JBIO 8-K
Jade Biosciences, Inc. (JBIO)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
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Aerovate Therapeutics, Inc.
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Item 7.01 | Regulation FD Disclosure. |
Aerovate Therapeutics, Inc. (the “Company”) is furnishing a corporate presentation, attached as Exhibit 99.1 to this Current Report on Form 8-K, which the Company intends to use from time to time in meetings with investors and others beginning on March 27, 2024. The corporate presentation will also be available in the “Events & Presentations” section of the Company’s website at https://ir.AerovateTx.com/events-presentations.
The information under this Item 7.01 of this Current Report on Form 8-K (including Exhibit 99.1) is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
99.1 |
| Corporate presentation of Aerovate Therapeutics, Inc., furnished herewith. | |||
104 | Cover Page Interactive Data File | ||||
SIGNATURE
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.
| Aerovate Therapeutics, Inc. | |||
Date: March 27, 2024 |
| By:
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| /s/ George A. Eldridge |
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| George A. Eldridge | ||
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| Chief Executive Officer | ||
| Meaningfully Improving the Lives of Patients with Rare Cardiopulmonary Disease Targeting the Hyperproliferative Cause of Pulmonary Arterial Hypertension March 2024 Nasdaq: AVTE |
| This presentation has been prepared by Aerovate Therapeutics, Inc. ("we," "us," "our," "Aerovate" or the “Company”) and is made for informational purposes only. The information set forth herein does not purport to be complete or to contain all of the information you may desire. Statements contained herein are made as of the date of this presentation unless stated otherwise, and this presentation shall not under any circumstances create an implication that the information contained herein is correct as of any time after such date or that information will be updated or revised to reflect information that subsequently becomes available or changes occurring after the date hereof. The following presentation contains forward-looking statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “future,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “seek,” “strategy,” “should,” “will,” “would” and similar expressions regarding future periods. These forward-looking statements include, but are not limited to, statements regarding Aerovate’s business plans and objectives; future plans for AV-101, including expectations regarding timing and success of the planned clinical trial, therapeutic potential, clinical benefits and safety thereof; growth as a company; the potential value and market for AV-101; and uses and need of capital, expenses and other financial results currently or in the future. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties and are based on our current beliefs, expectations and assumptions regarding future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Our actual results and financial condition may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Among the factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this presentation are those risks and uncertainties, without limitation, associated with the following: the impact of the COVID-19 pandemic on the company’s business, operations, patient enrollment and retention, strategy, goals and anticipated milestones; the therapeutic potential of AV-101, and the timing associated with the initiation, continuation or success of Aerovate’s ongoing or planned clinical trials of AV-101; Aerovate’s ability to execute on its strategy; positive results from a preclinical or clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; AV-101 may not be successfully developed and