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Inventiva Reports First-Half of 2021 Financial Information

On July 28, 2021 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 its cash position as of June 30, 2021 and its revenues for the first half of 20211 (Press release, Inventiva Pharma, JUL 28, 2021, View Source [SID1234585338]).

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Cash Position

As of June 30, 2021, Inventiva’s cash and cash equivalents stood at €93.6 million compared to €107.8 million as of March 31, 2021 and €113.02 million as of December 31, 2020.

Net cash used in operating activities amounted to €19.8 million in the first half of 2021 compared to €7.2 million for the same period in 2020. R&D expenses for the first half of 2021 were up 56% compared to the first half of 2020, mainly driven by the development of lanifibranor in NASH and costs linked to the preparation for the initiation of NATiV3, the Company’s Phase III clinical trial evaluating lanifibranor in NASH. This amount includes the payment received on June 30, 2021 of €8.0 million of Research Tax Credit (CIR – Crédit Impôt Recherche) (€3.8 million related to complementary filings following the 2020 Conseil d’État judgement covering prior years, and €4.2 million related to the 2020 Research Tax Credit), while the first half of 2020 had been positively impacted by the receipt of a €4.2 million non-recurrent late payment of the 2018 Research Tax Credit and a €4.2 million payment in respect of the 2019 Research Tax Credit.

Net cash used from investing activities amounted to €1.2 million in the first half of 2021 compared to 1.0 million for the same period in 2020.

No net cash from financing activities was generated over the first half of 2021 while Inventiva recorded
€24.6 million of net cash from financing activities for the same period in 2020, notably related to the issuance of €15 million (gross proceeds) of ordinary shares in February 2020 and the entry into a €10.0 million State-guaranteed loan, with a syndicate of French banks in May 2020.

Over the first semester of 2021, the Company recorded a positive exchange rate effect on cash and cash equivalent of €1.2 million.

Considering its current R&D and clinical development programs, and excluding additional financial resources, Inventiva’s cash runway will allow to fund its operating activities through the third quarter of 2022.

Revenues

The Company’s revenues for the first half of 2021 amounted to €0.2 million, as compared to €0.1 million for the same period in 2020.

As part of its collaboration with Abbvie in auto-immune diseases3, Inventiva is eligible to receive development, regulatory and commercial milestone payments as well as royalty payments. As such, the Company expects to receive another milestone payment upon the initiation by AbbVie of the Phase IIb clinical trial with cedirogant before the end of 2021.

Next key milestones expected

First patient first visit of NATiV3 Phase III clinical trial evaluating lanifibranor in NASH – planned for the third quarter of 2021
Initiation by AbbVie of a Phase IIb clinical trial with cedirogant – expected in the second half of 2021
Strategy update on the development of odiparcil – planned for the second half of 2021
Publication of the results of the Phase II clinical trial evaluating lanifibranor for the treatment of Non-Alcoholic Fatty Liver Disease (NAFLD) in patients with type 2 diabetes (T2DM) – planned for the first half of 2022
Upcoming investor conference participation

Citi’s 16th Annual BioPharma Conference 2021, September 8-9, 2021
KBC Virtual Life Science Conference, September 7-9, 2021
H.C. Wainwright 23rd Annual Global Investment Conference, September 13-15, 2021
Lyon Pôle Bourse Forum, September 27-28, 2021
HealthTech Innovation Days 2021, October 4-5, 2021
Portzamparc Health Biotech Seminar 2021, October 6, 2021
Guggenheim CV&M Disease Day, October 7, 2021
H.C. Wainwright 5th Annual NASH Investor Conference, October 11, 2021
Stifel Healthcare Conference 2021, November 16-17, 2021
Jefferies 2021 London Healthcare Conference, November 16-18, 2021
Upcoming scientific conference participation

Paris NASH Meeting, October 22-23, 2021
AASLD The Liver Meeting, November 12-15, 2021
Next financial results publication

H1 2021 Financial results: Monday, September 20, 2021 (after U.S. market close)

Abeona Therapeutics Reports Second Quarter Financial Results

On July 28, 2021 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported financial results for the second quarter 2021 and recent business progress (Press release, Abeona Therapeutics, JUL 28, 2021, View Source [SID1234585337]).

"The second quarter was a highly effective and productive quarter for Abeona, and we remain committed to delivering operational excellence and bringing our gene therapies to patients with no approved treatments," said Michael Amoroso, Chief Executive Officer of Abeona. "As we continue to advance our clinical programs, we are delivering on meaningful milestones. We are focusing our resources on completing registration-enabling studies for patients with RDEB and MPS IIIA, and are preparing for the potential of two BLA filings."

