On November 11, 2020 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported financial results for the quarter ended September 30, 2020 (Press release, Ascendis Pharma, NOV 11, 2020, View Source [SID1234570599]).

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"As Ascendis progresses toward closing out 2020, I am proud that we are on track or ahead of schedule for all 2020 corporate goals and to achieve Vision 3×3, moving us one step closer to becoming a fully integrated, global biopharmaceutical company," said Jan Mikkelsen, President and CEO. "As a company, we are driven by our core values to achieve our long-term strategic Vision 3×3, using the power of the TransCon technology. We push ourselves to continually deliver not only in endocrinology but also in oncology and to bring these products to patients as efficiently and safely as possible."

Corporate Highlights & Progress

TransCon hGH (lonapegsomatropin)
○ Submitted MAA to the EMA, which has now validated the application.
○ Filed a Clinical Trial Notification with the Pharmaceuticals and Medical Devices Agency in Japan to initiate the company’s phase 3 riGHt Trial of lonapegsomatropin for the treatment of pediatric GHD.
○ Announced the U.S. Food and Drug Administration (FDA) accepted the company’s Biologics License Application and set a PDUFA date for June 25, 2021.
TransCon PTH
○ Submitted regulatory filings to enable initiation of European and Canadian sites for phase 3 PaTHway Trial.
○ Received orphan designation by the European Commission (EC) for the treatment of hypoparathyroidism (HP).
○ Submitted an amendment to its IND with FDA for the PaTHway phase 3 clinical trial evaluating the safety, tolerability and efficacy of TransCon PTH in adults with HP.
○ Announced preliminary six-month results from the open-label extension portion of PaTH Forward, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH in adult subjects with HP. The preliminary results support potential use of TransCon PTH as a hormone replacement therapy for HP and demonstrated normalization of quality of life.
TransCon CNP
○ In collaboration with VISEN Pharmaceuticals, filed an IND application to initiate the phase 2 ACcomplisH China Trial.
○ Received orphan designation by the EC.
Oncology
○ On track for filing IND or similar for TransCon TLR7/8 Agonist by year-end. TransCon TLR7/8 Agonist is a long-acting prodrug of resiquimod. Administered as an intratumoral injection, TransCon TLR7/8 Agonist is designed to provide sustained release of unmodified resiquimod directly to the tumor.
Announced Mark A. Bach, M.D., Ph.D., as Senior Vice President of Clinical Development and Medical Affairs for Endocrinology Rare Diseases. Dr. Bach has 30 years of experience in clinical research and development.
Ended the third quarter 2020 with cash, cash equivalents and marketable securities totaling €957.5 million.
Third Quarter 2020 Financial Results

For the third quarter, Ascendis Pharma reported a net loss of €121.7 million, or €2.31 per share (basic and diluted) compared to a net loss of €25.1 million, or €0.53 per share (basic and diluted) for the same period in 2019.

Revenue for the third quarter was €2.8 million compared to €2.2 million in the same quarter of 2019. The increase was due to a higher sale of clinical supply and services to VISEN, partly offset by a lower amount of license revenue being recognized.

Research and development (R&D) costs for the third quarter were €64.1 million compared to €46.3 million during the same period in 2019. Higher R&D costs in 2020 reflect an increase in personnel-related costs, expansion of R&D facilities and the continued progress in development of the company’s product candidates.

Selling, general and administrative (SG&A) expenses for the third quarter were €17.5 million compared to €10.0 million during the same period in 2019. The increase is primarily due to additional personnel-related costs, higher IT and other infrastructure costs and continued build of the company’s commercial capabilities.

As of September 30, 2020, Ascendis Pharma had cash, cash equivalents and marketable securities totaling €957.5 million compared to €471.6 million as of June 30, 2020. As of September 30, 2020, Ascendis Pharma had 53,416,091 ordinary shares outstanding.

Conference Call and Webcast information

Ascendis Pharma will host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss its third quarter 2020 financial results. Details include:

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 also be available on this website shortly after conclusion of the event for 30 days.

