Full Press Release Details
Insmed Incorporated Commercial Presentation June 4, 2024
Forward Looking Statement Forward Looking Statements 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 timings discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: the risk that the full data set from the
ASPEN study or data generated in further clinical trials of brensocatib will not be consistent with the top-line results of the ASPEN study; failure to obtain, or delays in obtaining, regulatory approvals for brensocatib, TPIP or our other
product candidates in the U.S., Europe or Japan or regulatory approvals for potential future brensocatib or TPIP indications; failure to successfully commercialize brensocatib, TPIP or our other product candidates, if approved by applicable
regulatory authorities, in the U.S., Europe or Japan, or to maintain U.S., European or Japanese approval for brensocatib, TPIP or our other product candidates, if approved; uncertainties or changes in the degree of market acceptance of ARIKAYCE
or, if approved, brensocatib or TPIP by physicians, patients, third-party payors and others in the healthcare community; failure to continue to successfully commercialize ARIKAYCE, our 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; our inability to obtain full approval
of ARIKAYCE from the FDA, including the risk that we will not successfully or in a timely manner validate a patient reported outcome (PRO) tool and complete the confirmatory post-marketing clinical trial required for full approval of ARIKAYCE,
or our failure to obtain regulatory approval to expand ARIKAYCE's indication to a broader patient population; failure to obtain, or delays in obtaining, regulatory approvals for ARIKAYCE outside the U.S., Europe or Japan, including separate
regulatory approval for Lamira in each market and for each usage; failure of third parties on which the Company is dependent, including Esteve Pharmaceuticals, S.A., Thermo Fisher Scientific, Inc. or the Company's other third-party
manufacturers, to manufacture sufficient quantities of ARIKAYCE, brensocatib, or TPIP 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; our inability to obtain and maintain adequate reimbursement from government or third-party payors for ARIKAYCE or, if approved, brensocatib or TPIP, or acceptable prices for ARIKAYCE or, if
approved, brensocatib or TPIP; our inability to create or maintain an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of ARIKAYCE or, if approved,
brensocatib or TPIP; risk that our competitors may obtain orphan drug exclusivity for a product that is essentially the same as a product we are developing for a particular indication; inaccuracies in our estimates of the size of the potential
markets for ARIKAYCE, brensocatib, TPIP or our other product candidates, in our related estimates of peak sales potential, or in data we have used to identify physicians, expected rates of patient uptake, duration of expected treatment, or
expected patient adherence or discontinuation rates; development of unexpected safety or efficacy concerns related to ARIKAYCE, brensocatib, TPIP or our other product candidates; restrictions or other obligations imposed on us by agreements
related to brensocatib, including our license agreement with AstraZeneca AB, and failure to comply with our obligations under such agreements; failure to successfully conduct future clinical trials for ARIKAYCE, brensocatib, TPIP or our other
product candidates, including due to the Company's potential inability to enroll or retain sufficient patients to conduct and complete the trials or generate data necessary for regulatory approvals, among other things; risks that the Company's
clinical studies will be delayed, that serious side effects will be identified during drug development, or that any protocol amendments submitted will be rejected; risks that interim or partial data sets are not representative of a complete or
larger data set or that blinded data will not be predictive of unblinded data; 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 strength and enforceability of the Company's intellectual property rights or the rights of third parties; the cost and potential reputational damage resulting from litigation to which the
Company may become a party, including product liability claims; changes in laws and regulations applicable to our business, including any pricing reform and laws that impact our ability to utilize certain third parties in the research,
development or manufacture of our product candidates, and failure to comply with such laws and regulations; inability to adapt to our highly competitive and changing environment; risk that we are unable to maintain our significant
customers; our inability to attract and retain key personnel or to effectively manage our growth; risk that government healthcare reform materially increases our costs and damages our financial condition; risk that our operations are subject to
a material disruption in the event of a cybersecurity attack or issue; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; deterioration in general economic conditions in
the U.S., Europe, Japan and globally, including the effect of prolonged periods of inflation, affecting us, our suppliers, third-party service providers and potential partners; and inability to repay the Company's existing indebtedness and
uncertainties with respect to the Company's need and ability to access future capital. 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, 2023 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.
