1stOncology™: Cancer Intelligence Service – Page 10863 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Inventiva announces the appointment of Dr. Michael Cooreman as Chief Medical Officer

On November 5, 2020 Inventiva (Euronext Paris and Nasdaq: IVA), a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS) and other diseases with significant unmet medical need, reported the appointment of Dr. Michael Cooreman, M.D., as Chief Medical Officer (CMO) (Press release, Inventiva Pharma, NOV 5, 2020, View Source [SID1234570081]). He joins Inventiva’s Executive Committee and succeeds Dr. Marie-Paule Richard, M.D., who has decided to take her retirement as of December 17, 2020.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

Schedule Your 30 min Free Demo!

Dr. Michael Cooreman will oversee the Company’s medical and clinical activities and lead its medical development team, in particular with a view to the planned pivotal Phase III clinical trial evaluating Inventiva’s lead drug candidate lanifibranor for the treatment of NASH. Joining Dr. David Nikodem, Ph.D., Vice President of U.S. Operations, he will drive the establishment of Inventiva’s clinical team in the U.S. and manage the Company’s relationships with Key Opinion Leaders (KOL).

Dr. Cooreman joins Inventiva from Ferring Pharmaceuticals where he has been Vice President, Science and Medicine, in charge of Global Research and Development, Gastroenterology and Hepatology, since 2017. He will bring to Inventiva his extensive experience in translational medicine, clinical pharmacology and clinical product development, especially in the areas of liver diseases, including NASH, cirrhosis and viral hepatitis, metabolic and immune-mediated diseases and oncology. Over his career, Dr. Cooreman has worked for several companies in the United States of America (US) and been involved in numerous clinical trials. He has successfully led clinical and project teams developing a variety of investigational compounds, including small molecules and biologics, several of which have been approved by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and other health authorities. His strong industry knowledge, established network and international profile, together with his deep expertise as an academic internist and gastroenterologist-hepatologist, will be important assets for Inventiva’s future development.

Dr. Cooreman’s arrival will facilitate a smooth transition with Dr. Marie-Paule Richard ahead of the Company’s next key milestones, including NASH-related meetings with the FDA and the EMA, planned for the fourth quarter of 2020, as well as the initiation of the pivotal Phase III clinical trial evaluating lanifibranor in patients with NASH, planned for the first half of 2021.

Frédéric Cren, Chairman, CEO and cofounder of Inventiva, commented: "I am delighted to welcome Michael in the role of CMO at this exciting stage of Inventiva’s development. His experience as both health practitioner and senior executive in leading pharmaceutical and biotech companies will be key to the deployment of our strategy going forward. As such, he will play a crucial role in the development of Inventiva in the US, especially with regards to the upcoming planned Phase III clinical trial of lanifibranor in NASH. I would like to take this opportunity to warmly thank Marie-Paule for her dedication, remarkable contribution and professionalism over the last two years and I wish her all the best in the future."

Biography – Dr. Michael Cooreman, M.D.

Prior to joining Inventiva, Dr. Michael Cooreman has been Vice President, Science and Medicine, in charge of Global Research and Development, Gastroenterology and Hepatology, at Ferring Pharmaceuticals in the U.S. since 2017. He previously held numerous U.S.-based positions as CMO and Executive Director in global roles at leading pharmaceutical and biotechnology companies, including Takeda Pharmaceuticals, Merck, Mitsubishi Tanabe, ImmusanT and Novartis, covering the four major regulatory regions U.S., EU, Japan and China. Over the years, Dr. Cooreman has developed a strong expertise in translational medicine, clinical pharmacology and clinical product development.

Of dual US and Belgian citizenship, Dr. Cooreman is trained as an internist and gastroenterologist-hepatologist, with a special interest in metabolic and immune-mediated liver and gastrointestinal diseases, as well as viral hepatitis, cirrhosis and oncology.

He holds a Doctor of Medicine degree from the University of Louvain, Belgium, and a doctorate from the Heinrich Heine University in Dusseldorf, Germany.

Bellicum Reports Third Quarter 2020 Financial Results and Provides Operational Update

On November 5, 2020 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the third quarter 2020 and provided an operational update (Press release, Bellicum Pharmaceuticals, NOV 5, 2020, View Source [SID1234570080]).

"Bellicum recently took significant steps to strengthen our balance sheet, restructure, and focus on our clinical development programs: BPX-601 in mCRPC and BPX-603 in HER2+ solid tumors," said Rick Fair, President and Chief Executive Officer of Bellicum. "We believe we are well positioned to explore the value of GoCAR in these two different solid tumor settings."

