On February 17, 2022 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the fourth quarter and full year 2021 and provided an update on progress toward achieving key corporate objectives, as well as commercial, clinical and pipeline development milestones (Press release, Exelixis, FEB 17, 2022, View Source [SID1234608246]).

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"The Exelixis team made significant progress across our entire business in the fourth quarter and throughout 2021"

"The Exelixis team made significant progress across our entire business in the fourth quarter and throughout 2021," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer, Exelixis. "With strong performance from CABOMETYX in the fourth quarter, the cabozantinib franchise achieved two significant commercial milestones in 2021 with Exelixis full year U.S. net product revenues exceeding $1 billion and global net product revenues generated by Exelixis and its partners of more than $1.5 billion. These major milestones reflect the importance of our flagship product not only as a treatment for multiple serious cancers, but as the foundation for our company’s growth, enabling the buildout of our clinical pipeline and fueling our ambition to become a global, multiproduct oncology company."

Dr. Morrissey continued: "In late 2021 and early 2022, we achieved multiple key pipeline and discovery milestones, including expanding the phase 1b clinical program for XL092, advancing the phase 1 development of XB002 and XL102, in-licensing and progressing XL114 toward the clinic, and expanding our portfolio through new business development agreements. Collectively, our four clinical compounds and more than 10 discovery programs represent an exciting, diverse portfolio of therapeutic candidates with the potential to improve outcomes for patients with cancer. I’m grateful to the Exelixis team for their continued focus, commitment and hard work as we advance our mission to help cancer patients recover stronger and live longer."

Fourth Quarter and Full Year 2021 Financial Results

Total revenues for the quarter and year ended December 31, 2021 were $451.1 million and $1,435.0 million, respectively, compared to $270.1 million and $987.5 million for the comparable periods in 2020.

Total revenues for the quarter and year ended December 31, 2021 included net product revenues of $302.7 million and $1,077.3 million, respectively, compared to $200.4 million and $741.6 million for the comparable periods in 2020. The increases in net product revenues were primarily related to increases in sales volume driven by the strong uptake for the combination therapy of CABOMETYX (cabozantinib) and OPDIVO (nivolumab) following approval by the U.S. Food and Drug Administration (FDA) in January 2021.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $148.5 million and $357.7 million for the quarter and year ended December 31, 2021, respectively, compared to $69.7 million and $246.0 million for the comparable periods in 2020. The increases in collaboration revenues were primarily related to increases in the recognition of milestone-related revenues, increases in development cost reimbursements earned, and higher royalty revenues for the sales of cabozantinib outside of the U.S. generated by Exelixis’ collaboration partners, Ipsen Pharma SAS (Ipsen) and Takeda Pharmaceutical Company Limited (Takeda).

Research and development expenses for the quarter and year ended December 31, 2021 were $222.3 million and $693.7 million, respectively, compared to $154.3 million and $547.9 million for the comparable periods in 2020. The increases in research and development expenses were primarily related to increases in license and other collaboration costs, personnel expenses and stock-based compensation expense, which were partially offset by decreases in clinical trial costs.

Selling, general and administrative expenses for the quarter and year ended December 31, 2021 were $99.3 million and $401.7 million, respectively, compared to $82.4 million and $293.4 million for the comparable periods in 2020. The increases in selling, general and administrative expenses were primarily related to increases in personnel expenses, marketing costs, legal costs, corporate giving and stock-based compensation expense.

Provision for (benefit from) income taxes for the quarter and year ended December 31, 2021 was $22.9 million and $63.1 million, respectively, compared to $(0.3) million and $19.1 million for the comparable periods in 2020, primarily due to an increase in pre-tax income.

GAAP net income for the quarter ended December 31, 2021 was $95.2 million, or $0.30 per share, basic and $0.29 per share, diluted, compared to GAAP net income of $28.4 million, or $0.09 per share, basic and diluted, for the comparable period in 2020. GAAP net income for the year ended December 31, 2021 was $231.1 million, or $0.73 per share, basic and $0.72 per share, diluted, compared to GAAP net income of $111.8 million, or $0.36 per share, basic and $0.35 per share, diluted, for the comparable period in 2020.

Non-GAAP net income for the quarter ended December 31, 2021 was $113.3 million, or $0.36 per share, basic and $0.35 per share, diluted, compared to non-GAAP net income of $43.3 million, or $0.14 per share, basic and diluted, for the comparable period in 2020. Non-GAAP net income for the year ended December 31, 2021 was $324.2 million, or $1.03 per share, basic and $1.01 per share, diluted, compared to non-GAAP net income of $193.3 million, or $0.63 per share, basic and $0.61 per share, diluted, for the comparable period in 2020. Non-GAAP net income excludes stock-based compensation, adjusted for the related income tax effect.

Cash, cash equivalents, restricted cash equivalents and investments were $1.9 billion at December 31, 2021, compared to $1.5 billion at December 31, 2020.

Non-GAAP Financial Measures

To supplement Exelixis’ financial results presented in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Exelixis presents non-GAAP net income (and the related per share measures), which excludes from GAAP net income (and the related per share measures) stock-based compensation expense, adjusted for the related income tax effect for all periods presented.

Exelixis believes that the presentation of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors. In particular, Exelixis believes that these non-GAAP financial measures, when considered together with its financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare Exelixis’ results from period to period, and to identify operating trends in Exelixis’ business. Exelixis has excluded stock-based compensation expense, adjusted for the related income tax effect, because it is a non-cash item that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. Exelixis also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate its business and to make operating decisions.

These non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. Exelixis encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP financial information and the reconciliation between these presentations, to more fully understand Exelixis’ business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

2022 Financial Guidance

Exelixis is providing the following financial guidance for fiscal year 2022:

Total revenues

$1.525 billion – $1.625 billion

Net product revenues

$1.325 billion – $1.425 billion

Cost of goods sold

5% – 6% of net product revenues

Research and development expenses (1)

$725 million – $775 million

Selling, general and administrative expenses (2)

$400 million – $450 million

Effective tax rate

20% – 22%

____________________
(1) Includes $45 million of non-cash stock-based compensation expense.

(2) Includes $50 million of non-cash stock-based compensation expense.