Forward Looking Statement Additional Disclaimers With respect to the blended and
blinded data observed from the ongoing TPIP study in pulmonary arterial hypertension, the dose titration and efficacy analyses were based on data available as of April 1, 2024, and the safety analyses were based on data available as of January
25, 2024, respectively. These findings may not be representative of results after the study is completed and all data are collected and analyzed. As a result, later interim data readouts and final data from this study may be materially
different than the observations described herein, including with respect to efficacy, safety and tolerability of TPIP. Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data
obtained from third-party sources, as well as our own internal estimates and research. While we believe the information in these third-party sources to be reliable as of the date of this presentation, we have not independently verified any such
information or the underlying assumptions relied on in such third-party sources. In addition, while we believe our internal research is reliable, such research has not been verified by any independent source. Please be aware that brensocatib
and TPIP are investigational products that have not been approved for sale or found safe or effective by the FDA or any regulatory authority. In addition, ARIKAYCE has not been approved for the treatment of all patients with MAC lung disease.
This presentation is not promotion or advertisement of ARIKAYCE, brensocatib, or TPIP. Insmed and ARIKAYCE are registered trademarks of Insmed Incorporated. All other trademarks are property of their respective owner(s).
This presentation is intended to help frame the commercial potential for TPIP,
ARIKAYCE and brensocatib POSITIVE CLINICAL DATA have transformed Insmed's value In the last 9 months
Refractory MAC NTM** All MAC NTM Bronchiectasis CRSsNP HS PH-ILD PAH A
portfolio of multi-indication programs each with blockbuster potential* TPIP PH-ILD=Pulmonary hypertension due to interstitial lung disease; PAH=Pulmonary arterial hypertension MAC=Mycobacterium avium complex; NTM=nontuberculous
mycobacterial Bronchiectasis refers to non-cystic fibrosis bronchiectasis; CRSsNP=Chronic rhinosinusitis sans nasal polyps; HS=Hidradenitis suppurativa *If approved **ARIKAYCE is currently approved for refractory MAC
NTM ARIKAYCE BRENSOCATIB
30K brensocatib Bronchiectasis TPIP PH-ILD Potential addressable patients
anticipated to grow substantially with a steady cadence of launches* ARIKAYCE Refractory MAC 1.25M brensocatib CRSsNP 400K TPIP PAH 135K 90K future (through 2030) ~2.5M Bronchiectasis refers to non-cystic fibrosis bronchiectasis;
MAC/MAC LD=mycobacterium avium complex lung disease; CRSsNP=chronic rhinosinusitis without nasal polyps, PH-ILD= pulmonary hypertension due to interstitial lung disease; PAH=pulmonary arterial hypertension; HS=hidradenitis suppurativa *If
approved Note: Prevalence numbers depicted here are further detailed on slides 9, 10, 13, 17, 22, 24 today 275K ARIKAYCE MAC LD brensocatib HS 600K
Three therapies with positive clinical data in-hand have potential to be first-
and/or best-in-disease* >$2bn Max. dose well-tolerated Nominally stat. sig. benefit on clinical worsening TPIP PH-ILD Phase 2 Bronchiectasis refers to non-cystic fibrosis bronchiectasis PVR reduction including placebo similar to
treatment with other prostanoids PAH Phase 2** Peak sales potential *"Best-in-disease/best-in-class" indicates a profile that could be considered more attractive than other treatment options for a particular disease. Head-to-head clinical
trials are not anticipated. **Based on blended and blinded data observed from the ongoing Phase 2 study of TPIP in PAH. Efficacy analyses were based on data available as of April 1, 2024 >$1bn Validated a PRO tool Nominally stat. sig.
