United Therapeutics Corporation Reports Third Quarter 2021 Financial Results

On November 3, 2021 United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation with a purpose to provide a brighter future for patients, reported its financial results for the quarter ended September 30, 2021 (Press release, United Therapeutics, NOV 3, 2021, View Source [SID1234594158]). Total revenue in the third quarter of 2021 grew 17% year over year to $444.7 million, compared to $380.1 million in the third quarter of 2020.

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"Our momentum continues with growing patient use of our medicines, which are delivered by infusion, inhalation, and tablets," said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer of United Therapeutics. "I’m also very excited about the progress of our six phase 3 clinical trials across various forms of pulmonary hypertension and pulmonary fibrosis."

"We continue to add new Tyvaso patients following the approval in PH-ILD earlier this year, with approximately 4,000 U.S. patients on Tyvaso therapy at the end of the third quarter," said Michael Benkowitz, President and Chief Operating Officer of United Therapeutics. "Referrals for Tyvaso remain strong, and we’re well on our way toward our goal of reaching 6,000 U.S. patients on Tyvaso by the end of 2022."

THIRD QUARTER 2021 FINANCIAL RESULTS

Net product sales from our treprostinil-based products (Tyvaso, Remodulin, and Orenitram) grew by $46.1 million in the third quarter of 2021 compared to the third quarter of 2020. The growth in Tyvaso revenues resulted primarily from an increase in quantities sold, reflecting an increased number of patients following the pulmonary hypertension associated with interstitial lung disease (PH-ILD) label expansion. The growth in Orenitram revenues resulted from an increase in quantities sold, reflecting an increased number of patients and, to a lesser extent, a price increase. The growth in Unituxin revenues resulted from an increase in quantities sold and, to a lesser extent, price increases. Unituxin revenues in the third quarter of 2021 included $9.0 million related to the launch of Unituxin in Japan.

Expenses

Cost of product sales. The table below summarizes cost of product sales by major category (dollars in millions):

Research and development expense. The table below summarizes research and development expense by major category (dollars in millions):

Selling, general, and administrative expense. The table below summarizes selling, general, and administrative expense by major category (dollars in millions):

General and administrative, excluding share-based compensation. The increase in general and administrative expense for the three months ended September 30, 2021, as compared to the same period in 2020, was primarily due to: (1) an increase in legal expenses related to litigation matters; and (2) an increase in consulting expenses.

Share-based compensation. The table below summarizes share-based compensation expense (benefit) by major category (dollars in millions):

The increase in share-based compensation expense for the three months ended September 30, 2021, as compared to the same period in 2020, was primarily due to an increase in STAP expense driven by a three percent increase in our stock price for the three months ended September 30, 2021, as compared to a 17 percent decrease in our stock price for the same period in 2020, partially offset by a decrease in stock option expense due to fewer awards granted and outstanding in 2021.

Other expense, net. The change in other expense, net, for the three months ended September 30, 2021, as compared to the same period in 2020, was primarily due to an increase in net unrealized losses on equity securities.

Income tax expense. Income tax expense for the three months ended September 30, 2021 and 2020, was $41.7 million and $47.8 million, respectively. The effective income tax rate (ETR) for the three months ended September 30, 2021 and 2020 was 20 percent and 22 percent, respectively. The ETR for the three months ended September 30, 2021 decreased compared to the ETR for the three months ended September 30, 2020 primarily due to a decrease in valuation allowance, partially offset by increases in blended state income tax rates.

Non-GAAP Earnings

Non-GAAP earnings is defined as net income, adjusted for: (1) share-based compensation expense (benefit); (2) impairment charges; (3) unrealized gain on an investment in a privately-held company; (4) net changes in recurring fair value measurements; (5) license-related fees; (6) certain other costs incurred outside our normal course of business; and (7) tax impact on non-GAAP earnings adjustments.

A reconciliation of net income to non-GAAP earnings is presented below (in millions, except per share data):

PRODUCT COMMERCIALIZATION UPDATE

In 2021, we have launched one new product and one new product indication. In February 2021, we launched commercial sales of the Remunity Pump for Remodulin, and in April 2021, we launched a label expansion for Tyvaso to include an indication for PH-ILD following approval by the U.S. Food and Drug Administration (FDA) on March 31, 2021.

Remunity Pump for Remodulin. In February 2021, we launched sales of the Remunity Pump for Remodulin. The Remunity Pump is a pre-filled, semi-disposable system for subcutaneous delivery of treprostinil. The system consists of a small, lightweight, durable pump and separate controller. The pump uses disposable cartridges filled with Remodulin, which can be connected to the pump with less patient manipulation than is typically involved in filling other currently-available subcutaneous pumps.