Program Highlights and Current Updates

BPX-601 GoCAR-T

In October, Bellicum announced interim data from its BPX-601 Cohort 5C dose-escalation clinical trial in patients with relapsed/refractory metastatic pancreatic cancer. Consistent with previously reported findings of BPX-601 followed by single-dose rimiducid, BPX-601 followed by repeat rimiducid dosing was tolerated and exhibited evidence of rimiducid-mediated CAR-T cell activation in the first four patients treated. Clinically meaningful efficacy as measured by RECIST criteria was not observed.

Bellicum plans to explore the effects of BPX-601 in new tumor types and expects to begin enrolling patients with metastatic castration-resistant prostate cancer (mCRPC) in the ongoing Phase 1/2 clinical trial before the end of the year. The company intends to review its plans in pancreatic cancer upon completion of the current safety cohort.
BPX-603 GoCAR-T

BPX-603 is the company’s first dual-switch GoCAR-T product candidate, which incorporates Bellicum’s iMC activation and CaspaCIDe safety switch technologies. The company plans to initiate enrollment in a Phase 1/2 basket trial in solid tumors that express HER2 later this year.
Corporate Updates

In November, the company completed a financing consisting of 1,040,000 shares of common stock, pre-funded warrants to purchase 3,109,378 shares of common stock and accompanying warrants to purchase 4,149,378 shares of common stock, that raised gross proceeds of approximately $25.0 million, before deducting underwriting discounts and commissions and other offering expenses payable by Bellicum, and excluding any proceeds that may be received upon exercise of the warrants.
In October, Bellicum implemented a restructuring program to focus on the clinical development of BPX-601 and BPX-603, pause the BCMA GoCAR-NK program, and discontinue discovery research and new product development. The company plans to reduce headcount by 79%, from 68 to 14 full-time employees by the end of 2020, which is expected to reduce operating expenses and extend its cash runway.
In October, Bellicum paid down all of its Oxford Finance debt obligations with payment of $27.4 million in principal plus applicable fees and accrued interest.
Third Quarter 2020 Financial Results

R&D Expenses: Research and development (R&D) expenses were $8.1 million and $30.3 million for the three and nine months ended September 30, 2020, respectively, compared to $14.3 million and $51.2 million for the three and nine months ended September 30, 2019, respectively. The reduction in expenses in the third quarter resulted primarily from reduced expenses related to reduced rivo-cel related activities, reduced expenses resulting from the manufacturing facility sale and the reduction in force implemented during the second half of 2019, partially offset by an increase in expenses related to the GoCAR programs.

G&A Expenses: General and administrative (G&A) expenses were $4.2 million and $12.1 million for the three and nine months ended September 30, 2020, respectively, compared to $9.2 million and $24.3 million for the three and nine months ended September 30, 2019, respectively. The reduction in expenses during the third quarter relative to the comparable period in 2019 was primarily due to the reduction in rivo-cel related commercialization activities as well as the effects of the reduction in force that reduced employee-related charges.

Loss from Operations: Bellicum reported a loss from operations of $12.3 million and $38.7 million for the three and nine months ended September 30, 2020, respectively, compared to a loss from operations of $23.4 million and $73.5 million for the three and nine months ended September 30, 2019, respectively. The results for the nine months ended September 30, 2020 included a net gain on dispositions of $3.8 million due to the manufacturing facility sale. Cash used in operating activities was $43.3 million for the nine months ended September 30, 2020, compared to cash used in operating activities of $64.8 million for the nine months ended September 30, 2019.

Net Loss: Bellicum reported net loss of $0.9 million and $26.5 million for the three and nine months ended September 30, 2020, respectively, compared to a net loss of $32.0 million and $83.5 million for the three and nine months ended September 30, 2019, respectively. The results included a non-cash gain of $12.1 million and $14.3 million related to the change in fair value of warrant and private placement option liability for the three and nine months ended September 30, 2020, respectively.

Shares Outstanding: As of October 30, 2020, Bellicum had 5,059,779 shares of common stock and 534,000 shares of preferred stock outstanding. Each share of preferred stock can be converted into 10 shares of common stock. In the November 2020 financing, the company issued 1,040,000 shares of common stock and pre-funded warrants to purchase 3,109,378 shares of common stock.

Cash Position and Guidance: Bellicum reported cash and cash equivalents and restricted cash totaling $54.6 million as of September 30, 2020, compared to $93.8 million as of December 31, 2019. On a pro forma as adjusted basis to give effect to the net proceeds from the financing and the Oxford Finance payoff, cash and cash equivalents and restricted cash totaled $49.9 million as of September 30, 2020. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second quarter of 2022.

