Global Roundup: Denmark’s Ascendis Sees “Real Control” in Hypothyroidism

At ENDO 2022, Ascendis will share Phase II and III data showing clinical efficacy for its investigational product candidate for hypoparathyroidism. For this and more, see inside.

New clinical data from Denmark-based Ascendis Pharma A/S is showing significant potential in the treatment of hypothyroidism, a condition that can lower the levels of calcium in the blood.

Next week, at ENDO 2022, Ascendis will share Phase II and III data showing clinical efficacy for the company’s investigational product candidate TransCon PTH. Dr. Aliya Khan, clinical professor of medicine at McMaster University and director of the Calcium Disorders Clinic at McMaster University Medical Center, as well as an investigator in the study, said the Phase III data Ascendis will present at ENDO is the first late-stage trial in adult hypoparathyroidism in which the majority of treated patients achieved “real control of their disease,” meaning normalization of serum calcium and independence from conventional therapy.

“Additionally, TransCon PTH was generally well-tolerated with no discontinuations related to study drug, and TransCon PTH-treated patients showed a mean decrease in 24-hour urine calcium excretion into the normal range. I am hopeful that, with these encouraging results, we are closer to changing the treatment paradigm for patients living with this under-recognized and often debilitating disease,” Khan said.

The Phase II data that will be presented at ENDO will include long-term efficacy data that showed treatment with TransCon PTH provided durable benefit for adult hypothyroidism patients after 84 weeks of treatment.

Additionally, Ascendis posters will present new data showing the continued safety and efficacy of once-weekly TransCon hGH in children with growth hormone deficiency treated for 2.5 years in the EnliGHten Trial, and, for adults with a growth hormone deficiency, new research showing low treatment rates, increased medical risks, and higher healthcare costs.

Elsewhere Around the Globe:

Evaxion Biotech A/S: Also based in Denmark, Evaxion announced it entered into an equity purchase agreement with Lincoln Park Capital Fund for up to $40 million. Under the terms of the Agreement, Evaxion has the right, at its sole discretion, but not the obligation, to sell to LPC up to $40 million of its American Depository Shares over the 36-month term of the agreement. The investment will contribute to the progression of our exciting portfolio of assets, including our personalized cancer medicines, developed in programs EVX-01 and EVX-02, both of which are currently in Phase II clinical development.

Transgene: France-based Transgene presented Phase I data for its individualized neoantigen cancer vaccine TG4050 at the American Society of Clinical Oncology. Positive initial data, including molecular response, were generated from the first patients with ovarian cancer and HPV-negative head and neck cancer. TG4050 is based on Transgene’s myvac platform and powered by NEC’s cutting-edge AI capabilities.

Health Canada: The Canadian regulatory agency approved Orladeyo (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older. Orladeyo was developed by BioCryst Pharmaceuticals. Also this week, Swissmedic gave the nod to Orladeyo for HAE patients.

ScoutCam Inc.: Based in Israel, ScoutCam, a provider of image-based solutions, announced that it has completed the verification and validation (V&V) stage of its miniature camera solution. With this essential V&V phase complete, the company believes sales revenue could reach tens of millions of dollars as ongoing supply agreements with the customer accumulate in the coming years.

Ayala Pharmaceuticals: Also based in Israel, Ayala presented data on its investigational cancer drug AL101 at ASCO. Data from the Phase II ACCURACY trial provided dosing updates from cohorts. AL101 is a selective gamma-secretase inhibitor that is being assessed in subjects with recurrent/metastatic (R/M) adenoid cystic carcinoma who harbor Notch activating mutations. The Phase II trial is the first ACC study to select patients with ACC bearing defined NOTCH-activating mutations. Approximately 90% of the patients had metastatic disease at screening. This was a heavily pretreated patient population, with over half having previously received some form of systemic therapy.

SciSpark Ltd.: Another Israel-based company, SciSpark, announced initial positive pre-clinical results for treatment for cocaine addiction that harnessed the capabilities of MEAI, a novel psychedelic molecule developed by Canada’s Clearmind Medicine Inc. Using SciSparc’s CannAmide compound and Clearmind’s MEAI, the pre-clinical trials show possible positive results.

Masdar City: Based in the United Arab Emirates, Masdar City, a venture investment platform, signed a memorandum of understanding to boost agritech sustainability, and support the UAE in meeting key objectives in line with the National Food Security Strategy 2051. Under the agreement, Masdar City and DANA, which supports women-led startups across the sectors of agritech, energy and water solutions, and circular economy, will build its first Abu Dhabi-based beta site. The area will test the feasibility of early stage agritech projects that can be developed and piloted in Abu Dhabi and will focus on local and regional startups with solutions between the ‘pre-seed’ and ‘Series A’ development phases upon completion.

Axol Bioscience: A company that provides iPSC cells, U.K.-based Axol announced that its human induced pluripotent stem cell (iPSC)-derived ventricular cardiomyocytes have undergone comprehensive in vitro pro-arrhythmia assay (CiPA) validation. Using this assay, the cells were shown to be suitable for measuring cardiotoxicity, offering scientists a robust cardiac model for drug discovery and screening.

Olink Holding AB: Sweden’s Olink Holding announced a collaboration with Element Biosciences that will allow the use of the Olink Explore high-throughput proteomics platform in combination with Element’s next generation sequencing (NGS) platform. Element and Olink have performed a technical proof-of-concept study demonstrating high compatibility between the two platforms. Element and Olink are exploring more advanced means of seamlessly combining the existing Olink Explore product with Element’s AVITI System in additional use cases.

Olink also entered into a collaboration with Ultima Genomics for the use of Olink Explore. The partnership will allow for the combination of Olink Explore with Ultima’s sequencing architecture.

SOBI: Also based in Sweden, SOBI announced that the first patient has been dosed in the Phase III VALIANT study co-conducted with Apellis Pharmaceuticals. The study is investigating pegcetacoplan, a targeted C3 therapy, in primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), two rare and debilitating kidney diseases with similar underlying causes and no approved treatment.

AUM Biosciences: Singapore-based AUM Biosciences announced it received the green light from the U.S. Food and Drug Administration to begin a clinical trial evaluating AUM001, a selective translation inhibitor. As a result, AUM plans to evaluate AUM001, in combination with Merck’s anti-PD-1 therapy Keytruda (pembrolizumab), in a global Phase II clinical trial in patients with microsatellite stable colorectal cancer.

Cytena: Germany-based Cytena, a BICO group company, signed a long-term OEM agreement with PAIA biotech GmbH that supports the launch of F.QUANT Titer, a plate-based high-throughput titer quantification assay for antibodies, bispecific or Fc fusion proteins based of PAIA’s patented technology. The new F.QUANT Titer provides biopharma professionals with an easy-to-use and cost-effective solution for the identification of high-producer clones based on fluorescence intensity without a need for capital investment for a dedicated instrument.

Oryzon Genomics SA: Spain’s Oryzon was awarded the Seal of Excellence, a quality label awarded by the European Commission, and a non-refundable public grant of €1.87 million. The funding will support Oryzon’s project entitled “Development of new treatments against Acute Myeloid Leukemia (AML) using iadademstat, an epigenetic drug” for 30 months. The overall goal of this project is to supplement the execution costs of iadademstat’s ongoing FRIDA clinical study and develop the formulation and manufacturing processes for industrial production and worldwide supply of iadademstat.

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