8-K
INSMED Inc (INSM)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of
The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):February 15, 2022
INSMED INCORPORATED
Exact name of registrant as specified in its charter)
| Virginia | 000-30739 | 54-1972729 |
|---|---|---|
| (State or other jurisdiction | (Commission | (IRS Employer |
| of incorporation) | File Number) | Identification No.) |
| 700 US Highway 202/206<br><br> <br>Bridgewater,<br> New Jersey | 08807<br><br> <br>(Zip Code) | |
| --- | --- | |
| (Address of principal executive offices) |
Registrant’s telephone number, including area code: (908) 977-9900
Not Applicable
(Former name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
| ☐ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
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| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
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| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
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Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which<br><br> <br>registered |
|---|---|---|
| Common Stock, par value $0.01 per share | INSM | Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17
CFR 230.405\) or Rule 12b-2 of the Securities Exchange Act of 1934 \(17 CFR 240.12b-2\).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 2.02. | Results of Operations and Financial Condition. |
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On February 17, 2022, Insmed Incorporated (the “Company”) issued a press release regarding its financial results for the three months and fiscal year ended December 31, 2021. A copy of this press release is furnished herewith as Exhibit 99.1 pursuant to this Item 2.02. The slide presentation to be used during the conference call referenced in the press release is attached hereto as Exhibit 99.2 and incorporated herein by reference.
The information contained herein, including the exhibits attached hereto, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as shall be expressly set forth by specific reference in such a filing.
| Item 5.02. | Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers. |
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On February 15, 2022, Steinar J. Engelsen, M.D. notified the board of directors (the “Board”) of the Company that he will not stand for re-election to the Board upon the expiration of his current term as a Class I director, which occurs at the Company’s 2022 Annual Meeting of Shareholders (the “Annual Meeting”). Dr. Engelsen will continue to serve as a member of the Board and of the Audit Committee and Science and Technology Committee of the Board until the Annual Meeting. Dr. Engelsen’s decision not to stand for re-election to the Board was not a result of any disagreement with the Company on any matter relating to the Company’s operations, policies or practices.
| Item 9.01 | Financial Statements and Exhibits. |
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(d) Exhibits.
| Exhibit<br><br> <br>No. | Description |
|---|---|
| 99.1 | Press Release by Insmed Incorporated on February 17, 2022. |
| 99.2 | Insmed Incorporated February 17, 2022 Presentation. |
| 104 | Cover Page Interactive Date File (embedded within the Inline XBRL<br> document). |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| INSMED INCORPORATED | ||
|---|---|---|
| Dated: February 17, 2022 | By: | /s/ Michael Smith |
| Name: | Michael Smith | |
| Title: | General Counsel and Corporate Secretary |
Exhibit 99.1
Insmed Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business and Pipeline Update
—ARIKAYCE^®^ (amikacin liposome inhalation suspension) Total Revenue of $56.1 Million for the Fourth Quarter and $188.5 Million for the Full Year 2021; Anticipate at Least 30% Year Over Year Annual Growth for 2022—
—Enrollment Timelines Provided for Brensocatib Phase 3 ASPEN Study and ARIKAYCE Post-Marketing Studies (ARISE and ENCORE)—
—Planning to Advance Brensocatib in Two Additional Potential Indications: Chronic Rhinosinusitis without Nasal Polyps (CRS) and Hidradenitis Suppurativa (HS)—
—Cash Runway into 2024 to Support Company’s Ongoing Programs—
BRIDGEWATER, N.J., February 17, 2022 /PRNewswire/ — Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today 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.
“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.”
