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Cardiff Oncology to Present New Data from Lead Clinical Program in KRAS-mutated Metastatic Colorectal Cancer on September 8, 2021

On August 25, 2021 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage oncology company, developing new precision medicine treatment options for cancer patients in indications with the greatest unmet medical need including KRAS-mutated colorectal cancer, pancreatic cancer, and castrate-resistant prostate cancer, reported that it will host a virtual KOL event for analysts, investors, and the scientific community on Wednesday, September 8, 2021 (Press release, Cardiff Oncology, AUG 25, 2021, View Source [SID1234586875]).

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The webinar will include presentation of new data from Cardiff Oncology’s Phase 1b/2 clinical trial evaluating onvansertib in combination with standard-of-care FOLFIRI/bevacizumab in KRAS-mutated mCRC, featuring the clinical trial principal investigator, Heinz-Josef Lenz, M.D., FACP, USC Norris Comprehensive Cancer Center, key clinical advisor Afsaneh Barzi, M.D., Ph.D., City of Hope Comprehensive Cancer Center, and members of the Cardiff Oncology management team. A question & answer session will follow the formal presentations.

Heinz-Josef Lenz, M.D., FACP, is the associate director for clinical research and co-leader of the gastrointestinal (GI) cancers program at the University of Southern California Norris Comprehensive Cancer Center. Dr. Lenz is professor of medicine and preventive medicine, section head of gastrointestinal oncology in the division of medical oncology and co-director of the Colorectal Center at the Keck School of Medicine of the University of Southern California. Dr. Lenz received his medical degree from Johannes-Gutenberg Universität in Mainz, Germany, in 1985. He completed a residency in hematology and oncology at the University Hospital Tübingen in Germany, a clerkship in oncology at George Washington University in Washington, DC, and a clerkship in hematology at Beth Israel Hospital of Harvard Medical School in Boston, Massachusetts. He served subsequent fellowships in biochemistry and molecular biology at the University of Southern California Norris Comprehensive Cancer Center. An active researcher, Dr. Lenz focuses on topics including the regulation of gene expression involved in drug resistance, patients at high risk of developing colorectal cancer, and determination of carcinogenesis, methods of early detection, and better surveillance of these cancers. He is a member of several professional societies, including the American Association for Cancer Research (AACR) (Free AACR Whitepaper), the American Gastroenterology Association, and the National Society of Genetic Counselors. He also serves on the National Advisory Board of several professional organizations. Dr. Lenz is the author of numerous peer-reviewed publications and invited papers, reviews, and editorials. He also serves as co-chair of the GI Committee and Correlative Science Committee for SWOG. He is a member of the National Cancer Institute (NCI) Task Force for Gastroesophageal Cancer, the NCI Steering Committee, and the NCI Translational Science Committee.

Afsaneh Barzi, M.D., Ph.D., is a practicing medical oncologist, associate clinical professor for gastrointestinal oncology, and clinical director of AccessHope at City of Hope Comprehensive Cancer Center. Prior to joining City of Hope, Dr. Barzi was an associate professor of clinical medicine at the Keck School of Medicine of the University of Southern California. She earned her M.D. from Tehran University of Medical Sciences, then went on to earn a Master’s in Health Informatics and a Doctorate in Public Health Management and Policy Sciences from the University of Texas Health Science Center in Houston. Dr. Barzi completed a fellowship in hematology and oncology at the Cleveland Clinic’s Taussig Cancer Center. Her research and practice are focused on gastrointestinal malignancies with an emphasis on colorectal cancers. Her unique perspective on patterns of care in patients with colorectal cancer arises from the combination of her expertise in real-world data and her experience with biomarker discovery and the use of biomarkers for personalized care.

BridgeBio Pharma and LianBio Announce First Patient Treated in Phase 2a Trial of Infigratinib in Patients with Gastric Cancer and Other Advanced Solid Tumors

On August 25, 2021 LianBio, a biotechnology company dedicated to bringing paradigm-shifting medicines to patients in China and other major Asian markets, and BridgeBio Pharma, Inc. (Nasdaq: BBIO) reported the first patient has been treated in a Phase 2a clinical trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor-2 (FGFR2) gene amplification and other advanced solid tumors with FGFR genomic alterations (Press release, BridgeBio, AUG 25, 2021, View Source [SID1234586874]).

