Ryvu Therapeutics to Highlight HPK1 and STING Programs at the Upcoming Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting

On November 9, 2021 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology reported that it will present two research posters on its HPK1 and STING programs at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting (SITC 2021) taking place on November 10 – 14, 2021, in Washington, DC (Press release, Ryvu Therapeutics, NOV 9, 2021, View Source [SID1234594919]).

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Ryvu will present a poster on novel, orally administered HPK1 inhibitors which exhibit nanomolar activity in an immunosuppressive environment. These small molecules have shown favorable PK profiles allowing for in vivo target engagement after oral administration, and hold promising potential as a treatment in a variety of solid tumor indications.

The second poster will highlight a novel small-molecule STING agonist, RVU-27065. Selective STING pathway activation with RVU-27065 allows for repolarization of immunosuppressive tumor-associated macrophages into a pro-inflammatory phenotype without a negative impact on T-cell function. With favorable drug-like properties and a good safety profile, RVU-27065 is a promising candidate for standalone treatment as well as targeted delivery as a payload for antibodies.

Both posters will be available online as of November 12, at 7 AM ET, and presented during the Poster Session on November 13, at the Walter E. Washington Convention Center in Washington, D.C.

Novel, orally administered HPK1 inhibitors demonstrate anti-tumor efficacy and enhanced immune response (Abstract ID: 752), Maciej Kujawa et al.

On-site presentation session: November 13, 2021, 7:00 AM – 8:30 PM, Poster Hall E

Characterization of RVU-27065 a novel small-molecule STING agonist suitable for systemic administration (Abstract ID: 764), Maciej Rogacki et al.

On-site presentation session: November 13, 2021, 7:00 AM – 8:30 PM, Poster Hall E

To view the abstracts, please visit: View Source

Rarecells, Inc. to fund a clinical study at Columbia University of its ISET® -AML test for early detection of Acute Myeloid Leukemia (AML)

On November 9, 2021 Rarecells, Inc., a healthcare company in the field of liquid biopsy, focused on the development and commercialization of non-invasive tests for early cancer detection, reported that it has entered into an agreement with Columbia University to fund a clinical study evaluating its ISET -AML test for early detection of acute myeloid leukemia (AML) in patients with myelodysplastic syndrome (MDS) (Press release, RARECELLS, NOV 9, 2021, https://www.prnewswire.com/news-releases/rarecells-inc-to-fund-a-clinical-study-at-columbia-university-of-its-iset–aml-test-for-early-detection-of-acute-myeloid-leukemia-aml-301420287.html [SID1234594918]).

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The study, expected to begin in Q1 2022, will be the first clinical evaluation of the ISET -AML test. Azra Raza, M.D., Professor of Medicine at Columbia University Vagelos College of Physicians and Surgeons and Director of the MDS Center at Columbia University’s Herbert Irving Comprehensive Cancer Center, is the study’s principal investigator.

This clinical study aims to assess the clinical performance of the ISET -AML test in the context of early AML detection.

Myelodysplastic syndromes are a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells. For roughly 30% of the patients diagnosed with MDS, this type of bone marrow failure syndrome will progress to AML, which is often fatal. Currently, patients with MDS are monitored with blood tests to measure complete blood cell counts and bone marrow analyses to detect the appearance of leukemic blasts heralding the development of AML. However, blood tests have a very low sensitivity to detect rare blasts and bone marrow tests involve an invasive procedure that cannot be repeated frequently. As a result, AML is often diagnosed when it is already well established and may be resistant to available treatment options.

The ISET patented EC-marked platform is able to extract down to one single cancer cell of any type from 10 ml of blood, thus a tumor cell mixed with 100 million leukocytes and 50 billion erythrocytes, according to peer-reviewed published studies. The ISET -AML test uses the ISET platform to extract from blood the rare leukemic blast cells with unsurpassed sensitivity and detect them through suitable markers labelling, image analysis and AI.

Dr. Raza, who wrote the best-selling book "The First Cell and the Human Costs of Pursuing Cancer to the Last," said: "New non-invasive tests are needed to detect the first leukemic cells in the blood of MDS patients so that we can identify and treat patients at risk of developing AML before the disease becomes established."

