On November 14, 2022 Aptose Biosciences Inc. ("Aptose") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated therapeutics targeting the underlying mechanisms of cancer, reported dosing of the first patient to receive a continuous dosing regimen of the G3 formulation of luxeptinib, a potent, non-covalent oral inhibitor of BTK and FLT3, in the ongoing Phase 1a/b clinical trial in patients with relapsed or refractory (r/r) acute myeloid leukemia (AML) (Press release, Aptose Biosciences, NOV 14, 2022, View Source [SID1234623971]).

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The new G3 formulation thus far has been tested as a single dose in 20 patients from an ongoing Phase 1 clinical program of luxeptinib. Modeling of the pharmacokinetic (PK) properties of G3 predicts steady-state plasma exposure from continuous dosing with 50 mg of G3 (every 12 hours, Q12h) should be comparable to that of 900 mg of the original G1 formulation Q12h, representing a significant improvement in bioavailability with G3. Patients now will receive continuous dosing at the 50mg G3 Q12h dose, with the protocol allowing for further dose escalation of G3 in subsequent patients.

"We’re pleased to incorporate the new G3 formulation of luxeptinib into our clinical trial," said William G. Rice, Ph.D., Chairman, President, and Chief Executive Officer. "Preclinically, Lux is an extraordinary molecule, eradicating tumors with the absence of toxicity, and clinically, one patient administered the original G1 formulation in the AML trial achieved exposures that enabled a complete remission (CR). We are hopeful the G3 formulation will result in greater exposures of luxeptinib and additional responses in this difficult-to-treat patient population."

About Luxeptinib (formerly CG-806)
Luxeptinib is an oral, first-in-class FLT3 and BTK kinase inhibitor in Phase 1 a/b clinical studies for the treatment of myeloid hematologic malignancies. This small molecule demonstrates potent inhibition of wild type and all mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region) and cures animals of AML in the absence of toxicity in murine leukemia models. Likewise, luxeptinib demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser (C481S) mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting luxeptinib may be developed for various B cell malignancy patients that are resistant/refractory/intolerant to covalent or other non-covalent BTK inhibitors. Luxeptinib also inhibits NLRP3 inflammasome function in THP-1 monocytes and bone marrow-derived macrophages, suggesting potential indications in inflammatory conditions.

On November 14, 2022 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, reported financial results for the third quarter ended September 30, 2022 (Press release, Alpine Immune Sciences, NOV 14, 2022, View Source [SID1234623970]).

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"During our inaugural R&D Day and throughout subsequent scientific meetings this fall, we have shared promising nonclinical and clinical data that supports the best-in-class potential for our lead program ALPN-303 in multiple autoimmune and inflammatory indications. In particular, recent data from healthy volunteers presented at ASN’s Kidney Week demonstrated dose-dependent reductions in Gd-IgA1, a key effector molecule and clinical biomarker of disease progress in IgAN, and the first clinical disease-related biomarker data with ALPN-303," said Mitchell H. Gold, MD, Executive Chairman and Chief Executive Officer of Alpine. "To further accelerate development of this promising program in multiple indications, we completed a successful $113 million follow-on offering with top-tier investors in October, extending our cash runway through the end of 2025. We now look forward to beginning a broad development plan for ALPN-303, including a phase 2 study in systemic lupus erythematosus (SLE). In addition, we are particularly excited to begin open-label basket studies in glomerulonephritis and autoimmune cytopenias as they should provide a rapid assessment in multiple diseases and may potentially enable multiple accelerated development paths."

Gold continued, "As previously announced, we have voluntarily terminated enrollment in the davoceticept (ALPN-202) clinical studies. We would like to thank the patients and investigators who participated in the NEON studies. We remain focused on using our resources to further advance ALPN-303, as well as acazicolcept (ALPN-101) in SLE in collaboration with AbbVie."