ERYTECH Provides Business Update and Reports Financial Results for the Third Quarter of 2020

On November 5, 2020 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported a business and financial update (Press release, ERYtech Pharma, NOV 5, 2020, View Source [SID1234570079]).

TRYbeCA-1 Phase 3 trial in second-line metastatic pancreatic cancer
NOPHO-sponsored Phase 2 trial in acute lymphoblastic leukemia
Appointment of Dr. Stewart Craig as Chief Technical Officer
Cash and cash equivalents of €40.5 million ($47.5 million) at the end of September 2020
€10 million ($11.7 million) in non-dilutive financing secured in the form of state-guaranteed loans

Molecular Templates, Inc. Reports Third Quarter 2020 Financial Results and Provides a Corporate Update

On November 5, 2020 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates," "MTEM" or "the Company"), a clinical-stage biopharmaceutical company focused on the discovery and development of proprietary targeted biologic therapeutics, engineered toxin bodies (ETBs), reported financial results for the third quarter of 2020 and an update on its clinical pipeline (Press release, Molecular Templates, NOV 5, 2020, View Source [SID1234570078]).

On November 4, 2020, the U.S. Food and Drug Administration (FDA) notified MTEM that MT-3724 clinical studies have been placed on partial clinical hold following a treatment-related fatality in one subject who experienced Grade 5 capillary leak syndrome (CLS) in the Phase 2 MT-3724 monotherapy study. This subject and four others in the Phase 2 monotherapy study who were treated with material from the same MT-3724 product lot had markedly higher than expected peak drug exposure (Cmax). Subjects already enrolled in MT-3724 clinical studies will continue to be dosed but no new patients will be enrolled until the partial hold is removed. There are no changes to the trials or plans for other ETB product candidates, including MT-5111, TAK-169, and MT-6402, all of which utilize a next-generation ETB scaffold that has been designed to reduce or eliminate the propensity for innate immunity, including CLS.

"We are saddened to report the patient death in our MT-3724 monotherapy study. We are committed to working with the FDA to fulfill their information requests, resolve the partial clinical hold, and to resume enrollment of the affected MT-3724 clinical trials as quickly as possible," said Eric Poma, Ph.D., Molecular Templates’ Chief Executive and Chief Scientific Officer. "In the meantime, our other ETB programs, which are built on our proprietary, next generation de-immunized toxin scaffold with a simplified manufacturing process, are continuing as planned with study updates expected in late 2020 and into 2021."

Company Highlights, Pipeline Status, and Upcoming Milestones

MT-3724 (CD20 ETB)

The FDA has placed MT-3724 clinical studies on partial clinical hold pending further review of a treatment-related fatality in a single subject in the Phase 2 monotherapy study. Additional information on this subject is detailed below. The FDA has requested additional information around the event, a safety assessment of all data relevant to CLS, as well as additional information assessing attributes of the MT-3724 product lot in question that could have contributed to the high PK values observed.
The subject that experienced the Grade 5 CLS is one of five subjects in the monotherapy study with elevated pharmacokinetic (PK) findings, all of whom were treated with the same lot of MT-3724. An investigation is underway to determine the cause of the elevated PK in these subjects.
Until the partial clinical hold is removed, no new subjects will be enrolled in any MT-3724 study but patients currently receiving treatment and who are receiving clinical benefit may continue to be dosed.
As previously reported, in both Phase 2 combination studies, responses have been observed at MT-3724 doses (10 and 25 µg/kg) considerably lower than the dose used in the Phase 2 monotherapy study (50 µg/kg).
MTEM will provide an update on the status of the MT-3724 partial clinical hold and future MT-3724 development plans as soon as possible.
TAK-169 (CD38 ETB)

Takeda and MTEM are conducting an ongoing Phase 1 study evaluating TAK-169 in relapsed/refractory multiple myeloma. This study, which had started dosing in February, had a temporary pause in the activation of new study sites and new patient enrollment (along with most of Takeda’s other early stage studies) due to COVID-19 and was recently re-initiated.
Preclinical data on TAK-169 will be presented at the 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition being held virtually on December 5-8, 2020.
MT-5111 (HER2 ETB)

MTEM is conducting a Phase 1 study of MT-5111 in relapsed/refractory HER2-positive cancers.
Further to the interim update provided in June 2020, MTEM expects to provide an update on results from the dose escalation portion of the Phase 1 study in 4Q20.
Research

