On May 11, 2022 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the first quarter ended March 31, 2022 and provided a business update (Press release, Ascendis Pharma, MAY 11, 2022, View Source [SID1234614212]).

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"This continues to be a transformative time for Ascendis as we build on positive Phase 3 data for our first two endocrinology rare disease programs – TransCon hGH and TransCon PTH – and our first product approval and launch," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "2022 marks a key transition point for Ascendis. We have all the elements of success in place – a proven technology and algorithm for product innovation, the right capabilities, and a strong balance sheet that allows us to deliver on all components of our Vision 3×3 as we work to achieve sustainable growth through multiple approaches and long-term profitability."

Company Highlights & Progress

TransCon hGH:
TransCon hGH is commercially available in the U.S. under the brand name SKYTROFA (lonapegsomatropin-tcgd) as the only FDA-approved once-weekly treatment for pediatric patients one year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone. As of April 29, 2022, 1,231 unique patient prescriptions for SKYTROFA have been written by 404 prescribers and processed through Ascendis’ patient hub called the Ascendis Signature Access Program. Nearly 50% of the prescribers have written prescriptions for more than one patient.
Coverage for SKYTROFA continues to increase, with approximately 45% of U.S. lives covered at the end of April per MMIT, compared to approximately 36% at the end of February.
In January 2022, the Company received marketing authorization for Lonapegsomatropin Ascendis Pharma (developed under the brand name of TransCon hGH) in the European Union as a once-weekly subcutaneous injection for the treatment of children and adolescents ages 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone.
On track for FDA protocol submission during the second quarter of 2022 to evaluate TransCon hGH for Turner Syndrome.
The ongoing conflict in the region surrounding Ukraine, Russia, and Belarus has impacted our ability to conduct clinical trials or to treat and evaluate patients there. As a result, we have shifted focus for our foresiGHt Trial in adult growth hormone deficiency to other countries and are now targeting completion of enrollment during the fourth quarter of 2022.
TransCon PTH:
On March 13, 2022, we announced top-line data from a randomized, double-blind, placebo-controlled Phase 3 Trial (PaTHway) of TransCon PTH in adults with hypoparathyroidism (HP), demonstrating statistically significant improvement with TransCon PTH compared to control for the primary composite endpoint and all key secondary endpoints. As of May 1, 2022, all 79 patients continued in the open-label extension portion of the PaTHway Trial.
On track for planned U.S. NDA submission during the third quarter of 2022 and expected EU MAA submission during the fourth quarter of 2022.
After more than two years of treatment in the open-label extension portion of the PaTH Forward Trial, as of May 1, 2022, 57 out of 59 original subjects continue in the trial.
In April 2022, enrollment completed in the Phase 3 PaTHway Japan Trial. Top-line results expected later this year.
TransCon CNP:
Top-line data from the ACcomplisH Trial, a Phase 2 randomized, double-blind, placebo-controlled clinical trial in North America, Europe, and Oceania in children ages 2-10 years with achondroplasia are expected in the fourth quarter of 2022.
TransCon TLR7/8 Agonist:
Enrollment continues in transcendIT-101, a Phase 1/2 study of TransCon TLR7/8 Agonist with or without pembrolizumab in patients with advanced or metastatic solid tumors. We expect top-line monotherapy and combo-therapy dose escalation data during the third quarter of 2022.
TransCon IL-2 β/γ
The Phase 1/2 IL-βelieγe Trial evaluating TransCon IL-2 β/γ monotherapy in patients with locally advanced or metastatic solid tumors continues to enroll patients. Top-line data are expected in the fourth quarter of 2022.
On track to dose the first patient in checkpoint combination dose-escalation arm of the IL-βelieγe Trial in the second quarter of 2022.
TransCon TLR7/8 Agonist and TransCon IL-2 β/γ Combination:
We plan to submit an IND or similar for Phase 2 cohort expansion for TransCon TLR7/8 Agonist and TransCon IL-2 β/γ during the fourth quarter of 2022.
Ended the first quarter of 2022 with cash, cash equivalents, and marketable securities totaling €1,065 million.
First Quarter 2022 Financial Results

Total revenue for the first quarter was €6.8 million, an increase of €6.1 million compared to €0.7 million in the same quarter of 2021. Revenues for the first quarter include U.S. commercial SKYTROFA sales, sales of clinical supplies, rendering of services, and recognition of internal profit from the license agreements with VISEN. The increase in revenue was primarily attributable to the €1.9 million commercial SKYTROFA sales, and €3.9 million higher sales of clinical supply to VISEN compared to the same period last year.

