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Helsinn Group and BridgeBio Pharma’s Affiliate QED Therapeutics Announce Health Canada Conditional Approval of TRUSELTIQ™ (infigratinib) for Patients with Cholangiocarcinoma

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

AstraZeneca to fully acquire Caelum Biosciences

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]).

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.

INTERIM RESULTS ANNOUNCEMENT for the six months ended 30 June 2021

On September 29, 2021 ImmuPharma PLC (LSE:IMM), (Euronext Growth Brussels: ALIMM) ("ImmuPharma" or the "Company"), the specialist drug discovery and development company, reported its interim results for the six months ended 30 June 2021 (the "Period") (Press release, ImmuPharma, SEP 29, 2021, View Source [SID1234590452]).

Key Highlights (including post Period review)

Financials
Financial performance in line with expectations over the Period

Cash balance of £4.2m as at 30 June 2021 (31 December 2020: £5.9m)
Loss for the period of £3.7m (30 June 2020: £3m)
Research and development expenses of £1.3m (30 June 2020: £0.9m)
Administrative expenses of £1.5m (30 June 2020: £1m)
Derivative financial asset of £0.2m as at 30 June 2021 (31 December 2020: £1.2m)
Incanthera financial asset of £1.2m (£1.8m at 31 December 2020) and warrants financial asset of £0.2m (£0.6m at 31 December 2020)
Convertible loan notes of £0.7m (£0.6m at 31 December 2020)
Share based expense of £0.3m (30 June 2020: £1m)
Basic and diluted loss per share of 1.46p (30 June 2020: 1.69p)
‘Autoimmunity’: P140

Lupuzor (P140) – now entering a pharmacokinetic ("PK") study prior to the optimized Phase 3 study in lupus in conjunction with its licensing partner, Avion Pharmaceuticals.
P140 for Chronic Inflammatory Demyelinating Polyneuropathy ("CIDP") a neurological disorder targeting the body’s nerves. Active preparation for a phase 2/3 clinical study has now been initiated.
Potential further clinical applications based on further preclinical investigation include asthma, Sjogrens syndrome, renal inflammation in diabetes and periodontitis.
‘Anti-infection’

ioAMB, a novel peptide-based drug that offers a potential improvement on the limiting side effects of current Amphotericin-B ("AMB") formulations. AMB is one of a last line of agents against serious and life-threatening fungal infections caused by the aspergillus family of fungi.
BioCin, a novel peptide-based drug based on an existing potent antibacterial used in high medical need cases. BioCin has the potential to offer improved safety and/or administration benefits.
Board changes
New Board established:

Tim McCarthy appointed as Chief Executive Officer ("CEO")
Dr Tim Franklin appointed as Chief Operating Officer ("COO").
Non Executive Directors ("NED") appointed – Dr Sanjeev Pandya & Lisa Baderoon.

29 september 2021: Naventus Life Science Summit

On September 29, 2021 Sprint Bioscience reported that it will participate in the Naventus Life Science Summit, September 29, 2021 at the Grand Hôtel and CEO Erik Kinnman will present the company (Press release, Sprint Bioscience, SEP 29, 2021, View Source [SID1234590451]). The event is organized by Naventus Corporate Finance together with Setterwalls, Deloitte, FNCA, Nordnet and Nasdaq.

Alphamab Oncology and Raygene Pharmaceutical Announced Partnership to Develop Combination Therapy of Bispecific Antibody KN046 and Small Molecule Drug RG001

On September 28,2021 Alphamab Oncology (stock code: 9966.HK) reported, the company has entered a partnership agreement with Hangzhou Raygene Pharmaceutical Co., Ltd (Raygene Pharmaceutical) to jointly develop the combination therapy of the PD-L1/CTLA-4 bispecific antibody KN046 and the small molecule drug RG001 for the posterior line treatment of advanced HCC and liver metastasis CRC (Press release, Alphamab, SEP 28, 2021, View Source [SID1234650400]).

Liver cancer and colorectal cancer are the common types of gastrointestinal cancers. According to the latest global cancer burden data released by the World Health Organization in 2020, liver cancer is the sixth most common cancer worldwide, and the third leading cause of cancer-related death globally. Over 50% of new cases and deaths occurred in China. Colorectal cancer is the third most common cancer worldwide and the second most common tumor in China. 83% of colorectal cancer patients in China were at a middle or advanced stage when diagnosed, and 44% of them had liver/lung metastasis.

KN046 is a bispecific antibody independently developed by Alphamab Oncology, which simultaneously targets PD-L1 and CTLA-4 immune checkpoints and could effectively activate T cells and enhance immune anti-tumor ability. The Phase II clinical data of KN046 combined with Lenvatinib in the first-line treatment of HCC were published at the ESMO (Free ESMO Whitepaper) 2021. KN046 in combination with Lenvatinib in first line HCC achieved 57% and 76% ORR and 95% and 95% DCR by RECISCT 1.1 and imRECIST, respectively. Based on the results from this study, a global phase III clinical study of KN046 in combination with Lenvatinib for the first line treatment of hepatocellular carcinoma is justified and currently in the planning phase.

RG001 (TATE) is a proprietary anti-tumor small molecule drug licensed in from abroad by Ruizhen Pharmaceutical. It could be acted as prodrug and transformed to cytotoxic metabolites in hypoxic tumor cell, and resulted in tumor cells killed. According to pre-study, it could achieve a synergistical effects to enhance anti-cancer capacity when combined with other immune checkpoint inhibitors.

According to the agreement, Alphamab Oncology and Raygene Pharmaceutical will conduct clinical trials evaluating safety and efficacy of KN046 plus RG001 or in sequential combination for the posterior line treatment of advanced HCC, CRC liver metastasis and other indications jointly decided by both parties, and to bring new options to patients who have progressed after receiving chemotherapy, small molecule targeted therapy or PD-1 therapy.

About KN046

KN046 is PD-L1/CTLA-4 bispecific antibody independently developed by Jiangsu Alphamab. Its innovative designs include: a different mechanism CTLA-4 fused with PD-L1 single domain antibody; engineered to target the tumor microenvironment with high PD-L1 expression, and Treg（suppress tumor immunity） clearing function.

There are about 20 clinical trials of KN046 in different stages covering more than 10 types of tumors including NSCLC, thymic cancer, pancreatic cancer, HCC, ESCC and TNBC in Australia and China. The results of these clinical trials have shown an advantage in survival for patients. Alphamab has received FDA clearance to enter phase Ⅱ trial of KN046 based on the clinical results in China and Australia. Moreover, KN046 has obtained the U.S. FDA’s orphan drug designation for thymic epithelial tumor in September 2020. Four pivotal clinical trials are currently being conducted.