On February 24, 2022 United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, reported its financial results for the fourth quarter and year ended December 31, 2021 (Press release, United Therapeutics, FEB 24, 2022, View Source [SID1234608993]). Full year total revenues rose to $1,686 million, as U.S. patients being treated with the company’s treprostinil-based therapies reached an all-time high during the fourth quarter of 2021.

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"We continue to make strong progress with patient growth as we march toward our target of reaching 25,000 patients with our therapies by the end of 2025," said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer of United Therapeutics. "The recent transplants of our xenoheart and xenokidney products demonstrate the tremendous potential of our business model for the second half of the 2020s and beyond. Indeed, Mr. David Bennett, Sr., the first UHeart recipient, continues to have strong cardiovascular function seven weeks after his transplant."

"Our commercial teams continue to perform with double-digit percentage growth in revenue and patient counts in 2021 compared to 2020," said Michael Benkowitz, President and Chief Operating Officer of United Therapeutics. "Despite a setback with Tyvaso DPI, we remain on track to achieve 6,000 patients with Tyvaso by the end of 2022."

United Therapeutics recently received an information request letter from the U.S. Food and Drug Administration (FDA) requesting additional information regarding the pulmonary safety of Tyvaso DPI related to a pending Citizen’s Petition. United Therapeutics responded to the agency’s information request; however, the FDA has considered this response to be a major amendment to the NDA, extending FDA’s review deadline to May 2022.

FOURTH QUARTER AND FULL YEAR 2021 FINANCIAL RESULTS

(1) See definition of non-GAAP earnings, a non-GAAP financial measure, and a reconciliation of net income to non-GAAP earnings below.

Revenues

The table below summarizes the components of total revenues (dollars in millions):

Net product sales from our treprostinil-based products (Tyvaso, Remodulin, and Orenitram) grew by $23.5 million in the fourth quarter of 2021 compared to the fourth quarter of 2020. The growth in Tyvaso revenues resulted primarily from an increase in quantities sold, reflecting an increased number of patients following the expansion of the Tyvaso label to include pulmonary hypertension associated with interstitial lung disease (PH-ILD). The decrease in Remodulin revenues was primarily due to lower quantities sold in the U.S. The growth in Unituxin revenues primarily resulted from an increase in quantities sold. Unituxin revenues in the fourth quarter of 2021 included $6.3 million related to the launch of Unituxin in Japan.

Net product sales from our treprostinil-based products (Tyvaso, Remodulin, and Orenitram) grew by $134.2 million in 2021 compared to 2020. The growth in Tyvaso revenues resulted primarily from an increase in quantities sold, reflecting an increased number of patients following the PH-ILD label expansion and, to a lesser extent, price increases. The decrease in Remodulin revenues resulted from a decrease in U.S. Remodulin revenues of $28.9 million, partially offset by an increase in international Remodulin revenues of $25.9 million. The decrease in U.S. Remodulin revenues was primarily due to a decrease in quantities sold and, to a lesser extent, higher gross-to-net deductions. The increase in international Remodulin revenues was primarily due to reduced orders by an international distributor in 2020 in order to reduce its inventory as a result of the anticipated impact of generic competition. The growth in Unituxin revenues resulted from an increase in quantities sold and, to a lesser extent, price increases. Unituxin revenues in 2021 included $18.4 million related to the launch of Unituxin in Japan.

Cost of product sales, excluding share-based compensation. The increase in cost of product sales for the year ended December 31, 2021, as compared to the same period in 2020, was primarily attributable to shipments of the Remunity Pump following launch in February 2021.

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

Research and development expense, excluding share-based compensation. The decrease in research and development expense for the quarter ended December 31, 2021, as compared to the same period in 2020, was due to: (1) a decrease in milestone payments under our license and collaboration agreement with MannKind; (2) a decrease in pre-launch costs related to the Remunity Pump launch; and (3) a decrease in spending related to a facility we decided to repurpose in 2021.

