Regen BioPharma, Inc. to Present at the Emerging Growth Conference on August 10, 2023

On August 8, 2023 Regen BioPharma, Inc. (OTC PINK: RGBP) (OTC PINK: RGBPP), a biotechnology company advancing a diverse pre-clinical pipeline spanning cell therapies, RNA vaccines, RNA and DNA therapeutics and small molecule drugs reported that it will be presenting at the Emerging Growth Conference on August 10, 2023 (View Source) (Press release, Regen BioPharma, AUG 8, 2023, View Source [SID1234634014]).

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This live, interactive online event will give existing shareholders and the investment community the opportunity to interact with the Company’s CEO, Dr. David Koos, in real time. Please ask your questions during the event and Dr. Koos and his team will do their best to get through as many of them as possible.

"We plan to use this time to update our stakeholders on our future financing and programmatic plans, current state of our research program and to answer shareholder questions," says Dr. David Koos, Chairman and CEO of the Company.

Regen BioPharma, Inc. will be presenting at 11:25 – 11:55 Eastern time on Thursday August 10, 2023. Please register here to ensure you are able to attend the conference and receive any updates that are released View Source;tp_key=7656c5070a&sti=rgbp

If attendees are not able to join the event live on the day of the conference, an archived webcast will also be made available on EmergingGrowth.com.

Poseida Therapeutics Provides Financial Results for the Second Quarter of 2023

On August 8, 2023 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported financial results for the second quarter ended June 30, 2023 (Press release, Poseida Therapeutics, AUG 8, 2023, View Source [SID1234634013]).

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"In the second quarter, we continued to make strong progress while sharpening our focus on our clinical pipeline and research efforts," said Mark Gergen, Chief Executive Officer of the Company. "As announced yesterday, we received a strategic investment from Astellas, which is comprised of the purchase of 8,333,333 shares of common stock at $3.00 per share for an aggregate purchase price of $25 million and an additional $25 million one-time payment for certain strategic rights. This support from yet another premier biopharma strengthens our financial position and further validates our proprietary technologies and cell therapy approach. As we advance our clinical-stage allogeneic CAR-T portfolio, we continue to make improvements based upon learnings across our programs and look forward to providing data updates at a medical meeting later this year. In our gene therapy portfolio, we continued to validate our science, as highlighted by multiple presentations at the American Society of Gene & Cell Therapy annual meeting in May. In addition, we are excited by the return of our promising Hemophilia A and PKU gene therapy programs from Takeda. We are in the process of evaluating which gene therapy programs we may advance on our own or seek to re-partner, and we look forward to providing an update in due course."

Financial Results for the Second Quarter 2023

Revenues
Revenues were $20.0 million for the second quarter ended June 30, 2023, and $30.4 million for the six months ended June 30, 2023 compared to $2.7 million and $4.1 million for the same periods in 2022. The increase was due to revenues earned from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022 and $8.9 million of previously deferred revenue recognized as a result of the previously announced termination of our collaboration agreement with Takeda in the second quarter of 2023.

Research and Development Expenses
Research and development expenses were $39.2 million for the three months ended June 30, 2023, compared to $35.0 million for the same period in 2022. The increase was primarily due to an increase in personnel expenses as a result of increased headcount, an increase in preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity, and an increase in facilities expense, offset by a decrease in clinical stage programs, primarily driven by the wind-down of clinical development activities associated with autologous programs and related contract termination expense in the prior year and the transition of manufacturing to the Company’s internal pilot plant for P-BCMA-ALLO1.

For the six months ended June 30, 2023, research and development expenses were $77.2 million, compared to $83.9 million for the same period in 2022. The decrease was primarily due to a decrease in external costs related to clinical stage programs primarily driven by the wind-down of clinical development activities associated with autologous programs and related contract termination expense in the prior year and the transition of manufacturing to the Company’s internal pilot plant for P-BCMA-ALLO1, partially offset by an increase in external costs related to preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity, an increase in personnel expenses as a result of increased headcount and an increase in facilities expense related to an additional lease entered into in March 2022 to support headcount growth.

General and Administrative Expenses
General and administrative expenses for the three months ended June 30, 2023 were $8.7 million compared to $9.2 million for the same period in 2022. The decrease was primarily due to lower headcount and facility costs.

For the six months ended June 30, 2023 and 2022, general and administrative expenses were $20.5 million and $18.8 million, respectively. The increase was primarily due to an accelerated stock-based compensation expense in the first quarter of 2023 related to a one-time modification associated with the retirement of the Company’s former Executive Chairman.