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. |
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| • | 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<br> Italy, France, and England. |
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| • | 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<br> 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<br> newly diagnosed MAC lung disease using the PRO tool, by the end of 2023. |
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Brensocatib
| • | 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<br> anticipates completing enrollment in this study in early 2023. |
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| • | 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. |
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| • | 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<br> clinic in 2022. Both CRS and HS are serious diseases in which patients today face significant unmet needs. |
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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<br> 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<br> over a 16-week treatment period. As planned, site initiation for this study began in late 2021. |
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| • | 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<br> a 16-week treatment period. |
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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<br> indication for our first candidate from this portfolio by the end of 2022. |
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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. |
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| • | 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<br> in the U.S. and $6.9 million in Europe for the full year 2020. |
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| • | 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<br> product revenues (excluding amortization of intangible assets) was $44.2 million compared to $39.9 million in 2020. |
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| • | 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<br> million compared to $181.2 million in 2020. |
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| • | 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<br> $234.3 million, compared to $203.6 million in 2020. |
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| • | 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<br> 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. |
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Balance 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; |
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| (ii) | launch activities for ARIKAYCE in initial European countries and in Japan; and |
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| (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<br> bronchiectasis, (c) advancement of the Phase 2 clinical development programs of TPIP, and (d) advancement of our translational medicine efforts. |
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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.
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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<br><br> <br><br><br> <br>ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis,<br> bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases. |
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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.
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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.
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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.
Please see Full Prescribing Information.
About Insmed
Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product is a first-in-disease therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company is also progressing a robust pipeline of investigational therapies targeting areas of serious unmet need, including neutrophil-mediated inflammatory diseases and rare pulmonary disorders. Insmed is headquartered in Bridgewater, New Jersey, with a growing footprint across Europe and in Japan. For more information, visit www.insmed.com.
Forward-looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. “Forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “intends,” “potential,” “continues,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements.