The increase in research and development expense for the year ended December 31, 2021, as compared to the same period in 2020, was due to: (1) a $107.3 million IPR&D impairment charge related to our March 2021 decision to discontinue the U.S. development of Trevyent; (2) a $105.0 million purchase of a pediatric disease priority review voucher, which we redeemed upon submission of the Tyvaso DPI new drug application (NDA); and (3) an $11.6 million impairment charge related to repurposing one of our facilities. These increases were partially offset by a decrease in milestone payments under our license and collaboration agreement with MannKind and reduced costs following the completion of the phase 3 DISTINCT study of Unituxin in 2020.

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 year ended December 31, 2021, as compared to the same period in 2020, was primarily due to: (1) an increase in litigation expenses; and (2) an increase in consulting expenses.

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

The decrease in share-based compensation expense for the quarter and year ended December 31, 2021, as compared to the same periods in 2020, was due to a decrease in STAP expense driven by a 17 percent increase and a 42 percent increase in our stock price during the quarter and year ended December 31, 2021, respectively, as compared to a 50 percent increase and a 72 percent increase in our stock price for the quarter and year ended December 31, 2020, respectively. There was also a decrease in stock option expense for the year ended December 31, 2021 due to a reduction in awards granted and outstanding in 2021 compared to the same period in 2020. The decrease in share-based compensation expense for the year ended December 31, 2021 was partially offset by an increase in restricted stock unit expense.

Other (expense) income, net. The changes in other (expense) income, net for the quarter and year ended December 31, 2021, as compared to the same periods in 2020, were primarily due to the recognition of net unrealized and realized gains and losses on our investments in equity securities and net unrealized gains and losses on our contingent consideration assets.

Income tax expense. Income tax expense was $118.1 million for the year ended December 31, 2021, as compared to $124.1 million for the same period in 2020. For the years ended December 31, 2021 and 2020, our effective income tax rates (ETR) were approximately 20 percent and 19 percent, respectively. Our ETR for the year ended December 31, 2021 increased, as compared to our ETR for the year ended December 31, 2020, primarily due to increases in blended state income tax rates and decreases in tax credits, partially offset by a decrease in the valuation allowance on deferred taxes.

Non-GAAP Earnings

Non-GAAP earnings is defined as net income, adjusted for: (1) share-based compensation expense (including expenses relating to stock options, restricted stock units, share tracking awards, and our employee stock purchase plan); (2) unrealized gains on investments in privately-held companies; (3) impairment charges; (4) license-related fees; (5) net changes in recurring fair value measurements; (6) certain other costs incurred outside our normal course of business; and (7) tax impact on non-GAAP earnings adjustments.

(2) Recorded within other (expense) income, net in our consolidated statements of operations.

(3) For the quarter ended December 31, 2021, we recognized a $1.0 million impairment charge which was recorded within selling, general, and administrative in our consolidated statements of operations. For the year ended December 31, 2021, we recognized $134.9 million in impairment charges and recorded these charges within research and development, selling, general, and administrative, and impairments of investments in privately-held companies in our consolidated statements of operations. For the year ended December 31, 2020, we recognized impairment charges of $16.0 million and recorded these charges within impairments of investments in privately-held companies, selling, general, and administrative, and other (expense) income, net in our consolidated statements of operations.

(4) Recorded within research and development in our consolidated statements of operations.

(5) Net changes in the fair values of our contingent consideration liabilities were recorded within research and development in our consolidated statements of operations and net changes in all other recurring fair value measurements were recorded within other (expense) income, net in our consolidated statements of operations.

(6) For the year ended December 31, 2021, we expensed $105.0 million related to a pediatric disease priority review voucher and recorded the amount within research and development in our consolidated statements of operations.

PRODUCT COMMERCIALIZATION UPDATE

In 2021, we 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. 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 letter (CRL) 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 CRL noted, but did not cite as a deficiency, that the FDA had not yet completed its review of a Citizen Petition submitted to the FDA in July 2021 concerning the safety of an excipient in Tyvaso DPI.

