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]).

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"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.

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]).

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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.

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.

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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.

On August 2, 2021 Context Therapeutics Inc. (the "Company"), a women’s oncology company developing advanced small molecule and immunotherapy treatments to transform care for hormone-driven breast and gynecological cancers, and Tyligand Bioscience, Ltd. ("Tyligand"), a leader in small molecule drug discovery and development, reported the successful completion of ONA-XR (onapristone extended release) manufacturing process optimization pursuant to the collaboration agreement entered into between the parties in March 2020 (Press release, Context Therapeutics, AUG 25, 2021, View Source [SID1234586868]).

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ONA-XR is an orally administered, potent and specific antagonist of the progesterone receptor (PR) being evaluated in the clinic for PR-positive (PR+) breast, ovarian and endometrial cancers.

In connection with this successful optimization, Context and Tyligand entered into a license agreement whereby Tyligand was granted the exclusive right and sole responsibility for the development and commercialization of ONA-XR in China, Hong Kong and Macau (the "Territory"), and Context is eligible to receive royalties on net sales of ONA-XR in the Territory. Context retains rest of world rights to commercialize ONA-XR.

"We are thrilled to announce this successful optimization work completed for ONA-XR and the execution of the license agreement with Tyligand," said Martin Lehr, CEO of Context. "This work represents another major step forward for Context’s ability to efficiently scale our manufacturing and clinical capacity to support the development and future commercialization of ONA-XR to address the unmet need in treating women with PR+ cancers."

"Onapristone has an intriguing molecular structure, and it is no small feat to achieve such a significant improvement in its manufacturing process," said Peter Wuts, PhD, a pharmaceutical veteran and Senior Advisor to Context. "It is a testament to the drive and creativity of the Tyligand team of molecule builders and the power of chemistry in making better medicines."

"Tyligand and Context share the commitment of providing effective and affordable treatment options for cancer patients globally and this milestone brings us closer to realizing our goal," said Tony Zhang, PhD, CEO of Tyligand. "ONA-XR has the potential to be the first-in-class therapeutic agent specifically targeting progesterone receptors and we look forward to working with Context in further developing and commercializing this excellent molecule."

On August 25, 2021 XRad Therapeutics reported that first patient dosed in Phase 1a Clinical Trial of XRD-0394 for metastatic, locally advanced or recurrent solid tumours(Press release, Aptus Clinical, AUG 25, 2021, View Source [SID1234586867])

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Aptus Clinical is providing CRO support for this study

The dosing of the first patient into US based XRad Therapeutics’ Phase 1a study of XRD-0394 in metastatic, locally advanced or recurrent solid tumours, marks a major milestone for XRad and Aptus Clinical. For XRad, XRD-0394 is their first asset to reach clinical development, whilst for Aptus this is the first study utilising their US-based CRA team. As part of Aptus’ full-service delivery capability, the US CRAs, working alongside their UK headquartered clinical operations and data management colleagues, now provide significant geographical extension to Aptus’ current experience delivering early phase oncology, cell & gene therapy and rare disease clinical trials in in the UK and EU.

This Phase 1a trial will evaluate the safety and tolerability of single ascending doses of XRD-0394 in combination with radiation therapy. The trial is being conducted at two world renowned centres of excellence: Memorial Sloan Kettering Cancer Center in New York and the Stanford Cancer Institute in Palo Alto, California. XRD-0394 is a first-in-class, oral, dual kinase inhibitor of both ataxia-telangiectasia mutated and DNA-dependent protein kinase and based on preclinical studies is designed to enhance the effectiveness of radiation therapies. Results from this trial will inform the dosing regimen for the company’s planned Phase 1b study.

Tona Gilmer, PhD, President, Chief Executive Officer and co-founder of XRad said in a statement: "We are delighted to be working with our clinical CRO partners at Aptus who are managing the delivery of this vital first study in our development programme. Successful completion will allow us to rapidly move to the next step in our clinical development"

Jonathan Lewis, co-founder and Chief Commercial Officer at Aptus Clinical commented "We are thrilled that the XRad team entrusted the clinical delivery of their first asset to Aptus. Having worked initially with members of our consulting team, recognition of the strength and depth of experience in our operational delivery organisation has been a great endorsement of our capabilities. We look forward to working alongside the XRad team to demonstrate the full benefits of this important potential addition to the cancer treatment armamentarium"