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The forward-looking statements in this press release are based upon the Company’s current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company’s actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timing discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: failure to obtain, or delays in obtaining, regulatory approvals for ARIKAYCE outside the U.S., Europe or Japan, or for the Company’s product candidates in the U.S., Europe, Japan or other markets, including regulatory approval for the Lamira^®^ Nebulizer System and the drug delivery device for TPIP in each market and for each usage; failure to successfully commercialize ARIKAYCE, the Company's only approved product, in the U.S., Europe or Japan (amikacin liposome inhalation suspension, Liposomal 590 mg Nebuliser Dispersion, and amikacin sulfate inhalation drug product, respectively), or to maintain U.S., European or Japanese approval for ARIKAYCE; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; impact of the COVID-19 pandemic and efforts to reduce its spread on the Company’s business, employees, including key personnel, patients, partners and suppliers; risk that brensocatib does not prove effective or safe for patients in ongoing and future clinical studies, including the ASPEN study; risk that TPIP does not prove to be effective or safe for patients in ongoing and future clinical studies; uncertainties in the degree of market acceptance of ARIKAYCE by physicians, patients, third-party payors and others in the healthcare community; the Company’s inability to obtain full approval of ARIKAYCE from the U.S. Food and Drug Administration, including the risk that the Company will not successfully or in a timely manner complete the study to validate a PRO tool and the confirmatory post-marketing clinical trial required for full approval of ARIKAYCE; inability of the Company, PARI or the Company’s other third-party manufacturers to comply with regulatory requirements related to ARIKAYCE or the Lamira^®^ Nebulizer System; the Company’s inability to obtain adequate reimbursement from government or third-party payors for ARIKAYCE or acceptable prices for ARIKAYCE; development of unexpected safety or efficacy concerns related to ARIKAYCE or the Company’s product candidates; inaccuracies in the Company’s estimates of the size of the potential markets for ARIKAYCE, brensocatib, TPIP or the Company’s other product candidates or in data the Company has used to identify physicians, expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; the Company’s inability to create an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of ARIKAYCE or any of the Company’s product candidates that are approved in the future; failure to obtain regulatory approval to expand ARIKAYCE’s indication to a broader patient population; risk that the Company’s competitors may obtain orphan drug exclusivity for a product that is essentially the same as a product the Company is developing for a particular indication; failure
to successfully predict the time and cost of development, regulatory approval and commercialization for novel gene therapy products; failure to successfully conduct future clinical trials for
ARIKAYCE, brensocatib, TPIP and the Company’s other product candidates due to the Company’s limited experience in conducting preclinical development activities and clinical trials necessary for regulatory approval and its potential inability to
enroll or retain sufficient patients to conduct and complete the trials or generate data necessary for regulatory approval, among other things; risks that the Company’s clinical studies will
be delayed or that serious side effects will be identified during drug development; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE or the Company’s product candidates for commercial or
clinical needs, to conduct the Company’s clinical trials, or to comply with the Company’s agreements or laws and regulations that impact the Company’s business or agreements with the Company; failure
to comply with the Company’s obligations in the Company’s third party agreements; the Company’s inability to attract and retain key personnel or to effectively manage the Company’s growth;
the Company’s inability to successfully integrate its recent acquisitions and appropriately manage the amount of management’s time and attention devoted to integration activities; risks that the Company’s acquired technologies, products and product
candidates are not commercially successful; the Company’s inability to adapt to its highly competitive and changing environment; risk that the Company is unable to maintain its significant
customers; risk that government healthcare reform materially increases the Company’s costs and damages its financial condition; the Company’s inability to adequately protect its intellectual property rights or prevent disclosure of its trade
secrets and other proprietary information and costs associated with litigation or other proceedings related to such matters; restrictions or other obligations imposed on the Company by agreements related to ARIKAYCE or the Company’s product
candidates, including its license agreements with PARI and AstraZeneca AB, and failure of the Company to comply with its obligations under such agreements; the cost and potential reputational damage resulting from litigation to which the Company is
or may become a party, including product liability claims; risk that the Company’s operations are subject to a material disruption in the event of a cybersecurity attack or issue; business disruptions or expenses related to the upgrade to the