Net Loss
Net loss was $27.5 million and $66.3 million for the three and six months ended June 30, 2023, respectively, compared to net loss of $43.0 million and $101.1 million for the three and six months ended June 30, 2022, respectively.

Cash Position
As of June 30, 2023, the Company’s cash, cash equivalents and short-term investments balance was $214.6 million. The Company expects that its cash, cash equivalents and short-term investments together with the remaining near-term milestones and other payments from Roche as well as the proceeds from the Astellas strategic investment will be sufficient to fund operations into early 2025.

STORM Therapeutics publishes data in Cancer Discovery showing induction of anti-tumour immunity by METTL3 inhibition

On August 8, 2023 STORM Therapeutics Ltd. (STORM), a clinical stage biotechnology company focused on discovering and developing novel small molecule therapies targeting RNA modifying enzymes (RMEs) for oncology and other diseases, reported that it has published a scientific manuscript in the internationally recognised scientific journal Cancer Discovery (Press release, STORM Therapeutics, AUG 8, 2023, View Source [SID1234634012]).

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The paper, entitled ‘Inhibition of METTL3 results in a cell-intrinsic interferon response that enhances anti-tumour immunity’, has been published in collaboration with Professor Mark Dawson’s laboratory at the Peter MacCallum Cancer Centre in Melbourne, Australia, and reveals recent findings that demonstrate the therapeutic potential of drugs targeting METTL3 as immuno-oncology agents. Using pre-clinical cancer models, the study shows that pharmacological inhibition of METTL3 induces double-stranded RNA formation and a profound cell-intrinsic interferon response that potentiates T-cell killing of cancer cells. Whilst METTL3 inhibitors are equally efficacious to anti-PD1 therapy in pre-clinical models, the combination of the two agents has far greater activity, leading to tumour regression and durable anti-cancer immunity. A detailed investigation of the mechanism of action of the two treatments revealed that they act orthogonally and provides a strong rationale for their combination.

Oliver Rausch, Chief Scientific Officer of STORM Therapeutics and joint senior author of the study, said, "We are pleased to share this new data with the scientific community. Our study identifies a novel therapeutic mechanism triggered by METTL3 inhibition that opens a significant opportunity for METTL3 inhibitors in immuno-oncology. This work follows on from our previous work published in 2021 in Nature, demonstrating the efficacy of METTL3 inhibition in acute myeloid leukemia models, and illustrates the broad potential of this new class of drugs in oncology."

Dr. med. Josefin-Beate Holz, Chief Medical Officer of STORM Therapeutics, commented, "This exciting new data strengthens our hypothesis for the clinical development of STC-15 as a novel anticancer therapeutic in monotherapy and in combination with standard of care, including checkpoint inhibitors. We are looking forward to exploring these concepts following the conclusion of the current Phase 1 multiple ascending dose study with STC-15 in patients with relapsed solid tumours."

Jerry McMahon, Chief Executive Officer and President of STORM Therapeutics, added, "STORM is the first company devoted to pursuing RNA-modifying enzymes as drug targets, and our new study highlights the opportunities for new therapeutics targeting RNA modifications. We are encouraged by the progress of our ongoing Phase 1 trial of STC-15 in solid tumours and look forward to exploring its utility as an immuno-oncology drug in specific tumour types."

Citation:
Inhibition of METTL3 results in a cell-intrinsic interferon response that enhances anti-tumour immunity. Andrew A Guirguis, Yaara Ofir-Rosenfeld et al, 2023, Cancer Discovery (advanced online publication) View Source

ESSA Pharma Provides Corporate Update and Reports Financial Results for Fiscal Third Quarter Ended June 30, 2023

On August 8, 2023 ESSA Pharma Inc. ("ESSA", or the "Company") (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported a corporate update and reported financial results for the fiscal third quarter ended June 30, 2023 (Press release, ESSA, AUG 8, 2023, View Source [SID1234634011]). All references to "$" in this release refer to United States dollars, unless otherwise indicated.

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"Over the past months we have ramped up preparations to initiate the randomized Phase 2 combination study of EPI-7386 and Astellas and Pfizer’s antiandrogen Xtandi (enzalutamide), and we expect to complete the Phase 1 part of the study in the coming quarter," stated David Parkinson, MD, President and CEO of ESSA. "In the past quarter, we finalized our clinical collaboration with Janssen to evaluate EPI-7386 in combination with Janssen’s antiandrogens Erleada (apalutamide) and Zytiga (abiraterone acetate) in two Phase 1 cohorts, building on initial Phase 1 clinical data demonstrating promising prostate-specific antigen ("PSA") declines following combination treatment. ESSA is in a strong cash position as we advance our EPI-7386 studies, with our cash runway expected to fund operations and programs through 2025."