Cabozantinib Highlights

Cabozantinib Franchise Net Product Revenues and Royalties. Net product revenues generated by the cabozantinib franchise in the U.S. were $302.7 million during the fourth quarter of 2021, with net product revenues of $295.1 million from CABOMETYX and $7.6 million from COMETRIQ (cabozantinib). For the year ended December 31, 2021, net product revenues generated by the cabozantinib franchise in the U.S. were $1,077.3 million, with net product revenues of $1,054.1 million from CABOMETYX and $23.2 million from COMETRIQ. In 2021, global cabozantinib franchise net product revenues generated by Exelixis and its partners exceeded $1.5 billion. Based upon cabozantinib-related net product revenues generated by Exelixis’ collaboration partners during the quarter and year ended December 31, 2021, Exelixis earned $29.3 million and $105.1 million, respectively, in royalty revenues.

Achievement of Cabozantinib Sales-Based Milestone from Ipsen. In the fourth quarter of 2021, Exelixis recorded in license revenues a $100.0 million milestone from Ipsen in connection with the achievement of $400.0 million in net sales in its related license territory over four consecutive quarters. Exelixis expects to receive this payment in the first quarter of 2022.

Completion of Enrollment in CONTACT-01 Pivotal Trial of Cabozantinib in Combination with an Immune Checkpoint Inhibitor (ICI) in Previously Treated Metastatic Non-Small Cell Lung Cancer (NSCLC). In November 2021, Exelixis announced that enrollment was completed for CONTACT-01, the global phase 3 pivotal trial evaluating cabozantinib in combination with atezolizumab versus docetaxel in patients with metastatic NSCLC who have been previously treated with an ICI and platinum-containing chemotherapy. CONTACT-01 enrolled 366 patients who were randomized 1:1 to the experimental arm of cabozantinib in combination with atezolizumab and the control arm of docetaxel. The primary endpoint of the trial is overall survival (OS). Secondary endpoints include progression-free survival (PFS), objective response rate (ORR) and duration of response (DOR). CONTACT-01 is sponsored by F. Hoffmann-La Roche Ltd. (Roche) and co-funded by Exelixis. Interim data from the trial are anticipated in the second half of 2022.

Presentation of Detailed Results from Phase 3 COSMIC-312 Pivotal Trial of Cabozantinib in Combination with Atezolizumab in Previously Untreated Advanced Hepatocellular Carcinoma (HCC) at the European Society for Medical Oncology Asia Virtual Oncology Week 2021 (ESMO Asia 2021). In November 2021, Exelixis presented detailed results from the first planned analysis of COSMIC-312, the ongoing phase 3 pivotal trial evaluating cabozantinib in combination with atezolizumab versus sorafenib in patients with previously untreated advanced HCC, at ESMO (Free ESMO Whitepaper) Asia 2021. Exelixis expects the final OS data to be available in early 2022.

Completion of Enrollment in CONTACT-03 Pivotal Trial of Cabozantinib in Combination with Atezolizumab in Previously Treated Metastatic Renal Cell Carcinoma (RCC). In January 2022, Exelixis announced that enrollment was complete for CONTACT-03, the global phase 3 pivotal trial evaluating cabozantinib in combination with atezolizumab versus cabozantinib alone in patients with locally advanced or metastatic clear cell or non-clear cell RCC who progressed during or following treatment with an ICI. CONTACT-03 enrolled 523 patients who were randomized 1:1 to the experimental arm of cabozantinib in combination with atezolizumab and the control arm of cabozantinib alone. The primary endpoints of the trial are PFS per Response Evaluation Criteria in Solid Tumors v. 1.1 as assessed by independent radiology review and OS. Secondary endpoints include PFS, ORR and DOR as assessed by study investigators. CONTACT-03 is sponsored by Roche and co-funded by Exelixis. Interim data from the trial are anticipated in the second half of 2022.

Cabozantinib Data at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI 2022). In January 2022, investigators presented encouraging data from two trials of cabozantinib in combination with ICIs for the treatment of advanced colorectal cancer (CRC). The results reinforce Exelixis’ decision to pursue clinical development of XL092, which pairs a target profile similar to cabozantinib with a potentially significantly improved safety profile, in advanced CRC through the STELLAR-303 global phase 3 pivotal trial expected to initiate in the first half of 2022.

Cabozantinib Data at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU 2022). Later this week, cabozantinib will be the subject of multiple data presentations at ASCO (Free ASCO Whitepaper) GU 2022, which is being held in a hybrid virtual/in-person format from February 17-19. Notable presentations include two additional data sets from the phase 3 pivotal CheckMate -9ER study providing final OS analysis and organ-specific target lesion assessments with two-year follow-up, and updated health-related quality of life results.

Pipeline Highlights

In-Licensing of Second Anti-Cancer Compound from Aurigene Discovery Technologies Limited (Aurigene) Following FDA Acceptance of Investigational New Drug (IND) Application. In October 2021, Exelixis and Aurigene announced that Exelixis exercised its exclusive option to in-license XL114 (formerly AUR104) under the companies’ July 2019 collaboration, option and license agreement. As a result, Exelixis assumed responsibility for all subsequent clinical development, manufacturing and commercialization of the novel anti-cancer compound that inhibits the CARD11-BCL10-MALT1 (CBM) complex, a key component of signaling downstream of B- and T-cell receptors, which promotes B- and T-cell lymphoma survival and proliferation. Following the FDA’s recent acceptance of Exelixis’ IND, the company plans to initiate a phase 1 trial of XL114 as a monotherapy in patients with non-Hodgkin’s lymphoma (NHL) in the first half of 2022.

