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.

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]).

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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.

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.

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On September 28, 2021 XtalPi, a leading AI drug discovery company, reported a research collaboration with Acerand Therapeutics, a biotechnology company specialized in developing best-in-class and or first-in-class drugs (Press release, Acerand Therapeutics, SEP 28, 2021, View Source [SID1234641762]). The partnership will leverage XtalPi’s highly accurate physics-based models and machine learning models to develop potential novel chemical entities for a cancer target.

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The collaboration focuses on facilitating new scaffold identification and lead optimization for a promising oncology target using highly accurate physics-based methods and machine learning models. XtalPi will provide machine learning-based high throughput screening for identification of novel scaffold and new chemical entities on a large-scale cloud computing platform. The proprietary free energy perturbation method in combination with a highly automated design-calculation workflow will be used to predict and analyze the binding affinities and inhibition mechanism during lead optimization. Acerand Therapeutics will provide highly valuable medicinal chemistry expertise and key experimental data to further refine the computational models and improve estimation accuracy.

XtalPi’s automated calculation platform built with high accuracy free energy calculations can precisely depict the interaction between ligands and their targets. The utilization of this technology increases the success rate of lead optimization and novel scaffold identification, and reduces the time required for lead optimization.

XtalPi CEO Dr. Ma Jian’s remark:

The nature of lead optimization is a long and highly complex process involving optimizing molecule physical chemical, ADME, and PK properties. High speed and accurate prediction of ligand affinity and ADMET can significantly reduce the time and cost associated with the development of drugs, especially, the first-in-class drugs. XtalPi can simultaneously evaluate many molecules and speed up the design cycle using the in-house high precision computational chemistry and machine learning technologies combined with our high-capacity cloud computing platform. It is our pleasure to work with Acerand Therapeutics. By collaborating with Acerand Therapeutics, we take advantages of their medicinal chemistry and biology expertise in drug discovery combined with our stength in AI and computational chemistry to more effectively develop potential novel anti-cancer drugs to the patients across the globe.

Acerand Co-Founder and SVP Dr. Genshi Zhao’s remark:

We are thrilled to join forces with XtalPi to develop potential novel anti-cancer therapies for the patients. As we all know, the drug discovery and development is a long and expensive process and there is an urgent need for the efficiency in the development of novel therapies. By leveraging XtalPi’s leading physics-based models and machine learning capabilities, we can rapidly evaluate many molecules with high precision regarding their binding affinities and ADMET properties, which will significantly shorten the lead optimization process, thereby accelerating our drug discovery & development. Learnings from this target should apply to other targets. We believe this is a beginning of a long term and productive collaboration between Acerand Therapeutics and XtalPi.

On September 28, 2021 Hisun Biopharmaceutical Co., Ltd. (hereinafter referred to as "HisunBio"), a wholly-owned subsidiary of BioRay Pharmaceutical Co., Ltd. (hereinafter referred to as "Bioray") reported that the National Medical Products Administration (NMPA) of China approved the application for marketing of its own human-mouse chimeric monoclonal antibody targeting human tumor necrosis factor-α (TNF-α), Infliximab for Injection (Anbaite) on September 24 (Press release, Zhejiang Hisun Pharmaceutical, SEP 28, 2021, View Source;a=index&classid=43&id=5 [SID1234634621]). The National Medicine Permission Number (NMPN) of Anbaite is S20210039.

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The indications of Anbaite include rheumatoid arthritis, Crohn’s disease in adults and children over 6 years old, fistulizing Crohn’s disease, AS (ankylosing spondylitis), psoriasis, and adult ulcerative colitis. Anbaite is the second biosimilar for infliximab to enter China market. It also becomes the third commercialized biologics from BioRay following the launch of Anbainuo and Anjianning (Adalimumab). Including Anshuzheng (Tofacitinib Citrate Tablets), BioRay has entered the ‘Four-An’ era, expanding its leadership in the treatment of immune diseases.

Anbaite is a recombinant human-mouse chimeric monoclonal antibody targeting TNF-α. As an important cytokine, TNF-α is indispensable for immune homeostasis and fighting against various pathogens. However, it has also been found that the overexpression of TNF-α plays a great role in the pathogenesis of autoimmune diseases. The expression level of tumor necrosis factor-α is relatively low in healthy tissues. When its concentration increases pathologically, TNF-α could impair immune homeostasis by promoting excessive inflammation, resulting in damages to tissues and organs. Anbaite can specifically bind to both soluble (sTNF-α) and transmembrane tumor necrosis factor-α (tmTNF-α) with high-affinity, which blocks the binding of tumor necrosis factor with its receptor to alleviate inflammation and autoimmune disease.

Anbaite is the biosimilar of infliximab for injection (Remicade). Remicade was granted marketing authorization in China in 2006. The efficacy and safety of Remicade have been fully verified by its extensive clinical use. Its peak sale reached $9.24 billion. According to the data from Fierce Pharma, the global sale of Remicade was $4.196 billion in 2020, ranking among top 20 worldwide. The infliximab for injection has been recommended as one of the options to treat related diseases by guidelines such as those in the "Chinese experts’ consensus on the treatment of psoriasis with biological agents (2019)". Additionally, it has also been enlisted on the negotiated drug catalog by "China National Drug Catalog for Basic Medical Insurance, Work-Related Injury Insurance, and Maternity Insurance (2020 Edition)" to further guarantee the drug’s accessibility.

As a drug supported by National Science and Technology Programs, Anbaite has been approved for all indications of Remicade in China. The number of people suffering from those indications exceeds 10 million in China and keeps increasing. With its approval by National Medical Products Administration, Anbaite will help alleviate the huge unmet medical needs. BioRay will also take this opportunity to enrich its product pipelines for the treatment of immune diseases, sharpen its leading edge in the field, and continue to develop affordable high-quality products, bringing more treatment options to patients and helping them improve their health and life quality.