On September 29, 2021 Herantis Pharma Plc ("Herantis"), focusing on disease modifying therapies for debilitating neurodegenerative diseases, reported that Dr. Craig Cook, Chief Executive Officer, will hold 1×1 meetings and have a company presentation at the following investor conferences (Press release, Herantis Pharma, SEP 29, 2021, View Source,c3423626 [SID1234590460]):

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Sachs 21st Annual Biotech in Europe Forum, October 7th – 8th, 2021, Digital Conference

1×1 meetings and virtual presentation

Redeye Neurology Seminar CNS – October 13th, 2021

Virtual Presentations at 10:00 – 13:00 EEST / 9:00 – 12:00 CEST

Antti Vuolanto, Chief Operating Officer will have a company presentation at:

Turun Talouspäivät: Säästä ja Sijoita – October 22nd – 23rd, 2021

All presentations will be available via a digital library, which is accessible to event participants only. Please contact the organizers at the Sachs, Redeye, and Turun Talouspäivät if you wish to attend and/or schedule a meeting with Herantis.

On September 29, 2021 ChromaDex Corp. (NASDAQ: CDXC) reported a partnership with Sinopharm Xingsha Pharmaceuticals (Xiamen) Co., Ltd. ("Sinopharm Xingsha") to conduct cross-border sales of Tru Niagen in mainland China (Press release, ChromaDex, SEP 29, 2021, View Source [SID1234590456]). Sinopharm Xingsha is a subsidiary of Sinopharm Group, with businesses including pharmaceutical manufacturing, marketing and distribution of drugs, and food supplements and healthcare products. It is the main platform of Sinopharm Group for food supplements and healthcare products. Sinopharm Group is a large healthcare group directly under the State-owned Assets Supervision and Administration Commission (SASAC) of the State Council, with a full value chain in the industry covering R&D, manufacturing, logistics and distribution, retail chains, healthcare, engineering services, exhibitions and conferences, international business and financial services. Sinopharm Xingsha and ChromaDex will also collaborate to secure Health Food Registration in China.

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Tru Niagen is one of America’s top-selling brands for boosting NAD+ levels and healthy aging. The percentage of people in China over the age of 50 reached 32.8% in 2020 and is expected to reach 45.2% by 2040, according to United Nations Department of Economic & Social Affairs data. The 2020 Chinese Census and National Bureau of Statistics of China estimate that more than 260 million people in China are over the age of 60.

"We are honored and proud to be working with Sinopharm Xingsha and believe this partnership positions ChromaDex as an important player in addressing age-related health issues for the people of China," said ChromaDex CEO Rob Fried.

Tru Niagen is currently available for sale to Chinese consumers on several cross-border marketplaces including T-mall, JD, WeChat, and Kaola. It is also available in over 200 Watsons stores in Hong Kong and Macau.

Tru Niagen is the flagship consumer brand featuring Niagen (patented nicotinamide riboside or NR). Niagen is the world’s most efficient NAD+ precursor on the market, particularly over NMN (nicotinamide mononucleotide). Supplementation with Niagen is backed by 13 published and peer-reviewed clinical trials. Niagen has achieved regulatory acceptance by the world’s four leading regulatory bodies: the U.S. FDA, Health Canada, the European Commission, and the Therapeutic Goods Administration (TGA) of Australia.

On September 29, 2021 NuCana plc (NASDAQ: NCNA) reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Acelarin (NUC-1031), currently being evaluated in a Phase III study (NuTide:121) for the first-line treatment of patients with advanced biliary tract cancer (Press release, Nucana BioPharmaceuticals, SEP 29, 2021, View Source [SID1234590455]). Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address an unmet medical need.

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Acelarin (NUC-1031) | Nucleoside Analog | MedChemExpress
"We are very pleased that the FDA recognizes the potential of Acelarin to address the significant unmet need of patients with biliary tract cancer," said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. "We recently announced enrollment of 418 evaluable patients in our Phase III study, which is expected to enable the first interim analysis in the first half of 2022. This has the potential to allow for an accelerated approval of a new drug application (NDA) for Acelarin in the United States. With both Fast Track and Orphan Drug designations in place, we look forward to working closely with the FDA in our efforts to gain approval for Acelarin as the first approved front-line treatment option for patients with biliary tract cancer."