We resubmitted our NDA in December 2021 and the FDA issued an action date for February 2022. In February 2022, the FDA requested additional information concerning the pulmonary safety of Tyvaso DPI related to a pending Citizen’s Petition. We responded to the FDA’s request, and the FDA indicated that our response constitutes a major amendment to the Tyvaso DPI NDA, which extends the FDA’s anticipated deadline to review the pending NDA to May 2022.

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 1 and TETON 2. We are enrolling a phase 3 study called TETON 1, 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 1, called TETON 2, 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 February 18, 2022, we granted a total of 570 restricted stock units under our 2019 Inducement Stock Incentive Plan to four newly hired employees. These restricted stock units vest in three equal installments on February 28, 2023, February 28, 2024, and February 28, 2025, 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 fourth quarter and full year 2021 financial results on Thursday, February 24, 2022, 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.

UNITED THERAPEUTICS: ENABLING INSPIRATION

We build on the strength of our research and development expertise and a distinctive, entrepreneurial culture that encourages diversity, innovation, creativity, sustainability, and, simply, fun. Since inception, our mission has been to find a cure for pulmonary arterial hypertension and other life-threatening diseases. Toward this goal we have successfully gained FDA approval for five medicines, we are always conducting new clinical trials, and we are working to create an unlimited supply of manufactured organs for transplantation.

We are the first publicly-traded biotech or pharmaceutical company to take the form of a public benefit corporation (PBC). Our public benefit purpose is to provide a brighter future for patients through (a) the development of novel pharmaceutical therapies; and (b) technologies that expand the availability of transplantable organs. At the same time, we seek to provide our shareholders with superior financial performance and our communities with earth-sensitive energy utilization.

You can learn more about what it means to be a PBC here: unither.com/PBC.

On February 24, 2022 NantHealth, Inc. (NASDAQ-GS: NH), a provider of enterprise solutions that help transform complex data into actionable insights, reported financial results for its fourth quarter and full year ended December 31, 2021 (Press release, NantHealth, FEB 24, 2022, View Source [SID1234609012]).

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"The past year was marked by a number of operational highlights, including the continuing launch of new products and services, ongoing efforts to build and expand our business capabilities and successfully completing a major refinancing," said Ron Louks, Chief Operating Officer, NantHealth. "As expected, our 2021 fourth quarter revenues increased from the third quarter, returning to the average quarterly run rate for the year. We are encouraged to have ended 2021 on an upswing, with solid customer interest across our entire portfolio of products and services, and a strengthened cash position.

"We believe we have built a strong foundation that positioned NantHealth for meaningful top line growth in the coming year. This belief is in part based on the progress of our recently launched Eviti Connect program for autoimmune diseases and the exciting business opportunities for our OpenNMS platform. In addition, we continue to see growing interest in our NaviNet suite of authorization solutions from payers and network service providers, which we attribute to the robust product offering and ongoing investments in developing new features and enhanced functionality."

Software and Services Q4 Highlights:

Clinical Decision Support (Eviti):
Signed and went live with Eviti Connect for Autoimmune Diseases program with Maryland Physicians Care (MPC), to provide digital drug authorizations for members living with chronic autoimmune conditions
Continued expansion of Eviti Connect for Oncology services provided through a key Eviti channel partner with the addition of the country’s largest customer-owned health insurer
Expanded utilization management certification to include the states of Kentucky, Florida and Massachusetts, enabling the company to provide Eviti Connect customers with fully delegated end-to-end services
Signed a new partner agreement that enables NantHealth to offer care management services for complex, high risk and chronic diseases, as previously stated
Payer Engagement (NaviNet):
Signed a multi-year agreement with a new third party administrator that will use NaviNet Open to enhance the services it provides to self-insured health plan customers, as previously reported
Recorded 17% year-over-year revenue growth for NaviNet AllPayer Advantage, a direct-to-provider solution
Launched the new NantHealth Partner Portal that offers payer customers self-service, on-demand access to interactive reporting on workflows, trends, analytics and insights
Enhanced NaviNet Open Authorizations to guide users through submission of clinical criteria information and supporting documentation with smart workflow technology and dynamic instructional messaging
Entered into an alliance agreement with Change Healthcare to integrate InterQual Connect automation solution with NaviNet Open, allowing NaviNet payer customers to leverage InterQual medical review criteria in their NaviNet-based electronic prior authorization workflow
Signed agreement with PriorAuthNow that enables it to leverage NaviNet Open Authorizations APIs to automate and streamline prior authorization requests for PriorAuthNow clients
Network Monitoring and Management (The OpenNMS Group, Inc.)
Partnered with a Fortune 500 managed service provider to offer OpenNMS Meridian monitoring to over 150 customers
Released OpenNMS Horizon 29 featuring improved streaming analytics for flow data at scale, and enhancements in distributed monitoring and security: OpenNMS now runs without elevated privileges reducing security risk
Launched OpenNMS Minion virtual appliance and cloud-enabled service to help organizations quickly, reliably, and securely deploy OpenNMS Minion collectors to remote or adjacent private networks
Rapidly delivered updates to address the global Log4j vulnerability, collaborating closely with the OpenNMS open-source community
Business and Financial Highlights:

For the 2021 fourth quarter:

Total net revenue was $16.0 million compared with $18.6 million in the 2020 fourth quarter.
Gross profit was $9.1 million, or 57% of total net revenue, compared with $11.4 million, or 61% of total net revenue, for the prior year period.
Selling, general and administrative (SG&A) expenses increased to $14.8 million compared with $11.7 million in the 2020 fourth quarter.
Research and development (R&D) expenses increased to $5.2 million from $4.8 million mainly from the Company’s ongoing investments in expanding its product offerings.
Net loss from continuing operations, net of tax, of $16.7 million, or $0.14 per share, significantly decreased from $20.1 million, or $0.18 per share, for the 2020 fourth quarter.
On a non-GAAP basis, net loss from continuing operations was $11.8 million, or $0.10 per share, compared with $6.2 million, or $0.06 per share, for the fourth quarter of last year.
For the 2021 full year:

Total net revenue was $62.6 million, compared with $73.2 million.
Gross profit was $34.8 million, or 56% of total net revenue, from $43.9 million, or 60% of total net revenue, for the prior year.
SG&A expense was $52.1 million compared with $48.5 million.
R&D expense increased to $19.7 million from $17.3 million.
Net loss from continuing operations, net of tax, was $58.3 million, or $0.51 per share, compared with $88.3 million, or $0.80 per share, for the 2020 full year.
On a non-GAAP basis, net loss from continuing operations was $41.9 million, or $0.37 per share, up from $27.0 million, or $0.24 per share, for 2020.
At December 31, 2021, cash and cash equivalents totaled $29.1 million.

Conference Call Information and Forward-Looking Statements

Later today, the company will host a conference call at 1:30 p.m. PT (4:30 p.m. ET) to review its results of operations for the fourth quarter and full year ended December 31, 2021. The conference call will be available to interested parties by dialing 877-407-0312 from the U.S. or Canada, or 201-389-0899. The call will be broadcast via the Internet at www.nanthealth.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary audio software. A playback of the call will be archived and accessible on the same website for at least three months.

Discussion during the conference call may include forward-looking statements regarding topics such as the company’s financial status and performance, regulatory and operational developments, and other comments the company may make about its future plans or prospects in response to questions from participants on the conference call.

Use of Non-GAAP Financial Measures

This news release contains references to Non-GAAP financial measures, including adjusted net loss and adjusted net loss per share, which are financial measures that are not prepared in conformity with United States generally accepted accounting principles (U.S. GAAP). The Company’s management believes that the presentation of Non-GAAP financial measures provides useful supplementary information regarding operational performance, because it enhances an investor’s overall understanding of the financial results for the Company’s core business. Additionally, it provides a basis for the comparison of the financial results for the Company’s core business between current, past and future periods. Other companies may define these measures in different ways. Non-GAAP financial measures should be considered only as a supplement to, and not as a substitute for or as a superior measure to, financial measures prepared in accordance with U.S. GAAP. Non-GAAP per share numbers are calculated based on one class of common stock and do not incorporate the effects, if any, of using the two-class method.