commercialized; regulatory developments in the United States and foreign countries; Aerovate’s ability to protect and maintain its intellectual property position; as well as those risks and uncertainties set forth more fully under the caption "Risk Factors" in our Quarterly Report on Form 10-Q, as well as other risks detailed in our subsequent filings with the United States Securities and Exchange Commission (SEC). Any forward-looking statement made by us is based only on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data obtained from third-party sources and the Company’s own internal estimates and research. While the Company believes these third-party sources to be reliable as of the date of this presentation, it has not independently verified, and makes no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, all of the market data included in this presentation involves a number of assumptions and limitations, and there can be no guarantee as to the accuracy or reliability of such assumptions. Finally, while we believe our own internal research is reliable, such research has not been verified by any independent source. 2 Disclaimer; Forward-Looking Statements |
| Experienced Management Team 3 Ben Dake, PhD President & Founder • Entrepreneur, Cancer Biologist and Investor • Conceptualized AV-101 and secured up to $79M in financing for Aerovate Hunter Gillies, MB ChB Chief Medical Officer • Led AMBITION trial for Gilead that established current first-line PAH combination therapy • Led successful Phase 2 and 3 trials for PAH product candidates at Pfizer and Gilead Ralph Niven, PhD Chief Development Officer • 25 years’ broad expertise in translational medicine and inhalation dosage forms • Managed experimental and clinical development at public and private companies, including Amgen, AIR and Novartis Timothy Noyes Chief Executive Officer • 30 years’ commercial experience in pharma and biotech, including Merck, Genzyme, Proteon • Extensive launch planning and commercial launch experience George Eldridge Chief Financial Officer • 25 years’ experience in biotech, with both public and private companies, including Curis, Targanta, Proteon • Extensive background raising capital in private, IPO and follow-on settings, investment banking and M&A Timothy Pigot Chief Commercial Officer • 25 years’ experience in biotech and pharma working to launch and commercialize a range of products • 10 years’ experience in PAH overseeing the US launches of Revatio and Leairis while at Pfizer and Gilead Sciences, respectively Susan Fischer EVP, Clinical Ops. • 20 years’ experience in clinical operations in both academic and the pharmaceutical industry. • Previously built/led clinical ops teams at Syndax, EMD Serono, Acetylon Donna Dea Head of Regulatory • 35 years of pharmaceutical experience at AstraZeneca • 20 years of global regulatory experience designing/ implementing strategies resulting in approval of treatments for asthma, COPD, rhinitis and others Marco Verwijs Chief Technical Officer • 15 years’ experience developing drugs from clinical product development thru commercial launch • Proven leader in drug product scale-up and validation. |
| Aerovate: An Inventive Way Forward in PAH 4 Demonstrated Clinical Benefit Inventive Solution Significant Unmet Need Efficient Execution Established Market The molecule in AV-101, imatinib, already has shown clinical benefit in a Phase 3 clinical trial conducted by Novartis of oral imatinib mesylate in PAH patients on top of two or more standard of care therapies. Unfortunately, AEs with oral imatinib were common and development was discontinued Anti-proliferative targeted inhaled dry powder PAH product candidate AV-101 designed to provide robust clinical benefit of imatinib without systemic AEs observed with oral imatinib Despite three drug classes approved, 5-year survival for newly diagnosed PAH patients is 61% FDA and EMA orphan designation for AV-101 for the treatment of PAH Phase 1 SAD/MAD in healthy volunteers complete Phase 2b portion fully enrolled of seamless Ph2b/3 IMPAHCT trial and already enrolling in Phase 3 portion Ph2b data expected in June 2024 $6B+ market, yet outcomes are poor |