About Fast Track Designation

Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

About Biliary Tract Cancer

Biliary tract cancer, including cholangiocarcinoma, gallbladder and ampullary carcinoma, are a group of cancers originating in the biliary tract. The biliary tract is comprised of the gallbladder and interconnecting ducts responsible for the transport of bile from the liver to the gallbladder and small intestine. Approximately 178,000 new cases of biliary tract cancer are diagnosed each year worldwide, with more than 18,000 of those diagnoses in the United States. There are currently no agents approved for the first-line treatment of patients with advanced biliary tract cancer; however, the worldwide standard of care in these patients is the combination of gemcitabine and cisplatin. Patients receiving this regimen have a median overall survival of 11.7 months.

On September 29, 2021 Helsinn Group and BridgeBio Pharma, Inc. (Nasdaq: BBIO), through its affiliate QED Therapeutics, Inc., reported that Health Canada has approved TRUSELTIQ (infigratinib), a small molecule kinase inhibitor that targets fibroblast growth factor receptor (FGFR), under the Notice of Compliance with Conditions (NOC/c) policy, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or other rearrangement (Press release, BridgeBio, SEP 29, 2021, View Source [SID1234590454]).

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An NOC/c is a form of market approval granted to a product on the basis of promising evidence of clinical effectiveness following review of the submission by Health Canada. Products authorized under Health Canada’s NOC/c policy are intended for the treatment, prevention or diagnosis of a serious, life-threatening or severely debilitating illness. They have demonstrated promising benefit, are of high quality and possess an acceptable safety profile based on a benefit/risk assessment. In addition, they either respond to a serious unmet medical need in Canada or have demonstrated a significant improvement in the benefit/risk profile over existing therapies. Health Canada has provided access to this product on the condition that sponsors carry out additional clinical trials to verify the anticipated benefit within an agreed upon time frame.

"This is an important next step in growing TRUSELTIQ’s global reach. We are pleased to have achieved this milestone for patients with previously-treated locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or other rearrangement," said Riccardo Braglia, Helsinn Group Vice Chairman and CEO. "The conditional approvals from the U.S. FDA and Health Canada mark the beginning of our journey delivering this medicine to patients in need. We look forward to working to enter further markets in the months and years ahead and working closely with BridgeBio as we make strides to reach patients."

"We are grateful for our first international approval and the opportunity to reach patients outside the United States who are searching for options to treat FGFR2-fusion-driven cholangiocarcinoma. Helsinn has an impressive track record of advancing and commercializing oncology therapies around the globe and we partnered with them earlier this year in the hope of reaching as many patients with FGFR-driven cancers as possible," said BridgeBio CEO and Founder Neil Kumar, Ph.D. "We believe infigratinib may be able to treat other FGFR-driven conditions and we will continue to evaluate its safety and efficacy in urothelial carcinoma and other areas of unmet need."

Under Project Orbis, an initiative of the FDA, Oncology Center of Excellence that allows for concurrent submission and review of oncology drugs among participating international regulatory agencies, TRUSELTIQ received accelerated approval from the U.S. Food and Drug Administration (FDA) in May 2021. An additional marketing application for infigratinib is currently under review in Australia.

Helsinn Group has exclusive commercial rights for TRUSELTIQ in Canada with BridgeBio eligible for tiered royalties as a percentage of net sales as part of the global collaboration and license agreement entered into between the two companies in March 2021.

As part of this agreement, BridgeBio and Helsinn Group’s affiliate, Helsinn Therapeutics U.S., Inc., are jointly responsible for commercialization activities for TRUSELTIQ in the U.S. and will share U.S. profits and losses on an equal basis. Helsinn Group will have exclusive commercialization rights on infigratinib outside of the U.S., excluding China, Hong Kong and Macau. BridgeBio will be eligible for tiered royalties as a percentage of adjusted net sales, and payments totaling up to approximately $2.45 billion USD in the aggregate. Helsinn Group will fund the majority of ongoing and future research and development related to infigratinib in oncology. BridgeBio previously entered a strategic collaboration with LianBio for development and commercialization of infigratinib in oncology indications in China, Hong Kong and Macau.

About TRUSELTIQ (infigratinib)

TRUSELTIQ (infigratinib) is a small molecule kinase inhibitor that targets FGFR, which obtained accelerated approval by FDA and was conditionally approved by Health Canada for the treatment of adults with previously treated, unresectable locally advanced or metastatic CCA with a FGFR2 fusion or other rearrangement.