Second Quarter and Recent Highlights

Corporate Updates

Appointed Vishwas Seshadri, Ph.D., M.B.A., as Senior Vice President, Head of Research & Clinical Development. Dr. Seshadri joins Abeona from Celgene Corporation, now a subsidiary of Bristol-Myers Squibb Company, and brings substantial experience in the life sciences industry overseeing product development, regulatory submissions, and commercialization for novel therapies including personalized, autologous cell therapies.
EB-101 (Autologous, Gene-Corrected Cell Therapy)

Activated UMass Memorial Medical Center in Worcester, MA as the second clinical trial site in the pivotal Phase 3 VIITAL study of its investigational EB-101 treatment for recessive dystrophic epidermolysis bullosa (RDEB).
Presented updated Phase 1/2a clinical trial results at the Society for Pediatric Dermatology (SPD) 46th Annual Meeting, with EB-101 treatment of large, chronic RDEB wounds continuing to show a considerable reduction in both wound burden and associated long-term pain for up to six years.
ABO-102 and ABO-101 (AAV-based Gene Therapies)

Abeona completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal trial to support filing and approval for ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). Based on the Type B meeting, the ongoing Transpher A study will serve as the pivotal study for ABO-102 and could potentially support a Biologics License Application (BLA) submission depending on the data set. In addition, Abeona also aligned with the FDA on the definition of the primary endpoint for the study, neurocognitive assessment using the raw score from the Bayley Scales of Infant and Toddler Development (BSITD) and the Kauffman Assessment Battery for Children (KABC-2), which are already part of the assessment plan in the Transpher A protocol.
Presented new brain MRI data during an oral presentation at the 16th International Symposium on MPS and Related Diseases, held during July 23-25, 2021. The MRI data indicated that ABO-102 increased grey matter, corpus callosum, and amygdala volumes in the brain in the three young patients with MPS IIIA at 24 months as compared to afflicted patients without treatment. The MRI data is consistent with previously reported results of preservation of neurocognitive development in these three young patients in the Transpher A study.
The Company is in the process of closing enrollment for the Transpher B trial. To date, four patients have been treated in the higher dose cohort of Transpher B. An additional three patients have been treated and a fourth patient will be treated with the higher dose through the Named Patient Program (NPP) in Germany, a compassionate use program that allows for patients with high unmet need to be treated at the request of the treating physician. The patients treated in the NPP are followed for safety and efficacy with the same rigor and frequency as patients in Transpher B. The Company intends to pool all patients’ data from Transpher B and the NPP to assess therapeutic effect going forward. The Company expects 2-year neurocognitive data for the first patients treated in the high dose cohort of Transpher B beginning in the first half of 2022.
Preclinical Pipeline

Presented new data at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting supporting the potential of Cre-mediated dual AAV vector technology to enable delivery of large genes targeted for treatment of Stargardt disease.
Completed non-human primate (NHP) studies comparing several of the company’s AAV capsids with AAV8, the industry standard for intraocular administration. The results showed that AAV204, part of Abeona’s in-licensed AIM capsid library, was superior to AAV8 using a recently developed route of ocular administration. In a separate NHP experiment, the company’s AAV214 and AAV214D5 capsids demonstrated nearly identical levels of transduction compared with AAV8 of photoreceptor and retinal pigmented epithelium cells, which are the cell types most frequently affected in inherited retinal diseases. The NHP results support Abeona’s strategy to advance multiple preclinical eye programs onward toward the clinic.
Second Quarter Financial Results

Cash, cash equivalents and short-term investments totaled $77.6 million as of June 30, 2021, compared to $86.8 million as of March 31, 2021. Net cash used in operating activities was $11.5 million for the second quarter of 2021.

"While pursuing our key strategic priorities, we have thoughtfully and carefully managed our spending decisions. Across the organization, there is a balanced approach to not only focusing on moving towards our key milestones, but also utilizing our cash resources prudently and on time to deliver results," said Edward Carr, Chief Accounting Officer of Abeona.

Total research and development (R&D) spending was $7.4 million for the second quarter of 2021, which is consistent with the $7.2 million spent in the first quarter of 2021. R&D expenses include the cost of clinical development of the EB-101 and MPS programs, manufacturing of the drug products for EB-101 and ABO-102, and preclinical ophthalmic research activities. Total general and administrative (G&A) spending was $5.5 million in the second quarter of 2021, down from the $6.6 million spent in the first quarter of 2021. The decrease in G&A in the second quarter of 2021 is primarily due to lower professional fees. Net loss was $15.2 million for the second quarter of 2021.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast on Thursday, July 29, 2021 at 8:30 a.m. ET, to discuss its second quarter 2021 financial results and business update. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 7184522 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

Prescient takes its PTX-100 trial to next level after Phase 1b success

On July 28, 2021 Prescient Therapeutic’s (ASX:PTX) reported that anti-cancer drug PTX-100, the Company stated that the first-in-class compound demonstrated an excellent safety profile in Phase 1b clinical trial in solid and hematological cancers (Press release, Prescient Therapeutics, JUL 28, 2021, View Source;utm_medium=rss&utm_campaign=prescient-takes-its-ptx-100-trial-to-next-level-after-phase-1b-success [SID1234585336]).