Third Quarter 2022 and Recent Pipeline and Corporate Updates

ALPN-303

During the September R&D Day, the Company shared updated preliminary data from the phase 1 study (RUBY-1) of ALPN-303 in healthy volunteers and presented a broad development plan, including a proof-of-concept phase 2 study in SLE and basket studies in renal and hematologic autoimmune diseases, with initial clinical data from the basket studies expected in late 2023. (View Release)
At the American Society of Nephrology: Kidney Week Meeting, updated clinical data were presented in a poster titled, "Phase 1 Study in Healthy Adults of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALPN-303, a Dual BAFF/APRIL Antagonist for the Treatment of Autoimmune Glomerulonephritides (GN)". (View Release)
The data demonstrate that ALPN-303 continues to be well tolerated as single intravenous or subcutaneous doses of up to 960 mg and exhibits dose-related pharmacokinetic and on-target pharmacodynamic effects.
ALPN-303 maintains target coverage of free APRIL for 2-3 and ≥4 weeks with 80 and 240 mg, respectively, corresponding to reductions in serum Ig and antibody-secreting cells (CD38hi plasmablasts/plasma cells).
ALPN-303 dose-dependently reduces serum galactose-deficient IgA1 (Gd-IgA1), a critical molecule implicated in the pathogenesis of IgA nephropathy (IgAN).
These data support dose regimens of 80-240 mg SC every 4 weeks in future GN studies.
The Company also presented clinical data from the RUBY-1 study of healthy vs at the recent American College of Rheumatology in a poster titled, "A Randomized Placebo-Controlled Phase 1 Study in Healthy Adult Volunteers of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALPN-303, a Potent Dual BAFF/APRIL Antagonist for the Treatment of Systemic Lupus Erythematosus and Other Autoantibody-Associated Diseases". (View Poster)
Additional updates will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Conference in December. (View Release)
Corporate

The Company ended the third quarter with $277.1 million in cash, cash equivalents, restricted cash, and investments, following the successful completion of a $100.0 million underwritten public offering where we sold 13.6 million shares of our common stock with net proceeds of approximately $93.5 million, after deducting underwriting commissions and estimated offering expenses.
An additional 1.9 million shares of our common stock were sold pursuant to the underwriters’ partial exercise of their over-allotment option, with net proceeds of $13.1 million received upon closing on October 4, 2022.
The financing brings our pro-forma cash and investments balance to $290.2 million as of September 30, 2022, which should be sufficient to fund our planned operations through 2025.
On October 24, the Company announced that it had voluntarily terminated enrollment in the NEON studies of davoceticept as a monotherapy and in combination with pembrolizumab. (View Release)
Third Quarter 2022 Financial Results

As of September 30, 2022, Alpine’s cash, cash equivalents, restricted cash, and investments totaled $277.1 million. The Company recorded net losses of $13.3 million and $13.5 million for the quarters ended September 30, 2022, and 2021, respectively.

Collaboration revenue for the third quarter ended September 30, 2022, was $8.4 million compared to $8.5 million for the third quarter ended September 30, 2021. The 2022 amounts were primarily attributable to revenue recognized under the Company’s AbbVie and Horizon collaborations, while 2021 revenue recognized solely related to the AbbVie collaboration.

Research and development expenses for the third quarter ended September 30, 2022, were $17.6 million compared to $18.3 million for the third quarter ended September 30, 2021. The decrease was primarily attributable to decreased clinical development activities offset by higher personnel-related expenses due to increased headcount.

General and administrative expenses for the third quarter ended September 30, 2022, were $4.6 million compared to $3.5 million for the third quarter ended September 30, 2021. The increase was primarily attributable to increases in personnel costs.

About ALPN-303 and the RUBY-1 Study

ALPN-303 is a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in the activation and survival of B cells. Based upon an engineered TACI (transmembrane activator and CAML interactor) domain, ALPN-303 exhibits greater potency in preclinical studies versus wild-type TACI-based comparators, as well as other inhibitors of BAFF and/or APRIL alone. ALPN-303 is in development for multiple B cell and/or autoantibody-related diseases, such as systemic lupus erythematosus, glomerulonephritides, and autoimmune cytopenias.