Tyvaso Inhalation Solution in PH-ILD. The FDA approved Tyvaso for the PH-ILD indication on March 31, 2021, and we launched commercial efforts for the new indication shortly thereafter.

Tyvaso DPI. In April 2021, we submitted a new drug application (NDA) for Tyvaso DPI for pulmonary arterial hypertension (PAH) and PH-ILD indications. In October 2021 we received a complete response from the FDA noting a single deficiency preventing approval of Tyvaso DPI, related to an open inspection issue at a third-party facility that performs analytical testing of treprostinil drug substance. The FDA did not cite any deficiencies or issues related to operations performed at any United Therapeutics or MannKind Corporation facility for manufacturing, testing, and packaging of treprostinil drug substance, finished Tyvaso DPI drug product, or its associated device. All other requests from the agency have been addressed. We believe that the single deficiency identified in the complete response will be resolved quickly and that Tyvaso DPI can be approved by the summer of 2022, if not earlier.

Our Tyvaso DPI NDA includes the results of two clinical studies we conducted of Tyvaso DPI. One was a study in healthy volunteers, comparing the pharmacokinetics of Tyvaso DPI to Tyvaso Inhalation Solution. The study was completed in October 2020, and demonstrated comparable systemic treprostinil exposure between Tyvaso DPI and Tyvaso Inhalation Solution. In December 2020, we completed a clinical study (called BREEZE), which evaluated the safety and pharmacokinetics of switching PAH patients from Tyvaso Inhalation Solution to Tyvaso DPI. The BREEZE study demonstrated the safety and tolerability of Tyvaso DPI in subjects with PAH transitioning from Tyvaso Inhalation Solution, and comparable systemic treprostinil exposure between Tyvaso DPI and Tyvaso Inhalation Solution.

RESEARCH AND DEVELOPMENT UPDATE

Updates on select later-stage programs are below.

Tyvaso in chronic fibrosing interstitial lung diseases — TETON. We are enrolling a phase 3 study called TETON, which is a U.S. study of Tyvaso in patients with idiopathic pulmonary fibrosis (IPF). The primary endpoint of this study is the change in absolute forced vital capacity (FVC) from baseline to week 52. We are planning an additional phase 3 study of Tyvaso in IPF patients that will be similar to TETON, but will be conducted outside the United States.

The TETON program was prompted by data from the INCREASE study, which demonstrated improvements in certain key parameters of lung function in pulmonary hypertension patients with fibrotic lung disease. Specifically, in the INCREASE study, treatment with Tyvaso resulted in significant improvements in percent predicted FVC at weeks 8 and 16, with subjects having underlying etiologies of idiopathic interstitial pneumonias showing greater improvement. Consistent positive effects were also observed in patients with chronic hypersensitivity pneumonitis and environmental/occupational lung disease. These data points, combined with substantial preclinical evidence of antifibrotic activity of treprostinil, suggest that Tyvaso may offer a treatment option for patients with fibrotic lung disease.

Tyvaso in PH-COPD — PERFECT. Enrollment is ongoing for the phase 3 PERFECT study evaluating Tyvaso in patients with WHO Group 3 pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD). In a 30-week crossover study, 136 subjects will be randomized between inhaled treprostinil and placebo for a 26-week treatment period. The primary endpoint of the study is the change in 6MWD from baseline to week 12.

Ralinepag phase 3 clinical studies — ADVANCE CAPACITY and ADVANCE OUTCOMES. We are enrolling two phase 3 clinical studies to support the potential approval of oral ralinepag for PAH.

INDUCEMENT RESTRICTED STOCK UNITS

On October 29, 2021, we granted a total of 1,040 restricted stock units under our 2019 Inducement Stock Incentive Plan to five newly hired employees. These restricted stock units vest in three equal installments on October 31, 2022, 2023, and 2024, assuming continued employment on such dates, and are subject to the standard terms and conditions we filed with the SEC as Exhibit 10.2 to our Current Report on Form 8-K on March 1, 2019. We are providing this information in accordance with Nasdaq Listing Rule 5635(c)(4).

WEBCAST

We will host a webcast to discuss our third quarter 2021 financial results on Wednesday, November 3, 2021, at 9:00 a.m. Eastern Time. The webcast can be accessed live via our website at View Source A replay of the webcast will also be available at the same location on our website.

iOnctura to Share Preclinical Data on IOA-289 at SITC in November

On November 2, 2021 iOnctura SA, a clinical stage oncology company targeting core resistance and relapse mechanisms at the tumor-stroma-immune interface, reported that it will present preclinical data on its next generation autotaxin inhibitor, IOA-289, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting taking place on November 10–14, 2021 in Washington, D.C. and virtually (Press release, iOnctura, NOV 2, 2021, View Source [SID1234640244]).