| Comfortable at rest, but ordinary physical activity causes shortness of breath, fatigue, chest pain, fainting Comfortable at rest, but less than ordinary physical activity causes shortness of breath, fatigue, chest pain, fainting Symptoms at rest. Overt heart failure No limitation Relentless Disease Progression Impairs Daily Life 5 Activity limited Marked limitation Severe limitation Despite standard of care (SOC) most patients progress to overt heart failure |
| 6 ~ 70,000 People with PAH in the US/EU ~ 35,000 People with PAH in the US $6B+ Global annual drug spending 65-80% Female 61-65% 5-year survival Newly diagnosed and prevalent patients 53 years Average age at diagnosis Pulmonary Arterial Hypertension |
| Patient Outcomes Are Poor Despite $6B+ PAH Vasodilator Market 7 NO pathway $0.7B ERAs $2.4B Prostanoids $3.0B SOC Vasodilators 5-year survival 61-65% for newly diagnosed and prevalent patients ~65% patients take 2-3 drugs No approved PAH therapy primarily addresses abnormal cellular proliferation |
| Reinventing Imatinib From a Cancer Drug to a Potential PAH Therapy 88 |
| 9 Phase 3 IMPRES Trial: Oral Imatinib Demonstrated Improvement on Top of Maximal Background (Adapted from Circulation. 2013;127:1128-1138.) Change in 6MWD from baseline (m) Time (weeks) 0 4 8 12 16 20 24 32m 0 10 20 30 40 50 60 70 * * * * Imatinib Placebo *P<0.05 Novartis global Phase 3 trial (n=202) of oral imatinib Required to be on at least 2 SOC PAH drugs WHO functional class II- IV Statistically significant and clinically meaningful benefit on primary endpoint 6MWD after 24 weeks 6MWD is an accepted endpoint for approval in PAH |
| Phase 3 IMPRES Trial: Benefit Consistent Across Secondary Endpoints Secondary Endpoints (24wk) Demonstrated robust hemodynamic effect (A) Decreased mean pulmonary artery pressure (B) Increased cardiac output (C) Pulmonary vascular resistance (PVR) dropped 32% (P < 0.001), a validated efficacy endpoint typically used for dose-finding (D) Right atrial pressure lowered 10 PVR is the primary endpoint for Aerovate’s Phase 2b (Circulation. 2013;127:1128-1138.) |
| Imatinib n=103 (%) Placebo n=98 (%) Adverse Event + 100 (97) 94 (96) Nausea 57 (55) 23 (24) Peripheral edema 45 (44) 20 (20) Diarrhea 36 (35) 19 (19) Vomiting 31 (30) 10 (10) Periorbital edema 30 (29) 7 (7) Headache 25 (24) 22 (22) Dyspnea 19 (18) 13 (13) Nasopharyngitis 18 (18) 19 (19) Hypokalemia 16 (16) 3 (3) Anemia 14 (14) 3 (3) Cough 11 (11) 15 (15) Fatigue 11 (11) 7 (7) Face edema 10 (10) 1 (1) Muscle spasms 10 (10) 2 (2) Imatinib n=103 (%) Placebo n=98 (%) Serious Adverse Event + 45 (44) 29 (30) Worsening of pulmonary hypertension 6 (6) 8 (8) Anemia 7 (7) 1 (1) Dyspnea 6 (6) 2 (2) Peripheral edema 6 (6) 0 Presyncope 5 (5) 0 Diarrhea 3 (3) 2 (2) Device-related infection 3 (3) 0 Syncope 1 (1) 5 (5) Subdural hematoma * 2 (2) 0 Oral Imatinib Caused Systemic Adverse Events 11 (Circulation. 2013;127:1128-1138.) * In long-term extension portion of the trial, 6 additional patients experienced a subdural hematoma. + Individual adverse events are shown if they occurred in >10% in the imatinib group. Individual serious adverse events are shown if they occurred in ≥3 patients in either group. |
| AV-101 Potential Targeted Treatment for PAH 1212 |
| 13 AV-101 AV-101 is a combination product comprised of a proprietary dry powder imatinib formulation in a capsule delivered by a dry powder inhaler designed to: • Deliver drug to the lungs • Limit systemic exposure • Be easily administered Imatinib molecular structure unmodified |