Patrizia Paterlini, President and Chief Executive Officer of Rarecells, said: "We are pleased to be working with Columbia University’s Herbert Irving Comprehensive Cancer Center to conduct the first clinical evaluation of the ISET -AML test in patients with MDS. The strategic decision to transition the ISET -AML test to an investigator-sponsored study at Columbia will provide access to world-class expertise and has the potential to accelerate patients’ recruitment in order to expedite our clinical study. We look forward to a continued progress across our ISET Early Cancer Detection program, which also includes ongoing studies in patients at risk of Prostate Cancer".

Curis Reports Third Quarter 2021 Financial Results and Business Update

On November 9, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the third quarter ended September 30, 2021 and provided business updates (Press release, Curis, NOV 9, 2021, View Source [SID1234594917]).

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"During the third quarter of 2021, we continued to advance our clinical trials of CA-4948, our first-in-class, small molecule IRAK4 inhibitor in nine distinct patient populations across AML, MDS and B cell cancers, including the recent initiation of our Phase 1 combination study in AML/MDS. At the AACR (Free AACR Whitepaper)-NCI-EORTC Conference in October, we shared exciting preclinical data highlighting potential applications for CA-4948 across different hematologic malignancies, including pCNS lymphoma, an aggressive lymphoma with severe unmet need for patients, further highlighting the potential broad applicability of CA-4948," said James Dentzer, President and Chief Executive Officer of Curis.

"As we look ahead, we plan to provide a clinical data update in January from our ongoing clinical studies, including safety data from our Phase 1 study of CI-8993 and the latest safety and efficacy data from our CA-4948 study in AML/MDS patients with spliceosome or FLT3 mutations."

"In our ongoing monotherapy AML/MDS study, we expect to achieve our enrollment target of having 10-20 total patients with a spliceosome mutation by year-end. We believe data from these patients may provide for an opportunity to explore discussions with the FDA on a registrational path forward in the first half of 2022," concluded Mr. Dentzer.

Third Quarter 2021 and Recent Operational Highlights

Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

Enrollment remains on track in nine distinct patient populations across AML, MDS and B cell cancers.
In November 2021, Curis initiated dosing in the combination stage of the Phase 1/2 study of CA-4948 plus azacitidine and CA-4948 plus venetoclax.
The combination therapy portion includes two arms: CA-4948 plus azacitidine, for patients naïve to HMA, and CA-4948 plus venetoclax, for patients naïve to venetoclax.
When combined with azacitidine, CA-4948 will be dosed at 200 mg twice daily for 21 days of a 28-day cycle, followed by a 300 mg dose cohort if tolerability allows. Azacitidine will be given in 7 consecutive doses or split doses starting at 75 mg/m2.
When combined with venetoclax, the starting dose level for CA-4948 will be 200 mg twice daily for 21 days of a 28-day cycle, followed by a 300 mg dose cohort if tolerability allows. Venetoclax will be administered at 100 mg orally with a ramp up over 3 days to 400 mg for 21 days of a 28-day cycle.
In October 2021, Curis announced new preclinical data highlighting the potential of CA-4948 in additional hematologic malignancies in two presentations at the AACR (Free AACR Whitepaper)-NCI-EORTC Virtual Conference on Molecular Targets and Cancer Therapeutics.
Preclinical data concluded CA-4948 is synergistic with small molecule BTKi therapies that target BCR signaling and suggest the combination can overcome or reduce secondary resistance to BTKi therapies in marginal zone lymphoma.
CA-4948 is able to cross the blood-brain barrier in a preclinical murine model of CNS lymphoma, producing significant and dose-dependent anti-tumor activity and survival advantage.
Curis expects to have 10-20 patients in AML/MDS patients with SF3B1 or U2AF1 spliceosome mutation enrolled by year-end 2021.
Curis plans to provide an update on efficacy and safety from these patients in January. Data may not be fully matured or complete at this time.
Curis expects to present additional data from the 10-20 patients with spliceosome mutations in more detail in the first half of 2022. Data from these patients may provide for an opportunity to explore discussions with the FDA on a registrational path forward in the first half of 2022.
Immuno-oncology, CI-8993 (anti-VISTA antibody; ImmuNext collaboration):