Company’s enterprise resource planning \(ERP\) system; the Company’s limited experience operating internationally; changes in laws and regulations applicable to the Company’s business, including any pricing reform, and failure to comply with such
laws and regulations; the Company’s history of operating losses, and the possibility that the Company may never achieve or maintain profitability; goodwill impairment charges affecting the Company’s results of operations and financial condition;
inability to repay the Company’s existing indebtedness and uncertainties with respect to the Company’s ability to access future capital; and delays in the execution of plans to build out an additional third-party manufacturing facility approved by
the appropriate regulatory authorities and unexpected expenses associated with those plans.
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The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company’s forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company’s business, please see the factors discussed in Item 1A, “Risk Factors,” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021 and any subsequent Company filings with the Securities and Exchange Commission (SEC).
The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
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Financial Statements Follow
INSMED INCORPORATED
Consolidated Statements of Net Loss
(in thousands, except per share data)
(unaudited)
| Three Months Ended<br><br> <br>December 31, | Twelve Months Ended<br><br> <br>December 31, | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2021 | 2020 | 2021 | 2020 | |||||||||
| Product revenues, net | $ | 56,124 | $ | 41,415 | $ | 188,461 | $ | 164,413 | ||||
| Operating expenses: | ||||||||||||
| Cost of product revenues (excluding amortization of intangible assets) | 13,288 | 10,862 | 44,152 | 39,872 | ||||||||
| Research and development | 76,352 | 67,814 | 272,744 | 181,157 | ||||||||
| Selling, general and administrative | 65,266 | 56,019 | 234,273 | 203,613 | ||||||||
| Amortization of intangible assets | 1,262 | 1,258 | 5,052 | 5,003 | ||||||||
| Change in fair value of deferred and contingent consideration liabilities | (966 | ) | - | 7,334 | - | |||||||
| Total operating expenses | 155,202 | 135,953 | 563,555 | 429,645 | ||||||||
| Operating loss | (99,078 | ) | (94,538 | ) | (375,094 | ) | (265,232 | ) | ||||
| Investment income | 61 | 26 | 174 | 1,703 | ||||||||
| Interest expense | (11,350 | ) | (7,499 | ) | (40,473 | ) | (29,564 | ) | ||||
| Loss on extingushment of debt | - | - | (17,689 | ) | - | |||||||
| Other (expense) income, net | (2,652 | ) | 419 | (3,330 | ) | 405 | ||||||
| Loss before income taxes | (113,019 | ) | (101,592 | ) | (436,412 | ) | (292,688 | ) | ||||
| (Benefit) provision for income taxes | (41 | ) | 621 | (1,758 | ) | 1,402 | ||||||
| Net loss | $ | (112,978 | ) | $ | (102,213 | ) | $ | (434,654 | ) | $ | (294,090 | ) |
| Basic and diluted net loss per share | $ | (0.95 | ) | $ | (1.00 | ) | $ | (3.88 | ) | $ | (3.01 | ) |
| Weighted average basic and diluted common shares outstanding | 118,502 | 102,297 | 112,111 | 97,605 |
9
INSMED INCORPORATED
Consolidated Balance Sheets
(in thousands, except par value and share data)
| As of<br><br> <br>December 31, 2020 | |||||
|---|---|---|---|---|---|
| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | 716,782 | $ | 532,756 | ||
| Accounts receivable | 24,351 | 16,562 | |||
| Inventory | 67,009 | 49,592 | |||
| Prepaid expenses and other current assets | 28,898 | 23,982 | |||
| Total current assets | 837,040 | 622,892 | |||
| Marketable securities | 50,043 | - | |||
| Fixed assets, net | 52,955 | 53,953 | |||
| Finance lease right-of-use assets | 9,256 | 10,334 | |||
| Operating lease right-of-use assets | 33,305 | 32,946 | |||
| Intangibles, net | 73,809 | 49,261 | |||
| Goodwill | 136,110 | - | |||
| Other assets | 50,990 | 26,769 | |||
| Total assets | 1,243,508 | $ | 796,155 | ||
| Liabilities and shareholders' equity | |||||
| Current liabilities: | |||||
| Accounts payable | 35,784 | $ | 42,853 | ||
| Accrued liabilities | 60,665 | 37,807 | |||
| Accrued compensation | 28,581 | 25,591 | |||
| Finance lease liabilities | 609 | 1,081 | |||
| Operating lease liabilities | 9,527 | 11,475 | |||
| Total current liabilities | 135,166 | 118,807 | |||
| Debt, long-term | 566,588 | 356,318 | |||
| Contingent consideration | 75,668 | - | |||
| Finance lease liabilities, long-term | 14,103 | 14,713 | |||
| Operating lease liabilities, long-term | 21,441 | 21,255 | |||
| Other long-term liabilities | 20,074 | 9,178 | |||
| Total liabilities | 833,040 | 520,271 | |||
| Shareholders' equity: | |||||
| Common stock, 0.01 par value; 500,000,000 authorized shares, 118,738,266 and 102,763,060 issued and outstanding shares at<br> December 31, 2021 and December 31, 2020, respectively | 1,187 | 1,028 | |||
| Additional paid-in capital | 2,673,556 | 2,105,252 | |||
| Accumulated deficit | (2,265,243 | ) | (1,830,589 | ) | |
| Accumulated other comprehensive income | 968 | 193 | |||
| Total shareholders' equity | 410,468 | 275,884 | |||
| Total liabilities and shareholders' equity | 1,243,508 | $ | 796,155 |
All values are in US Dollars.