| PAH is a Disease of All Layers of the Pulmonary Arteries 14 1. Adapted from J Am Coll Cardiol. 2015 May 12;65(18):1976-97. 2. Eur Respir J. 2019 Jan 24;53(1):1801887. 3. J Appl Physiol (1985). 2011 Apr;110(4):1119-29.) Latex cast of human airway (white), arterial tree (red), and venous tree (blue)3 1 |
| 15 AV-101 Phase 1 Trial A single and multiple ascending dose Phase 1 study (n=82) in healthy volunteers to determine safety and tolerability MAD n=12 per cohort; 9 Active / 3 Placebo Low dose 10 mg Med dose 30 mg High dose 90 mg Seven days of BID dosing SAD STUDY ASSESSMENTS n=8 per cohort Five AV-101 cohorts from 1 mg to 90 mg 400 mg imatinib tablet for systemic exposure comparison Vital Signs Pulmonary Function Testing (FEV1 and FVC) Oxygen Saturations QTc Adverse Events n=6 Active / 2 Placebo n=8 |
| 16 AV-101 Phase 1 Trial: Lower Systemic Exposures Observed 0 500 1000 1500 2000 2500 0 12 24 Imatinib plasma concentration (ng/ml) Hours post-dose Plasma levels of AV-101 at final dose (steady state) 400 mg oral simulated steady state 90 mg AV-101 day 7 90 mg projected additional dose to simulate BID 30 mg AV-101 day 7 10 mg AV-101 day 7 Systemic exposures for all doses of AV-101 observed to be lower than simulated steady-state exposure of 400 mg of oral imatinib |
| 17 AV-101 Phase 1 Tolerability Profile AV-101 was generally well tolerated • No serious adverse events were reported • No change in pulmonary function, oxygen saturation, and QTc interval • Most AEs mild to moderate • One discontinuation due to vomiting on Day 1 in the MAD 90 mg Cohort • Most common AE, cough, was at max dose and limited to within 30 minutes of dosing Adverse Event n(%) 10 mg (n=8) 30 mg (n=9) 90 mg* (n=9) Cough 1 (13) 1 (11) 5 (56) Persistent cough - - - Headache - - 4 (44) Nausea - - 2 (22) Chest discomfort - - 2 (22) Throat irritation - 1 (11) 1 (11) Musculoskeletal pain - - 2 (22) Single incidence AEs: Vomiting (discontinued), Dysgeusia, Musculoskeletal chest pain, Nasal congestion, Oropharyngeal pain, Back pain, Abdominal pain, Covid-19, Presyncope, Alanine aminotransferase increased *90mg dose was administered as 9 x 10mg capsules |
| Phase 2b Dose-Ranging Trial • Placebo controlled dose-ranging study – 10 mg, 35 mg and 70 mg AV-101 BID for 24 weeks – Targeting 200 patients across 4 dose groups • Multicenter international trial • Two or more background PAH therapies • Functional class II - IV • Primary endpoint: change in pulmonary vascular resistance (PVR) at 24 weeks – Statistically significant improvement on PVR already demonstrated with oral imatinib in multiple third-party clinical trials – Validated endpoint – Powered to detect statistically significant change • Key secondary endpoints: Change in 6MWD, NTproBNP, QoL 18 |
| 19 Expected AV-101 Lung Exposures of IMPAHCT Doses, Extrapolated based on Phase 1 Results 0 5000 10000 15000 20000 25000 0 12 24 Imatinib lungs concentration (ng/ml) Hours post-dose 70 mg AV-101 35 mg AV-101 10 mg AV-101 400 mg oral Predicted lung concentrations of Phase 2b doses of AV-101 in IMPAHCT trial overlap or surpass 400 mg oral imatinib dose Predicted lung concentrations extrapolation based on a published PK model using plasma levels observed in Phase 1 trial CPT Pharmacometrics Syst Pharmacol. 2018 Mar;7(3):147-157. doi: 10.1002/psp4.12270. ERJ Open Res. 2023 Mar 13;9(2):00433-2022. doi: 10.1183/23120541.00433-2022. |
| Phase 2b Phase 3 IMPAHCT Phase 2b/3 Seamless Trial of AV-101 20 • All patients are treated for 24 weeks • 6MWD is primary endpoint in Phase 3 • Phase 3 enrollment began immediately upon completion of Phase 2b enrollment • Phase 3 dose selected based on Phase 2b data after 24 weeks of follow up PART 1 PART 2 PART 3 10 mg (n=50) 35 mg (n=50) 70 mg (n=50) placebo (n=50) 10 mg 35 mg 70 mg Placebo Continue Optimal Dose 11/2023 6/2024 https://clinicaltrials.gov/study/NCT05036135 |