Prior to initiation of TRUSELTIQ therapy, FGFR2 fusion or rearrangement should be established using a validated test.

Clinical effectiveness of TRUSELTIQ is based on overall response rate (ORR) and duration of response (DoR) from a single-arm Phase 2 trial in patients with specific FGFR2 fusion or other rearrangements.

Infigratinib is not FDA- or Health Canada-approved for any other indication in the United States and Canada, and is not approved for use by any other health authority.

About Cholangiocarcinoma (CCA)

CCA represents an aggressive group of malignancies that form in the bile ducts. The incidence of this serious and fatal disease varies considerably worldwide. As the disease is usually asymptomatic at early-stages, CCA typically presents at diagnosis as locally advanced or metastatic disease with a poor prognosis. In this respect, the five-year survival rate for patients affected by metastatic CCA is 2%. Depending on the anatomical site of origin, CCAs are classified into two subtypes: intrahepatic (iCCA – 10% of total) and extrahepatic (eCCA – 90% of total) CCA. Approximately 10% to 16% of iCCA harbor FGFR2 genetic alterations.1, 2, 3

On September 29, 2021 AstraZeneca reported it’s Alexion has exercised its option to acquire all remaining equity in Caelum Biosciences for CAEL-101, a potentially first-in-class fibril-reactive monoclonal antibody (mAb) for the treatment of light chain (AL) amyloidosis (Press release, AstraZeneca, SEP 29, 2021, View Source [SID1234590453]).

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AL amyloidosis is a rare disease in which misfolded amyloid proteins build up in organs throughout the body, including the heart and kidneys, causing significant organ damage and failure that may ultimately be fatal.1,2 Approximately 20,000 people across the US, France, Germany, Italy, Spain and the UK live with AL amyloidosis classified as Mayo stage IIIa or IIIb disease.3

CAEL-101 is currently being evaluated in the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase III clinical programme in combination with standard-of-care (SoC) therapy in AL amyloidosis. Two parallel Phase III trials in patients with Mayo stage IIIa disease and in patients with Mayo stage IIIb disease respectively are ongoing.4,5

Marc Dunoyer, Chief Executive Officer, Alexion, said: "With a median survival time of less than 18 months following diagnosis, there is an urgent need for new treatments for this devastating disease. CAEL-101 has the potential to be the first therapy to target and remove amyloid deposits from organ tissues, improve organ function, and, ultimately, lead to longer lives for these patients."

Financial considerations
In 2019 Caelum and Alexion first entered into a collaboration whereby Alexion acquired a minority equity interest and an exclusive option to acquire the remaining equity in Caelum. Alexion currently consolidates Caelum and reflects a non-controlling interest of $150m. Upon closing the acquisition, which is expected to take place on 5 October 2021, Alexion will pay Caelum the agreed option exercise price of approximately $150m, with the potential for additional payments of up to $350m upon achievement of regulatory and commercial milestones.

AL amyloidosis
AL amyloidosis is a rare disease caused by defective plasma cells in the bone marrow that produce abnormal antibody (immunoglobulin) proteins.1 These abnormal proteins misfold and aggregate to form amyloids that may deposit in tissues and/or organs.1,2 Amyloid accumulation in organs, particularly in the heart and kidneys, can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure.1,2

CAEL-101
CAEL-101 is a potentially first-in-class mAb designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain proteins and amyloid and shows binding to both kappa and lambda subtypes. CAEL-101 has received Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Commission as a potential therapy for patients with AL amyloidosis. Additionally, the US FDA granted Fast Track Designation to CAEL-101 for AL amyloidosis in June 2021.

CARES Phase III clinical programme
The CARES clinical programme consists of two parallel global Phase III trials which are evaluating the efficacy and safety of CAEL-101 in AL amyloidosis patients who are newly diagnosed and naïve to SoC treatment (based on a cyclophosphamide-bortezomib-dexamethasone regimen). One trial is enrolling approximately 270 patients with Mayo stage IIIa disease and one trial is enrolling approximately 110 patients with Mayo stage IIIb disease. The primary endpoint for both clinical trials is overall survival and enrolment is underway.

In each study, participants are being randomised in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks and will subsequently be enrolled in an open-label extension study.