Clinical and Corporate Highlights for the Third Quarter Ended June 30, 2023
EPI-7386 Clinical Collaborations

The Company is preparing to initiate the open-label, randomized Phase 2 study of EPI-7386 and Astellas and Pfizer’s antiandrogen Xtandi (enzalutamide) in patients with metastatic castration-resistant prostate cancer ("mCRPC") naïve to second-generation antiandrogens. The Phase 2 study will assess the anti-tumor activity of EPI-7386 in combination with enzalutamide at the recommended Phase 2 doses versus single agent enzalutamide at the standard-of-care dose. The study is expected to enroll approximately 120 patients. The Company expects to complete the Phase 1 part of the study and establish the recommended Phase 2 combination doses (for both EPI-7386 and enzalutamide when used in combination) in the third calendar quarter of 2023, followed by initiation of the Phase 2 part of the study.

In April 2023, the Company entered into a clinical trial support agreement with Janssen Research & Development, LLC ("Janssen") under which Janssen will supply apalutamide and abiraterone acetate for a Phase 1 clinical study sponsored and conducted by ESSA evaluating EPI-7386 combination therapies in two cohorts. The two cohorts will be evaluated as additional cohorts in the Company’s ongoing Phase 1 study of EPI-7386 (Clinical Trials Identifier: NCT04421222). Cohort 1 will assess EPI-7386 in combination with abiraterone acetate plus prednisone in patients with mCRPC and high-risk metastatic castration-sensitive prostate cancer. Cohort 2 is a Window of Opportunity study in which patients with non-metastatic castration-resistant prostate cancer ("nmCRPC") will receive up to 12 weeks of single agent EPI-7386 before adding standard-of-care apalutamide. ESSA will retain all rights to EPI-7386. The Company expects enrollment to begin in the second half of calendar 2023.
EPI-7386 Monotherapy

The Phase 1b EPI-7386 monotherapy dose expansion study is ongoing and is evaluating two doses/schedules of single agent EPI-7386 in mCRPC patients with less than three prior lines of therapy, no visceral disease and no prior chemotherapy who have progressed on at least one second-generation antiandrogen. The Company is also enrolling nmCRPC patients in the Window of Opportunity cohort of the study, in which patients will receive 12 weeks of EPI-7386 monotherapy treatment before starting standard-of-care therapy.
Corporate Updates

In June 2023, the Company announced the appointment of Lauren Merendino, M.B.A., to its Board of Directors. Ms. Merendino is a leading biopharmaceutical executive who brings over 25 years of commercial experience spanning 20+ disease states, including 15 years of leadership for oncology-specific portfolios.
Summary Financial Results

Net Loss. ESSA recorded a comprehensive loss of $7.3 million for the third quarter ended June 30, 2023, compared to a comprehensive loss of $8.8 million for the third quarter ended June 30, 2022. For the third quarter ended June 30, 2023, this included non-cash share-based payments of $1.2 million compared to $1.6 million for the prior year, recognized for stock options granted and vesting. The decrease in the third quarter was primarily attributed to decreases in research and development expenditures and general and administration expenditures in addition to an increase of $1.2 in interest and other income.

Research and Development ("R&D") expenditures. R&D expenditures for the third quarter ended June 30, 2023 were $6.3 million compared to $6.4 million for the third quarter ended June 30, 2022 and include non-cash costs related to share-based payments ($599,621 for the third quarter ended 2023 compared to $872,531 for the third quarter ended 2022). The decrease in R&D expenditures for the year ended June 30, 2023 is the result of decreased non-cash share-based payments, legal patents and license fees and manufacturing costs related to the Phase 1 clinical trial of EPI-7386.

General and administration ("G&A") expenditures. G&A expenditures for the third quarter ended June 30, 2023 were $2.6 million compared to $2.9 million for the third quarter ended June 30, 2022 and include non-cash costs related to share-based payments of $561,452 for the third quarter ended 2023 compared to $718,469 for the third quarter ended 2022. The decrease in the third quarter is the result of decreased non-cash share-based payments, salaries and benefits and consulting and subcontractor fees.
Liquidity and Outstanding Share Capital
At June 30, 2023, the Company had available cash reserves and short-term investments of $152.5 million reflecting the gross proceeds of the February 2021 financing of approximately $150.0 million, less operating expenses in the intervening period. The Company’s cash position is expected to be sufficient to fund current and planned operations through 2025.