Initiation of STELLAR-002 Phase 1b Trial Evaluating XL092 in Combination with Immuno-Oncology (IO) Therapies in Patients with Advanced Solid Tumors. In December 2021, Exelixis announced the initiation of the dose-escalation stage of STELLAR-002, a phase 1b trial evaluating XL092 in combination with IO therapies in advanced solid tumors. The objective of the study is to evaluate the safety, tolerability and efficacy of XL092, Exelixis’ novel next-generation tyrosine kinase inhibitor, in combination with: nivolumab; nivolumab and ipilimumab; and nivolumab and bempegaldesleukin. Exelixis expects to expand the STELLAR-002 study, as well as the ongoing phase 1b STELLAR-001 study, which are evaluating XL092 in combination with several IO therapies, into potential new tumor types, and IO and other targeted therapy combination regimens throughout 2022. Clinical updates are expected in 2022.

Amendment of Option and License Agreement for XB002, an Antibody-Drug Conjugate (ADC) Targeting Tissue Factor (TF). In January 2022, Exelixis and Iconic Therapeutics, Inc. (Iconic) announced amended terms to their May 2019 exclusive option and license agreement for XB002, a next-generation TF-targeting ADC. Under the amended agreement, Exelixis acquired broad rights to use the anti-TF antibody incorporated into XB002 for any application, including conjugated to other payloads, as well as rights within oncology to a number of other anti-TF antibodies developed by Iconic, including for use in ADCs and multispecific biotherapeutics. The single-agent dose escalation cohort for Exelixis’ ongoing phase 1 trial of XB002 continues enrolling, with the study moving into its cohort expansion and combination phase as the next step. Based on early clinical data supportive of a potentially differentiated and best-in-class profile, Exelixis intends to aggressively expand development of XB002, both as a monotherapy and in combination with ICIs and other targeted therapies, across a wide range of tumor types, including indications other than those currently addressed by commercially available TF-targeted therapies. Exelixis expects to provide clinical updates from the ongoing phase 1 study of XB002 in 2022.

Corporate Updates

Exclusive Collaboration and License Agreement with STORM Therapeutics LTD (STORM) to Discover and Develop Inhibitors of Novel RNA Modifying Enzymes. In October 2021, Exelixis and STORM entered into an exclusive collaboration and license agreement under which the companies will discover and advance novel drug leads intended for the treatment of cancer. The collaboration will focus initially on ADAR1, advancing early work by STORM applying its proprietary RNA epigenetic platform, as well as explore an additional undisclosed target.

Appointment of Jacqueline Wright to the Exelixis Board of Directors. In December 2021, Exelixis announced that Jacqueline (Jacky) Wright was appointed to the company’s Board of Directors. The appointment took effect on December 16, 2021. Ms. Wright is an accomplished technology executive with decades of technology experience and is widely recognized for her expertise in digital transformation, both in the public and private sectors. She currently serves as Corporate Vice President & Chief Digital Officer, U.S. Business at Microsoft Corporation.

Appointment of Vicki L. Goodman, M.D., as Executive Vice President, Product Development & Medical Affairs and Chief Medical Officer (CMO). In January 2022, Exelixis announced the appointment of Vicki L. Goodman, M.D., as Executive Vice President, Product Development & Medical Affairs and CMO. Dr. Goodman has more than 20 years of oncology experience as a drug development leader at global biopharmaceutical organizations, regulator and clinician. She joined from Merck & Co., where she served as Vice President, Clinical Research and Therapeutic Area Head, Late Stage Oncology. Dr. Goodman will be based in the Greater Philadelphia area. As part of her role overseeing the company’s product development operations, she will play a leadership role in building a new Exelixis team that will expand the company’s development activities on the East Coast. Exelixis’ East Coast presence will complement the company’s growing West Coast development team and enable the company to lay additional groundwork for potential future growth outside the U.S.

Announcement of Key Priorities and Anticipated Milestones for 2022. In January 2022, Exelixis announced its key priorities and anticipated milestones for 2022, including: multiple pivotal clinical trial readouts for cabozantinib across the COSMIC and CONTACT clinical studies; the launch of the XL092 phase 3 pivotal trial program; the expansion of the ongoing phase 1b STELLAR-001/-002 clinical trials of XL092, with plans to provide clinical updates; accelerated development and expansion of the XB002 clinical program, with plans to provide clinical updates from the ongoing phase 1 study; expansion of the XL102 phase 1 clinical program into the cohort expansion phase, with plans to provide clinical updates; initiation of the phase 1 trial for XL114, an inhibitor of the CBM complex, in patients with NHL; plans to expand development operations to the East Coast under the leadership of Dr. Goodman, the company’s newly hired CMO; and the progression of up to five new development candidates into preclinical development from the company’s more than 10 discovery programs currently advancing through internal and collaborative efforts. Exelixis presented the details of its key priorities and anticipated milestones at the 40th Annual J.P. Morgan Healthcare Conference.

Basis of Presentation

Exelixis has adopted a 52- or 53-week fiscal year policy that ends on the Friday closest to December 31st. For convenience, references in this press release as of and for the fiscal periods ended January 1, 2021 are indicated as being as of and for the periods ended December 31, 2020.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the fourth quarter and full year of 2021 and provide a general business update during a conference call beginning at 5:00 p.m. ET / 2:00 p.m. PT today, Thursday, February 17, 2022.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 855-793-2457 (domestic) or 631-485-4921 (international) and provide the conference call passcode 8647777 to join by phone.

A telephone replay will be available until 8:00 p.m. ET on Saturday, February 19, 2022. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 8647777. A webcast replay will also be archived on www.exelixis.com for one year.

On February 17, 2022 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the fourth quarter and full year ended December 31, 2021 and provided an update on the Company’s business and pipeline (Press release, Insmed, FEB 17, 2022, View Source [SID1234608212]).

"Insmed made tremendous progress across our four pillars throughout 2021, and we begin 2022 from a position of strength, with seven ongoing clinical trials, commercial operations in three major territories, a pathway to ARIKAYCE growth, and a highly innovative research engine to identify what we believe to be the most promising next set of candidates," commented Will Lewis, Chair and Chief Executive Officer of Insmed. "Importantly, our strong cash position will support advancement across our programs as we continue to lay the groundwork for what we believe will be a meaningful inflection point for the Company. I am enormously proud of our world-class team and what we have been able to achieve on behalf of patients with serious and rare diseases."

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Recent Corporate Developments & Program Highlights

ARIKAYCE

•Insmed launched ARIKAYCE in Japan in July of 2021, and early launch progress has been strong, with several positive trends.