Enrollment remains on track in the ongoing Phase 1 dose escalation study of CI-8993, Curis’s first-in-class monoclonal anti-VISTA antibody for the treatment of R/R solid tumors.
Curis expects to report initial safety data from this trial in January.
Curis will hold a poster presentation with new preclinical data on CI-8993 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting being held from November 12-14, 2021.
Title: Preclinical evaluation of anti-VISTA antibody CI-8993 in a syngeneic huVISTA-KI model
Presenting Author: Andrew M. Scott, MD Olivia Newton-John Cancer Research Institute, Tumour Targeting Laboratory, Melbourne, VIC, Australia
Abstract Number: 324
Abstracts were made available Tuesday, November 9, 2021, at 8:00 a.m.
Virtual ePoster presentations will be available Friday, November 12, 2021, at 7:00 a.m.
Upcoming Planned Milestones for 2022

In January, provide a clinical data update on our ongoing studies, including initial safety data from the ongoing Phase 1 monotherapy study of CI-8993 for the treatment of R/R solid tumors and the latest safety and efficacy data from the Phase 1/2 monotherapy study of CA-4948 in AML/MDS patients with spliceosome mutations that result in aberrant splicing of oncogenic IRAK4-L and patients with FLT3 mutations.
In the first half of the year, provide additional data from the ongoing Phase 1/2 monotherapy study of CA-4948 in patients with R/R AML/MDS at a medical meeting.
In the first half of the year, report initial data at a medical meeting from the ongoing Phase 1/2 combination study of CA-4948 plus ibrutinib in patients with B cell cancers.
Third Quarter 2021 Financial Results

For the third quarter of 2021, Curis reported a net loss of $11.1 million or $0.12 per share on both a basic and diluted basis, as compared to a net loss of $6.0 million, or $0.11 per share on both a basic and diluted basis for the same period in 2020. Curis reported a net loss of $31.8 million or $0.35 per share on both a basic and diluted basis, for the nine months ended September 30, 2021, as compared to a net loss of $22.4 million, or $0.52 per share on both a basic and diluted basis, for the same period in 2020.

Revenues for the third quarter of 2021 and 2020 were $3.0 million and $2.7 million, respectively. Revenues for the nine months ended September 30, 2021 were $7.5 million, as compared to $7.8 million for the same period in 2020. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses for the third quarter of 2021 were $13.1 million, as compared to $7.5 million for the same period in 2020. Operating expenses for the nine months ended September 30, 2021 were $37.0 million, as compared to $26.4 million for the same period in 2020, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.2 million for the third quarter of 2021, as compared to $0.1 million for the same period in 2020. Cost of royalty revenues for the nine months ended September 30, 2021 were $0.4 million, as compared to $0.4 million for the same period in 2020.

Research and Development Expenses. Research and development expenses were $8.6 million for the third quarter of 2021 as compared to $4.7 million for the same period in 2020. The increase in direct research and development expenses for the quarter is primarily attributable to increased clinical and manufacturing costs for our programs. Additionally, employee related costs increased by $2.3 million, primarily attributable to increased stock compensation and personnel costs as a result of additional headcount. Research and development expenses were $24.1 million for the nine months ended September 30, 2021 as compared to $17.5 million for the same period in 2020.

General and Administrative Expenses. General and administrative expenses were $4.3 million for the third quarter of 2021, as compared to $2.6 million for the same period in 2020. The increase in general administrative expense was driven primarily by higher costs for stock-based compensation, personnel, and professional and consulting services. General and administrative expenses were $12.5 million for the nine months ended September 30, 2021, as compared to $8.6 million for the same period in 2020.

Other Expense, Net. For the third quarter of 2021 and 2020, net other expense was $1.0 million and $1.3 million, respectively. Net other expense primarily consisted of imputed interest expense related to future royalty payments. Net other expense was $2.3 million for the nine months ended September 30, 2021, as compared to $3.8 million for the same period in 2020.

As of September 30, 2021, Curis’s cash, cash equivalents and investments totaled $149.8 million, and there were approximately 91.6 million shares of common stock outstanding. Curis expects that its existing cash, cash equivalents and investments should enable it to maintain its planned operations into 2024.

Conference Call Information

Curis management will host a conference call today, November 9, 2021, at 4:30 p.m. ET, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

Cumberland Pharmaceuticals Reports Third Quarter 2021 Financial Results & Company Update

On November 9, 2021 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, reported that is providing a company update and third quarter 2021 financial results (Press release, Cumberland Pharmaceuticals, NOV 9, 2021, View Source;company-update-301420388.html [SID1234594916]). Net revenues from continuing operations during the quarter were $8.1 million and totaled $27.7 million for the first nine months of 2021, up 1.8% compared to the same period in 2020. The company also recorded an additional $0.5 million in revenue during the third quarter and $1.5 million year to date, associated with divested product rights for two brands it is no longer distributing.