Research and development (R&D) costs for the first quarter were €83.2 million compared to €88.1 million during the same period in 2021. Following FDA approval of SKYTROFA (lonapegsomatropin-tcgd) in August 2021, commercial manufacturing is recognized as inventory while such costs were recognized as R&D costs prior to the FDA approval.

Selling, general, and administrative (SG&A) expenses for the first quarter were €47.4 million compared to €37.2 million during the same period in 2021. Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel costs as well as an increase in commercial costs.

Net loss of associate was €4.9 million in the first quarter, compared to a net profit of €28.1 million during the same period in 2021.

Net finance income was €7.6 million in the first quarter compared to €33.6 million in the same period in 2021.

For the first quarter of 2022, Ascendis Pharma reported a net loss of €125.5 million, or €2.21 per share (basic and diluted) compared to a net loss of €62.8 million, or €1.17 per share (basic and diluted) for the same period in 2021.

As of March 31, 2022, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €1,065 million compared to €790 million as of December 31, 2021. As of March 31, 2022, Ascendis Pharma had 56,958,391 ordinary shares outstanding.

Conference Call and Webcast Information

Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its first quarter 2022 financial results. Details include:

A live webcast of the conference call will be accessible from the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A replay of the webcast will be available on this website shortly after conclusion of the event for 30 days.

On May 11, 2022 Amber Specialty Pharmacy reported that they will begin dispensing LENVIMA (lenvatinib). Used by itself, LENVIMA treats differentiated thyroid cancer (DTC) when it can no longer be treated with radioactive iodine, and hepatocellular carcinoma (HCC) when it cannot be removed by surgery (Press release, Amber Specialty Pharmacy, MAY 11, 2022, View Source [SID1234614245]). LENVIMA is used with other medications to treat additional types of cancers, including advanced renal cell carcinoma (RCC) and advanced endometrial carcinoma. LENVIMA is manufactured by Eisai.

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Amber Specialty Pharmacy’s high-touch service model will support patients, caregivers, and oncology specialists throughout the country. Their Oncology Center of Excellence provides an enhanced level of care throughout a patient’s treatment journey.

"Supporting patients who are navigating a cancer diagnosis is one of the most important things we do," said Kristin Williams, president of Amber Specialty Pharmacy. "We are honored Eisai recognizes the value our whole-health approach offers oncology patients. From reducing or eliminating financial barriers, to one-on-one counseling with our registered dietitian – and everything in between – oncology professionals can trust Amber Specialty Pharmacy to make sure patients have the medicine and support they deserve."

On May 11, 2022 Scandion Oncology (Scandion), a biotech company developing first-in-class medicines aimed at treating cancer which is resistant to current treatment options, reported ramps up its clinical development capabilities with the appointment of Dr. Alfredo Zurlo as Chief Medical Officer (CMO) (Press release, Scandion Oncology, MAY 11, 2022, View Source,c3564960 [SID1234614185]). Alfredo Zurlo joins Scandion immediately as part of the company’s Executive Management-team.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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A medical doctor by training, Alfredo Zurlo has more than 20 years of experience in planning and conducting early and late-stage clinical programs in oncology (cancer). He has held several global management positions in a seven-year long career with Roche, including serving as International Medical Director of Oncology and heading the launch of bevacizumab (Avastin) in Europe and the rest of the world for the colorectal indication.