•In Europe, ARIKAYCE has now been launched in Germany, the Netherlands, Wales, and Scotland. The Company is pursuing country-by-country reimbursement and launches throughout Europe, with a near-term focus on Italy, France, and England.

•Insmed continues to advance the post-marketing confirmatory frontline clinical trial program of ARIKAYCE in patients with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC). The ARISE trial, an interventional study designed to validate a patient-reported outcome (PRO) tool in MAC lung disease, is now 50% enrolled. The Company anticipates completing enrollment in the ARISE study in 2022 and having topline data in the first half of 2023. Insmed anticipates completing enrollment in ENCORE, a pivotal study evaluating the clinical benefits and safety of ARIKAYCE in patients with newly diagnosed MAC lung disease using the PRO tool, by the end of 2023.

1Brensocatib

•The Phase 3 ASPEN study, a global, randomized, double-blind, placebo-controlled trial to assess the efficacy, safety, and tolerability of brensocatib in patients with bronchiectasis, is now 50% enrolled. Insmed anticipates completing enrollment in this study in early 2023.

•A Phase 2 pharmacokinetic/pharmacodynamic study of brensocatib in patients with cystic fibrosis is underway and Insmed anticipates sharing data from this study by early 2023.

•Insmed plans to develop brensocatib in two additional neutrophil-mediated diseases – chronic rhinosinusitis without nasal polyps (CRS) and hidradenitis suppurativa (HS) – and to advance one indication into the clinic in 2022. Both CRS and HS are serious diseases in which patients today face significant unmet needs.

TPIP

•Insmed is advancing two Phase 2 studies of treprostinil palmitil inhalation powder (TPIP) in patients with pulmonary arterial hypertension (PAH). The Phase 2a study will measure the impact of TPIP on pulmonary vascular resistance (PVR) over a 24-hour period. The Company anticipates having preliminary data from a small number of patients in this study this year. The Phase 2b study will evaluate the effect of TPIP on PVR and 6-minute walk distance over a 16-week treatment period. As planned, site initiation for this study began in late 2021.

•Site initiation is underway for a Phase 2 study of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD). This study will assess the safety and tolerability of TPIP over a 16-week treatment period.

Translational Medicine

•Insmed is advancing a translational medicine portfolio consisting of several technology programs running in parallel. The Company anticipates filing an Investigational New Drug Application in a non-pulmonary indication for our first candidate from this portfolio by the end of 2022.

Fourth Quarter and Full-Year 2021 Financial Results

•Total revenue for the fourth quarter ended December 31, 2021 was $56.1 million, compared to total revenue of $41.4 million for the fourth quarter of 2020. Total revenue for the full year 2021 was $188.5 million, compared to total revenue of $164.4 million for the full year 2020.

•Total revenue for the full year 2021 comprised ARIKAYCE net sales of $159.5 million in the U.S., $16.0 million in Japan, and $12.9 million in Europe and rest of world. This compares to net sales of $157.5 million in the U.S. and $6.9 million in Europe for the full year 2020.

•Cost of product revenues (excluding amortization of intangible assets) was $13.3 million for the fourth quarter of 2021, compared to $10.9 million for the fourth quarter of 2020. For the full year 2021, cost of product revenues (excluding amortization of intangible assets) was $44.2 million compared to $39.9 million in 2020.

•Research and development (R&D) expenses were $76.4 million for the fourth quarter of 2021, compared to $67.8 million for the fourth quarter of 2020. For the full year 2021, R&D expenses were $272.7 million compared to $181.2 million in 2020.

•Selling, general and administrative (SG&A) expenses for the fourth quarter of 2021 were $65.3 million, compared to $56.0 million for the fourth quarter of 2020. For the full year 2021, SG&A expenses were $234.3 million, compared to $203.6 million in 2020.

•For the fourth quarter of 2021, Insmed reported a net loss of $113.0 million, or $0.95 per share, compared to a net loss of $102.2 million, or $1.00 per share, for the fourth quarter of 2020. For the full year 2021, Insmed reported a net loss of $434.7 million, or $3.88 per share, compared to a net loss of $294.1 million, or $3.01 per share, in 2020.

2Balance Sheet, Financial Guidance, and Planned Investments

As of December 31, 2021, Insmed had cash and cash equivalents and marketable securities of $766.8 million. The Company’s total operating expenses for the fourth quarter of 2021 were $155.2 million and for the full year 2021 were $563.6 million.

Insmed expects full-year 2022 revenues for ARIKAYCE to increase at least 30% year over year from 2021. The Company anticipates that its cash on hand will support its ongoing programs into 2024.

The Company plans to invest in the following key activities in 2022:

(i)commercialization and expansion of ARIKAYCE globally;

(ii)launch activities for ARIKAYCE in initial European countries and in Japan; and

(iii)clinical trial activities, including (a) advancement of the frontline clinical trial program for ARIKAYCE (ARISE and ENCORE), (b) advancement of brensocatib, including the Phase 3 ASPEN study in patients with bronchiectasis, (c) advancement of the Phase 2 clinical development programs of TPIP, and (d) advancement of our translational medicine efforts.

Conference Call

Insmed will host a conference call beginning today at 8:00 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing (844) 200-6205 (U.S. toll free), (646) 904-5544 (U.S. local), or +1-929-526-1599 (international) and referencing access code 359415. The call will also be webcast live on the company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately 1 hour after its completion through March 17, 2022, by dialing (866) 813-9403 (U.S. toll free), (929) 458-6194 (U.S. local), or +44-204-525-0658 (international) and referencing access code 838814. A webcast of the call will also be archived for 90 days under the Investor Relations section of the company’s website at www.insmed.com.

About ARIKAYCE

ARIKAYCE is approved in the United States as ARIKAYCE (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion, and in Japan as ARIKAYCE inhalation 590 mg (amikacin sulfate inhalation drug product). Current international treatment guidelines recommend the use of ARIKAYCE for appropriate patients. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed’s proprietary PULMOVANCE liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides, while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira Nebulizer System manufactured by PARI Pharma GmbH (PARI).

