On May 10, 2023 Aadi Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company focused on developing and commercializing precision therapies for genetically defined cancers with alterations in mTOR pathway genes, reported financial results for the first quarter of 2023 and provided an initial update on the tumor-agnostic PRECISION 1 trial, a registration-directed Phase 2 study of nab-sirolimus in patients with solid tumors with pathogenic inactivating alterations in TSC1 or TSC2 genes (Press release, Aadi Bioscience, MAY 10, 2023, View Source [SID1234631415]).

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"We are excited about the uptake of FYARRO and its continued sales growth after our first year on the market and believe we are operating from a position of strength with solid cash balances into 2025," said Scott Giacobello, Interim CEO and President and CFO of Aadi. "We are also encouraged by initial PRECISION 1 enrollment data, indicating more than 15 different tumor types on study, shaping the foundation for a truly tumor agnostic trial. We believe in the potential of this study and given the importance of maintaining its integrity, we will provide the results of an efficacy analysis later in the year when response data is unblinded in conjunction with a pre-planned interim analysis on 40 patients with appropriate follow-up."

First Quarter 2023 Updates and Recent Operational Highlights

Based on initial information from the PRECISION 1 trial, enrollment is well-balanced, and the trial is accruing evenly between the TSC1 and TSC2 arms. More than 15 discreet tumor types have been enrolled with no more than three of any type, supporting the thesis that TSC1 and TSC2 alterations occur broadly across different solid tumors. A pre-planned interim analysis on 40 patients with appropriate follow-up is expected by the end of 2023.
FYARRO net product sales were $5.9 million in the first quarter, continued double-digit growth of 12% quarter-over-quarter.
The appointment of Mohammad Hirmand, M.D., to Board of Directors. Dr. Hirmand, previously Chief Medical Officer of Turning Point Therapeutics prior to its acquisition by BMS, is the co-founder of Avenzo Therapeutics, Inc., and serves as executive vice president and chief medical officer of this privately held biotechnology company focused on oncology therapeutics.
Multiple presentations at AACR (Free AACR Whitepaper) Annual Meeting 2023. Aadi presented an encore trials-in-progress (TIP) poster for the ongoing PRECISION 1 trial; results on the anti-tumor activity of nab-sirolimus in combination with KRASG12C inhibitors in xenograft models; and results of a biomarker analysis from AMPECT correlating response to nab-sirolimus with TSC1 and TSC2 inactivating alterations. The AACR (Free AACR Whitepaper) posters can be found here on the Aadi website.
Multiple presentations at SGO Annual Meeting 2023. Aadi presented a TIP poster for the ongoing PRECISION 1 trial and additional data with nab-sirolimus from its AMPECT trial at SGO 2023. The SGO posters can be found here on the Aadi website.
Upcoming presentations at ASCO (Free ASCO Whitepaper) Annual Meeting 2023. Aadi will present a company-sponsored TIP update from the PRECISION 1 Phase 2 study and combination data of nab-sirolimus and pazopanib (PAZO) from an ongoing Investigator Initiated Trial at ASCO (Free ASCO Whitepaper) 2023, taking place June 2-6, 2023, in Chicago. Following Aadi’s presentation at ASCO (Free ASCO Whitepaper), the posters will be made available on the investor relations page of the Aadi website at www.aadibio.com.
Initiation of Phase 1/2 trial in KRASG12C is expected to begin with first patient dosing in the second quarter of 2023. The study will evaluate the combination of adagrasib with nab-sirolimus in collaboration with Mirati Therapeutics. The open-label Phase 1/2 trial is intended to determine the optimal dose and recommended Phase 2 dose in patients with KRASG12C mutant solid tumors.
First Quarter 2023 Financial Results

Total revenue resulting from sales of FYARRO for the quarter ended March 31, 2023 was $5.9 million. This compares to the prior year period of $2.3 million and $5.2 million in the fourth quarter 2022.
Cash, cash equivalents and short-term investments as of March 31, 2023 were $151.2 million as compared to $172.6 million as of December 31, 2022, which is expected to fund operations into 2025 based on current plans.
Net loss for the three months ended March 31, 2023 was $15.2 million as compared to $13.9 million for the three months ended March 31, 2022.
Conference Call Information

The Aadi management team is hosting a conference call and webcast today at 8:30 am ET (5:30 am PT) to provide a corporate update and discuss results for the first quarter 2023.