Following his tenure at Roche, Alfredo Zurlo has served as CMO in the two biotech companies Glycotope and Mologen. In addition to his extensive strategic and operational clinical trial experience, he brings a large international network of health care professionals, research scientists and industry experts to Scandion.

"I am delighted to welcome Alfredo Zurlo to Scandion. As we develop both our company and development pipeline, most notably our lead asset SCO-101, it is critical to further strengthen our capabilities in clinical development. With his experience in the field of cancer drug development, Alfredo is an excellent addition to our team, and he will play a key role in the execution of our clinical trials and the planning of pivotal development", says Bo Rode Hansen, President & CEO of Scandion.

"Scandion has a tremendous potential to help address cancer’s resistance to existing treatments, which is perhaps the biggest problem in modern cancer treatment. I am excited by the opportunity to join this company and contribute to the development of new treatments to the benefit of patients, their relatives, health care professionals and society", says Alfredo Zurlo, CMO at Scandion.

Alfredo Zurlo replaces Peter Michael Vestlev, who will continue to work for Scandion in the position of Distinguished Medical Scientist, ensuring an effective handover to Alfredo Zurlo.

On May 11, 2022 TRACON Pharmaceuticals, Inc. (Nasdaq:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with companies to develop and commercialize innovative products in the U.S., reported financial results for the first quarter ended March 31, 2022 (Press release, Tracon Pharmaceuticals, MAY 11, 2022, View Source [SID1234614213]). The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time.

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"We are pleased with the pace of enrollment under the amended ENVASARC protocol and remain on track to deliver interim efficacy data in the second half of this year," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Additionally, with the arbitration hearing now completed we look forward to the outcome of the binding arbitration for the TJ4309 and Bispecific Antibody agreements during the second half of this year. We also continue preparations to initiate dosing of the Phase 1/2 clinical trial of envafolimab with our potential best-in-class CTLA-4 antibody, YH001, as well as doxorubicin chemotherapy in the second half of 2022."

Recent Corporate Highlights

In March, we announced the amended ENVASARC protocol using a higher dose of envafolimab was approved by the FDA.

In April, we announced the amended ENVASARC protocol was approved by internal review boards or ethic committees at each of the 30 clinical sites in the U.S. and U.K. and that all sites were open for enrolment, with more than 10 patients enrolled.
Expected Key Upcoming Milestones

Two interim safety reviews and the interim efficacy data review by the ENVASARC Independent Data Monitoring Committee (IDMC) in the second half of 2022.

Initiate dosing of a Phase 1/2 clinical trial of envafolimab with our potential best in class CTLA-4 antibody YH001 as well as with doxorubicin chemotherapy in the second half of 2022.

Report the International Chamber of Commerce (ICC) Arbitration Panel’s binding decisions on the legal disputes involving the TJ4309 and bispecific antibody agreements with I-Mab this year.

Complete the TJ4309 Phase 1 trial permitting I-Mab the opportunity to terminate the license for $9M this year.

Initiate dosing of a randomized Phase 2 trial of TRC102 in locally advanced non-small cell lung cancer sponsored and funded by the National Cancer Institute this year.
First Quarter 2022 Financial Results

Cash, cash equivalents and short-term investments were $16.6 million at March 31, 2022, compared to $24.1 million at December 31, 2021. The Company expects that its current cash and cash equivalents will fund operations into 2023.

Research and development expenses for the first quarter of 2022 were $3.0 million, compared to $2.3 million for the first quarter of 2021.

General and administrative expenses for the first quarter of 2022 were $6.5 million, compared to $2.7 million for the first quarter of 2021. The increase was primarily attributable to legal expenses incurred due to the arbitration hearing held in February 2022 with I-Mab related to the TJ4309 and bispecific antibody agreements, and the Company expects general and administrative expenses to decrease significantly for the remainder of the year.

Net loss for the first quarter of 2022 was $9.5 million, compared to $5.1 million for the first quarter of 2021.
Conference Call Details

A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab (KN035), a single-domain antibody against PD-L1 invented by Alphamab Oncology, is the first approved subcutaneously injected PD-(L)1 inhibitor. Envafolimab was approved by the Chinese NMPA in November 2021 in adult patients with MSI-H/dMMR advanced solid tumors who failed systemic treatment and have no satisfactory alternative treatment options. In December 2019, Alphamab Oncology, 3D Medicines and TRACON entered into a collaboration whereby TRACON has the right to develop and commercialize envafolimab in soft tissue sarcoma in North America. Envafolimab is currently being studied in the pivotal ENVASARC Phase 2 trial in the United States sponsored by TRACON and a Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at 30 top cancer centers in the United States and the United Kingdom that began dosing in December 2020. TRACON expects the trial to enroll more than 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into a cohort of treatment with single agent envafolimab at 600 mg every three weeks and 80 patients enrolled into a cohort of treatment with envafolimab at 600 mg every three weeks with Yervoy. The primary endpoint is objective response rate by central review with duration of response a key secondary endpoint.

About YH001

YH001 is an IgG1 antibody against CTLA-4 that has shown enhanced antibody dependent cellular cytotoxicity (ADCC) and complement dependent cytotoxicity (CDC) in vitro. In preclinical studies YH001 demonstrated superior T cell activation and superior tumor growth inhibition activity compared to ipilimumab. YH001 also demonstrated superior activity compared to ipilimumab in human transgenic mouse tumor models when combined with a PD-(L)1 antibody. In these models, single agent YH001 depleted regulatory T cells and increased CD8+ T cells in tumor tissue. YH001 is being dosed as a single agent in a Phase 1 trial in China (NCT04699929) and in combination with the PD-1 antibody toripalimab in a Phase 1 trial in Australia (NCT04357756).

About TRC102

TRC102 (methoxyamine) is a novel, small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA) and has orphan drug designation from the FDA in malignant glioma, including glioblastoma.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in an ongoing Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.

On May 11, 2022 Cleveland Diagnostics, Inc., a clinical-stage biotechnology company developing next-generation diagnostic tests for the early detection of cancers, reported that its prostate cancer test, IsoPSA, has been added to the National Comprehensive Cancer Network (NCCN) Guidelines for Prostate Cancer Early Detection (version 1.2022) (Press release, Cleveland Diagnostics, MAY 11, 2022, View Source [SID1234614246]). The NCCN panel now recommends the use of IsoPSA as an option to further define the probability of high-grade prostate cancer (Gleason score ≥ 3+4, Grade Group 2 or higher) prior to a first biopsy or after a negative biopsy.

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"We are honored that NCCN, an organization that provides valuable guidelines based on the latest evidence and expert consensus, now includes our IsoPSA test in its guidelines for prostate cancer early detection," said Arnon Chait, Ph.D., Chief Executive Officer at Cleveland Diagnostics. "The NCCN’s new recommendation highlights years of extensive clinical validation and clinical utility studies that all point to how IsoPSA could change the way prostate cancer is diagnosed and subsequently treated."

"Most urologists follow NCCN prostate cancer guidelines as a standard in the diagnosis and treatment of the disease," said Mark Stovsky, M.D., Chief Medical Officer at Cleveland Diagnostics. "We are pleased to have IsoPSA included in these guidelines as it will give urologists more insight into the best course of action for patients for which they are evaluating for prostate cancer."

IsoPSA is a non-invasive, blood-based test that has demonstrated in large, multicenter studies superior diagnostic accuracy and clinical utility when compared to prostate-specific antigen (PSA), the current standard of care in prostate cancer, in men being considered for prostate biopsy.

"In the Introduction section of the new guidelines, the NCCN panel recognizes that maximizing early detection of prostate cancer will increase the detection of both indolent (slower-growing) and aggressive (faster-growing) prostate cancers," adds Bob Rochelle, Chief Commercial Officer at Cleveland Diagnostics. "The panel also describes a goal of minimizing immediate treatment or overtreatment of indolent cancers by accurately characterizing the biology of detected cancer, and that there are some indolent cancers and benign prostate conditions that can be monitored rather than immediately treated. This is precisely the rationale for our IsoPSA test."