About PARI Pharma and the Lamira Nebulizer System

ARIKAYCE is delivered by a novel inhalation device, the Lamira Nebulizer System, developed by PARI. Lamira is a quiet, portable nebulizer that enables efficient aerosolization of ARIKAYCE via a vibrating, perforated membrane. Based on PARI’s 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms to improve patient care.

3
About Brensocatib

Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis and other neutrophil-mediated diseases. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. Brensocatib is an investigational drug product that has not been approved for any indication in any jurisdiction.

About TPIP

Treprostinil palmitil inhalation powder (TPIP) is a dry powder formulation of treprostinil palmitil, a treprostinil prodrug consisting of treprostinil linked by an ester bond to a 16-carbon chain. Developed entirely in Insmed’s laboratories, TPIP is a potentially highly differentiated prostanoid being evaluated for the treatment of patients with PAH, PH-ILD, and other rare and serious pulmonary disorders. TPIP is administered in a capsule-based inhalation device. TPIP is an investigational drug product that has not been approved for any indication in any jurisdiction.

IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.

WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS

ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases.

Hypersensitivity Pneumonitis has been reported with the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis, pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.

Hemoptysis has been reported with the use of ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis occurs, manage patients as medically appropriate.

Bronchospasm has been reported with the use of ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7%). If bronchospasm occurs during the use of ARIKAYCE, treat patients as medically appropriate.

Exacerbations of underlying pulmonary disease has been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying pulmonary disease (reported as chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation of bronchiectasis) have been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%) compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease occur during the use of ARIKAYCE, treat patients as medically appropriate.

4
Anaphylaxis and Hypersensitivity Reactions: Serious and potentially life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients taking ARIKAYCE. Signs and symptoms include acute onset of skin and mucosal tissue hypersensitivity reactions (hives, itching, flushing, swollen lips/tongue/uvula), respiratory difficulty (shortness of breath, wheezing, stridor, cough), gastrointestinal symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and cardiovascular signs and symptoms of anaphylaxis (tachycardia, low blood pressure, syncope, incontinence, dizziness). Before therapy with ARIKAYCE is instituted, evaluate for previous hypersensitivity reactions to aminoglycosides. If anaphylaxis or a hypersensitivity reaction occurs, discontinue ARIKAYCE and institute appropriate supportive measures.

Ototoxicity has been reported with the use of ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus, and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17%) compared to patients treated with background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background regimen vs 0.9% in the background regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background regimen alone arm). Closely monitor patients with known or suspected auditory or vestibular dysfunction during treatment with ARIKAYCE. If ototoxicity occurs, manage patients as medically appropriate, including potentially discontinuing ARIKAYCE.

Nephrotoxicity was observed during the clinical trials of ARIKAYCE in patients with MAC lung disease but not at a higher frequency than background regimen alone. Nephrotoxicity has been associated with the aminoglycosides. Close monitoring of patients with known or suspected renal dysfunction may be needed when prescribing ARIKAYCE.

Neuromuscular Blockade: Patients with neuromuscular disorders were not enrolled in ARIKAYCE clinical trials. Patients with known or suspected neuromuscular disorders, such as myasthenia gravis, should be closely monitored since aminoglycosides may aggravate muscle weakness by blocking the release of acetylcholine at neuromuscular junctions.

Embryo-Fetal Toxicity: Aminoglycosides can cause fetal harm when administered to a pregnant woman. Aminoglycosides, including ARIKAYCE, may be associated with total, irreversible, bilateral congenital deafness in pediatric patients exposed in utero. Patients who use ARIKAYCE during pregnancy, or become pregnant while taking ARIKAYCE should be apprised of the potential hazard to the fetus.

Contraindications: ARIKAYCE is contraindicated in patients with known hypersensitivity to any aminoglycoside.

Most Common Adverse Reactions: The most common adverse reactions in Trial 1 at an incidence ≥5% for patients using ARIKAYCE plus background regimen compared to patients treated with background regimen alone were dysphonia (47% vs 1%), cough (39% vs 17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%), ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%), musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs 10%), exacerbation of underlying pulmonary disease (15% vs 10%), diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%), headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash (6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs 1%), and chest discomfort (5% vs 3%).

Drug Interactions: Avoid concomitant use of ARIKAYCE with medications associated with neurotoxicity, nephrotoxicity, and ototoxicity. Some diuretics can enhance aminoglycoside toxicity by altering aminoglycoside concentrations in serum and tissue. Avoid concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea, or intravenous mannitol.

Overdosage: Adverse reactions specifically associated with overdose of ARIKAYCE have not been identified. Acute toxicity should be treated with immediate withdrawal of ARIKAYCE, and baseline tests of renal function should be undertaken. Hemodialysis may be helpful in removing amikacin from the body. In all cases of suspected overdosage, physicians should contact the Regional Poison Control Center for information about effective treatment.

5
U.S. INDICATION

LIMITED POPULATION: ARIKAYCE is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.

This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088. You can also call the Company at 1-844-4-INSMED.

On February 17, 2022 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported that financial results for the three and 12 months ended December 31, 2021, and provided an operating forecast and business updates (Press release, Ligand, FEB 17, 2022, View Source [SID1234608231]). Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

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"2021 was an outstanding year for Ligand, both operationally and financially. The execution by the team and the performance of the business throughout the past several months position us very well for a transformative 2022. Last year, we had the most substantial calendar of product and market approvals in Ligand’s history, including five approvals of partners’ drugs that were developed using Ligand’s technology. Each of these approvals is a scientific and medical success, establishing important treatment options for patients in need," said John Higgins, CEO of Ligand.

"Notably, OmniAb continues to excel as a leading antibody discovery platform, and we have made good progress toward our goal of establishing the business as a separate public company. OmniAb had its most prolific year ever as nine antibodies derived from the platform recently into the clinic, including the first OmniChicken-derived antibody, and two antibodies received regulatory approval and we expect will begin generating royalties. We believe more than ever that OmniAb offers one of the industry’s leading antibody discovery platforms and that the business is primed for continued growth and success," continued Higgins.