MTEM expects to file an investigational new drug (IND) application in 4Q20 for MT-6402, its ETB targeting PD-L1 (with antigen seeding). A Phase 1 study in relapsed/refractory patients with PD-L1 expressing tumors is expected to be initiated in 1H21.
MTEM expects to file an IND application for its ETB targeting CTLA-4 in 2021.
COVID-19 Impact

The COVID-19 pandemic has resulted in a significant slowdown in the pace of site initiations and patient enrollment across our MT-3724 Phase 2 programs. As with other sponsors with studies in patients with hematologic malignancies, we are working with sites to determine when a patient is suitable for each research study and to ensure the continued safety of all research participants.
To date, screening and enrollment for the MT-5111 Phase 1 study has been less adversely affected than the MT-3724 studies but it is enrolling at a slower pace than was projected pre-COVID-19.
To date, MTEM has continued to operate its cGMP manufacturing facility and laboratories without interruption from COVID-19. As a result, manufacturing of product supply for clinical trials and research activities to support advancement of our preclinical pipeline (including partnered programs) have not been adversely affected by COVID-19 to date.
Details on MT-3724 Update

On November 4, 2020, the FDA notified MTEM that MT-3724 clinical studies have been placed on partial clinical hold following a fatality in one subject in the Phase 2 monotherapy study due to treatment-related capillary leak syndrome (CLS) on October 20, 2020. The fatality occurred in a diffuse large B-cell lymphoma (DLBCL) subject who had been treated with six prior lines of therapy including rapid progression through three lines of therapy in the six months prior to MT-3724 dosing (including most recently a first generation anti-CD19 CAR T-cell). The subject had transformed DLBCL from Waldenstrom’s Macroglobulinemia and came onto the MT-3724 study with a CD4/CD8 T-cell ratio of 0.47. The subject did not have a radiographic assessment of response but an elevated LDH was thought by the principal investigator to represent disease progression. The subject initially had Grade 2 CLS following treatment with MT-3724, recovered after a dosing interruption, resumed dosing and then had CLS that was ultimately fatal. While Grade 1 and 2 CLS is an expected potential adverse reaction of MT-3724, this was the only subject in any MT-3724 study to date with CLS that was more severe than Grade 2.

In addition, markedly higher than expected peak drug exposure (Cmax) was observed in five of the last six subjects enrolled in the monotherapy study, including the subject with the fatal CLS. All of these subjects had been treated with drug product from the same lot of MT-3724 and while the other four subjects with higher than predicted exposure exhibited signs or symptoms of innate immunity, none experienced any unexpected serious adverse events. This lot of drug product met all specifications for drug product release as well as its ongoing stability testing specifications. MTEM is investigating the higher than expected drug exposure findings to determine if this was caused by an issue with this specific lot of MT-3724.

MTEM is working to address the clinical and MT-3724 product lot information requests from the FDA and will then seek agreement from FDA to remove the partial clinical hold. In the meantime, no new patients will be enrolled in any MT-3724 study. MTEM’s trials and plans for its other ETB product candidates, including MT-5111, TAK-169, and MT-6402, which utilize next-generation ETB technology, are not affected. Next-generation ETB scaffolds have been designed to reduce or eliminate the propensity for innate immunity or CLS; no cases of CLS have been observed in human subjects who have been dosed with any next-generation ETBs.

Financial Results

The net loss attributable to common shareholders for the third quarter of 2020 was $23.2 million, or $0.47 per basic and diluted share. This compares with a net loss attributable to common shareholders of $38.2 million, or $1.03 per basic and diluted share, for the same period in 2019.

Revenues for the third quarter of 2020 were $4.3 million, compared to $3.6 million for the same period in 2019. Revenues for the third quarter of 2020 were comprised of revenues from collaborative research and development agreements with Takeda and Vertex. Total research and development expenses for the third quarter of 2020 were $19.6 million, compared with $15.2 million for the same period in 2019. Total general and administrative expenses for the third quarter of 2020 were $7.5 million, compared with $4.5 million for the same period in 2019.

As of September 30, 2020, MTEM’s cash and investments totaled $118.2 million, which is expected to fund operations into 2H22.

Aprea Therapeutics Reports Third Quarter 2020 Financial Results and Provides Update on Business Operations

On November 5, 2020 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, reported financial results for the three and nine months ended September 30, 2020 and provided a business update (Press release, Aprea, NOV 5, 2020, View Source [SID1234570077]).