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IOA-289 will be the first autotaxin inhibitor to be clinically investigated in oncology. It is currently being evaluated in healthy volunteers as a lead-in to a Phase I clinical study in pancreatic cancer.

The presentation at SITC (Free SITC Whitepaper) is entitled "A novel autotaxin inhibitor, IOA-289, modulates tumor, immune and stromal cell function and has monotherapy activity in fibrotic cancer models." The poster will be presented on Saturday November 13th between 7.00 and 8.30 am EST (13.00-14.30 CET), 12.30-2.00 pm EST (18.30-20.00 CET) and 7.00-8.30 pm EST (01.00-02.30 CET on November 14). It will be available for viewing on the SITC (Free SITC Whitepaper) virtual meeting platform from 7.00 am EST (13.00 CET) on Friday November 12, 2021 until the close of the platform on January 9, 2022.

Contacts

iOnctura
Catherine Pickering
Chief Executive Officer
T : +41 79 952 72 52
E: [email protected]

Press Relations
Jeremy Nieckowski
LifeSci Advisors
T: +41 79 699 97 27
E: [email protected]
iOnctura SA is clinical stage oncology company targeting core resistance and relapse mechanisms at the tumor-stroma-immune interface. iOnctura’s best-in-class drug development programs combine immune-mediated and direct anti-tumour activity to deliver molecules with superior clinical efficacy and safety in oncology. Its lead program, IOA-244 is the only semi-allosteric PI3Kdelta specific, orally dosed, small molecule inhibitor that is being developed in solid and hematological malignancies to address tumor and stroma induced immune suppression. IOA-244 is currently in Part B of a Phase 1 study. iOnctura’s second program, IOA-289, is an oral small molecule that inhibits the cross-talk between the tumor and its stroma and is in a Phase 1 clinical study. iOnctura is backed by blue chip investors including M Ventures, Inkef Capital, VI Partners, Schroders Capital, and 3B Future Health Fund. For more information, please visit www.ionctura.com

IOA-289, originally licensed from Cancer Research UK, is iOnctura’s second clinical compound, a next generation oral small molecule autotaxin inhibitor that is currently being investigated in the healthy volunteer stage of the AION 01 trial, a phase 1 clinical study in pancreatic cancer. iOnctura has undertaken extensive validation of the autotaxin inhibition mechanism in multiple preclinical solid tumor models.

Carcell Biopharma closes an oversubscribed financing round led by Hyfinity Investments

On November 1, 2021 Carcell Biopharma reported the close of an over-subscribed financing (Press release, Carcell Biopharma, NOV 2, 2021, View Source [SID1234637814]). The round was led by Hyfinity Investments, with participation from BioTrack Capital, Legendstar Capital and other investors.

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Carcell Biopharma is a pre-clinical biotech company incubated by EVX Ventures. Following a "platform of platforms" model, Carcell’s vision is to house multiple platform technologies that will lead to next-generation cell and gene therapies. Two lead platforms have been established around engineered red blood cells for immunomodulation and non-viral gene delivery using lipid nanoparticles. The company is founded by XQ Lin, the chairman and founder of EVX Ventures and Dr. Jiahai Shi, Associate Professor at the City University of Hong Kong. Joining the founding team are Lanlin Wu, the former CEO of Dendreon China, together with other biotech industry veterans with previous work experience at marquee biotech companies in Boston and Singapore.

Carcell is powered by a top-notch R&D team in Singapore and has plans to establish a site in Boston in the near future. The funds will also be used to construct a 6000 sqm integrated R&D and manufacturing centre in Shanghai, supporting the pre-clinical development of the drug candidates towards IND-enabling studies. Operating through the Singapore-Shanghai-Boston axis, Carcell leverages all the advantages from each region in the discovery, manufacturing, and clinical phases of drug development.

XQ Lin, Chairman and founder of Carcell Biopharma, commented, "Carcell builds on EVX Ventures’ global track record in incubating novel modality platform companies. Our innovative business model will enable us to accelerate the development of innovative medicines to address unmet medical needs."