"In terms of the separation process, we initially outlined plans that favored pursuing an OmniAb IPO, while also evaluating other listing alternatives. We diligently explored those paths and engaged with dozens of high-quality investors. Both existing Ligand holders and potential new investors have shown strong interest in our plan to operate two independent public companies. Given our confidence in OmniAb’s ability to thrive as an independent publicly traded company, we have decided to pursue a direct spin-off that will result in the separation occurring in the soonest possible execution window as compared to other alternatives. Our plan now is for Ligand to directly fund the OmniAb business at the time of the spin-off. This strategy is intended to best serve our science and partners, and to maximize value for our shareholders," he added.

Fourth Quarter 2021 Financial Results

Total revenues for the fourth quarter of 2021 were $72.5 million, compared with $70.0 million for the same period in 2020. Royalties for the fourth quarter of 2021 were $17.6 million, compared with $11.0 million for the same period in 2020, with the increase primarily attributable to the additional royalties from the sale of drugs using the Pelican platform. Captisol sales were $35.4 million for the fourth quarter of 2021, compared with $41.0 million for the same period in 2020, with the decrease due to lower sales of Captisol for manufacturing remdesivir, a COVID-19 treatment. Contract revenue was $19.5 million for the fourth quarter of 2021, compared with $18.0 million for the same period in 2020.

Cost of Captisol was $12.0 million for the fourth quarter of 2021, compared with $11.7 million for the same period in 2020. Cost of Captisol was higher in 2021 primarily as a result of expenses associated with manufacturing Captisol for use with remdesivir and customer mix. Amortization of intangibles was $11.8 million, compared with $12.2 million for the same period in 2020. Research and development expense was $18.2 million, compared with $21.9 million for the same period in 2020, with the decrease primarily attributed to the sale of Vernalis in December 2020. General and administrative expense was $17.7 million, compared with $30.1 million for the same period in 2020, with the decrease primarily attributable to Pfenex acquisition-related expenses in the prior-year period.

Net loss for the fourth quarter of 2021 was $(5.0) million, or $(0.30) per share, compared with net income of $5.8 million, or $0.35 per diluted share, for the same period in 2020. Net income (loss) for the fourth quarter of 2021 and 2020 was impacted by a non-cash loss of $(13.0) million and a non-cash gain of $0.07 million, respectively, from the value of Ligand’s short-term investments. Adjusted net income for the fourth quarter of 2021 was $31.3 million, or $1.80 per diluted share, compared with adjusted net income of $27.1 million, or $1.62 per diluted share, for the same period in 2020. See the table below for a reconciliation of net income (loss) to adjusted net income.

As of December 31, 2021, Ligand had cash, cash equivalents and short-term investments of $341.1 million.

Full Year 2021 Financial Results

Total revenues for 2021 were $277.1 million, compared with $186.4 million for 2020. Royalties for 2021 were $48.9 million, compared with $33.8 million for 2020, with the increase primarily attributable to the additional royalties from the sale of drugs using the Pelican platform. Captisol sales for 2021 were $164.3 million, compared with $110.0 million for 2020, primarily reflecting higher sales of Captisol for use with remdesivir. Contract revenue for 2021 was $64.0 million, compared with $42.7 million for 2020, with the increase primarily due to revenue from the acquisitions of Icagen in April 2020 and Pfenex in October 2020.

Cost of Captisol was $62.2 million for 2021, compared with $30.4 million for 2020, with the increase due primarily to higher sales of Captisol. Amortization of intangibles was $47.2 million for 2021, compared with $23.4 million for 2020, with the increase attributable to the Icagen and Pfenex acquisitions. Research and development expense was $69.0 million for 2021, compared with $59.4 million for 2020, with the increase primarily due to the Icagen and Pfenex acquisitions. General and administrative expense was $57.5 million for 2021, compared with $64.4 million for 2020, with the decrease primarily attributable to acquisition-related expenses in the prior year.

Other operating income was $37.6 million for 2021, which represented a non-cash valuation adjustment related to eliminating the remaining Pfenex CVR liability. There was no other operating income for 2020.

Net income for 2021 was $57.6 million, or $3.34 per diluted share, compared with net loss of $(3.0) million, or $(0.18) per share, for 2020. Net income for 2021 included a $(10.6) million net non-cash loss from the value of Ligand’s short-term investments, while net loss for 2020 included a net non-cash loss from the value of Ligand’s short-term investments of $(17.9) million. Adjusted net income for 2021 was $110.8 million, or $6.42 per diluted share, compared with adjusted net income of $76.5 million, or $4.55 per diluted share, for 2020. See the table below for a reconciliation of net income (loss) to adjusted net income.

2022 Financial Guidance

Ligand is providing 2022 revenue guidance for the combined business as well as some information about revenue that is estimated to be attributable to the OmniAb business anticipating the spin-off later this year. Ligand expects 2022 royalties of $55 million to $60 million, material sales of $40 million to $50 million, and contract revenue of $52 million to $62 million. These revenue components result in total revenue of $147 million to $172 million for the combined Ligand business. Ligand estimates two-thirds of the contract revenue guidance to be attributable to OmniAb and a couple million of the royalty revenue will be attributable to OmniAb. Following the completion of the separation process, Ligand will provide more detailed guidance on expenses and earnings.

Update on the Separation Process

In November 2021, Ligand announced plans to explore multiple paths for OmniAb to become a stand-alone public company, with the leading option under consideration at that time being an IPO and eventual distribution of OmniAb shares to Ligand shareholders. Ligand now expects to pursue separation of OmniAb through a direct spin-off of 100% of OmniAb equity to shareholders with Ligand capitalizing the OmniAb business directly with $70 million. OmniAb expects to file a Form 10 with the Securities and Exchange Commission and complete its separation in the first half of 2022. The distribution is expected to qualify as a tax-free transaction for U.S. federal income tax purposes to both Ligand and its shareholders. The separation remains subject to final approval by Ligand’s Board of Directors, and Ligand will continue to evaluate other options to optimize value and ensure flexibility to invest in growth. There can be no assurance that this process will result in Ligand pursuing a particular transaction or consummating any such transaction, or that the anticipated benefits of a separation will materialize should the separation be completed.