10
Contact:
Investors:
Eleanor Barisser
Associate Director, Investor Relations
Insmed
(718) 594-5332
eleanor.barisser@insmed.com
Media:
Mandy Fahey
Executive Director, Corporate Communications
Insmed
(732) 718-3621
amanda.fahey@insmed.com
11
Exhibit 99.2
February 17, 2022
This presentation contains forward-looking statements that involve substantial risks and uncertainties. “Forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “intends,” “potential,” “continues,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements.The forward-looking statements in this presentation are based upon the Company’s current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company’s actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timing discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: failure to obtain, or delays in obtaining, regulatory approvals for ARIKAYCE outside the U.S., Europe or Japan, or for the Company’s product candidates in the U.S., Europe, Japan or other markets, including regulatory approval for the Lamira® Nebulizer System and the drug delivery device for TPIP in each market and for each usage; failure to successfully commercialize ARIKAYCE, the Company's only approved product, in the U.S., Europe or Japan (amikacin liposome inhalation suspension, Liposomal 590 mg Nebuliser Dispersion, and amikacin sulfate inhalation drug product, respectively), or to maintain U.S., European or Japanese approval for ARIKAYCE; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; impact of the COVID-19 pandemic and efforts to reduce its spread on the Company’s business, employees, including key personnel, patients, partners and suppliers; risk that brensocatib does not prove effective or safe for patients in ongoing and future clinical studies, including the ASPEN study; risk that TPIP does not prove to be effective or safe for patients in ongoing and future clinical studies; uncertainties in the degree of market acceptance of ARIKAYCE by physicians, patients, third-party payors and others in the healthcare community; the Company’s inability to obtain full approval of ARIKAYCE from the U.S. Food and Drug Administration, including the risk that the Company will not successfully or in a timely manner complete the study to validate a PRO tool and the confirmatory post-marketing clinical trial required for full approval of ARIKAYCE; inability of the Company, PARI or the Company’s other third-party manufacturers to comply with regulatory requirements related to ARIKAYCE or the Lamira® Nebulizer System; the Company’s inability to obtain adequate reimbursement from government or third-party payors for ARIKAYCE or acceptable prices for ARIKAYCE; development of unexpected safety or efficacy concerns related to ARIKAYCE or the Company’s product candidates; inaccuracies in the Company’s estimates of the size of the potential markets for ARIKAYCE, brensocatib, TPIP or the Company’s other product candidates or in data the Company has used to identify physicians, expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; the Company’s inability to create an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of ARIKAYCE or any of the Company’s product candidates that are approved in the future; failure to obtain regulatory approval to expand ARIKAYCE’s indication to a broader patient population; risk that the Company’s competitors may obtain orphan drug exclusivity for a product that is essentially the same as a product the Company is developing for a particular indication; failure to successfully predict the time and cost of development, regulatory approval and commercialization for novel gene therapy products; failure to successfully conduct future clinical trials for ARIKAYCE, brensocatib, TPIP and the Company’s other product candidates due to the Company’s limited experience in conducting preclinical development activities and clinical trials necessary for regulatory approval and its potential inability to enroll or retain sufficient patients to conduct and complete the trials or generate data necessary for regulatory approval, among other things; risks that the Company’s clinical studies will be delayed or that serious side effects will be identified during drug development; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE or the Company’s product candidates for commercial or