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The Company’s financial position included $87.6 million in total assets, with $25.8 million in cash, $41 million of total liabilities, and $46.8 million of shareholders’ equity at the end of the quarter.

"We delivered another steady quarterly performance when factoring in the challenges of operating during the pandemic," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "Our diversified portfolio has been critical in keeping our forward progress during this time, and we are seeing signs that things are slowly starting to return to a more normal operating environment. Our team remains committed to advancing our mission of improving patient care through the delivery of high-quality medicines through the end of the year and beyond."

RECENT COMPANY DEVELOPMENTS:

RediTrex Launch

In late 2019, Cumberland received approval from the U.S. Food and Drug Administration ("FDA") for its New Drug Application for RediTrex, the Company’s methotrexate product line. RediTrex is a new line of pre-filled syringes specifically designed for ease of handling and dosing accuracy for the subcutaneous administration of methotrexate in patients with arthritis and psoriasis.

In late 2020, the Company received initial product supplies and then provided shipments of RediTrex to select accounts. Due to the pandemic, Cumberland delayed the national launch of the product, which was implemented during the third quarter of 2021.

RediTrex treats patients with severe, active rheumatoid arthritis, and polyarticular juvenile idiopathic arthritis who have difficulty tolerating or responding to orally delivered methotrexate. It is also approved for symptomatic control of severe, recalcitrant, disabling psoriasis in adults who are not adequately responsive to other forms of therapy.

With more than 54 million Americans living with some form of arthritis, the disease is among the most common causes of work disability in the U.S., according to the CDC. The oral form of methotrexate is typically the first line of treatment for rheumatoid arthritis. As the disease progresses, the dose must be increased to stay effective, often causing intolerable gastrointestinal side effects. Injectable methotrexate has been proven to be more effective than oral delivery, with fewer gastrointestinal reactions. Because of the increased efficacy and tolerability, injectable methotrexate can delay the need to move to costly biologics, lowering overall patient treatment costs. Once disease progression requires the use of biologics, continuing the treatment of injectable methotrexate along with the biologic has been shown to increase overall efficacy.

Other injectable methotrexate options available may not optimally meet the needs of an arthritis patient. Patients are offered either a vial and syringe for self-injection, or the use of an expensive autoinjector. The vial and syringe method can be difficult for a patient to handle due to limited dexterity in their hands. Additionally, obtaining the exact dose needed while preventing skin exposure to the caustic methotrexate can be quite challenging for many patients. The autoinjectors provide a better alternative to the vial and syringe, but they remove injection control from the patient and can be painful to administer. They are also the most expensive methotrexate delivery.

ESG Report

In July 2021, Cumberland released its second annual Sustainability Report (the "2020 Sustainability Report"), which details the Company’s activities pertaining to its environmental, social and governance ("ESG") matters. After issuing its inaugural ESG report last year (the "2019 Sustainability Report"), Cumberland remains committed to sustainability and to maintaining transparency of its corporate operations. As the largest biopharmaceutical company founded and headquartered in the Mid-South, the Company holds itself to the highest standards of ethical practices and understand the importance of recognizing and addressing its impact on its constituents, the community and the environment.

The 2020 Sustainability Report notes that Cumberland provided nearly 2.5 million patient doses of its products, safely disposed of over 4,000 pounds of expired and damaged products and had no product recalls. Cumberland also had no Company brands listed on the FDA’s MedWatch Safety Alerts for Human Medical Products, no Company product issues identified by FDA from their Adverse Event Reporting System and no clinical trials terminated due to failure to practice good clinical standards.

The 2020 Sustainability Report also highlights several initiatives Cumberland implemented as part of its commitment to delivering high-quality pharmaceutical products to improve patient care. For example, the Company continued a program to serialize all commercial products sold in the United States, allowing it to track every unit distributed, which helps to prevent counterfeit drugs from entering the market under the Cumberland brand. In addition, through its coupon program, Cumberland can cover up to 90% of patient prescription costs for its gastrointestinal products.

The 2020 Sustainability Report also highlights Cumberland’s investment in its employees through its continuing education programs, employee development initiatives and employee recognition awards. Cumberland’s workforce is 46% women – and 18% of its employees are minorities.

Ifetroban Clinical Studies

Cumberland has been evaluating its ifetroban product candidate in a series of clinical studies. The Company is sponsoring Phase II clinical programs to evaluate its ifetroban product candidates in 1) patients with cardiomyopathy associated with Duchenne Muscular Dystrophy, a rare, fatal, genetic neuromuscular disease that results in deterioration of the skeletal, heart and lung muscles, 2) Systemic Sclerosis or scleroderma, a debilitating autoimmune disorder characterized by diffuse fibrosis of the skin and internal organs and 3) Aspirin-Exacerbated Respiratory Disease, a severe form of asthma.

Enrollment in these clinical studies was interrupted due to the COVID-19 pandemic. However, many of Cumberland’s clinical study sites have reopened and resumed screening of patients for potential participation into its studies. The Company is awaiting results from the studies underway before deciding on the best development path for the registration of ifetroban, its first new chemical entity.

In September 2021, Cumberland’s Board of Directors approved a new clinical program for the use of ifetroban to treat Progressive Fibrosing Interstitial Lung Diseases ("PF-ILDs"). Nonclinical studies are complete, and the resulting manuscript has been prepared and submitted for publication. A Phase II clinical study is planned and an application to the FDA is in preparation to support this new clinical program.

Additional pilot preclinical and clinical studies of ifetroban are underway, including several investigator-initiated trials.

Hyponatremia Publication

The Health Outcome Predictive Evaluation (HOPE) COVID-19 Registry Analysis, an international study of over 4,000 patients published in November 2020, found that patients hospitalized with COVID-19 had a high risk of developing hyponatremia. These COVID-19 patients also had a higher incidence of mortality due to their hyponatremia. The study results support the use of an intravenous vaptan to treat hyponatremia in critically ill patients afflicted with COVID-19.

Hyponatremia, an imbalance of serum sodium to body water, is the most common electrolyte disorder among hospitalized patients. Cumberland’s Vaprisol product is one of two branded prescription products indicated for the treatment of hyponatremia, and the only intravenously administered branded treatment. Vaprisol has a proven day-1 response rate to normalize serum sodium levels in hyponatremic patients and move them out of the Intensive Care Unit as efficiently as possible.

New Line of Credit

On October 28, 2021, Cumberland entered into a Fourth Amendment to the Revolving Credit Note and Fifth Amendment ("Fifth Amendment") to the Revolving Credit Loan Agreement with Pinnacle Bank (the "Pinnacle Agreement"). The original Pinnacle Agreement was dated July 31, 2017 and the Fifth Amendment extended the term of the loan agreement for a three-year period ending October 1, 2024. The facility provides for a principal available for borrowing of up to $15 million and an opportunity to request an increase in availability to $20 million, upon the satisfaction of certain conditions and approval by Pinnacle Bank. The interest rate on funds borrowed under the facility ranges from 30-day LIBOR plus 175 to 275 basis points depending on the funded debt ratio.

Vibativ International Agreements

On August 25, 2021, Cumberland signed an agreement with Verity Pharmaceuticals International Limited to license and commercialize Vibativ in Puerto Rico. Verity is a specialty pharmaceutical company with commercial operations in the U.S. and Canada.

Vibativ is a patented, FDA-approved injectable anti-infective for the treatment of certain serious bacterial infections, including hospital-acquired and ventilator-associated bacterial pneumonia, and complicated skin and skin structure infections. It addresses a range of Gram-positive bacterial pathogens, including those that are considered difficult-to-treat and multidrug-resistant. In November 2018, Cumberland reached an agreement to acquire Vibativ from Theravance Biopharma and assume global responsibility for the product.

SciClone Pharmaceuticals (Holdings) Limited has licensed Cumberland’s Vibativ product for sale and distribution in China. In February 2021, SciClone completed an initial public offering and listing of their shares on the Hong Kong stock exchange. During the third quarter of 2021, SciClone submitted an application to the Chinese regulatory authority for the approval of Vibativ in that country. In September 2021, the filing was accepted by the regulatory agency for review. SciClone expects a review period of up to twelve months for their application and believes that the potential for Vibativ in China may be significant.
FINANCIAL RESULTS:

Net Revenues: For the three months ended September 30, 2021, net revenues from ongoing operations were $8.1 million, compared to $9.3 million for the prior year period. The company also recorded an additional $0.5 million in revenue during the third quarter associated with divested rights to products that the company no longer distributes.