As of June 30, 2023, the Company had 44,092,374 common shares issued and outstanding.

In addition, as of June 30, 2023 there were 2,927,477 common shares issuable upon the exercise of warrants and broker warrants. This includes 2,920,000 prefunded warrants at an exercise price of $0.0001, and 7,477 warrants at a weighted average exercise price of $42.80. There were 8,150,274 common shares issuable upon the exercise of outstanding stock options at a weighted-average exercise price of $5.05 per common share.

Cumberland Pharmaceuticals Reports Revenue and Earnings Growth Second Quarter 2023

On August 8, 2023 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, reported significantly improved financial results and a favorable overall company performance for the second quarter of 2023 (Press release, Cumberland Pharmaceuticals, AUG 8, 2023, View Source [SID1234634010]).

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Highlights include:

$10.9 million in revenue during the second quarter, an increase of 6% over the prior year period, and an increase of 18% sequentially from the first quarter of this year.
Positive earnings for the second consecutive quarter, with $1.1 million in net income for the first half of the year.
Adjusted earnings for the first half of 2023 of $4.0 million, or $0.27 a share, which is up significantly from the same period last year.
$89.4 million in total assets, $52.5 million in total liabilities, and $36.8 million of shareholders’ equity at the end of June 2023.
Cumberland will report its full second quarter 2023 financial results and provide a company update via a conference call today at 4:30 p.m. Eastern Time.

"We have had an overall successful first half of the year, with several key developments, including FDA approval for the use of our Caldolor product for treating infants," said Cumberland’s CEO, A.J. Kazimi. "We look forward to building on this success throughout the remainder of the year, as we continue to provide innovative products to improve patients’ lives."

Recent Company developments include:

Caldolor for Treating Infants & Supporting Study Publication

In May 2023, Cumberland announced that the FDA approved expanded labeling for its Caldolor product, an intravenously delivered formulation of ibuprofen, to now include use in infants. The non-narcotic agent may now be administered for the treatment of pain and fever in patients 3 to 6 months of age. With this newly approved labeling, Caldolor is the only non-opioid product approved to treat pain in infants that is delivered through injection.

In June 2023, the Company shared the positive results from a clinical study investigating the safety and pharmacokinetics of Caldolor in newborn infants. The clinical study evaluated the safety and drug exposure profile of Caldolor in 24 hospitalized infants between the ages of 1 and 6 months who required treatment for pain or fever. The results of the study support the growing body of evidence that demonstrates Caldolor is a safe therapeutic option available to practitioners for the treatment of fever and pain in infants and children.

Clinical Development Programs

Cumberland has been evaluating its ifetroban product candidate, a selective thromboxane-prostanoid receptor antagonist, in a series of clinical studies. It has been dosed in nearly 1,400 subjects and has been found to be safe and well tolerated in healthy volunteers and various patient populations.

Patient enrollment is well underway in two of the company’s sponsored Phase II clinical programs to evaluate ifetroban in Systemic Sclerosis, or scleroderma, a debilitating autoimmune disorder characterized by diffuse fibrosis of the skin and internal organs; and the cardiomyopathy associated with Duchenne Muscular Dystrophy ("DMD"), a rare and fatal genetic neuromuscular disease that results in deterioration of the skeletal, heart and lung muscles.

In June 2023, Cumberland presented results from an interim analysis for the FIGHT DMDTM trial at the 29th annual Parent Project Muscular Dystrophy Conference in Dallas. The interim analysis was conducted on data from 25 patients with DMD who completed six of the 12 total months of treatment and assessments. Both doses of ifetroban were reported well tolerated in DMD participants ages 7 years of age or older. The FDA Orphan Product Division previously awarded Cumberland $1.0 million in funding under its Orphan Products Grants Program to support this trial. This was the first DMD trial awarded such funding.

In May 2023, Cumberland announced that the FDA had cleared the Investigational New Drug Application for a Phase II study in patients with Idiopathic Pulmonary Fibrosis ("IPF"), the most common form of progressive fibrosing interstitial lung disease. As a result, the company will launch its FIGHTING FIBROSIS trial designed to enroll 128 patients in over 20 medical centers of excellence across the U.S. This Phase II clinical trial will study the safety, tolerability and efficacy of oral ifetroban in patients with IPF. Recent studies have shown ifetroban can both prevent and enhance resolution of lung fibrosis in multiple preclinical models.