On February 24, 2022 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported that it will host a webcast/conference call in conjunction with its fourth quarter and year end 2021 update and financial results on Wednesday, March 9 at 4:30 p.m. ET (Press release, Acorda Therapeutics, FEB 24, 2022, View Source [SID1234608946]).

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To participate in the Webcast, please use the following pre-registration link:

To register for the Webcast, use the link below:
View Source

If you register for the Webcast, you will have the opportunity to submit a written question for the Q&A portion of the presentation. Once you have registered, you will receive a confirmation email with Webcast/Conference Call details. For the Webcast, you will receive an email 2 hours prior to the start of the call with the link to join. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 7:30 p.m. ET on March 9, 2022 until 11:59 p.m. ET on April 8, 2022. To access the replay, please dial 1 866 813 9403 (domestic) or +44 204 525 0658 (international); reference code 309853. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On February 24, 2022 MilliporeSigma, the U.S. and Canada Life Science business sector of Merck KGaA, Darmstadt, Germany, a leading science and technology company, reported the closing of the transaction to acquire Exelead, following regulatory clearances and the fulfillment of other customary closing conditions, for approximately USD 780 million in cash (Press release, MilliporeSigma, FEB 24, 2022, View Source [SID1234608963]). The business combination is expected to enable the Life Science business to provide its customers with comprehensive end-to-end contract development and manufacturing organization (CDMO) services across the mRNA value chain. The Life Science business plans to further invest over € 500 million to scale up Exelead’s technology over the next ten years.

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MilliporeSigma Announces Close of Exelead Acquisition

"With the addition of Exelead’s leading capabilities and highly experienced team, our Life Science business achieves an important milestone in becoming one of the leading CDMO players in mRNA vaccines and therapeutics, offering an integrated CDMO across the mRNA value chain from pre-clinical to commercial," said Matthias Heinzel, Member of the Executive Board of Merck KGaA, Darmstadt, Germany, and CEO Life Science. "mRNA holds much promise as a treatment well beyond Covid-19 and we will further invest in this technology to help realize its potential."

Exelead, a biopharmaceutical CDMO, specializes in PEGylated products and complex injectable formulations, including Lipid Nanoparticle (LNP) based drug delivery technology, which is key in mRNA vaccines and therapeutics for use in Covid-19 and many other indications. The company has experience in all development phases from pre-clinical development to commercial contract manufacturing for LNP formulations, including fill and finish. Exelead will complement the Life Science business sector’s more than 20 years’ experience in producing lipids as well as its mRNA manufacturing capabilities acquired through AmpTec in 2020. This integrated offering will accelerate the Life Science business sector’s ability to bring life-enhancing vaccines and treatments to patients faster by simplifying supply chain complexity and enhancing speed to market through its end-to-end portfolio.

Over the past two years, the Life Science business sector has made significant investments to advance traditional and novel modalities (mAb, ADC, HP-API, viral vector, and mRNA) through acquisitions and expansions. The acquisition of Exelead is another milestone to accelerate innovation in the company’s Process Solutions and Life Science Services businesses, one of the company’s three growth engines ("Big 3"), through targeted smaller to medium-sized acquisitions with high impact.

Follow MilliporeSigma on Twitter @MilliporeSigma, on Facebook @MilliporeSigma and on LinkedIn.

All Merck KGaA, Darmstadt, Germany news releases are distributed by email at the same time they become available on the EMD Group website. In case you are a resident of the U.S. or Canada please go to www.emdgroup.com/subscribe to register again for your online subscription of this service as our newly introduced geo-targeting requires new links in the email. You may later change your selection or discontinue this service.

On February 24, 2022 -IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the fourth quarter and full year ended December 31, 2021 and provided a general business update (Press release, Ophthotech, FEB 24, 2022, View Source [SID1234608979]).