"Infigratinib is a potent and selective FGFR inhibitor that has demonstrated compelling clinical activity across multiple tumor types with FGFR alterations," said Yizhe Wang, Ph.D., chief executive officer of LianBio. "Given the disproportionately high prevalence rate of gastric cancer in China, LianBio is pursuing a region-specific development strategy focused on this area of great unmet need. This study marks LianBio’s first trial initiation and demonstrates our continued progress in delivering potentially transformational medicines to patients in Asia."

TRUSELTIQ (infigratinib) is an oral selective inhibitor of FGFR1-3 that is approved in the United States for the treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement as detected by an FDA-approved test. It is also being further evaluated in clinical trials based on demonstration of clinical activity in patients with advanced urothelial carcinoma with FGFR3 genomic alterations. LianBio in-licensed rights from BridgeBio for infigratinib for development and commercialization in Mainland China, Hong Kong and Macau.

The Phase 2a trial is a multicenter, open-label, single-arm study in China designed to evaluate the safety and efficacy of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with FGFR2 gene amplification and other advanced solid tumors with FGFR alterations. The primary endpoint is objective response rate (ORR). Secondary endpoints include duration of response, safety, disease control rate, progression-free survival and overall survival.

Preclinical data have demonstrated the potential infigratinib may have for patients with gastric cancer. These results, published in Cancer Discovery, demonstrated tumor regression in multiple in vivo FGFR2 amplified gastric models.1

"We believe that infigratinib could have a meaningful impact for people living with gastric cancer as well as many other cancers with FGFR alterations, and are pleased LianBio is initiating this clinical trial in China where more therapeutic options are needed to match the growing diagnosis rate," said BridgeBio founder and chief executive officer Neil Kumar, Ph.D. "On the heels of TRUSELTIQ recently obtaining accelerated approval in the United States, we are hopeful that this trial will yield pivotal results in another subset of cancer patients as we continue to build our portfolio of oncology indications with the aim of reaching as many people in need as possible."

About TRUSELTIQ (infigratinib)

TRUSELTIQ (infigratinib) is an orally administered, ATP-competitive, tyrosine kinase inhibitor of fibroblast growth factor receptor (FGFR) that received accelerated approval from the FDA in the United States for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. TRUSELTIQ targets the FGFR protein, blocking downstream activity. In clinical studies, TRUSELTIQ demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response. TRUSELTIQ is not FDA-approved for any other indication in the United States and is not approved for use by any other health authority, including any Chinese or other Asian health authority. It is currently being evaluated in clinical studies for first-line cholangiocarcinoma, urothelial carcinoma (bladder cancer), locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma, and other advanced solid tumors with FGFR genomic alterations.

Ascendis Pharma A/S Reports Second Quarter 2021 Financial Results

On August 25, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to potentially create new treatments that make a meaningful difference in patients’ lives, reported financial results for the second quarter ended June 30, 2021 (Press release, Ascendis Pharma, AUG 25, 2021, View Source [SID1234586873]).

"We are actively preparing for the U.S. commercial launch of SKYTROFA for the treatment of children with GHD, which is now the first FDA-approved once-weekly treatment for pediatric GHD. SKTROFA is also the first FDA-approved product utilizing our innovative TransCon technology. Our pivotal heiGHt Trial demonstrated that once-weekly TransCon hGH increased annualized height velocity in treatment-naïve subjects at 52 weeks compared to a daily growth hormone with comparable safety and tolerability," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We see this approval as the first step in creating a market leading product and building a fully integrated global biopharmaceutical company guided by our values of patients, science, and passion."