On November 11, 2020 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody technology platform, reported that Sean McCarthy, D.Phil., president, chief executive officer, and chairman, will participate in the following virtual healthcare conferences in November (Press release, CytomX Therapeutics, NOV 11, 2020, View Source [SID1234570597]).

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Jefferies Virtual London Healthcare Conference
Date: Wednesday, November 18, 2020
Presentation Time: 10:15a.m. ET

32nd Annual Piper Sandler Virtual Healthcare Conference
Date: Monday, November 30, 2020
Presentation Time: November 23, 2020 at 10:00 a.m. ET. The fireside chat will be available for on-demand viewing before the conference.

Webcasts from both conferences will be available on the Events and Presentations page of CytomX’s website at www.cytomx.com. An archived replay will be available for 90 days following the events.

On November 11, 2020 Five Prime Therapeutics, Inc. (Nasdaq: FPRX), a clinical-stage biotechnology company focused on developing immune modulators and precision therapies for solid tumor cancers, reported that it plans to offer and sell, subject to market and other conditions, 5,000,000 shares of its common stock in an underwritten public offering (Press release, Five Prime Therapeutics, NOV 11, 2020, View Source [SID1234570596]). In connection with the offering, Five Prime intends to grant the underwriters in the offering a 30-day option to purchase up to an additional 750,000 shares of common stock offered in the public offering.

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Five Prime plans to use the net proceeds of the offering, together with other available funds, to fund ongoing clinical development of bemarituzumab and FPT155, to advance FPA157 through preclinical and into clinical development, to advance its late-stage research programs and for working capital and general corporate purposes.

Cowen and SVB Leerink are acting as joint book-running managers for the offering. Wedbush PacGrow is acting as co-manager for the offering.

The shares of common stock are being offered pursuant to a "shelf" registration statement previously filed with and declared effective by the Securities and Exchange Commission (SEC). A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the website of the SEC at www.sec.gov. When available, copies of the preliminary prospectus supplement and accompanying prospectus relating to the offering may be obtained from: Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, by telephone at (833) 297-2926 or by email at [email protected], or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132 or by e-mail at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. Any offer, if at all, will be made only by means of a prospectus supplement and accompanying prospectus, which are a part of the effective registration statement.

On November 11, 2020 MD Anderson reported that Promising clinical results with combination treatments for patients with melanoma and lung cancer highlight immunotherapy advances being presented by researchers from The University of Texas MD Anderson Cancer Center at The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting & Pre-Conference Programs (SITC 2020) (Press release, MD Anderson, NOV 11, 2020, View Source [SID1234570595]).

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"The SITC (Free SITC Whitepaper) Annual Meeting provides us the opportunity to learn about and share groundbreaking new discoveries in tumor immunology and clinical advances for treating patients," said Giulio Draetta, M.D., Ph.D., chief scientific officer. "As we strive to expand the use of immunotherapy for more patients, we look forward to seeing these results presented at this year’s meeting."

Combination immunotherapy shows activity in metastatic melanoma (Abstract 420)

First-line treatment with a combination of nivolumab and interleukin-2 agonist bempegaldesleukin (BEMPEG) resulted in encouraging clinical activity for patients with metastatic melanoma in the Phase I/II PIVOT-02 trial. Results from the ongoing trial will be presented by Adi Diab, M.D., associate professor of Melanoma Medical Oncology.
Forty-one patients with previously untreated stage IV melanoma were enrolled on the trial, 38 of whom were able to be evaluated on the study. The treatment was well tolerated, as described in previous studies with this combination.

Twenty of the 38 efficacy evaluable patients (53%) had an overall response, or tumor shrinkage, and 13 (34%) had a complete response. Sixteen patients (80%) had ongoing responses and the median duration of response had not been reached at a follow-up of 29 months.

Median progression-free survival was 30.9 months, and median overall survival had not yet been reached.

"Despite the tremendous successes we have seen with immunotherapy in treating patients with advanced melanoma, current therapies are not effective for all patients. We have an unmet clinical need for new therapies that can provide prolonged benefit to more patients," said Diab. "We are encouraged by the safety profile and durable responses seen with this combination therapy, and we look forward to continued evaluation in Phase III studies, which are enrolling now."