"We made solid progress during the third quarter across our development pipeline as we approach top-line data from the Phase 3 clinical trial evaluating eprenetapopt with azacitidine for the treatment of front-line TP53 mutant myelodysplastic syndromes (MDS) patients by year-end 2020" said Christian S. Schade, Chairman and Chief Executive Officer of Aprea. "We continue to execute on our goal of expanding the clinical opportunities for our p53 reactivator programs, including expansion of our front-line AML clinical trials, enrollment of the first patients in our solid tumor trial, initiation of our lymphoma trial and clearance from FDA to proceed with the Phase 1 trial of our next-generation p53 reactivator, APR-548."

Business Operations Update:

The Company is conducting, supporting, and planning multiple clinical trials of eprenetapopt (APR-246):

Pivotal Phase 3 MDS Trial—The Company has completed the full enrollment of 154 patients in its pivotal Phase 3 randomized, controlled trial evaluating eprenetapopt with azacitidine as frontline therapy in HMA-naïve TP53 mutant myelodysplastic syndromes (MDS) patients with a primary endpoint of complete remission (CR) rate. The Company remains confident it will have top-line data available by year-end 2020.

Phase 2 MDS/AML Post-Transplant Trial—The Company has completed the target enrollment of 31 patients in its single-arm, open-label Phase 2 trial evaluating eprenetapopt with azacitidine as post-transplant maintenance therapy in TP53 mutant MDS and acute myeloid leukemia (AML) patients who have received an allogeneic stem cell transplant.

Phase 1/2 AML Trial—The Company is currently enrolling its Phase 1/2 trial evaluating the safety, tolerability, and preliminary efficacy of eprenetapopt therapy in TP53 mutant AML patients. The lead-in portion of the trial evaluated the tolerability of eprenetapopt with venetoclax, with or without azacitidine, and no dose-limiting toxicities were observed in 12 patients receiving either regimen. Based on these results, the Company has expanded the trial to treat approximately 30 additional frontline TP53 mutant AML patients with the combination of eprenetapopt, venetoclax and azacitidine. The Company also plans to activate a separate cohort in the trial to evaluate the combination of eprenetapopt with azacitidine in approximately 30 frontline TP53 mutant AML patients.

Phase 1 NHL Trial—The Company has designed and plans to conduct a Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab, and eprenetapopt with ibrutinib in order to further assess eprenetapopt in hematological malignancies. A poster describing the clinical trial has been accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (abstract # 1311) on December 5, 2020 from 10:00 am to 6:30 pm eastern time. The Company is targeting the first patient to be enrolled in the fourth quarter of 2020.

Phase 1/2 Solid Tumor Trial—Based on in vivo data suggesting synergistic activity between eprenetapopt and immuno-therapy agents including anti-PD-1 antibody, the Company has designed and is conducting Phase 1/2 clinical trials in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy. Five patients have been enrolled in the safety review cohort of this trial.

APR-548 — The Company’s second product candidate, APR-548, is a pre-clinical, next-generation p53 reactivator with the potential for oral administration. APR-548 exhibits high oral bioavailability in preclinical testing and is being developed in an oral dosage form. The Company completed Investigational New Drug, or IND, enabling preclinical studies of APR-548 and filed an IND with the FDA. The Company received clearance from the FDA in October 2020 to initiate Phase 1 clinical trials for APR-548. The Company anticipates enrollment in the Phase 1 clinical trial to begin in the first quarter of 2021.
Third Quarter Financial Results

Cash and cash equivalents: As of September 30, 2020, the Company had $101.1 million of cash and cash equivalents compared to $130.1 million of cash and cash equivalents as of December 31, 2019. The Company expects cash burn for the full year 2020 to be between $35.0 million $40.0 million. The Company believes its cash and cash equivalents as of September 30, 2020 will be sufficient to meet its current projected operating requirements into 2023.

Research and Development (R&D) expenses: R&D expenses were $8.8 million for the quarter ended September 30, 2020, compared to $4.9 million for the comparable period in 2019. The increase in R&D expenses was primarily related to the continued development of the Company’s lead product candidate, eprenetapopt in the following ongoing clinical trials; our pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for frontline treatment of TP53 mutant MDS, our Phase 1/2 clinical trials in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy, our Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab, and eprenetapopt with ibrutinib and our Phase 2 post-transplant MDS/AML clinical trial.

General and Administrative (G&A) expenses: G&A expenses were $3.5 million for the quarter ended September 30, 2020, compared to $2.3 million for the comparable period in 2019. The increase in G&A expenses was primarily due to increased non-cash stock-based compensation, increased insurance expense and increased commercial development expense.

Net loss: Net loss was $12.3 million, or $0.58 per share for the quarter ended September 30, 2020, compared to a net loss of $6.2 million, or $5.29 per share for the quarter ended September 30, 2019. The Company had 21,186,827 shares of common stock outstanding as of September 30, 2020.