Participants may access a live webcast of the call on the "Investors & News" page of the Aadi Bioscience website at aadibio.com. To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the Company’s website for at least 30 days.

About FYARRO

FYARRO is an mTOR inhibitor indicated for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

About the PRECISION 1 Trial

The PRECISION 1 trial is a multi-center, open-label, tumor-agnostic registrational clinical trial of nab-sirolimus. This tumor agnostic study will evaluate approximately 60 mTOR inhibitor naïve patients in each of two independent study arms, or approximately 120 in total, comprised of patients with solid tumors harboring pathogenic inactivating alterations in either TSC1 or TSC2 genes. In November 2022, the FDA granted Fast Track designation to evaluate nab-sirolimus for this patient population.

nab-Sirolimus 100 mg/m2 is given weekly intravenously over 30 minutes on Days 1 and 8 of each 21-day cycle. The primary endpoint is overall response rate per independent radiographic review (IRR) using RECIST v1.1. Other endpoints include duration of response, time to response, progression-free survival by IRR, overall survival, patient-reported quality of life, and safety.

On May 10, 2023 Servier, a global pharmaceutical group, reported that the European Commission (EC) has approved Tibsovo (ivosidenib tablets) as a targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy; as well as in monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy (Press release, Servier, MAY 10, 2023, View Source [SID1234631414]).

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Tibsovo is the first and only IDH1 inhibitor approved in Europe. It has received orphan medicine designation recognizing the significant benefit brought to patients by Tibsovo over available therapies for both CCA and AML.

"The prognosis for patients diagnosed with acute myeloid leukemia or cholangiocarcinoma has historically been poor with very limited treatment options. With today’s approval by the European Commission, Tibsovo is now the first targeted IDH1 inhibitor approved in Europe. This further affirms our unparalleled scientific leadership in harnessing the IDH mutation and commitment to finding new therapeutic solutions for patients with difficult and hard-to-treat cancers," said Arnaud Lallouette, M.D., Executive Vice President, Global Medical & Patient Affairs at Servier.

"IDH1 mutations are major drivers of disease progression in acute myeloid leukemia and cholangiocarcinoma, which are usually diagnosed at an advanced stage, highlighting the urgent need for a targeted therapeutic option. The development of new targeted therapies such as Tibsovo, which works differently from traditional chemotherapies, is now providing treatment options that may increase the life expectancy and quality of life for patients," said Philippe Gonnard, M.D., Executive Vice President, Global Product Strategy at Servier.

AML is a cancer of the blood and bone marrow marked by rapid disease progression. It is the most common acute leukemia in adults and affects 5/100,000 inhabitants in Europe, i.e., more than 20,000 new cases each year.i The two-year survival rate of 75 years-old patients with AML is below 10%.ii

The approval by the European Commission in AML is supported by data from the AGILE study, a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial published in the New England Journal of Medicine. Results demonstrated a statistically significant improvement in event-free survival (EFS) (hazard ratio [HR] 0.33; 95% CI [0.16, 0.69]) and overall survival (OS) (HR 0.44; 95% CI [0.27, 0.73] of patients with IDH1-mutated AML treated with Tibsovo in combination with azacitidine compared to azacitidine plus placebo. The median OS (95% CI) for Tibsovo + azacitidine and placebo + azacitidine was 24.0 (11.3, 34.1) and 7.9 (4.1, 11.3) months, respectively. In addition to the primary endpoint of EFS, the study met all key secondary endpoints, including complete remission (CR) rate, OS, and complete remission with partial hematologic recovery (CRh) rate, as well as objective response rate (ORR). These results prove that Tibsovo, in combination with azacitidine, is an effective combination treatment option for patients with newly diagnosed IDH1-mutated AML. The most common adverse reactions were vomiting, neutropenia, thrombocytopenia, electrocardiogram QT prolonged, and insomnia.