Prescient also highlighted that the targeted therapy demonstrated biological activity in two highly pre-treated T cell lymphoma (TCL) patients with aggressive disease. TCL is a type of cancer that forms in T lymphocytes (or T cells).

MUST READ: Positive results from immunogenicity testing send Prescient’s shares higher

PTX highlighted that PTX-100 demonstrated an excellent safety profile for two reasons: firstly, the drug may have efficiency in fragile patients who cannot tolerate treatments with high toxicities, and secondly, because of the low toxicity profile of PTX-100.

Prescient now intends to advance PTX-100 to an expansion cohort study that will focus on TCL, an area of significant unmet medical need.

Patients’ enrolment for Phase 1b trial
A total of ten patients were enrolled for the Phase 1b basket trial-

Five with solid tumours, including pancreatic and colorectal cancers.
Five with haematological malignancies, including multiple myeloma and T cell lymphomas.
In this trial, patients received a median of three prior lines of treatment and up to five prior lines of treatment. PTX-100 was administered to the patients at doses ranging from 500 mg/m2 to 2,000 mg/m2.

PTX-100’s impressive safety profile
In the Phase 1b basket trial, PTX-100 showed an outstanding safety profile and was well tolerated up to and including the highest dose of 2,000mg/m2.

PTX also notified that several Grade 3 or 4 adverse events were observed in the study, including a reduction in platelets (observed in three patients) as well as a reduction in neutrophils or neutropenia (observed in two patients). However, none of these were deemed serious and were not related to PTX-100.

Serious adverse events included nose bleeds (one patient); osteomyelitis (bone infection; one patient); hip fracture (one patient), and subarachnoid haemorrhage (one patient). However, none of these adverse events were found to be related to PTX-100.

PTX-100 demonstrates clinical benefit in Phase 1 trial
Although the primary goal of the basket trial was to evaluate safety, clinical benefit was observed in two TCL patients with aggressive disease who had failed 3-5 prior therapies.

One patient with peripheral T cell lymphoma (PTCL) had an aggressive form of the disease and had failed five prior treatments. These treatments weren’t able to control the disease for more than a couple of months. However, when treated with PTX-100, the patient experienced a partial response (reduction in cancer burden) without disease progression for 17 months and still counting. The patient has undergone 24 cycles of therapy and continues to receive PTX-100.

Another patient with cutaneous T cell lymphoma (CTCL) with K-Ras mutation also had aggressive cancer and had failed three prior therapies. This patient had a partial response to the study, with reduced cancerous lesions and symptomatic relief. The patient was on therapy for 12 months, receiving 19 cycles of therapy.

In cases where refractory TCL patients are treated with standard of care therapies, disease progression is generally expected within four months. This highlights the reassuring nature of responses when treated with PTX-100.

DO READ: Prescient Therapeutics inks new deal with Peter Mac to rev up its OmniCAR programs

PTX to conduct expansion cohort study for PTX-100
Following the encouraging results, Prescient will progress the development of PTX-100 as a monotherapy in an expansion cohort study in relapsed and refractory TCL, with a particular focus on peripheral T cell lymphoma (PTCL).

WATCH NOW: Expert Talks With Mr Steven Yatomi Clarke, CEO and Managing Director of Prescient Therapeutics

Relatively small clinical trials may be adequate for registration compared to typical Phase 3 clinical trials, as indicated by previous drug approvals. PTCL is an indication with substantial unmet medical need. The survival rate following relapse is low and has not significantly improved in the last two decades.

While PTCL is not a common malignancy, the nature of this form of cancer and the absence of efficient therapy options for refractory patients generates a potentially shorter regulatory path for PTX-100. Notably, this will be the fastest route to market in a high-value area of unmet medical need.

Prescient will provide additional details on the expansion cohort study in the upcoming quarter.

ALSO READ: Prescient Therapeutics’ major strides in anti-cancer programs set the stage for a sturdy 2021

New Study Validates the Signatera® MRD Test and Demonstrates its Clinical Utility in Early-Stage Esophageal Cancer

On July 28, 2021 Natera,Inc. (NASDAQ: NTRA), a pioneer and global leader in cell-free DNA testing, reported a new peer-reviewed paper published in Gastroenterology validating its personalized and tumor-informed molecular residual disease (MRD) assay, Signatera, in resected esophageal adenocarcinoma (EAC) (Press release, Natera, JUL 28, 2021, View Source [SID1234585334]). The paper can be found here.