Cumberland has also completed Phase II clinical programs with ifetroban in patients with Hepatorenal Syndrome, Portal Hypertension and Aspirin Exasperated Respiratory Disease. Additional preclinical and pilot clinical studies of ifetroban are underway, including several investigator-initiated trials.

The company plans to complete each of its company-sponsored studies, analyze their final data and announce top-line results before deciding on the best development path for the registration of ifetroban.

FDA Fee Waivers

During the second quarter of 2023, the FDA informed Cumberland that it had granted two barrier-to-innovation waivers that would result in refunds totaling approximately $2.8 million that the company previously paid for prescription drug program fees.

The FDA granted each waiver after concluding that Cumberland met the statutory criteria based on the innovation associated with its ifetroban clinical development programs, as the funds could be better used to advance those studies, which are designed to address a series of unmet medical needs. Cumberland received both refunds in June 2023.

Sancuso Acquisition and Approval of New Manufacturing Plant

In early 2022, Cumberland announced its acquisition of the U.S. rights to oncology-supportive drug Sancuso from the U.S. subsidiary of Kyowa Kirin, Inc., a Japan-based specialty pharmaceutical company. Sancuso is the first and only FDA-approved prescription patch for the prevention of nausea and vomiting in patients receiving certain types of chemotherapy treatment. The active drug in Sancuso, granisetron, slowly dissolves in the thin layer of adhesive that sticks to the patient’s skin and is released into their bloodstream over several days, working continuously to prevent chemotherapy-induced nausea and vomiting ("CINV"). It is applied 24 to 48 hours before receiving chemotherapy and can prevent CINV for up to five consecutive days. Alternative oral treatments must be taken several times (day and night) to deliver the same therapeutic doses.

Cumberland assumed commercial responsibility for the product in the U.S. – including its marketing, promotion, distribution, manufacturing and medical support activities – early last year and largely completed the transition of Sancuso to Cumberland throughout the remainder of the year. In late 2022, the FDA approved moving the product’s manufacture to a new facility, which will be source of future product supplies. In June 2023, Cumberland launched an expanded oncology sales division to feature the product, which continues to be a significant contributor to Cumberland’s business.

Nordic Pharma RediTrex Agreement Restructured

In 2022, Cumberland restructured its agreement with Nordic Pharma, who previously provided Cumberland with the license for the U.S. rights associated with the RediTrex product line. Nordic has assumed responsibility for the product in the U.S. as of July 1, 2023.

FINANCIAL RESULTS:

Net Revenue: For the three months ended June 30, 2023, net revenues were $10.9 million. Net revenue by product for the second quarter of 2023, included $4.1 million for Kristalose, $2.1 million for Vibativ, $1.9 million for Sancuso and $1.2 million for Caldolor.

Year-to-date 2023 net revenues were $20.1 million. Year-to-date net revenues by product were $8.4 million for Kristalose, $4.0 million for Vibativ, $3.8 million for Sancuso and $2.2 million for Caldolor.

Operating Expenses: Total operating expenses were $10.9 million for the second quarter of 2023 and $21.6 million for the first half of the year.

Net Income: The Net Income for the second quarter of 2023 was $0.9 million, or $0.06 a share, and $1.1 million year to date, or $0.07 a share.

Adjusted earnings: Adjusted earnings for the second quarter of 2023 were $2.3 million, or $0.16 a share and $4.0 million year to date, or $0.27 a share. The adjusted earnings calculation does not include the benefit of the $0.2 million of Vibativ cost of goods, which were received with the product acquisition. It also does not include the benefit of the $0.3 million of Sancuso cost of goods, which were received with that product’s acquisition.

Balance Sheet: At June 30, 2023, Cumberland had $89.4 million in total assets, including $18.2 million in cash and cash equivalents.

Total liabilities were $52.5 million, including $13.1 million outstanding on the Company’s revolving line of credit. Total shareholders’ equity was $36.8 million.

EARNINGS REPORT CALL:

A conference call will be held on August 8 at 4:30 p.m. Eastern Time, to discuss the results. To participate in the call, please register at: https://register.vevent.com/register/BIeb03c2e461914b7d94f99de2f7535170.

Registered participants can dial in from their phone using a dial-in and PIN number that will be provided to them. Alternatively, they can choose a "Call Me" option to have the system automatically call them at the start of the conference.

A replay of the call will be available for one year and can be accessed via Cumberland’s website or by visiting View Source