manufacturing of initial lots of nafamostat. Quarterly combined R&D and SG&A expense is expected to be approximately $9-$10 million (and $8-$9 million excluding stock compensation and depreciation). Annual debt service is expected to approximate $10 million as the Company continues to pay down amounts outstanding under its senior debt facility that matures in June 2023. Annual capital expenditures are expected to approximate $2 million attributed mainly to the final validation of the automated packaging line at AcelRx’s contract manufacturer.

2022 financial guidance is based on the Company’s current expectations and are forward-looking statements. Actual results could differ materially depending on market conditions and the factors set forth under Forward-Looking Statements below.

Webcast and Conference Call Information
As previously announced, AcelRx will host a live webcast Thursday, March 10th at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss these financial results and provide other corporate updates. The webcast is accessible by visiting the Investors page of AcelRx’s website at View Source and clicking on the webcast link. The webcast will be accompanied by a slide presentation. Investors who wish to participate in the conference call may do so by dialing (866) 361-2335 for domestic callers, (855) 669-9657 for Canadian callers or (412) 902-4204 for international callers. A webcast replay will be available on the AcelRx website for 90 days following the call by visiting the Investor page of AcelRx’s website at View Source.

About DSUVIA (sufentanil sublingual tablet), 30 mcg
DSUVIA, to be marketed as DZUVEO in Europe, is indicated for use in adults in certified medically supervised healthcare settings, such as hospitals, surgical centers, and emergency departments, for the management of acute pain severe enough to require an opioid analgesic, and for which alternative treatments are inadequate. DSUVIA was designed to provide rapid analgesia via a non-invasive route of administration and to eliminate dosing errors associated with intravenous (IV) administration of opioid analgesics. DSUVIA is a single-strength solid dosage form administered sublingually via a single-dose applicator (SDA) by healthcare professionals. Sufentanil is an opioid analgesic previously only marketed for IV and epidural anesthesia and analgesia. The sufentanil pharmacokinetic profile when delivered sublingually avoids the high peak plasma levels and short duration of action observed with IV administration. The European Commission approved DZUVEO for marketing in Europe and it will be commercialized by AcelRx’s European partner, Aguettant.

For more information, including important safety information and black box warning for DSUVIA, please visit www.DSUVIA.com.

About nafamostat Nafamostat is a broad spectrum, synthetic serine protease inhibitor with anticoagulant, anti-inflammatory and potential anti-viral activities. Niyad is a lyophilized formulation of nafamostat and is currently being studied under an investigational device exemption, or IDE, as an anticoagulant for the extracorporeal circuit. Niyad has received Breakthrough Device Designation Status from the FDA. LTX-608 is a proprietary nafamostat formulation for direct IV infusion that we plan to potentially develop as a COVID anti-viral treatment, as well as for the treatment of disseminated intravascular coagulation (DIC), acute respiratory distress syndrome (ARDS), and acute pancreatitis.

Myeloid Therapeutics to Present at the Oppenheimer 32nd Annual Healthcare Conference

On March 10, 2022 Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage mRNA-immunotherapy company developing novel therapies for cancer and autoimmune diseases, reported that company management will present a corporate overview at Oppenheimer’s 32nd Annual Healthcare Conference and will participate in one-on-one meetings with investors (Press release, Myeloid Therapeutics, MAR 10, 2022, View Source [SID1234609936]). The conference is being held in a virtual format, March 15-17, 2022.