| Key Baseline Characteristics Among Recent PAH Clinical Trials Measure IMPAHCT N=147 PULSAR N=106 STELLAR N=323 TORREY N=86 PVR, dynes.sec.cm-5, mean 790 779 764 669 6MWD, m, mean 394 397 401 408 WHO FC II/III 49%; 51% 53%; 47% 49%; 51% 68%; 32% NTproBNP, pg/mL, mean 754 908 1121 628 Age, years, mean 47 48 48 49 Gender F82% ; M18% F87%; M13% F 79%; M21% F91%; M9% IPAH or HPAH 71% 74% 77% 66% Non-IPAH or HPAH 29% 26% 23% 34% Mono/Double/triple background 0%; 40%; 59% 9%; 35%; 56% 4%; 35%; 61% 3%; 40%; 57% Mean PA pressure, mmHg 50.9 52.4 52.6 Not reported Cardiac index, L/min/m2 2.7 2.6 2.7 Not reported Baseline characteristics with first 147 patients from IMPAHCT Ph2b compared with select PAH trials 21 The data in the chart above are based on a cross-trial comparison and not a head-to-head clinical trial. Such data may not be directly comparable due to differences in trial design, study protocols, conditions and patient populations. |
| 22 Oral Imatinib Merck/Acceleron Sotatercept (activin trap) Gossamer Seralutinib (TKI) Phase 2 PULSAR (Ph2) TORREY (Ph2) Low dose High dose PVR -26.0% -18.5% -31.8% -14.3% 6MWD +21.7 m P=0.21 +29.4 m P=0.02 +21.4 m P=0.08 +6.5m P=0.597 Phase 2 Trials for Non-Vasodilator Therapies The data in the chart above are based on a cross-trial comparison and not a head-to-head clinical trial. Such data may not be directly comparable due to differences in trial design, study protocols, conditions and patient populations. The percent reduction in PVR for the Oral Imatinib Phase 2 study is calculated using the placebo corrected arithmetic means. The STELLAR and TORREY study percent reduction in PVR are calculated using least square means (LSM) corrected for placebo. |
| AV-101: Potential to Be An Ideal Add-On Agent • Al Launch, AV-101 has the potential to be an ideal add on agent* – Compelling clinical profile in combination with current therapies – Simplicity and ease-of-use – Potential to be part of the future standard of care • Potential positioning within target populations* – In front of inhaled and oral prostacyclin therapy – In front of sotatercept for patients who prefer inhalation vs injection and for sotatercept ineligible patients – Patients who remain at moderate to high risk of poor outcomes despite sotatercept and prostacyclin therapy – Patients who do not tolerate sotatercept and prostacyclin therapy 23 *Assumes data and FDA discussions support FDA approval with supportive labeling |
| AVTE’s Perspective on Current And Future PAH Landscape Informed by Extensive Experience, Stakeholder Engagement, and Market Research • Global Advisory Boards with PAH Experts and PAH Patients • Interactions with Investigators, KOL’s, PAH Treaters, Nurses, Patients, Advocacy Associations* • Robust Market Research – 100+ U.S. Physician Market Landscape Research (Oct 2021) – 100+ European Physician Market Landscape Research (June 2022) – 150+ U.S. Physician Conjoint Demand Study post Sotatercept Data (Aug 2023) *Individual interactions focus solely on treatment landscape unless seeking bona-fide advice with an appropriate consulting agreement 24 Prof. Vizza MD investigator discussion |
| PAH Treatment Pathways Have Not Changed Since 2005 and Outcomes Remain Poor Epoprostenol Treprostinil Bosentan Ambrisentan Sildenafil Tadalafil Prostacyclin Pathway Endothelin Pathway Nitric Oxide Pathway 61%-65% 5 Year Survival among Newly Diagnosed and Prevalent Patients Multiple Agents Within Pathways More Convenient Prostacyclin Pathway Routes of Administration Infusion Inhalation Oral More Aggressive Treatment Approaches Initial Monotherapy Upfront Combination Therapy Treat to Low Risk Significant Efforts to Optimize Traditional Pathways 25 25 |
| Unmet Need Beyond Sotatercept The STELLAR trial represents significant progress in therapy for PAH however… • 60% of patients did not meet multi-component improvement measure • 60% of patients did not achieve low risk on the simplified French risk model • 70% of patients did not improve functional class • Long term effects not fully established 26 |