clinical needs, to conduct the Company’s clinical trials, or to comply with the Company’s agreements or laws and regulations that impact the Company’s business or agreements with the Company; failure to comply with the Company’s obligations in the Company’s third party agreements; the Company’s inability to attract and retain key personnel or to effectively manage the Company’s growth; the Company’s inability to successfully integrate its recent acquisitions and appropriately manage the amount of management’s time and attention devoted to integration activities; risks that the Company’s acquired technologies, products and product candidates are not commercially successful; the Company’s inability to adapt to its highly competitive and changing environment; risk that the Company is unable to maintain its significant customers; risk that government healthcare reform materially increases the Company’s costs and damages its financial condition; the Company’s inability to adequately protect its intellectual property rights or prevent disclosure of its trade secrets and other proprietary information and costs associated with litigation or other proceedings related to such matters; restrictions or other obligations imposed on the Company by agreements related to ARIKAYCE or the Company’s product candidates, including its license agreements with PARI and AstraZeneca AB, and failure of the Company to comply with its obligations under such agreements; the cost and potential reputational damage resulting from litigation to which the Company is or may become a party, including product liability claims; risk that the Company’s operations are subject to a material disruption in the event of a cybersecurity attack or issue; business disruptions or expenses related to the upgrade to the Company’s enterprise resource planning (ERP) system; the Company’s limited experience operating internationally; changes in laws and regulations applicable to the Company’s business, including any pricing reform, and failure to comply with such laws and regulations; the Company’s history of operating losses, and the possibility that the Company may never achieve or maintain profitability; goodwill impairment charges affecting the Company’s results of operations and financial condition; inability to repay the Company’s existing indebtedness and uncertainties with respect to the Company’s ability to access future capital; and delays in the execution of plans to build out an additional third-party manufacturing facility approved by the appropriate regulatory authorities and unexpected expenses associated with those plans.The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company’s forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company’s business, please see the factors discussed in Item 1A, “Risk Factors,” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021 and any subsequent Company filings with the Securities and Exchange Commission (SEC).The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this presentation. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
Refractory MAC lung disease Front Line MAC lung disease: ARISE & ENCORE Bronchiectasis: ASPEN Cystic Fibrosis (CF)Chronic Rhinosinusitis without Nasal Polyps (CRS) Hidradenitis Suppurativa (HS) Pulmonary arterial hypertension (PAH) Pulmonary hypertension associated with interstitial lung disease (PH-ILD)Other rare pulmonary disorders Gene TherapyProtein De-immunization Breakthrough Manufacturing Not for promotional use
$766.8M $188.5M $159.5M $16M $12.9M 13.2%$44.2M$272.7M$234.3M Cash to support ongoing business into 2024, driven by three factors: ARIKAYCE revenue How fully and broadly we pursuepipeline programs How we resource launch readiness
Currently 50% Enrolled Anticipated Enrollment CompletionEarly 2023 40 Countries ~460 Sites Patient Enrollment Initiated December 2020 DSMB Approval of Study Continuation
EOS, end of study; EOT, end of treatment; QD, once daily; Follow-up Period 4 weeks Single-Blind Treatment period 4 weeks Baselineday 1 randomization EOTday 28 EOSday 56 Up to 4 weeks PK (Cmax and AUC) of brensocatib in CF subjects after 28 days of QD oral administration Safety and tolerability of brensocatib Change from baseline in sputum concentration of NE, Cat G and PR3 over 4-week treatment periodChange from baseline in blood concentration of NE, Cat G and PR3 over 4-week treatment periodChange from baseline in lung function atDay 28 Change from baseline in CFQ-R (Cystic Fibrosis Questionnaire-Revised) at week 4To evaluate the PK/PD relationship between dose/exposure and NE activityTo evaluate the PK/PD relationship between dose/exposure and FEV1