RUBY-1 (NCT05034484) is a phase 1, randomized, placebo-controlled study in healthy adult volunteers designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single doses of intravenously and subcutaneously administered ALPN-303. Initial data show ALPN-303 to be well tolerated up to 960 mg with dose-dependent pharmacokinetics and reductions in circulating immunoglobulins and antibody-secreting cells, supporting the use of a once every four-week dose regimen for subsequent studies.

On November 14, 2022 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP), a commercial-stage biopharmaceutical company primarily focused on developing and commercializing products in various therapeutic areas, including allergy, opioid overdose, respiratory and inflammatory disease, reported financial results for the third quarter of 2022 and provided an update on recent corporate developments (Press release, Adamis Pharmaceuticals, NOV 14, 2022, View Source [SID1234623969]).

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Process Update

In October, the Company announced that it had initiated a process to explore a range of strategic and financing alternatives and had retained an investment bank to assist in evaluating certain alternatives focused on maximizing stockholder value. Potential alternatives include a sale, partnership, distribution or other agreement regarding one or both of the Company’s commercial products, a sale, merger, or reverse merger of the company, and/or seeking additional financing. As of today, the Company is engaged in communications with third parties regarding one or more possible transactions.

"The strategic process is well under way with the goal of maximizing shareholder value and preserving cash resources," stated David J. Marguglio, CEO of Adamis. "Concurrently, we have implemented significant expense reduction measures including employee headcount reductions and pausing all product development programs. While engaged in this process, we continue to work closely with our commercial partner to continue the momentum of the ZIMHI launch and the return of SYMJEPI to the market."

There can be no assurance regarding the schedule for completion of the strategic review process, that this strategic review process will result in the Company pursuing any transaction or that any transaction, if pursued, will be completed.

Business Updates in Q3 2022

ZIMHI (naloxone) Injection

●Following its introduction to the market at the end of March, our commercial partner continues to make steady progress on the ZIMHI commercial launch plan.

●Unit sales increased 90% in the third quarter compared to the second quarter of 2022.

SYMJEPI (epinephrine) Injection

●In March 2022, the Company announced that manufacturing issues at one of its contract manufacturers, Catalent Belgium, led to a voluntary recall of four lots of SYMJEPI. Following corrective and preventative actions, Catalent resumed operations at its Belgium facility in November 2022.

●Assuming no additional interruptions or delays, the Company anticipates having SYMJEPI relaunched and commercially available before the end of the first quarter of 2023.

TEMPOL

●The Company announced that following a review of interim data, it’s Phase 2/3 clinical trial examining the effects of Tempol in COVID-19 treatment failed to achieve its primary endpoint and further development of Tempol has been halted.

Discontinued Operations

●The Company is continuing to liquidate the assets of the former US Compounding business and, although there are no assurances, the Company expects to receive additional proceeds which could range from approximately $3 to $4 million between now and the end of the first quarter of 2023.

Q3 2022 Financial Highlights

●Total net revenue for the third quarter of 2022 was approximately $1.5 million compared to approximately $760,000 in the third quarter of 2021, an increase of 98%. The increase in revenues was primarily due to sales of ZIMHI, the absence of sales for SYMJEPI resulting from the March 2022 product recall and the recognition of deferred revenue under our commercial distribution agreement, offset by cost of the SYMJEPI recall.

●Operating expense (selling, general and administrative expenses and research and development expenses) for the third quarter of 2022 was approximately $4.5 million compared to $9.4 million in the third quarter of 2021, a decrease of 52%. The decrease was primarily due to lower development spending for Tempol and ZIMHI, decreases in legal and advisory fees, and a reversal of 2022 accrued bonus expenses.

●Net loss for the combined (continued and discontinued) operations for the third quarter of 2022 was approximately $4.4 million compared to a net loss of $12.4 million in the third quarter of 2021, a 64% decrease.

●Cash and cash equivalents as of September 30, 2022 were approximately $2.4 million.

Conference Call Information

Management will host a live webcast/conference call today, November 14, 2022, at 2:00 p.m. PT / 5:00 p.m. ET, during which Company executives will review financial information for the third quarter of 2022 and provide a corporate update.

A live audio webcast of the conference call will also be available via this link. If you are unable to participate in the live call, a replay will be available shortly after the live event. To listen to the replay please visit the events page of the Adamis investor relations section of the company website at View Source

On November 14, 2022 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results for the third quarter of 2022 (Press release, Abeona Therapeutics, NOV 14, 2022, View Source [SID1234623968]).

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"The positive topline data from the Phase 3 VIITAL study provides strong support for EB-101’s potential and validation of the Abeona team’s extensive efforts," said Vish Seshadri, Chief Executive Officer of Abeona. "This is an exciting time for Abeona as we are sharply focused on submitting a Biologics License Application for EB-101 to the U.S. FDA. With the additional capital raised after quarter-end, we are now well-funded into the third quarter of 2024, beyond the anticipated timing for potential BLA approval."

Third Quarter and Recent Operating Highlights

EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB)

●On November 3, 2022, Abeona announced positive topline data from the pivotal Phase 3 VIITAL study of investigational EB-101 in RDEB. The VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. The Company intends to present more detailed results from this study at future medical meetings and in a peer-reviewed journal.
●Based on the positive VIITAL topline results, the Company plans to submit a Biologics License Application (BLA) for EB-101 to the U.S. Food and Drug Administration (FDA) in the second quarter of 2023. If the BLA is approved, Abeona may be eligible for a Priority Review Voucher (PRV), which can be used to receive expedited review by the FDA of a subsequent marketing application for a different product or sold to another company.
●Long-term follow up data up to eight years and quality of life data from a completed Phase 1/2a study of EB-101 in RDEB were published in Orphanet Journal of Rare Diseases. The data showed that large chronic RDEB wounds treated with EB-101 had sustained wound healing with mean 5.9 years of follow-up, and long-term symptomatic relief, including reduction in pain and itch.

Preclinical programs

●Abeona’s preclinicalors, and started dosing mice in proof-of-concept studies to support possible pre-Investigational New Drug Application (IND) meetings with the FDA in early 2023.

Corporate highlights

●On November 3, 2022, the Company announced a private placement financing with gross proceeds of $35.0 million. The private placement included participation from new and existing institutional investors.

Third Quarter Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $23.5 million as of September 30, 2022. Net cash used in operating activities was $6.8 million for the third quarter of 2022, compared to $9.0 million in the second quarter of 2022. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments plus the net proceeds from the private placement financing on November 3, 2022 are sufficient resources to fund operations into the third quarter of 2024.

Research and development (R&D) expenses for the three months ended September 30, 2022 were $5.5 million, compared to $9.1 million for the same period of 2021. General and administrative (G&A) expenses were $3.9 million for the three months ended September 30, 2022, compared to $5.8 million for the same period of 2021.

Net loss attributable to common shareholders for the third quarter of 2022 was $9.5 million, or $1.48 loss per common share as compared to $7.0 million, or $1.80 loss per common share, in the third quarter of 2021.

On November 14, 2022 Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results and provided a business update for the three months ended September 30, 2022 (Press release, Galectin Therapeutics, NOV 14, 2022, View Source [SID1234623933]). These results are included in the Company’s Quarterly Report on Form 10-Q, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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Joel Lewis, Chief Executive Officer and President, stated: "The Company made outstanding progress this quarter. In addition to securing the largest financing in the Company’s history, which extended our cash runway for planned trial expenditures through 2024, we accelerated recruitment on NAVIGATE, our global pivotal NASH cirrhosis trial, and continue to progress towards our goal of full enrollment around the end of 2022. We presented at multiple conferences culminating in our submission of five scientific presentations that were accepted and presented at the American Association for the Study of Liver Diseases (AASLD) in the first week of November. During AASLD, we had the opportunity to host some of our investigators and their staff. I want to thank all of those who attended for their dedication to our program and for the insight they shared with us. I truly believe the knowledge you shared will enable us to reach our enrollment goals, as well as our overall goals for our study.

"Additionally, as recently announced, our team successfully completed an Investigational New Drug (IND) application and received a Study May Proceed letter from FDA, for belapectin in combination with a Keytruda for the treatment of Head and Neck cancers."

Dr. Pol Boudes, Chief Medical Officer, stated: "I, along with several other team members, have visited multiple sites and investigators over the past few months, in addition to meeting with several more at recent industry conferences. We continue to receive consistent and supportive feedback from investigators regarding the importance and uniqueness of NAVIGATE and the potential to bring a therapy to patients with cirrhosis and portal hypertension for this large unmet medical need. We have now randomized 279 patients of the planned 315 patients with an additional 74 patients currently in screening."

Financial Results

For the three months ended September 30, 2022, the Company reported a net loss applicable to common stockholders of $8.6 million, or ($0.14) per share, compared to a net loss applicable to common stockholders of $8.6 million, or ($0.14) per share for the three months ended September 30, 2021.

Research and development expenses for the three months ended September 30, 2022, were $6.6 million compared with $6.6 million for the three months ended September 30, 2021. These are primarily due to costs related to the NAVIGATE clinical trial and other supportive activities. General and administrative expenses for the three months ended September 30, 2022 were $1.5 million, compared to $1.6 million for the three months ended September 30, 2021. The decrease was primarily due to a decrease in legal expenses.

As of September 30, 2022, the Company had $15.8 million of cash and cash equivalents. The Company believes it has sufficient cash, including availability under its $60 million line of credit, to fund currently planned operations and research and development activities through at least December 31, 2024.

About Belapectin

Belapectin is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of NASH and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs, including fibrotic disorders of the liver, lung, kidney, heart and vascular system. Belapectin binds to galectin-3 and disrupts its function. Preclinical data in animals have shown that belapectin has robust treatment effects in reversing liver fibrosis and cirrhosis. A Phase 2 study showed belapectin may prevent the development of esophageal varices in NASH cirrhosis, and these results provide the basis for the conduct of the NAVIGATE trial. The NAVIGATE trial (www.NAVIGATEnash.com), titled "A Seamless Adaptive Phase 2b/3, Double-Blind, Randomized, Placebo-controlled Multicenter, International Study Evaluating the Efficacy and Safety of Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in NASH Cirrhosis," began enrolling patients in June 2020, and is posted on www.clinicaltrials.gov (NCT04365868). Galectin-3 has a significant role in cancer, and the Company has supported a Phase 1b study in combined immunotherapy of belapectin and KEYTRUDA in advanced melanoma and in head and neck cancer. This trial provided a strong rationale for moving forward into a Company-sponsored Phase 2 development program, which the company is exploring.

About Fatty Liver Disease with Advanced Fibrosis and Cirrhosis

Non-alcoholic steatohepatitis (NASH) has become a common disease of the liver with the rise in obesity and other metabolic diseases. NASH is estimated to affect up to 28 million people in the U.S. It is characterized by the presence of excess fat in the liver along with inflammation and hepatocyte damage (ballooning) in people who consume little or no alcohol. Over time, patients with NASH can develop excessive fibrosis, or scarring of the liver, and ultimately liver cirrhosis. It is estimated that as many as 1 to 2 million individuals in the U.S. will develop cirrhosis as a result of NASH, for which liver transplantation is the only curative treatment available. Approximately 9,000 liver transplants are performed annually in the U.S. There are no drug therapies approved for the treatment of liver fibrosis or cirrhosis.