Fourth Quarter 2021 and Recent Business Highlights

OmniAb Platform and Partner Updates

The OmniAb discovery platform provides Ligand’s pharmaceutical industry partners with access to diverse antibody repertoires and high-throughput screening technologies to enable discovery of next-generation therapeutics. At the heart of the OmniAb platform is the Biological Intelligence (BI) of our proprietary transgenic animals, including OmniRat, OmniChicken and OmniMouse that have been genetically modified to generate antibodies with human sequences to facilitate development of human therapeutic candidates. Over 55 partners have access to OmniAb-derived antibodies and more than 250 programs are being actively developed or commercialized. As of December 31, 2021, there were 25 active clinical- or commercial-stage OmniAb-derived antibodies, compared with 16 a year earlier.

CStone Pharmaceuticals received approval from China’s NMPA for Celjemy (sugemalimab), an OmniAb-derived anti-PD-L1 monoclonal antibody for the first-line treatment of advanced non-small cell lung cancer (NSCLC) in combination with chemotherapy. Sugemalimab is the second OmniAb-derived antibody to receive regulatory approval. CStone announced complete enrollment in two Phase 3 registrational clinical trials investigating sugemalimab in combination with chemotherapy for the first-line treatment of metastatic gastric adenocarcinoma/gastroesophageal junction adenocarcinoma or esophageal squamous cell carcinoma. CStone’s partner EQRx announced the publication of positive results from two Phase 3 trials with sugemalimab in Stage III and Stage IV NSCLC in Lancet Oncology. CStone announced the Phase 2 GEMSTONE-201 trial met its primary endpoint of objective response rate in patients with relapsed or refractory (R/R) extranodal natural killer/T-cell lymphoma.

In December 2021, Janssen Biotech, Inc. (Janssen) announced submission of a Biologics License Application (BLA) to the FDA seeking U.S. approval of teclistamab for the treatment of patients with R/R multiple myeloma. Teclistamab is an OmniAb-derived bispecific antibody targeting BCMA and CD3. Ligand is entitled to receive a $25 million milestone payment upon first commercial sale of teclistamab in the U.S.

Immunovant announced alignment with the FDA to initiate a Phase 3 trial for batoclimab in myasthenia gravis. Immunovant plans to start the Phase 3 study in the first half of this year, and also expects to initiate pivotal trials in two additional indications this year.

We expanded an existing collaboration and license agreement with GlaxoSmithKline (GSK) to leverage our Icagen Ion Channel Technology to target neurological diseases. We received an upfront payment of $10 million and are eligible for milestones of up to $247.5 million, and tiered royalties on net sales of any drug from the collaboration commercialized by GSK.

We recently entered into new OmniAb platform licensing agreements with Paragon Therapeutics, LTZ Therapeutics, and Seismic Therapeutics.

Pelican Platform Updates

Merck announced European Commission approval of VAXNEUVANCE for adults 18 years of age and older. VAXNEUVANCE is a 15-valent pneumococcal vaccine utilizing Ligand’s CRM197 vaccine carrier protein produced using the Pelican Expression Technology platform. Additionally, Merck announced the FDA accepted for priority review the supplemental BLA (sBLA) for VAXNEUVANCE in infants and children.

Jazz Pharmaceuticals announced submission of an sBLA to the FDA seeking approval for a Monday/Wednesday/Friday (M/W/F) intramuscular dosing schedule for Rylaze, as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients one month of age and older who have developed hypersensitivity to E. coli-derived asparaginase. Jazz presented initial results at ASH (Free ASH Whitepaper) from a Phase 2/3 study of Rylaze in adult and pediatric ALL and LBL patients showing Rylaze maintains a clinically meaningful level of asparaginase activity throughout the entire duration of treatment on a M/W/F dosing schedule.

Arcellx announced the pricing of a $124 million IPO with proceeds planned to advance their pipeline. Arcellx uses the Pelican Expression Technology platform for the expression of certain proprietary sparX proteins used in their ARC-SparX platform.

Captisol

Amgen announced FDA approval of a new Kyprolis combination regimen with DARZALEX FASPRO and dexamethasone for patients with multiple myeloma at first or subsequent relapse. Additionally, Amgen presented results from a Phase 1b study at ASH (Free ASH Whitepaper) showing Captisol-enabled Kyprolis in combination with vincristine, dexamethasone, PEG-asparaginase and daunorubicin (VXLD) induction therapy showed promising efficacy in highly advanced relapsed/refractory pediatric ALL.

Gilead Sciences announced the FDA granted accelerated approval of a supplemental New Drug Application (NDA) for Veklury in non-hospitalized patients at high risk of disease progression.

Other

Travere Therapeutics announced plans to submit an NDA to the FDA for accelerated approval of sparsentan for IgA nephropathy in the first quarter of 2022 and for focal segmental glomerulosclerosis (FSGS) in mid-2022. Travere, in collaboration with its partner Vifor Pharma, plans to submit a combined IgA nephropathy and FSGS Marketing Authorization Application in mid-2022 for conditional marketing authorization in Europe.

Verona Pharma announced completion of enrollment in the Phase 3 ENHANCE-1 and ENHANCE-2 trials evaluating ensifentrine for the maintenance treatment of chronic obstructive pulmonary disease (COPD) with topline data expected by the end of 2022.

Sermonix Pharmaceuticals closed a $40 million financing to fund lasofoxifene through late-stage clinical development as an oral SERM to treat women with ESR1 breast cancer mutations. Topline data are expected in the first half of 2022 for the Phase 2 ELAINE 1 trial assessing oral lasofoxifene versus intramuscular fulvestrant and the Phase 2 ELAINE 2 trial of oral lasofoxifene in combination with Eli Lilly and Company’s CDK4 and 6 inhibitor Verzenio (abemaciclib) for the treatment of ER+/HER2- breast cancer in patients with an ESR1 mutation.

Ligand provides regular updates on individual partner events through its Twitter account, @Ligand_LGND.