Cholangiocarcinoma, a cancer of the bile duct, is a rare and aggressive tumor often linked to medical history such as cirrhosis or liver infection. Cholangiocarcinoma affects 1–3 in 100,000 people in Europe, with approximately 10,000 new cases each year.iii The five-year survival rate is 9%, but 0% if metastasized.iv Only surgery has been shown to cure patients, but this treatment option is only possible for a limited number of patients, and the risk of relapse remains high. Chemotherapy and immunotherapy are the standard therapy for patients with cholangiocarcinoma who are not eligible for surgery or whose disease has progressed after surgery.

The European Commission’s approval in cholangiocarcinoma is supported by data from the ClarIDHy trial, the first and only randomized Phase 3 trial for previously treated IDH1-mutated cholangiocarcinoma. Results from the ClarIDHy study demonstrated a statistically significant improvement in the primary endpoint of progression-free survival (PFS) by an independent review committee (HR 0.37; 95% CI [0.25, 0.54], p<0.001)v. The median PFS (95% CI) for Tibsovo and placebo was 2.7 (1.6, 4.2) and 1.4 (1.4, 1.6) months, respectively. Thirty-two percent and 22% of patients randomized to Tibsovo remained free of progression or death at 6 and 12 months, respectively, versus none on the placebo arm. The most common adverse reactions were fatigue, nausea, abdominal pain, diarrhea, decreased appetite, ascites, vomiting, anemia, and rash.

Tibsovo is currently approved in the U.S. as monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory AML and in monotherapy or in combination with azacitidine for adults with newly diagnosed IDH1-mutant AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy. Tibsovo has also been approved in the U.S. and Australia for patients with previously treated IDH1-mutated cholangiocarcinoma. Tibsovo is also approved in Chinavi for the treatment of adult patients with relapsed or refractory AML who have a susceptible IDH1 mutation.

The Marketing Authorization covers the 27 countriesvii of the European Union as well as Iceland, Liechtenstein and Norway.

Find out more about cholangiocarcinoma and acute myeloid leukemia on servier.com.

On May 10, 2023 Coeptis Therapeutics Holdings, Inc. (NASDAQ: COEP) ("Coeptis" or "the Company"), a biopharmaceutical company developing innovative cell therapy platforms for cancer, reported that research involving its SNAP-CAR technology was detailed in a peer-reviewed article published in the peer-reviewed journal, Nature Communications (Press release, Coeptis Pharmaceuticals, MAY 10, 2023, View Source [SID1234631413]). SNAP-CAR is a multi-antigen chimeric antigen receptor T cell (CAR T) technology that can be adapted to different cancer indications, including hematologic and solid tumors.

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The article titled, "Post-translational covalent assembly of CAR and synNotch receptors for programmable antigen targeting," describes key advances in antigen receptor design to develop "universal" receptor systems for diverse programming of cell behaviors using covalent chemistry. Using in vitro and in vivo models, the authors demonstrated that the SNAP-CAR platform provides a powerful adaptor strategy for fully programmable targeting of engineered cells to multiple antigens. Further, it was concluded that these systems have the potential for clinical application and biotechnological utility by providing researchers with the ability to rapidly screen CAR and synNotch antibody candidates and to rewire and activate cellular programs in response to highly specific antibody-antigen interactions.

"This research highlights key advances in antigen receptor design, including the creation of a universal adaptor synNotch system and a universal CAR system that act through self-labeling enzyme chemistry, representing a potential breakthrough in programmable antigen targeting," said Jason Lohmueller, Ph.D., Assistant Professor of Surgery and Immunology in the Division of Surgical Oncology Research, University of Pittsburgh. "These ‘universal’ receptor systems, for which receptor specificity can be directed post-translationally via covalent attachment of a co-administered antibody, offer the potential to allow for one population of T cells to target multiple tumor antigens, enabling the development of cell therapies for a wide range of cancers, including hematologic and solid tumors."

"SNAP-CAR represents a powerful technology with the potential to be engineered to address numerous cancers, including HER2-expressing cancer, which we are targeting as our potential first-in-human clinical development program," said Dave Mehalick, President and CEO of Coeptis Therapeutics. "Having research on SNAP-CAR published in such a prestigious scientific journal as Nature Communications provides further validation of the technology’s potential and the groundbreaking work being conducted by Dr. Lohmueller and his team at the University of Pittsburgh."