No approved treatments Many patients do not respond to corticosteroids and/or endoscopic sinus surgery (ESS) ~33M patients with CRS1 and ~26Mpatients with CRSsNP in the U.S. Targeting severe patient population Approximately 155K patients require ESS annually in the U.S.; Approximately 15%, or 23K patients require repeat surgery annually.1 Only one approved treatment Patients may need multiple therapies and/or surgery to maintain control of disease ~300K diagnosed patients in theU.S.2,3 Targeting moderate to severe patient population ~1/3 of diagnosed patients havemoderate to severe disease Addressable market of ~100K patients with moderate to severe disease in the U.S. 2 Addressable market of ~23K -~155K annually in the U.S.1Cho et. Al, Chronic Rhinosinusitis without Nasal Polyps J Allergy Clin Immunol Pract. 2016 ; 4(4): 575–582. doi:10.1016/j.jaip.2016.04.0152Phan et al, Global prevalence of hidradenitis suppurativa and geographical variation—systematic review and metaanalysis Biomedical Dermatology (2020) 4:23Puri, Ajay: Hidradenitis Suppurativa Executive Insights, DRG Nov 2019 Inflamed lesions and nodulesDeep abscessesDraining fistulasSevere scars Underarm Breast area Groin
4 weeks Treatment period 24 weeks Baseline EOT(primary analysis) EOS 4 weeks Change in Total Symptom Score (TSS) NSPs in blood Change in LMK-CT (Lund-MacKaycomputed tomography) scoreChange in Sino-nasal Outcome Test 22 scoreChange in VAS (visual analog scale) Score ≥2 of the following symptoms for 12 weeks:Nasal congestion/blockage/ obstruction with moderate or severe symptom severity (score 2 or 3) and a weekly average severity of >1 at the time of randomization ANDLoss of smell ORRhinorrhea (anterior/posterior)Sino-nasal Outcome Test 22 of at least 30Bilateral LMK-CT score ≥6 EOS, end of study; EOT, end of treatment; QD, once daily
Period 2OLE 36 weeks Period 116 weeks Baseline EOT(primary analysis) EOS Up to 4 weeks Percentage of subjects achieving 50% improvement as measured by Hidradenitis Suppurativa Clinical Response (HiSCR) at Week 16 Mean change in total AN (total abscesses and inflammatory nodules) count at Week 16% patients with HS flaresHiSCR 75 and 90Mean Change from Baseline in Skin Pain on the HS Numeric Rating Scale (NRS) at Week 16IHS4Proportion of patients requiring rescue therapy over the 16-week treatment period Hurley Stage II or IIIPrevious anti-TNF α Treatment Adults with mod/severe disease Excludes patients on TNFα therapy in previous 6 months Inadequate response to systemic antibiotics EOS, end of study; EOT, end of treatment; QD, once daily.
Currently 50% enrolled Anticipated enrollment completion in 2022 ARISE ENCORE Enrollment Completion Anticipated in 2023Once ARISE is fully enrolled, anticipate enrollment in ENCORE to accelerate Data Expected1H23
TPIP is an investigational drug product that has not been approved for any indication in any jurisdiction. Simplifying dosing with once daily administration Providing prolonged, localized pulmonary vasodilationOffering improved tolerability,with fewer prostanoid-related side effectsEnabling higher dosing for superior efficacywith the potential for disease-modifying effect
4 weeks Treatment period 16 weeks Baseline EOT(primary analysis) EOS 4 weeks 2:1 active : placebo Change from baseline in pulmonaryvascular resistance (PVR) at week 16 Change from baseline in exercise capacity (6MWD)Change from baseline in WHO Functional classChange from baseline in Quality ofLife (CAMPHOR questionnaire)Change from baseline in biomarkers of cardiac stress (NT-proBNP) EOS, end of study; EOT, end of treatment; QD, once daily.
4 weeks Treatment period 16 weeks Baseline EOT(primary analysis) EOS 4 weeks PK 3:1 active : placebo Safety and tolerabilityOxygenation Improvement in exercise capacity (6MWD)Improvement in biomarkers of cardiac stress (NT-proBNP)Improvement in lung function andpulmonary vascular volume (FRI)Improvements in Quality of Life (CAMPHOR questionnaire) EOS, end of study; EOT, end of treatment; QD, once daily.