The study represents the first published data on the use of Signatera in EAC and demonstrates the potential of the Signatera technology in esophageal cancer, which sees around 20,000 new cases per year in the U.S.1

Key findings from this retrospective study of 20 EAC patients after resection:

Signatera detected recurrence with a sensitivity of 80% (4/5). The only missed recurrence had the last sample drawn two years before recurrence. The specificity was 100% (12/12) and the PPV was 100%.
Signatera detected recurrence with a median lead time of almost one year before clinical or radiological recurrence.
Postsurgical MRD-positive patients had disease-free survival of 14.2 months, compared to 51.2 months in MRD-negative patients, indicating prognostic value in this setting.
Patients who were MRD-positive preoperatively and became MRD-negative after surgery had a good prognosis, indicating that Signatera can potentially be used to risk-stratify patients for adjuvant therapy.
"The incidence of esophageal cancer is on the rise globally and more than half of these patients experience recurrence after surgery or treatment with curative intent,"2,3 said Alexey Aleshin, M.D., M.B.A., vice president of medical affairs, oncology at Natera. "For these patients, a sensitive prognostic biomarker, such as Signatera status, could have significant clinical utility in predicting relapse and guiding adjuvant treatment decisions."

This study comes on the heels of another peer-reviewed study recently published in JCO Precision Oncology validating Signatera in oligometastatic cancer, adding to the growing body of evidence behind Signatera in gastrointestinal cancers. To date, Signatera has been published in 14 peer-reviewed publications, in a wide range of cancer types, including colorectal, breast, lung, bladder, pancreatic and more.

About Signatera

Signatera is a custom-built circulating tumor DNA (ctDNA) test for treatment monitoring and molecular residual disease (MRD) assessment in patients previously diagnosed with cancer. The test is available for both clinical and research use, and has been granted three Breakthrough Device Designations by the FDA for multiple cancer types and indications. The Signatera test is personalized and tumor-informed, providing each individual with a customized blood test tailored to fit the unique signature of clonal mutations found in that individual’s tumor. This maximizes Signatera’s accuracy for detecting the presence or absence of residual disease in a blood sample, even at levels down to a single tumor molecule in a tube of blood. Signatera is intended to detect and quantify how much cancer is left in the body, to detect recurrence earlier and to help optimize treatment decisions.

Signatera test performance has been clinically validated in multiple cancer types including colorectal, non-small cell lung, breast, and bladder cancers. Signatera has been developed and its performance characteristics determined by Natera, the CLIA-certified laboratory performing the test. The test has not been cleared or approved by the US Food and Drug Administration (FDA). CAP accredited, ISO 13485 certified, and CLIA certified.

Strata Oncology Completes $90 Million Series C Financing

On July 28, 2021 Strata Oncology, Inc., a precision oncology company advancing molecular indications for cancer therapies, reported the completion of a $90 million Series C financing, bringing the company’s total financing to over $130 million since the company’s inception (Press release, Strata Oncology, JUL 28, 2021, View Source [SID1234585333]). The proceeds will be used to accelerate the development of Strata Oncology’s personalized minimal residual disease (MRD) assay for early-stage cancers and advance its pipeline of novel RNA-based treatment selection tests for solid tumors.

Wellington Management led the financing and was joined by other new investors including Cormorant Asset Management, Monashee Investment Management and Highside Capital Management. Strata Oncology’s existing investors, including Pfizer Ventures, Merck Global Health Innovation Fund, Arboretum Ventures, Deerfield Management, Baird Capital and Renaissance Venture Capital Fund also participated in the financing. J.P. Morgan served as sole placement agent to Strata Oncology on the financing.

"We are grateful to our new and existing investors for their commitment to our bold mission of delivering the best possible treatment for each patient, as early as possible," said Dan Rhodes, Ph.D., co-founder and Chief Executive Officer, Strata Oncology. "This financing will enable us to increase our investment in our platform, programs, and team, as we combine highly sensitive MRD testing with DNA and quantitative RNA-informed treatment selection testing."

The financing supports Strata Oncology’s efforts to expand its comprehensive genomic and transcriptomic profiling platform to deliver a portfolio of industry-leading treatment selection tests for solid tumors. The company’s platform, which integrates comprehensive genomic profiling and highly quantitative RNA expression profiling, enables comprehensive molecular insights from a single, minute tumor tissue specimen. In a recent study shared at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, Strata Oncology demonstrated the potential of its platform, identifying a new multifactorial biomarker that can predict immune checkpoint inhibitor benefit.