Net revenue by product for the third quarter 2021, included $4.0 million for Kristalose, $1.9 million for Vibativ, $1.3 million for Caldolor, and $0.3 million for Vaprisol.

Year-to-date 2021 net revenues were $27.7 million, up 1.8% from $27.2 million during the first nine months of 2020. There were additional revenues of $1.5 million in 2021 associated with the divested product rights.

Year-to-date 2021 net revenues by product were $12.3 million for Kristalose, $8.8 million for Vibativ, $3.7 million for Caldolor and $1.9 million for Vaprisol.

Operating Expenses: Total operating expenses for the three months ended September 30, 2021, were $9.6 million, compared to $10.5 million during the prior year period.

Earnings: Net income (loss) for the third quarter 2021 was $(1.1) million or $(0.07) a share, compared to $(0.5) million or $(0.03) a share for the prior year period. Adjusted earnings (loss) for the third quarter was $(0.3) million, compared to $0.2 million for the prior year period.

Year-to-date net income (loss) in 2021 was $0.3 million, compared to $(2.5) million for the same period last year. Adjusted earnings for the first nine months of 2021 were $0.7 million, compared to an adjusted loss of $(0.3) million during the same period in 2020.

Balance Sheet: At September 30, 2021, Cumberland had $87.6 million in total assets including $25.8 million in cash and cash equivalents. Total liabilities were $41 million, including $15 million outstanding on the Company’s revolving line of credit, resulting in total shareholders’ equity of $46.8 million.

CONFERENCE CALL & WEBCAST:

A conference call and live internet webcast will be held on Tuesday, November 9, at 4:30 p.m. Eastern Time to discuss the results. To participate in the call, please dial 877-303-1298 (for U.S. callers) or 253-237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing 855-859-2056 (for U.S. callers) or 404-537-3406 (for international callers). The Conference ID for the rebroadcast is 9476299. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

Poseida Therapeutics Provides Program Updates and Financial Results for the Third Quarter of 2021

On November 9, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported program updates and financial results for the third quarter ended September 30, 2021 (Press release, Poseida Therapeutics, NOV 9, 2021, View Source [SID1234594915]).

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"The last quarter saw continued progress as Poseida reached multiple key strategic milestones including FDA clearance of our first fully allogeneic CAR-T IND for P-BCMA-ALLO1, the presentation of strong CAR-T data in a solid tumor indication with our P-PSMA-101 program and, shortly after the quarter, the announcement of a strategic collaboration with Takeda focused on non-viral in vivo gene therapy programs utilizing our platform technologies," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida.

"The initiation of our P-BCMA-ALLO1 clinical trial represents the beginning of a long-planned strategic transition to what we believe is the ‘holy grail’ of cell therapy for oncology, a fully allogeneic CAR-T with a fully humanized heavy chain BCMA binder and a high percentage of TSCM cells which we believe are the key to success," continued Ostertag. "While we believe the P-BCMA-101 autologous program has competitive advantages and would be approvable, one long-term strategic benefit of that program has been to inform our highly-differentiated allogeneic approach. With the P-BCMA-ALLO1 clinical program now underway and with very high confidence in our allogeneic platform, we will begin a planned wind down of P-BCMA-101."

Program Updates

BCMA Program
P-BCMA-ALLO1, the Company’s first fully allogeneic CAR-T product candidate, is in development for the treatment of relapsed/refractory multiple myeloma. In August of 2021, the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for P-BCMA-ALLO1. The IND clearance and the start-up of the Phase 1 clinical trial mark the beginning of the Company’s strategic shift toward focusing on P-BCMA-ALLO1 rather than the autologous P-BCMA-101 program.

While data in the autologous trial showed meaningful responses and a favorable safety profile, the Company’s strategic focus has long been on allogeneic CAR-T therapies, leveraging the learnings of the autologous CAR-T program to provide benefits beyond those of autologous CAR-T, including a more desirable off-the-shelf product profile for future commercialization while maintaining the tolerability advantage of our autologous product candidate. P-BCMA-ALLO1 has the potential to deliver up to hundreds of doses per manufacturing run, thereby dramatically reducing both clinical trial costs and ultimately commercial product cost compared to the autologous P-BCMA-101 program.

PSMA Program
P-PSMA-101 is a solid tumor autologous CAR-T product candidate being developed to treat patients with metastatic castrate-resistant prostate cancer (mCRPC) currently in an ongoing Phase 1 dose escalation trial.