Phase 2aData expected in 2022 Phase 2bFirst site initiation late 2021 PAH Phase 2Site initiation underwayPH-ILD
IND Candidate IdentifiedManufacturing & IND Enabling Studies IND IND Manufacturing & IND Enabling StudiesDeimmunization and POC Studies IND Enabling StudiesDeimmunization and POC Studies IND Enabling Studies Deimmunization and POC Studies IND Enabling Studies IND Capsid Deimmunization IND Transgene Deimmunization and POC StudiesIND Enabling Studies IND IND Enabling Studies IND IND Enabling Studies IND GMP Manufacturing Candidate Identified Deimmunization and POC Studies IND Enabling Studies IND GMP Manufacturing
U.S.JapanEurope CRSHSBronchiectasisCF
12K-17KRefractory MAC patients(2019E)* *Source: Internal analysis of published NTM epidemiology, primary market research with treating HCPs, and anonymized patient level claims data in US U.S. ARIKAYCE franchise has remained resilient throughout the ongoing pandemic
15K-18KRefractory MACpatients(2018E)* *Source: Internal analysis of published NTM epidemiology, primary market research with treating HCPs, and anonymized patient level claims data in US Japan expected to be thesecond largest revenue generator of the markets we are pursuing
*Source: Internal analysis of published NTM epidemiology, primary market research with treating HCPs, and anonymized patient level claims data in US† European 5 comprised of France, Germany, Italy, Spain and the United Kingdom 1,400Refractory MACpatients(2018E)* ARIKAYCE currently available and reimbursed in Germany, the Netherlands, Wales, and Scotland Anticipate reimbursement decisions in England, France, and Italy in 2022
~340K - ~540K diagnosed patients in U.S. ~350K - ~500K diagnosed patients in Europe ~1M - ~5M diagnosed patients in Asia-Pacific ~1.7M – 6M total diagnosed patients worldwide ~35K diagnosed patients in U.S.~33K diagnosed patients in Europe~70K total diagnosed patientsworldwide ~26M patients with CRSsNP in the U.S.*155K patients require ESS annually in the US. 15%, or 23K patients, 1 require repeat surgery each yearAddressable incidence market of severe patients of ~23K-~155K annually in U.S.Europe market estimated to be~20% smaller than U.S. marketJapan market estimated to be~80% smaller than U.S. market ~300K diagnosed patients in the U.S.2,3~1/3 of diagnosed patients have moderate to severe disease.2 Addressable market of ~100K patients with moderate to severe disease in U.S.Europe market estimated to be 3-4x U.S. marketJapan market estimated to be~10% of U.S. market No approved treatments CFTR modulators, mucolytics, antibiotics No approved treatmentsDupixent, Nucala, and Xolair approved for CRS with nasal polyps (targeting eosinophilic- driven disease) Humira Treatment regimens arebuilt around airway clearance and antibiotic therapy to reduce symptom burden and risk of exacerbations No approved anti-inflammatory treatments targeting neutrophil- mediated inflammation Significant burden for patients who do not respond to corticosteroids and/or endoscopic sinus surgery Significant burden for patients who require multiple therapies and/or surgery to manage disease Targeting neutrophil- mediated inflammation,the key driver of exacerbations and disease progression Targeting neutrophil- mediated inflammation,the key driver of exacerbations and disease progression Potential symptom reliefby reducing the contribution of neutrophil serine proteasesPotential for first-in-disease therapy Targeting a key component of the inflammatory process observed in HS *CRSsNP prevalence proxy based on incidence rate1Cho et. Al, Chronic Rhinosinusitis without Nasal Polyps J Allergy Clin Immunol Pract. 2016 ; 4(4): 575–582. doi:10.1016/j.jaip.2016.04.0152Phan et al, Global prevalence of hidradenitis suppurativa and geographical variation—systematic review and metaanalysis Biomedical Dermatology (2020) 4:23Puri, Ajay: Hidradenitis Suppurativa Executive Insights, DRG Nov 2019
2022 2023 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 ARISE ENCORE ASPEN Phase 2 in CF Phase 2a in PAH Enrollment Completion Top-line Data Enrollment Completion Enrollment Completion Top-line Data Top-line Data Begin IND-enabling Studies Gene Therapy ProteinDe-immunization Manufacturing IND Filing in New Non-pulmonary Indication Begin GMP Manufacturing Continue De-immunization and Proof-of-Concept Studies