About SNAP-CAR
SNAP-CAR, which Coeptis Therapeutics licensed from the University of Pittsburgh, is designed to be a "universal" CAR T cell therapy platform that can be adapted to different cancer indications. Instead of directly binding to a target on the tumor cell, CAR T cells are co-administered with one or more antibody adaptors that bind to the tumor cells and are fitted with a chemical group that irreversibly connects them to the SNAP-CAR on the therapeutic cells via a covalent bond. Pre-clinical studies in mice have demonstrated that by targeting tumors via antibody adaptor molecules, the SNAP-CAR therapy provides a highly programmable therapeutic platform.

On May 10, 2023 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported a business update, including its financial position as of March 31, 2023 (Press release, Transgene, MAY 10, 2023, View Source [SID1234631412]).

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Key events and upcoming milestones

Since the beginning of the year, all of Transgene’s clinical and preclinical assets have progressed in line with expectations. Promising data were presented on all clinical-stage immunotherapies at the AACR (Free AACR Whitepaper) 2023 conference (April 2023). New data will be communicated in Q2 2023 on TG4001, TG4050 and BT-001.

Therapeutic cancer vaccines

TG4001: New data to be presented at the ASCO (Free ASCO Whitepaper) 2023 conference — Preparing for a planned potentially registrational trial in an HPV-positive indication

Transgene anticipates that the last patient will be randomized in the current Phase II clinical study in the first half of 2024. This study is comparing TG4001 in combination with avelumab vs avelumab alone in patients with HPV16-positive anogenital tumors. Final results will be communicated in 2024.

A trial-in-progress poster was presented at the AACR (Free AACR Whitepaper) conference in April 2023. New translational data from TG4001 will be presented in another poster at the upcoming ASCO (Free ASCO Whitepaper) conference (June 2–6, 2023). Transgene is working on the design of a potentially registrational trial to further confirm the benefit of this novel therapeutic cancer vaccine.

TG4050: Strong clinical potential confirmed by initial data from the two ongoing Phase I trials — Transgene is preparing a Phase II trial in head and neck cancers

New data were presented on TG4050 at AACR (Free AACR Whitepaper) 2023. These data show that this individualized neoantigen cancer vaccine is able to induce strong immune responses, which are expected to result in longer remission periods for patients.

All evaluable patients developed a specific immune response against multiple cancer neoantigens after treatment with TG4050 and remained disease-free in the head and neck cancer trial. These data suggest that TG4050 can boost the immune system of patients in the absence of pre-existing response and despite a challenging tumor micro-environment at baseline.

Transgene hosted a key opinion leader (KOL) event with the participation of Professor Christian Ottensmeier, MD, PhD, FRCP (University of Liverpool, La Jolla Institute for Immunology) who highlighted the medical need in head and neck cancer and the potential of a virus-based immunotherapy such as TG4050.

Transgene expects the last patient to be treated in the Phase I study in head and neck cancer in the coming weeks. Final results from this trial are expected mid-2024.

Transgene and NEC are preparing for a Phase II trial in head and neck cancers which could be initiated in H2 2023.

Transgene will present new data from TG4050 at the upcoming ASCO (Free ASCO Whitepaper) conference in June.

Oncolytic Viruses

TG6002: New data presented at AACR (Free AACR Whitepaper) support the potential of intravenous administration (IV) of Invir.IO-based oncolytic viruses, which offers a competitive advantage

Clinical data presented at AACR (Free AACR Whitepaper) 2023 confirmed the mechanism of action and the safety of our Invir.IO based oncolytic viruses, which offers a key competitive advantage.

These findings support the potential of Invir.IO-based oncolytic viruses to be given by IV administration. This extends their potential use to a much broader range of solid tumors. At present, the use of oncolytic viruses is limited by their intratumoral administration.

BT-001: Ongoing Phase I trial — Monotherapy data expected in H1 2023

Transgene and BioInvent intend to communicate data from the Part A (monotherapy) of the ongoing Phase I trial in H1 2023. The Part B of the Phase I trial (in combination with pembrolizumab) is expected to start in the second half of 2023. KEYTRUDA (pembrolizumab) will be provided to the trial by MSD (Merck & Co).

TG6050: First patient treated with novel Invir.IO candidate designed to express IL-12 and be administered intravenously

In early 2023, Transgene announced the regulatory approval to initiate a clinical trial of TG6050, a novel oncolytic virus from its Invir.IO platform. This innovative candidate has been designed to express human IL-12, a cytokine known to trigger a potent antitumor immune response, and an anti-CTLA4 antibody.

The Delivir trial is evaluating TG6050 in patients with advanced non-small cell lung cancer who have failed standard therapeutic options. The first patient has been dosed. Completion of the trial is expected in H2 2024.

AstraZeneca collaboration update

As previously announced on 5 May 2023, the Company was informed by AstraZeneca of its decision to terminate its oncolytic virus research and development collaboration with Transgene that was signed in 2019. The decision was made by AstraZeneca following a strategic review of its pipeline. Following termination, Transgene will regain the global rights to the oncolytic virus drug candidate that was in-licensed by AstraZeneca in December 2021. This intravenous drug candidate has been granted a US IND.

Operating revenue

Q1

In millions of euros

2023

2022

Revenue from collaborative and licensing agreements

0.1

0.4

Government financing for research expenditures

1.5

1.7

Other income

–

0.1

Operating revenue

1.6

2.2

During the first quarter of 2023, revenue from collaborative and licensing agreements was mainly composed of revenue from the collaboration with AstraZeneca.

As of March 31, 2023, government financing for research expenditures mainly consisted of accrual of 25% of the research tax credit expected for 2023 (€1.5 million in the first quarter of 2023 compared to €1.7 million for the same period in 2022).

Cash, cash equivalents and other financial assets

Cash, cash equivalents and other financial assets stood at €17.0 million as of March 31, 2023, compared to €26.8 million as of December 31, 2022. In the first quarter of 2023, Transgene’s net cash burn was €9.8 million, compared to €2.8 million for the same period in 2022. In Q1 2022, cash burn was reduced due to an $8 million payment from AstraZeneca following the exercise of a license option for an oncolytic virus developed by Transgene.

The Company holds shares of Tasly BioPharmaceuticals valued at €14.3 million at the end of December 2022. The Company is expecting to sell its shareholding in Tasly BioPharmaceuticals in mid-2023.

As a result, the Company confirms its financial visibility until early 2024.

New leadership structure appointed to accelerate the development of Transgene’s innovative immunotherapy portfolio

On May 5, 2023, Transgene announced its Board of Directors’ decision to appoint Dr. Alessandro Riva, MD, as the Company’s new Chairman and CEO to accelerate the development of Transgene’s innovative immunotherapy portfolio. Alessandro Riva has been the Chairman of the Company’s Board of Directors since May 2022. Dr. Riva has an outstanding track record in the pharmaceutical and biotechnology industry, leading to the approval of innovative oncology treatments in the US and in Europe. Dr. Riva’s appointment will be effective on June 1st, 2023. Hedi Ben Brahim will retire from the CEO position and will stay n as a strategic advisor until the transition is complete.

In addition, on May 5, 2023, the Combined General Meeting adopted all resolutions recommended by the Board of Directors, including the appointment of Ms. Carol Stuckley, MBA, as an independent Director of the Company. Ms. Carol Stuckley brings more than 35 years of experience as a strategic and international financial executive, with proven success leading finance teams and creating shareholder value for healthcare companies.

In March 2023, Transgene appointed Dr. John C. Bell and Dr. Pedro Romero as key scientific advisors. These key opinion leaders in cancer immunotherapy bring considerable expertise to Transgene.

On May 10, 2023 Avid Bioservices, Inc. (NASDAQ:CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that the company will participate in the RBC Capital Markets Global Healthcare Conference (Press release, Avid Bioservices, MAY 10, 2023, View Source [SID1234631411]). Nick Green, president and chief executive officer, will be the featured speaker in a fireside chat at the conference, which will take place May 16-17, 2023.

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Details of the company’s participation are as follows:

RBC Capital Markets Global Healthcare Conference
Conference Date: May 16-17, 2023
Fireside Chat Time/Date: 8:30 – 8:55 a.m. Eastern on Wednesday, May 17, 2023