On August 8, 2023 Aleta Biotherapeutics (Aleta), a privately-held immuno-oncology company with a platform of CAR T-Cell Engagers (CTE) which enable cell therapies to improve outcomes for patients with cancer, reported that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted clinical trial authorization (CTA) to evaluate investigational biologic ALETA-001 in a Phase 1/2 clinical trial in the treatment of patients with B-cell malignancies who are relapsed/refractory to CD19 CAR T-cell therapy (Press release, Aleta Biotherapeutics, AUG 8, 2023, View Source [SID1234634016]).

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Aleta’s lead program, ALETA-001 has clinical support and funding from its collaboration partner, Cancer Research UK’s Centre for Drug Development, which has sponsored and will conduct the Phase 1/2 clinical trial.

"The authorization to clinically evaluate, in collaboration with Cancer Research UK, the potent activity of ALETA-001, our lead biologic CAR T-Cell Therapy Engager, marks important progress for the entire global oncology community – especially patients," commented Paul Rennert, Ph.D., Chief Executive Officer and Chief Scientific Officer, Aleta Biotherapeutics. "Aleta’s CTEs act by transforming the expression of any cancer tumor cell to match the CAR T-cell therapies circulating in a patient’s blood, importantly restoring and increasing the effectiveness with which CAR T-Cells can kill cancer cells," continued Dr. Rennert.

Dr. Nigel Blackburn, Cancer Research UK’s Director of Drug Development, stated, "CAR-T cell therapy has been transformative for some cancer patients but there remains a critical need to ensure that this therapy is an option for all. ALETA-001 is working to address this treatment gap for people with blood cancers by advancing a potentially life-saving CAR-T cell therapy into the clinic. We are delighted to play a key role in this trial and look forward to seeing this treatment become available for more patients in the future."

About CAR T-Cell Therapy Engager (CTE) ALETA-001

Aleta’s lead development program, ALETA-001, contains the CD19 target protein which is further linked to an CD20 antibody domain. ALETA-001 is designed to improve the effectiveness of CD19-directed CAR T therapies by increasing CD19 antigen density and restoring lost CD19 expression on the cancer cell. This allows CD19+/CD20+ cancer cells to be easily recognized and killed by CD19-directed CAR T-cells that were previously administered and are already circulating within a patient.

Aleta previously secured landmark clinical support and funding from Cancer Research UK for the ALETA-001 Phase I/II clinical trials, and ALETA-001 has received a UK Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). ILAP designation is granted to medicines that address life-threatening or seriously debilitating conditions, and where there exists a significant patient or public health need.

On August 8, 2023 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that it has been notified by the U.S. Food and Drug Administration (FDA) about its Investigational New Drug (IND) submission has been reviewed, and Puma can proceed with the clinical development of alisertib monotherapy for the treatment of patients with extensive stage small cell lung cancer (SCLC) (Press release, Puma Biotechnology, AUG 8, 2023, View Source [SID1234634015]). Puma’s Phase II trial (Study PUMA-ALI-4201) will enroll up to 60 patients with extensive stage small cell lung cancer who have progressed after first-line platinum-based chemotherapy and immunotherapy. Patients must provide tissue-based biopsies so that biomarkers can be analyzed. Alisertib will be dosed at 50 mg BID on days 1-7 of every 21 day cycle. The Company anticipates initiating the Phase II trial in the second half of 2023.

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The primary endpoint of the trial will be objective response rate with secondary endpoints of duration of response, disease control rate, progression free survival and overall survival. The Company will also be looking at each of these endpoints within selected pre-specified biomarker subgroups as well as to assess whether there is enhanced efficacy in any biomarker subgroup. Puma will be performing its biomarker analysis of the ALI-4201 trial in parallel with the execution of the clinical trial. Puma plans to perform an initial interim analysis for the evaluation of the biomarkers as well as an evaluation of the efficacy. Based upon the outcomes of the study, the Company anticipates meeting with the FDA to explore the potential for an accelerated approval pathway for alisertib in small cell lung cancer.

"Treatment options for patients with small cell lung cancer that has progressed on or after platinum-based chemotherapy are limited, and there is an urgent need for new drugs to treat this patient population," said Taofeek K. Owonikoko, MD, PhD, Chief of the Division of Hematology/Oncology and Associate Director for Translational Research and Co-Leader of the Cancer Therapeutics Program at the UPMC Hillman Cancer Center. "The results from the previous clinical trials of alisertib in small cell lung cancer suggest that the drug may represent a potentially promising treatment option for these patients and, more specifically, for patient subsets whose tumors harbor potential molecular markers that are likely associated with the clinical activity of an aurora kinase A inhibitor such as alisertib," said Dr. Owonikoko.

Alan H. Auerbach, Chief Executive Officer, President and Founder of Puma, stated, "We are pleased to move forward with the clinical development of alisertib in small cell lung cancer. We are eagerly awaiting the start of this Phase II trial, and we hope that the study will provide much needed insight into the clinical activity of alisertib in small cell lung cancer and, more specifically, in patients with molecularly defined tumors that may be targetable with an aurora kinase A inhibitor like alisertib."

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.

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.

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