Adjusted Financial Measures

Ligand reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, transaction costs and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call

Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (833) 540-1167 from the U.S. or (929) 517-0358 from outside the U.S., using the conference ID 4832757. To participate via live or replay webcast, a link is available at www.ligand.com.

About OmniAb

The OmniAb discovery platform provides Ligand’s pharmaceutical industry partners with access to diverse antibody repertoires and high-throughput screening technologies to enable discovery of next-generation therapeutics. At the heart of the OmniAb platform is the Biological Intelligence (BI) of our proprietary transgenic animals, including OmniRat, OmniChicken and OmniMouse, which have been genetically modified to generate antibodies with human sequences to facilitate development of human therapeutic candidates. OmniFlic (transgenic rat) and OmniClic (transgenic chicken) address industry needs for bispecific antibody applications though a common light chain approach, and OmniTaur features unique structural attributes of cow antibodies for complex targets. OmniAb animals comprise the most diverse host systems available in the industry and they are optimally leveraged through computational antigen design and immunization methods, paired with high-throughput microfluidic-based single B cell screening and deep computational analysis of next-generation sequencing datasets to identify fully human antibodies with superior performance and developability characteristics. An established core competency focused on ion channels and transporters further differentiates our technology and creates opportunities to further leverage across modalities, including antibody-drug conjugates and others. The OmniAb suite of technologies and differentiating computational capabilities and BI features are combined to offer a highly efficient and customizable end-to-end solution for the growing discovery needs of the global pharmaceutical industry.

About Pelican Expression Technology Platform

Pelican is a robust, validated, cost-effective and scalable platform for recombinant protein production that is especially well-suited for complex, large-scale protein production where traditional systems are not. Multiple global manufacturers have demonstrated consistent success with the platform and the technology is currently out-licensed for numerous commercial and development-stage programs. The versatility of the platform has been demonstrated in the production of enzymes, peptides, antibody derivatives and engineered non-natural proteins. Partners seek the platform as it can contribute significant value to biopharmaceutical development programs by reducing development timelines and costs for manufacturing therapeutics and vaccines. Given pharmaceutical industry trends toward large molecules with increasing structural complexities, Pelican is well positioned to meet these growing needs as the most comprehensive broadly available protein production platform in the industry.

About Captisol

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella, University Distinguished Professor at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled several FDA-approved products, including Gilead Sciences’ VEKLURY, Amgen’s KYPROLIS, Baxter International’s NEXTERONE, Acrotech Biopharma L.L.C.’s and CASI Pharmaceuticals’ EVOMELA, Melinta Therapeutics’ BAXDELA and Sage Therapeutics’ ZULRESSO. There are many Captisol-enabled products currently in various stages of development. Ligand maintains a broad global patent portfolio for Captisol with more than 400 issued patents worldwide relating to the technology (including over 40 in the U.S.) and with the latest expiration date in 2033. Other patent applications covering methods of making Captisol, if issued, extend to 2040.

On February 17, 2022 Lunaphore, a Swiss life sciences company developing technology to enable spatial biology in every laboratory, reported it has entered a collaboration with the Pathology Department at Massachusetts General Hospital to develop an in vitro diagnostic (IVD) that evaluates sensitivity of solid tumors to poly-ADP ribose polymerase (PARP) inhibitors, with an initial focus on ovarian, breast and prostate cancers (Press release, Lunaphore Technologies, FEB 17, 2022, View Source [SID1234608247]). The project will be led by Markus D. Herrmann, M.D., Ph.D., Director of Computational Pathology at Massachusetts General Hospital, who is also Assistant Professor of Pathology at Harvard Medical School. Lunaphore and Dr. Herrmann will collaborate to develop a multiplexed immunofluorescence assay that can measure the expression of multiple proteins using the COMET platform currently installed at Massachusetts General Hospital.

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"Spatial biology tools enhance the visualization and quantification of the expression of multiple proteins at once, providing valuable information for treatment decisions," said Diego G. Dupouy, Ph.D., Chief Technology Officer of Lunaphore. "This partnership will further solidify spatial biology’s role in both improved diagnostics and treatment selection with targeted therapies."

PARP inhibitors are targeted therapies that are designed to kill cancer cells by preventing them from repairing their damaged DNA. The DNA repair pathway called homologous recombination is of clinical interest as tumors with homologous recombination deficiency (HRD) have been found to be sensitive to PARP inhibitors. However, current methods of identifying HRD in tumors have been varied and imperfect. For example, current biomarker assays are not able to identify epigenetic changes in HRD genes with traditional genotyping assays.

"We look forward to developing a quantitative image-based proteomic assay to comprehensively assess DNA repair at the single-cell level in spatial tissue context and to better predict response to PARP inhibitor therapy," said Dr. Herrmann. "Adopting spatial biology methods can help us gain pathophysiological insight into the biological processes involved in DNA repair in tumor tissue that may yield clinically actionable biomarkers to improve treatment selection and patient outcomes."

On February 17, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics leveraging CRISPR-based technologies, reported that it will be hosting two virtual investor events in February (Press release, Intellia Therapeutics, FEB 17, 2022, View Source [SID1234608213]).

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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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Fourth Quarter and Full-Year 2021 Earnings – February 24, at 8:00 a.m. ET
Intellia will present its fourth quarter and full-year 2021 financial results.

To join the call, U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
NTLA-2001 Interim Clinical Data Update – February 28, at 4:30 p.m. ET
Intellia will present additional interim clinical data from the ongoing Phase 1 study of NTLA-2001 in patients with transthyretin (ATTR) amyloidosis with polyneuropathy. The event will include a presentation by Ed Gane, MBChB, MD, FRACP, MNZM, Professor of Medicine at the University of Auckland, New Zealand and Chief Hepatologist, Transplant Physician and Deputy Director of the New Zealand Liver Transplant Unit at Auckland City Hospital and an investigator in the ongoing NTLA-2001 Phase 1 study, along with members of Intellia’s management team.

To join the webcast, please visit this link, or the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com.
A replay of the events will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com for at least 30 days following the event.

About NTLA-2001
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first single-dose treatment for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001’s potential as a single-administration therapeutic. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron.