On November 10, 2021 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported financial results for the third quarter and nine months ended September 30, 2021, and provided a corporate update (Press release, Lipocine, NOV 10, 2021, View Source [SID1234595156]).

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Third Quarter and Recent Corporate Highlights

Entered into a license agreement with Antares Pharma to commercialize TLANDO in the US
Lipocine to receive up to $21.0 million in licensing fees, including $11.0 million payable immediately and $10.0 million to be paid in the future subject to certain conditions
Lipocine is entitled to commercial sales milestone payments of up to $160.0 million and tiered royalties on net sales of TLANDO from mid-teens up to 20%
Antares Pharma to undertake all commercialization, post-marketing study obligations, and sourcing of TLANDO in the U.S.
Antares Pharma was also granted an option to license TLANDO XR for development and commercialization in the U.S. for additional licensing fees ($4.0 million), clinical and regulatory milestone payments ($35.0 million), sales milestone payments and royalties (mid-teens up to 20%)
The U.S. Food and Drug Administration ("FDA") granted Fast Track Designation to LPCN 1144 for the treatment of non-cirrhotic non-alcoholic steatohepatitis ("NASH")
The FDA has affirmed that the resubmission of the New Drug Application ("NDA") for TLANDO will be a Class 1 resubmission, with a two-month FDA review goal period
The FDA previously granted tentative approval to TLANDO in adult males indicated for conditions associated with a deficiency or absence of endogenous testosterone: primary hypogonadism (congenital or acquired) and hypogonadotropic hypogonadism (congenital or acquired)
The product is not eligible for final approval and marketing in the U.S. until the expiration of the FDA’s Orange Book listed exclusivity period previously granted to Clarus Therapeutics, Inc. with respect to Jatenzo, which expires on March 27, 2022
Announced positive topline 36-week results from its Phase 2 proof-of-concept Liver Fat intervention with oral Testosterone ("LiFT") clinical study, NCT04134091, investigating LPCN 1144 in men with biopsy-confirmed NASH
Study met its primary endpoint. At 12 weeks, treatment with LPCN 1144 resulted in statistically significant liver fat reduction, assessed by MRI-PDFF
Both LPCN 1144 treatment arms showed significant improvement in NASH without worsening of fibrosis
Efficacy and safety results from the LiFT study have been accepted for late-breaking presentations at the American Association for the Study of Liver Diseases ("AASLD") The Liver Meeting on November 12-15, 2021
Company intends to meet with the FDA regarding the path forward for an accelerated approval and to discuss Phase 3 study requirements
Third Quarter Ended September 30, 2021 Financial Results

Lipocine reported a net loss of $3.1 million, or ($0.03) per diluted share, for the third quarter ended September 30, 2021, compared with a net loss of $4.3 million, or ($0.07) per diluted share, for the third quarter ended September 30, 2020.

Research and development expenses were $2.4 million for the third quarter ended September 30, 2021, compared with $2.5 million for the third quarter ended September 30, 2020. The decrease for the third quarter of 2021 was primarily due to a decrease in contract research organization expense and outside consulting costs related to our LPCN 1144 LiFT clinical study as well as decrease costs related to TLANDO. These decreases were offset by increases in costs associated with our LPCN 1154 and LPCN 1148 programs.

General and administrative expenses were $1.2 million for the third quarter ended September 30, 2021, compared with $1.9 million for the third quarter ended September 30, 2020. The decrease in general and administrative was primarily related to a decrease in our legal costs in 2021 as well as decreased personnel costs primarily related to reduced stock compensation expense.

As of September 30, 2021, the Company had $38.7 million of unrestricted cash, cash equivalents, and marketable investments, compared to $19.7 million of unrestricted cash, cash equivalents and marketable investment securities as of December 31, 2020.

Subsequent to the end of the third quarter, the Company received an $11.0 million upfront license fee as part of the licensing agreement with Antares Pharma to commercialize TLANDO.

Nine Months Ended September 30, 2021 Financial Results

Lipocine reported a net loss of $13.3 million, or ($0.15) per diluted share, for the nine months ended September 30, 2021, compared with a net loss of $16.5 million, or ($0.32) per diluted share, for the nine months ended September 30, 2020.

Research and development expenses were $5.4 million for the nine months ended September 30, 2021, compared with $7.3 million for the nine months ended September 30, 2020. The decrease in research and development expenses was primarily due to a decrease in contract research organization expense and outside consulting costs related to our LPCN 1144 LiFT clinical study, a decrease in costs related to TLANDO and a decrease in personnel costs primarily related to reduced stock compensation expense. These decreases were offset by increases in costs associated with our LPCN 1154 and LPCN 1148 programs.

General and administrative expenses were $4.3 million for the nine months ended September 30, 2021, compared with $5.9 million for the nine months ended September 30, 2020. The decrease in general and administrative expenses was primarily due to a decrease in our legal costs in 2021 as well as decreased personnel costs primarily related to reduced stock compensation expense. These decreases were offset by an increase in corporate insurance expense.

On November 10, 2021 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that Søren Bregenholt, Chief Executive Officer, will participate and host one-on-one investor meetings at the following upcoming investor conferences (Press release, Alligator Bioscience, NOV 10, 2021, View Source [SID1234595045]):

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Redeye Life Science Day 2021
Location: Hybrid Event
Date: Thursday, November 11, 2021
Type: Live Presentation

Time: 16:30 – 16:50 CET

Live presentations can be viewed via the following link: View Source

H.C. Wainwright 7th Annual Israel Conference
Location: Virtual

Date: Monday, November 15, 2021
Type: Live Presentation & 1×1’s

Time: 14:00 – 14:30 CET

Inv€$tival Showcase 2021
Location: London and Virtual

Dates: November 8 – 15, 2021
Type: In Person 1×1’s, Nov 15, 2021

Jefferies HealthCare Conference
Location: London and Virtual
Date: November 16, 2021

Type: In Person 1×1’s

ØU Life Science Life Science Investor Konference Summit
Location: Copenhagen
Date: Wednesday, November 24, 2021
Type: Live Presentation & 1×1’s

Time: 15:50 – 16:20 CET

Presentation can be viewed via the following link: View Source

All presentations will be available via a digital library, which is accessible to event participants only. Please contact the organizers to if you wish to attend or Julie Silber if you wish to schedule a meeting with Alligator.

On November 10, 2021 Ayala Pharmaceuticals, Inc. (NASDAQ: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, reported that Ayala management will participate in a fireside chat at the Jefferies London Healthcare Conference on Wednesday, November 17, 2021 at 5:00pm GMT (12:00pm ET) (Press release, Ayala Pharmaceuticals, NOV 10, 2021, View Source [SID1234595060]).

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A live webcast of the fireside chat may be accessed by visiting the Events & Presentations section of Ayala’s website at ir.ayalapharma.com. An archived replay of the webcast will be available on the website for approximately 90 days following the presentations.

On November 10, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Regulus, NOV 10, 2021, View Source [SID1234595092]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"We are pleased with the progress we have made in advancing our next generation candidate RGLS8429 as a potential treatment for ADPKD, as the compound has been shown to have the favorable properties of RGLS4326 without the limitations of the first-generation compound," commented Jay Hagan, CEO of Regulus Therapeutics. "RGLS8429 has demonstrated comparable potency, as well as similar pharmacokinetic and pharmacodynamic profiles, without the off-target CNS effects seen in chronic preclinical toxicology studies with RGLS4326 at the top doses tested. We look forward to our pre-IND meeting in December with the FDA to help finalize our IND submission and reach another important milestone in our mission to improve the lives of ADPKD patients."

Program Updates

RGLS8429 for ADPKD: In October 2021, the Company discontinued development of its first-generation compound, RGLS4326, to allocate resources and efforts towards the development of its more promising next-generation compound, RGLS8429. The Company believes RGLS8429 has demonstrated a superior pharmacological profile, with the absence of the off-target central nervous system (CNS) effects that were seen with RGLS4326 at the top doses tested in chronic preclinical toxicology studies. RGLS8429 has also shown equal potency to RGLS4326 for its molecular target (miR-17) in both in-vitro and in-vivo efficacy studies. The Company expects to have a pre-IND meeting with the U.S. Food and Drug Administration (FDA) for RGLS8429 before year-end and is on track for an IND submission and initiation of clinical development in the second quarter of 2022, subject to FDA clearance of the IND.

The Company’s Phase 1 plans include a single dose escalation study in healthy volunteers to enable a multi-dose escalation study in ADPKD patients around the dose levels where robust clinical biomarker effects were demonstrated with RGLS4326. The Company anticipates reporting top-line biomarker data in the first cohort of RGLS8429-treated patients in early 2023.

RGLS4326 for ADPKD: On November 4 and November 9, data from the first cohort of patients in the Phase 1b clinical trial of RGLS4326, the Company’s first-generation compound, for the treatment of ADPKD, was presented at the American Society of Nephrology (ASN) Kidney Week, and at the Biomarkers for Rare Diseases Summit. In the first cohort, nine patients were enrolled and received 1 mg/kg of RGLS4326 subcutaneously every other week for four doses. The mean increase in polycystins 1 and 2 at the end of study compared to baseline levels for all nine patients in the first cohort were 58% (p=.0004) and 38% (p=.026), respectively. These data demonstrate clinical evidence that treatment with RGLS4326 increased PC1 and PC2, most likely through inhibition of miR-17 in the kidney of patients with ADPKD. These results also imply that overexpressed miR-17 in ADPKD patients represses Pkd1 and Pkd2 expression, further validating miR-17 as a therapeutic target for ADPKD treatment. The details for each presentation are below:

ASN Kidney Week ePoster:
Poster Title: RGLS4326 Increases Urinary PC1 and PC2 Levels in Individuals with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Poster Date and Time: Thursday, November 4, 2021, 10:00 AM PDT
Poster Number: PO1244

Biomarkers for Rare Diseases Summit Presentation:
Presentation Title: Results from the First Cohort of Phase1b Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Presenter: Edmund Lee, PhD, Executive Director, Biology, Regulus Therapeutics
Presentation Date and Time: Tuesday, November 9, 2021, 9:30 AM PDT

A copy of each presentation is available at www.regulusrx.com/publications/

Financial Results

Cash Position: As of September 30, 2021, Regulus had $35.8 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $5.9 million and $13.4 million for the three and nine months ended September 30, 2021, respectively, compared to $4.0 million and $11.4 million for the same periods in 2020, respectively. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.5 million and $7.5 million for the three and nine months ended September 30, 2021, respectively, compared to $2.1 million and $6.7 million for the same periods in 2020, respectively. These amounts reflect personnel-related and ongoing general business operating costs.

Net Loss: Net loss was $8.6 million, or $0.10 per share (basic and diluted), and $20.7 million, or $0.26 per share (basic and diluted), for the three and nine months ended September 30, 2021, compared to $1.5 million, or $0.04 per share (basic and diluted), and $14.4 million, or $0.47 per share (basic and diluted), for the same periods in 2020.

Conference Call and Webcast Information:
The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Daylight Time to discuss its third quarter 2021 financial results and corporate update. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 2108429. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 2108429. The webcast and telephone replay will be archived on the Company’s website at www.regulusrx.com following the call.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS8429

RGLS8429 is a novel, second generation oligonucleotide designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity, as well as a superior pharmacologic profile have been demonstrated. Regulus has nominated RGLS8429 as a development candidate for the treatment of ADPKD.

On November 10, 2021 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported financial results for the third quarter ended September 30, 2021 (Press release, TapImmune, NOV 10, 2021, View Source [SID1234595108]).

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"This quarter, we continued our momentum in advancing Marker’s Phase 2 trial of MT-401, Marker’s lead MultiTAA-specific T cell therapy, for the treatment of post-transplant acute myeloid leukemia, or AML," said Peter L. Hoang, President & CEO of Marker Therapeutics. "We are pleased to announce that the first patients in Marker’s Phase 2 AML trial have been dosed with study drug. Further, we are on track to enroll the first 20 patients of the trial in the fourth quarter, with the first data readout expected in the first quarter of 2022. We look forward to providing year-end updates in a conference call and webcast early next year."

PROGRAM UPDATES

The Company continues to enroll patients and activate clinical sites across the U.S. in Marker’s Phase 2 trial of MT-401, its lead MultiTAA-specific T cell product candidate, for the treatment of post-transplant AML. The trial is expected to enroll approximately 120 patients in the adjuvant setting and 40 patients with active disease at approximately 20 clinical sites.
BUSINESS UPDATES

The Company’s new cGMP manufacturing facility in Houston, Texas is fully operational and is supporting ongoing operations. The facility will also manufacture Marker’s MultiTAA-specific T cell products for future hematological and solid tumor trials, in addition to producing the potential commercial supply of any products, if approved.
In August, the Company announced that it received notice of a Product Development Research award totaling approximately $13.1 million from the Cancer Prevention and Research Institute of Texas (CPRIT) to support the Company’s Phase 2 AML trial.
ANTICIPATED PROGRAM MILESTONES

AML Trial Milestones

Enrollment of first 20 patients of the Phase 2 AML trial expected in Q4 2021
Topline readout of Group 2 active disease anticipated in Q1 2022
THIRD QUARTER 2021 FINANCIAL RESULTS

Cash Position and Guidance: At September 30, 2021, Marker had cash and cash equivalents of $48.7 million. The Company believes that its existing cash and cash equivalents will fund its operating expenses and capital expenditure requirements into the first quarter of 2023.
R&D Expenses: Research and development expenses were $6.8 million for the quarter ended September 30, 2021 compared to $4.8 million for the quarter ended September 30, 2020. The increase was primarily attributable to increases in clinical trial and sponsored research expenses and headcount-related expenses due to growth of research and development operations.
G&A Expenses: General and administrative expenses were $3.2 million for the quarter ended September 30, 2021 compared to $2.6 million for the quarter ended September 30, 2020.
Net Loss: Marker reported a net loss of $12.4 million for the quarter ended September 30, 2021, compared to a net loss of $7.4 million for the quarter ended September 30, 2020.