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"In 2021, we successfully achieved a number of major milestones that we believe have laid the groundwork for 2022 to be a banner year for Iveric Bio," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "We continue to focus on execution as evidenced by GATHER2, our second Phase 3 clinical trial for Zimura (avacincaptad pegol), a novel complement inhibitor, for the treatment of geographic atrophy (GA), which continues to exceed expectations with a 12-month injection fidelity rate target of greater than 90%, despite a global pandemic. We are excited to be more than 84% complete with year one of the trial, based on the number of scheduled patient visits. We look forward to sharing topline GATHER2 data in the second half of this year, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis."

"This is a pivotal time for the Company as we continue our internal efforts to prepare for a potential filing of a New Drug Application (NDA) for Zimura for the treatment of GA," stated Pravin U. Dugel, MD, President of Iveric Bio. "We continue to gain momentum in building out our medical affairs and commercial infrastructure as we prepare for the potential launch of Zimura in the US. We plan to initiate a Phase 3 clinical trial studying Zimura in patients with intermediate AMD during the second half of this year. We are pursuing additional lifecycle initiatives, including evaluating multiple sustained-release delivery technologies for Zimura. Further, the U.S. Patent and Trademark Office (USPTO) recently allowed claims for a patent covering methods of treating GA with Zimura, an important addition to our intellectual property portfolio."

Therapeutics Programs Targeting Geographic Atrophy (GA) and other Stages of Age-Related Macular Degeneration (AMD)

Zimura (avacincaptad pegol): Complement C5 Inhibitor

In February 2022, the USPTO allowed claims for methods of using Zimura for the treatment of GA. The patent, when issued, is expected to expire in 2034.
In February 2022, results from a post-hoc analysis that evaluated various GA growth parameters to explore the rate of disease progression within various regions in the fovea in a subset of patients from GATHER1, the Company’s Phase 3 clinical trial for the treatment of Zimura in GA, were presented at the Angiogenesis, Exudation and Degeneration conference. Consistent with the overall results of GATHER1, in the new analysis a reduction in lesion growth in five standardized regions surrounding and including the central foveal area was observed for patients receiving Zimura 2 mg as compared to patients receiving sham over a period of 18 months. We believe the preservation of the central fovea region that was observed in this post-hoc analysis has the potential to be a corollary to a functional benefit.
In July 2021, the Company announced the completion of patient enrollment in GATHER2, four months ahead of the Company’s original schedule.
In July 2021, the Company received a written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the overall design of GATHER2. The agreement further solidifies the Company’s plans to file an NDA with the FDA for marketing approval of Zimura for GA, if the ongoing GATHER2 clinical trial meets its primary endpoint at 12 months. Zimura met its pre-specified primary efficacy endpoint at 12 months with statistical significance in the previously completed GATHER1 pivotal clinical trial.
In June 2021, the Company announced data from post-hoc analyses from the GATHER1 trial, in which the Company evaluated the progression of incomplete Retinal Pigment Epithelial and Outer Retinal Atrophy (iRORA) to complete Retinal Pigment Epithelial and Outer Retinal Atrophy (cRORA) and the progression of drusen to iRORA or cRORA, in patients receiving Zimura 2 mg as compared to patients in the corresponding sham group. Based on the Company’s hypothesis regarding complement inhibition as a mechanism of action to treat AMD and the results of the analyses, the Company plans to initiate a Phase 3 clinical trial studying Zimura in patients with intermediate AMD in the second half of 2022. The development strategy in this indication is subject to regulatory feedback, which the Company plans to obtain before initiating this trial.
Patient enrollment in STAR, the Company’s Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease, is ongoing. The results of this trial are expected after the topline results of GATHER2.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

In 2021, the Company initiated a number of preclinical tolerability and pharmacokinetic studies for IC-500. The Company anticipates that the start of IND-enabling toxicology studies for IC-500 will be later than originally planned, primarily due to the limited availability of study slots at contract research organizations in the wake of the COVID-19 pandemic. The Company expects to submit an investigational new drug application (IND) to the FDA for IC-500 during mid-2023.
Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