Based on these results, the combination of BEMPEG and nivolumab was awarded Breakthrough Therapy designation by the U.S. Food and Drug Administration.

The study was supported by Nektar Therapeutics. A complete list of collaborating authors can be found within the abstract here.

Diab reports research support and consulting fees from both Nektar and Bristol Myers Squibb.

BEMPEG plus targeted therapy stimulates anti-tumor immune activation in advanced cancers (Abstract 368)

In the Phase I REVEAL trial for patients with advanced refractory solid tumors, combination treatment with BEMPEG and NKTR-262, a small-molecule agonist of Toll-like receptors (TLR) 7/8, led to activation of immune pathways associated with antitumor response. Results from the dose-escalation study will be presented by Diab.
NKTR-262 is designed to promote stimulation of an immune response in the tumor microenvironment, which can lead to antitumor activity when combined with BEMPEG in preclinical studies, explained Diab.

In 36 enrolled patients, the most frequent side effects were flu-like symptoms, fatigue, nausea and itching, all of which are consistent with the known safety profile of BEMPEG. One dose-limiting toxicity of elevated transaminase levels was observed at the highest dosage.

Two patients experienced partial responses, and seven patients had disease control (partial response or stable disease). Analysis of the immune response revealed increased activation of both T cells and natural killer (NK) cells in patients as well as of cytokines and interferon genes.

Based on these results, this combination will be evaluated in a Phase Ib dose-expansion with or without nivolumab for patients with advanced melanoma.

The study was supported by Nektar Therapeutics. A complete list of collaborating authors can be found within the abstract here.

Presurgical nivolumab and chemotherapy achieves high rates of tumor downstaging in patients with resectable lung cancer (Abstract 277)

Combining platinum-based doublet chemotherapy with nivolumab as a neoadjuvant, or presurgical, therapy for patients with stage I-IIIA non-small cell lung cancer (NSCLC) resulted in the highest rates of tumor downstaging at surgery, relative to other neoadjuvant regimens. Results of the study will be presented by Boris Sepesi, M.D., associate professor of Thoracic and Cardiovascular Surgery.
Patient cohorts on the study included 302 MD Anderson lung cancer patients treated with neoadjuvant platinum doublet chemotherapy and patients treated on the Phase II NEOSTAR study, led by Tina Cascone, M.D., Ph.D., assistant professor of Thoracic/Head & Neck Medical Oncology. NEOSTAR participants include 21 patients receiving nivolumab only, 16 patients receiving nivolumab plus ipilimumab and 22 patients receiving nivolumab with platinum doublet chemotherapy.
Among all cohorts, the combination of chemotherapy with nivolumab achieved the highest rates of clinical downstaging at 68%, compared to 38% in each additional cohort, a statistically significant difference.

"Maximizing the effectiveness of neoadjuvant therapy will help us to prevent relapse after surgery and improve outcomes for our patients," said Sepesi. "The robust clinical downstaging rates seen after platinum doublet chemotherapy in combination with nivolumab are encouraging in patients with operable non-small cell lung cancer, and we look forward to learning if there are survival benefits in long-term follow-up data."

This study was supported by Bristol Myers Squibb and MD Anderson’s Lung Cancer Moon Shot, part of the institution’s Moon Shots Program. A complete list of collaborating authors can be found within the abstract here.

On November 11, 2020 Pfizer Inc. (NYSE: PFE) reported that investors and the general public to listen to a webcast of a discussion with Angela Hwang, Group President, Biopharmaceuticals Group, at the 2nd Annual Wolfe Research Healthcare Conference on Wednesday, November 18, 2020 at 9:50 a.m. EST (Press release, Pfizer, NOV 11, 2020, View Source [SID1234570591]).

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To listen to the webcast, visit our web site at www.pfizer.com/investors. Information on accessing and registering for the webcast will be available at www.pfizer.com/investors beginning today.

Visitors will be able to listen to an archived copy of the webcast at www.pfizer.com/investors.