In August of 2021, the Company presented preliminary data at the 6th Annual CAR-TCR Summit virtual meeting that demonstrated meaningful patient responses while maintaining a favorable safety and tolerability profile with modest overall rates of CRS and no neurotoxicity observed at low doses. An additional update on the P-PSMA-101 program is expected in the first half of 2022.

MUC1-C Program
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate in preclinical development with the potential to treat a wide range of solid tumors, including breast and ovarian cancers. P-MUC1C-ALLO1 is proceeding, with an anticipated IND filing and initiation of a Phase 1 clinical trial by the end of 2021.

Liver-Directed Gene Therapy Program
P-OTC-101 is the Company’s first liver-directed gene therapy program for the in vivo treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene, a condition characterized by high unmet medical need. The Company is currently evaluating whether to modify the P-OTC-101 program to move to the fully non-viral nanoparticle delivery system. The Company will update expected timing on program advancement once that evaluation is complete.

Other Operational Updates and Upcoming Events

Gene Therapy Research Collaboration with Takeda
In October of 2021, the Company entered into a collaboration and license agreement with Takeda Pharmaceuticals USA, Inc. to utilize Poseida’s proprietary genetic engineering platforms for the research and development of up to eight gene therapies.

The collaboration will focus on developing non-viral in vivo gene therapy programs, including Poseida’s Hemophilia A program. The Company will receive an upfront payment of $45.0 million, of which $5.0 million is for prepaid research. Per the agreement, Takeda will fund all ongoing partnered program research performed by Poseida. The collaboration may utilize all of Poseida’s novel genetic engineering platform technologies, including the piggyBac DNA Modification System for gene addition, the Cas-CLOVER Site-specific Gene Editing System for ultra-precise gene editing, biodegradable nanoparticle technologies for gene delivery and other emerging technologies. Poseida will lead research activities up to candidate selection, after which Takeda will assume responsibility for further development and commercialization.

GMP Facility
In the third quarter, the Company completed qualification and commenced GMP activity in its internal pilot manufacturing plant. The pilot plant is designed to support and speed the development of allogeneic product candidates including manufacturing clinical material for early-stage trials. The first product candidate to be produced out of the pilot plant will be P-MUC1C-ALLO1.

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting
The Company will give two virtual poster presentations at the upcoming SITC (Free SITC Whitepaper) Annual Meeting, being held in Washington, D.C., and virtually November 10-14, 2021. The full abstracts were made available on the SITC (Free SITC Whitepaper) website earlier today, with presentations taking place on November 12, 2021.

63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition
The Company will present data from the Company’s differentiated P-BCMA-101 autologous CAR-T program including how it has informed the P-BCMA-ALLO1 allogeneic CAR-T program in a poster publication at the ASH (Free ASH Whitepaper) Annual Meeting on Monday, December 13, 2021:

Session Name: 704. Cellular Immunotherapies: Clinical: Poster III
Presentation Time: 6:00 PM – 8:00 PM Eastern time
Location: Georgia World Congress Center, Hall B5

Financial Results for the Third Quarter 2021

Research and Development Expenses
Research and development expenses were $32.5 million for the third quarter ended September 30, 2021, compared to $27.0 million for the same period in 2020. For the nine months ended September 30, 2021, research and development expenses were $97.6 million, compared to $75.6 million for the same period in 2020. The increase was primarily due to increased stock-based compensation expense, headcount, external costs related to our preclinical programs and clinical stage programs, including the ongoing enrollment and manufacturing associated with our P-BCMA-101, P-BCMA-ALLO1 and P-PSMA-101 clinical trials, and internal costs related to facilities development.

General and Administrative Expenses
General and administrative expenses were $9.1 million for the third quarter ended September 30, 2021, compared to $6.5 million for the same period in 2020. General and administrative expenses were $26.3 million for the nine months ended September 30, 2021, compared to $15.6 million for the same period in 2020. The increase was primarily due to increased stock-based compensation expense, headcount, insurance costs and professional fees.

Net Loss
Net loss was $42.4 million and $126.4 million for the three and nine months ended September 30, 2021, respectively, and $34.4 million and $93.6 million for the three and nine months ended September 30, 2020, respectively.

Cash Position
As of September 30, 2021, our cash and cash equivalents balance was $197.8 million.