As the Company focuses its efforts and resources on the development and potential commercialization of Zimura, the Company is exploring potential collaborations for the future development and potential commercialization of IC-100, the Company’s product candidate for Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) and IC-200, the Company’s product candidate for BEST1-Related IRDs.
In the second half of 2021, the Company transitioned the Stargardt Disease (ABCA4) and USH2A minigene research programs from the University of Massachusetts Medical School (UMMS) to the Company with plans to continue these programs internally. The Company has established a laboratory for continuing the work on its minigene research programs and other preclinical ocular research activities.
Corporate Updates

The Company expanded its Board of Directors and management by adding a number of industry leaders:

Christine Ann Miller, a pharmaceutical veteran, joined the Company’s board of directors in January 2022.
Tony Gibney joined the Company as Executive Vice President and Chief Business and Strategy Officer in December 2021. Mr. Gibney is an experienced biotechnology executive and former investment banker.
Christopher Simms joined the Company as Senior Vice President and Chief Commercial Officer in August 2021. Mr. Simms has commercial leadership experience in retina, ophthalmology, and optometry.
In October 2021, the Company raised approximately $163 million in net proceeds in an underwritten public offering of its common stock. In July 2021, the Company raised approximately $108 million in net proceeds in an underwritten public offering of its common stock.

Fourth Quarter and Year Ended 2021 Operational Update and 2022 Cash Guidance

As of December 31, 2021, the Company had approximately $381.7 million in cash, cash equivalents and marketable securities.
The Company estimates its year-end 2022 cash, cash equivalents and marketable securities will range between $215 million and $225 million. The Company also estimates that its cash, cash equivalents and available for sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses through at least mid-2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for Zimura in GA and STGD1 and the initiation of an intermediate AMD clinical trial, preparation and potential filing of an NDA and a MAA for Zimura in GA, continuing preparations for potential commercial launch of Zimura in GA, investing in sustained release delivery technologies for Zimura, and the advancement of its IC-500 development program. Excluded from these estimates are any potential approval or sales milestones payable to Archemix Corp. or any potential expenses for actual commercial launch of Zimura, such as associated sales force expenses, any additional expenditures related to potentially studying Zimura in indications outside of GA, STGD1 and intermediate AMD, or resulting from the potential in-licensing or acquisition of additional product candidates or technologies, or any associated development the Company may pursue.
2021 Q4 Financial Highlights

R&D Expenses: Research and development expenses were $25.1 million for the quarter ended December 31, 2021, compared to $17.5 million for the same period in 2020. For the year ended December 31, 2021, research and development expenses were $85.1 million compared to $62.8 million for the same period in 2020. Research and development expenses increased year over year primarily due to the commencement and completion of patient enrollment for the GATHER2 clinical trial, increased manufacturing activities for Zimura and increases in personnel costs, including share-based compensation associated with additional research and development staffing. This increase in costs was partially offset by decreases in costs associated with the Company’s gene therapy programs.
G&A Expenses: General and administrative expenses were $8.0 million for the quarter ended December 31, 2021 and for the same period in 2020. For the year ended December 31, 2021, general and administration expenses were $29.7 million, compared to $26.0 million for the same period in 2020. General and administration expenses increased year over year primarily due to an increase in external costs, including legal and consulting costs associated with litigation, pre-commercialization activities and other administrative costs necessary to support the Company’s operations.
Income Tax Benefit: The Company recorded no income tax benefit for the three months ended December 31, 2021 and 2020 and the year ended December 31, 2021. Income tax benefit of $3.7 million for the year ended December 31, 2020, was recognized to reflect a favorable settlement of a state corporate income tax audit.
Net Loss: The Company reported a net loss for the quarter ended December 31, 2021 of $33.0 million, or ($0.29) per diluted share, compared to a net loss of $25.4 million, or $(0.27) per diluted share, for the same period in 2020. For the year ended December 31, 2021, the Company reported a net loss of $114.5 million or ($1.12) per diluted share, compared to a net loss of $84.5 million or ($1.14) for the same period in 2020.
Conference Call/Web Cast Information
Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for February 24, 2022 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 1-888-317-6003 (USA) or 1-412-317-6061 (International), passcode 8865993. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 1-877-344-7529 (USA Toll Free), passcode 8000837.