Company Highlights & Progress

TransCon hGH (lonapegsomatropin)
TransCon hGH is now FDA approved in the U.S. under the brand name SKYTROFA. Continued preparation for commercial launch for the treatment of pediatric patients with GHD in the U.S.
European Commission decision on the company’s Marketing Authorisation Application (MAA) for the treatment of pediatric patients with GHD is anticipated in the fourth quarter of 2021.
Ongoing enrollment in the foresiGHt Trial, a global phase 3 trial in adults with GHD, and the riGHt Trial, a phase 3 trial in Japan in pediatric patients with GHD.
Patient follow-up continues in enliGHten, a multi-center phase 3, long-term open-label trial investigating safety and efficacy of SKYTROFA in pediatric patients with GHD.
Comprehensive results from the heiGHt Trial recently published on-line in the Journal of Clinical Endocrinology & Metabolism, an official journal of the Endocrine Society.
TransCon PTH (palopegteriparatide)
Exceeded target enrollment in the PaTHway Trial, a phase 3 trial evaluating the safety, tolerability, and efficacy of palopegteriparatide in adult subjects with hypoparathyroidism with similar demographics as enrolled in the phase 2 trial including broad representation of different non-surgical disease etiologies and leading influential clinical sites balanced between North America and Europe.
On track to announce 84-week top line results from the open label extension (OLE) portion of the PaTH Forward Trial in the fourth quarter of 2021. Continued strong long-term subject retention with 58 out of the 59 randomized subjects continuing in the OLE portion of the trial as of August 23, 2021.
Clinical trial notification for the PaTHway Japan Trial was accepted by the Japanese Pharmaceuticals and Medical Device Agency. The single-arm, phase 3 study will enroll a minimum of 12 Japanese subjects with HP.
Received Orphan Drug Designation (ODD) from the Japanese Ministry of Health, Labor and Welfare.
VISEN Pharmaceuticals (VISEN) obtained investigational new drug (IND) approval to initiate the phase 3 PaTHway China Trial.
TransCon CNP
Continued execution in the ongoing phase 2 ACcomplisH Trial and ACcomplisH China Trial to evaluate the safety and efficacy of TransCon CNP in children ages two to ten years with achondroplasia.
Clinical program update planned for the fourth quarter of 2021.
TransCon TLR7/8 Agonist
Initiated combination therapy arm in transcendIT-101 with TLR7/8 Agonist and a CPI.
TransCon IL-2 ß/y
IND filing on track for this quarter.
Ended the second quarter of 2021 with cash, cash equivalents and marketable securities totaling €641.3 million.
Second Quarter 2021 Financial Results

For the second quarter, Ascendis Pharma reported a net loss of €134.4 million, or €2.50 per share (basic and diluted) compared to a net loss of €94.9 million, or €1.97 per share (basic and diluted) for the same period in 2020.

Revenue for the second quarter was €1.0 million compared to €1.4 million in the same quarter of 2020. The decrease was due to a lower amount of license revenue being recognized, partly offset by higher sale of clinical supplies and services to VISEN and recognition of revenue from services rendered to another collaboration partner.

Research and development (R&D) costs for the second quarter were €83.3 million compared to €63.6 million during the same period in 2020. Higher R&D costs in 2021 reflect an increase in external development costs of the company’s product candidates and an increase in personnel-related costs.

Selling, general and administrative expenses for the second quarter were €35.3 million compared to €20.8 million during the same period in 2020. The increase is primarily due to higher personnel-related costs and an increase in IT costs.

Net loss of associate for the second quarter was €4.8 million compared to a net loss of €1.9 million in the same quarter of 2020. The net loss of associate represents our share of the net result from VISEN.

As of June 30, 2021, Ascendis Pharma had cash, cash equivalents and marketable securities of €641.3 million compared to €771.1 million as of March 31, 2021. As of June 30, 2021, Ascendis Pharma had 53,900,990 ordinary shares outstanding.

Conference Call Details

A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will be available on this website shortly after conclusion of the event for 30 days.

About Ascendis Pharma’s Pipeline

Ascendis Pharma currently has three product candidates in clinical development in rare endocrine diseases and one oncology product candidate in clinical development:

TransCon hGH (lonapegsomatropin-tcgd), an investigational long-acting prodrug of somatropin (human growth hormone or hGH) that releases somatropin with the identical amino acid sequence and size as daily growth hormone, is designed as a once-weekly treatment for GHD and is approved for pediatric GHD by the U.S. Food and Drug Administration and under review by the European Medicines Agency.
TransCon PTH (palopegteriparatide), an investigational long-acting prodrug of parathyroid hormone (PTH) in phase 3 development as a once-daily replacement therapy for adults with hypoparathyroidism designed to replace PTH at physiologic levels for 24 hours, and address both short-term symptoms and long-term complications of the disease.
TransCon CNP, an investigational long-acting prodrug of C-type natriuretic peptide (CNP) in phase 2 development as a therapy for children with achondroplasia (ACH), the most common form of dwarfism, for which there is no FDA-approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose.
TransCon TLR7/8 Agonist is an investigational long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8. Administered as an intratumoral injection, TransCon TLR7/8 Agonist is designed to provide sustained activation of intratumoral antigen presenting cells driving tumor antigen presentation and induction of immune stimulatory cytokines in the tumor.
TransCon IL-2 ß/y is an investigational long-acting prodrug of IL-2 ß/y designed for optimized IL-2R ß/y bias and potency, combined with low Cmax and long exposure.

First US patent granted to Scandion Oncology on combination treatment with its candidate drug SCO-101

On August 25, 2021 Scandion Oncology A/S, the Cancer Drug Resistance Company, reported that the US Patent and Trademark Office (USPTO) will grant the company’s patent US11,103,481 directed to the use of SCO-101, on August 31, 2021 (Press release, Scandion Oncology, AUG 25, 2021, View Source,c3402649 [SID1234586871]).

The granted patent covers combination therapy with Scandion Oncology’s first-in-class lead candidate SCO-101 and is part of the company’s patent family originating from the international application PCT/EP2017/061823. The patent claims the use of SCO-101 in combination with different anti-cancer agents across many cancer indications. The Patent Term for the US patent has been extended by 275 days, prolonging the expiry of this patent until January 2038.

"We are pleased that the USPTO has recognized the uniqueness of SCO-101 and granted this patent. This is an important milestone on our journey of developing SCO-101 and internationalizing the company. We have obtained valuable patent protection for SCO-101 in the US, one of our projected key markets, increasing the commercial potential of SCO-101 and creating value for shareholders," said Bo Rode Hansen, President and CEO.

This information is information that Scandion Oncology A/S is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, on August 25, 2021, at 8:30 CET

Scandion Oncology A/S is a clinical Phase II biotechnology company currently developing first-in-class, oral add-on drugs to existing market leading anti-cancer therapies. As add-on to standard anti-cancer therapies, it introduces an effective treatment approach for cancer, which is or has become resistant to cancer-fighting drugs, offering the potential for better response rates, longer survival and improved quality of life. The first-in-class lead candidate, SCO-101, is currently in clinical Phase II. The Company is targeting cancer drug resistance in various treatment modalities including chemotherapy, anti-hormonal therapy and immunotherapy. Scandion Oncology is listed on Nasdaq First North Growth Market Sweden. Ticker: SCOL.

FDA accelerates review of Novartis STAMP inhibitor asciminib (ABL001) for patients with chronic myeloid leukemia (CML)

On August 25, 2021 Novartis reported that the US Food and Drug Administration (FDA) accepted and granted Priority Review to the company’s New Drug Application (NDA) for asciminib (ABL001) in chronic myeloid leukemia (CML), following its submission under the FDA’s Real-Time Oncology Review (RTOR) program. Priority Review is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions, as determined by the FDA1 (Press release, Novartis, AUG 25, 2021, View Source [SID1234586869]). This designation could shorten the FDA review period to eight months compared to the 12 months under Standard Review1.

Priority Review granted based on positive data from the pivotal, Phase III ASCEMBL trial, where asciminib was compared to Bosulif (bosutinib)* in patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs), and data from a Phase I trial that included patients with Ph+ CML-CP harboring the T315I mutation2,3
Despite available treatments, many patients with CML remain at risk of disease progression, and sequential therapy with currently available TKIs may be associated with increased resistance and/or intolerance4-10
Asciminib, a novel investigational therapy specifically targeting the ABL myristoyl pocket – also known as a STAMP inhibitor –, is in development across multiple treatment lines for CML11-17
Novartis has previously received Orphan Drug, Fast Track and two Breakthrough Therapy designations for asciminib. Breakthrough Therapy designations were granted for asciminib for the treatment of adult patients with Ph+ CML-CP previously treated with two or more TKIs, as well as adult patients with Ph+ CML-CP harboring the T315I mutation.