Dr. Xin He, Managing Partner and founder of Hyfinity Investments, stated, "Hyfinity is glad to support Carcell financially and also in assembling a world class team in China. The company is a rare combination of a strong leadership team, with a solid foundation in engineered red blood cell technologies, and in non-viral delivery know-how. We look forward to being a long term investor, supporting the leadership team in their efforts of building a globally competitive cell and gene therapy platform company."

OncoResponse Announces Initiation of Phase 1 Trial of OR2805 – Human Monoclonal Antibody Derived from Elite Responder to Cancer Immunotherapy – in Patients with Advanced Cancer

On November 2, 2021 – OncoResponse, a clinical-stage biotech company advancing immunotherapies derived from the immune systems of elite cancer responders, reported initiation of a Phase 1/2 study of OR2805 in patients with advanced cancer. OR2805 is a fully human monoclonal antibody identified from an elite cancer responder using OncoResponse’s proprietary B-cell discovery platform (Press release, OncoResponse, NOV 2, 2021, View Source [SID1234609566]).

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The study will evaluate the safety, pharmacokinetics and preliminary anti-cancer activity of OR2805 in patients with advanced solid tumors alone and in combination with anti-PD-1 therapy. The trial will include a dose escalation phase, followed by several expansion cohorts. The role of potential biomarkers will be evaluated throughout the study, and more intensively in a separate biology cohort. ClinicalTrials.gov identifier: NCT05094804

"The initiation of our Phase 1 clinical trial of OR2805 represents a significant milestone for OncoResponse. OR2805 has the potential to reverse the inhibitory effect of tumor associated macrophages, or TAMs, and unlock a potent anti-tumor response," said Bob Lechleider, MD, Chief Medical Officer of OncoResponse. "Preclinical studies have demonstrated the ability of OR2805 to effectively counter the inhibitory activity of M2 macrophages, and we now intend to determine how that will translate to clinical activity. OncoResponse is dedicated to bringing novel and effective therapies that modulate the tumor microenvironment into the clinic to help patients who desperately need additional treatment options."

OR2805 is a fully human antibody discovered using B cells derived from an elite responder to checkpoint inhibitor (CPI) therapy. This antibody binds to CD163 which is highly expressed on tumor associated macrophages (TAMs) that create an immunosuppressive tumor microenvironment and inhibit anti-tumor T-cell responses. High frequency of CD163-expressing TAMs generally predicts an unfavorable prognosis in solid tumors.improve anti-tumor T-cell responses, the immunosuppression of TAMs, as a therapeutic strategy for monotherapy and in combination with CPI.

OncoResponse recently highlighted platform and preclinical data on OR2805 in an oral presentation "Using the Human Immune System to Identify Antibodies that Modulate the Tumor Microenvironment" at the Macrophage-Directed Therapies Summit. The presentation, by Kamal D. Puri, PhD, Chief Scientific Officer, can be accessed from the Publications page of the OncoResponse website.

BioTheryX Receives IND Clearance from FDA to Proceed with Phase 1 Study of BTX-1188, a Molecular Glue, for the Treatment of Hematologic and Solid Malignancies

On November 2, 2021 BioTheryX, Inc., a clinical-stage company focused on targeted protein degradation to create life-saving medicines, reported that the U.S. Food and Drug Administration (FDA) cleared the company to proceed with a Phase 1 clinical trial of BTX-1188 in hematologic and solid malignancies (Press release, BioTheryX, NOV 2, 2021, View Source [SID1234596452]).

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BTX-1188 is a novel oral small molecule cereblon binder that has immunomodulatory properties and promotes the degradation of a neosubstrate G1 to S phase transition 1 (GSPT1), a translation termination factor, as well as IKZF1/3, zinc finger transcription factors important in hematological function. By degrading multiple proteins including GSPT1 and IKZF1/3, BTX-1188’s profile is expected to be differentiated from protein degraders that exclusively target GSPT1. BTX-1188 has demonstrated promising preclinical activity against a variety of leukemias and solid tumors.

"The FDA clearance to begin our Phase 1 study for BTX-1188 is a major milestone for BioTheryX, which represents our first molecular glue to enter clinical development, and the second IND clearance for our pipeline," said Zung Thai, M.D., Ph.D., Chief Medical Officer of BioTheryX. "We plan to study BTX-1188 in patients with hematological and solid malignancies who have limited treatment options and for whom new therapies are desperately needed. We expect to initiate patient enrollment in the Phase I study by year end."

The Phase I clinical trial is designed to assess the safety and tolerability of BTX-1188 and to determine the recommended Phase 2 dose. Following determination of the recommended Phase 2 dose, the company plans to further evaluate the safety and efficacy of BTX-1188 in expansion cohorts in both hematological and solid tumor malignancies.