culture conversion benefit at Month 7 compared to active control ARIKAYCE ARISE >$5bn Potential for a new standard of care in bronchiectasis Unlocks DPP1 mechanism for neutrophil-mediated diseases BRENSOCATIB ASPEN
TPIP Pulmonary Arterial Hypertension (PAH) A devastating and debilitating
disease that pervades all aspects of a patient's daily life Pulmonary Hypertension due to Interstitial Lung Disease (PH-ILD) A rapidly progressing disease associated with poor survival and decreased quality of life Image Source: Valentini
A, Franchi P, Cicchetti G, Messana G, Chiffi G, Strappa C, Calandriello L, del Ciello A, Farchione A, Preda L, et al. Pulmonary Hypertension in Chronic Lung Diseases: What Role Do Radiologists Play? Diagnostics. 2023; 13(9):1607.
TPIP has the potential to clearly differentiate from other treatments in PH-ILD
and PAH Higher Dosing Tolerability may lead to improved outcomes Safety** Favorable safety profile Convenience* Once daily inhalation TPIP TPIP Remodulin Tyvaso Yutrepia Orenitram Uptravi Route of administration Inhaled
(dry powder) IV orSubcutaneous Inhaled (nebulized and dry powder) Inhaled (dry powder) Oral Oral Dosing frequency Once daily Continuous 4x per day 4x per day 2x or 3x per day 2x per day Favorable tolerability for higher
dose Yes** Yes No Yes No No Efficacy in PAH (WHO Group 1) To be evaluated in Phase 2 and Phase 3 Yes Yes Yes Yes Yes Efficacy in PH-ILD (WHO Group 3) Pursuing in parallel to PAH No data Yes Yes No data No data *No
head-to-head or convenience studies have been conducted or planned. **Safety analysis based on topline safety and tolerability data from the Phase 2 PH-ILD study of TPIP disclosed on May 6, 2024 Based on most recent publicly available data
Diagnosed with BE15 TPIP TPIP - PH-ILD PH-ILD is a significant potential
commercial opportunity, with ~135k patients 65K 50K 20K 190K 150K 45K PH-ILD1 Diagnosed PH1 Japan TAM US TAM EU5 TAM Tyvaso DPI List Price* U.S. Pricing Benchmark $300K *Wholesale acquisition cost (WAC) as of March 12,
2024 EU5 comprised of France, Germany, Italy, Spain and the United Kingdom Note: Diagnosed pulmonary hypertension (PH) consists of pulmonary arterial hypertension (PAH), pulmonary hypertension due to left heart diseases (PH-LHD), pulmonary
hypertension due to interstitial lung disease (PH-ILD), chronic thromboembolic pulmonary hypertension (CTEPH), Idiopathic PH For references, please refer to slides 29-32 TAM=Total Addressable Market
Diagnosed with BE15 40K 35K 15K 190K 150K 45K PAH2 Diagnosed PH2 TPIP
represents potential best-in-class* prostanoid for ~90k patients with PAH Japan TAM US TAM Tyvaso DPI List Price* EU5 TAM U.S. Pricing Benchmark $300K *Wholesale acquisition cost (WAC) as of March 12, 2024 For references, please refer
to slides 29-32 EU5 comprised of France, Germany, Italy, Spain and the United Kingdom Note: Diagnosed pulmonary hypertension (PH) consists of pulmonary arterial hypertension (PAH), pulmonary hypertension due to left heart diseases (PH-LHD),
pulmonary hypertension due to interstitial lung disease (PH-ILD), chronic thromboembolic pulmonary hypertension (CTEPH), Idiopathic PH TPIP - PAH *"Best-in-disease/best-in-class" indicates a profile that could be considered more attractive
than other treatment options for a particular disease. Head-to-head clinical trials are not anticipated. TAM=Total Addressable Market
Phase 3 STELLAR trial suggests that sotatercept will be complementary with