| 80% of Physicians Would Prescribe an “IMPRES” Like Inhaled TKI if it Were Available Alongside Sotatercept 80% 89% 57% 0% 20% 40% 60% 80% 100% IMPRES Like Inhaled TKI STELLAR Like Activin Signaling Inhibitor TORREY Like Inhaled TKI Market Research with >150 U.S. PAH Treating Physicians Conducted Post STELLAR Data* % Who Would Prescribe Each Hypothetical Agent to Their Patients 27 *Proprietary Market Research with U.S. PAH Treating Physicians; Aug 2023. Data based on product allocation exercise assuming all hypothetical agents were available |
| AV-101: Unmet Need, Blockbuster Potential 28 Positioning Added to two agents before prostacyclin Added to three agents before parenteral prostacyclin ~ 35,000 Patients are treated for PAH in the US We estimate to be taking 2-3 drugs ~65% (~22,750) |
| Positive Regulatory Interactions and Feedback Regulatory Interactions Completed • FDA Pre-IND meeting in January 2020 • FDA and EMA Orphan Designation granted for AV-101 for the treatment of PAH • Received EMA Scientific Advice in March 2021 • End-of-Phase-1 Meeting with FDA in April 2021 Feedback from FDA and EMA on Clinical Development • A single Phase 2b/3 trial could support NDA • Aligned on Phase 2b/3 endpoints for potential NDA/MAA submission 29 |
| Intellectual Property Protection for AV-101 to 2040+ Multiple issued US patents covering AV-101 drug product and methods of use • Patent coverage extends at least until 2040 • Current US patents being extended worldwide Multiple additional pending applications pursuing • Methods of manufacture • Filing on other aerosol compositions • Filing on mechanism of action (composition agnostic) Regular reviews enacted to extend or file new IP based on discoveries in research, clinical trials and CMC 30 |
| Chemistry, Manufacturing and Controls Stability of AV-101 • Release and stability testing supports – API stability of at least 36 months – Drug product stability of at least 30 months • Filled product stability testing ongoing 31 Proprietary Methods Several bulk supplier options Contracted manufacturing and filling in place Contracted with supplier AV-101 manufacturing and supply chain GMP Imatinib Mesylate Processed to novel dry powder formulation and capsules filled Co-packaged with device |
| 32 AV-101 Device and Delivery Performance AV-101 Device • Off-the-shelf commercial scale dry powder inhaler – No batteries, compressors or cords – No sterile vials containing solutions or suspensions – CE mark in EU and Device Master File registered with FDA • Designed for ease of use and convenience • Intended dosing of 2 capsules twice a day • Device replaced weekly AV-101 Delivery Performance • Consistently high delivered and fine particle dose • Ideal size and size distribution for lung penetration NOTE: Device in IMPAHCT was not used in Phase 1 trial, but delivery performance was assessed. |
| Aerovate: An Inventive Way Forward in PAH 33 Demonstrated Clinical Benefit Inventive Solution Significant Unmet Need Efficient Execution Established Market The molecule in AV-101, imatinib, already has shown clinical benefit in a Phase 3 clinical trial conducted by Novartis of oral imatinib mesylate in PAH patients on top of two or more standard of care therapies. Unfortunately, AEs with oral imatinib were common and development was discontinued Anti-proliferative targeted inhaled dry powder PAH product candidate AV-101 designed to provide robust clinical benefit of imatinib without systemic AEs observed with oral imatinib Despite three drug classes approved, 5-year survival for newly diagnosed PAH patients is 61% FDA and EMA orphan designation for AV-101 for the treatment of PAH Phase 1 SAD/MAD in healthy volunteers complete Phase 2b portion fully enrolled of seamless Ph2b/3 IMPAHCT trial and already enrolling in Phase 3 portion Ph2b data expected in June 2024 $6B+ market, yet outcomes are poor |
| 3434 Thank you! |