treprostinil products Note: Prostacyclins are part of the prostacyclin receptor agonist class Prostacyclins expected to remain cornerstone of PAH therapy; TPIP has potential to be prostacyclin of choice Regardless of sotatercept's efficacy,
the prostacyclin pathway will remain important to PAH Thoughts from Key Opinion Leaders in the PAH Space3,4 TPIP really sounds like something that could be very valuable to the patient TPIP - PAH For references, please refer to slides
ARIKAYCE Refractory Mycobacterium avium complex lung disease (rMAC LD) A rare
and chronic disease that can cause irreversible lung damage and is the most common form of NTM respiratory pathogen Mycobacterium avium complex lung disease (MAC LD) Image source: Hendrix C, McCrary M, Hou R, Abate G. Diagnosis and Management
of Pulmonary NTM with a Focus on Mycobacterium avium Complex and Mycobacterium abscessus: Challenges and Prospects. Microorganisms. 2023; 11(1):47. https://doi.org/10.3390/microorganisms11010047
Japan TAM US TAM EU5 TAM Diagnosed with
BE15 1.4K 12-17K 15-18K 95-115K 14K 125-145K ARIKAYCE Refractory MAC5 MAC LD5 ARIKAYCE ARIKAYCE has the potential to be best in MAC LD treatment regimen to address ~275K patients TAM=Total Addressable Market For references,
please refer to slides 29-32 EU5 comprised of France, Germany, Italy, Spain and the United Kingdom If approved, we anticipate marketing ARIKAYCE in MAC LD at same price and dosage as currently available ARIKAYCE
ARIKAYCE brensocatib Physician and patient overlap between NTM and BE is
high Insmed has strong relationships in this space, spanning 6+ years Bronchiectasis refers to non-cystic fibrosis bronchiectasis For references, please refer to slides 29-32 Bronchiectasis (BE) and nontuberculous mycobacterial (NTM) lung
disease are inextricably linked pathophysiologically6
In 2018, ARIKAYCE delivered a top 10 non-oncology rare disease
launch ARIKAYCE brensocatib 2018 2025 94% Current sales reps who have sold ARIKAYCE for more than 1 year 5x HCP targets 31x Patient volume Increase in stakeholders7 Consistency of field 67% Current field team that launched
ARIKAYCE Patient overlap8 5K+ Healthcare Professional (HCP) targets Focused engagement7 In-house patient services High touch patient services and specialty distribution Patient services heritage One in-house patient support model
with consistent specialty distribution model 44% Of NTM patients also have bronchiectasis For references, please refer to slides 29-32 Bronchiectasis refers to non-cystic fibrosis bronchiectasis and we expect to deliver again
BRENSOCATIB Chronic Rhinosinusitis without Nasal Polyps (CRSsNP) Hidradenitis
Suppurativa (HS) Bronchiectasis (BE) A chronic, progressive inflammatory disease that causes permanent lung damage Bronchiectasis refers to non-cystic fibrosis bronchiectasis Image Source: 1) Fraser CS, Jos RJ. Insights into Personalised
Medicine in Bronchiectasis. Journal of Personalized Medicine. 2023; 13(1):133. https://doi.org/10.3390/jpm13010133. 2) Rethinkbronchiectasis.com
Japan TAM *Includes misdiagnosed, miscoded, undiagnosed EU5 comprised of
France, Germany, Italy, Spain and the United Kingdom Note: COPD and asthma may be comorbid with bronchiectasis and not all patients with bronchiectasis have comorbid asthma or COPD Bronchiectasis could represent a >1M patient indication
at launch, with significant growth potential Asthma or COPD11 Undiagnosed* BE10 Diagnosed with BE9 600K 500K 150K US TAM 2.4M 2.1M 0.8M 32M 27M 17M brensocatib - BE EU5 TAM Bronchiectasis refers to non-cystic fibrosis