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|Item 2.02|| |
Results of Operations and Financial Condition.
On January 6, 2021, Chiasma, Inc. (the “Company”) issued a press release (the “Press Release”) providing a year-end corporate update and preliminary 2021 outlook, which Press Release included the Company’s preliminary approximate net revenue for the year ended December 31, 2020 and preliminary approximate cash, cash equivalents, marketable securities and restricted cash as of December 31, 2020 (the “Financial Information”). The Financial Information is unaudited and does not present all information necessary for an understanding of the Company’s financial condition as of December 31, 2020 and its results of operations for the three or twelve months ended December 31, 2020. A copy of the Press Release is furnished herewith as Exhibit 99.1.
The Financial Information set forth in Item 2.02 and 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 a filing.
|Item 7.01|| |
Regulation FD Disclosure.
A copy of the slide presentation that will be used by representatives of the Company in connection with investor meetings and presentations from time to time (the “Corporate Presentation”) is attached to this Current Report on Form 8-K as Exhibit 99.2. The Corporate Presentation is current as of January 6, 2021, and the Company disclaims any obligation to correct or update this material in the future.
The information set forth in Item 7.01 and Exhibit 99.2 is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of 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 a filing.
|Item 9.01|| |
Financial Statements and Exhibits.
|99.1||Press release dated January 6, 2021.|
|99.2||Corporate presentation current as of January 6, 2021.|
|104||Cover Page Interactive Data File (formatted as Inline XBRL).|
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.
|Date: January 6, 2021||Chiasma, Inc.|
Chiasma Provides Corporate Update and Previews Expected 2021 Milestones
MYCAPSSA® launch in US continuing to gain traction with physicians, patients, and payers
MYCAPSSA® EMA submission for EU marketing approval on track for mid-2021 following positive results of MPOWERED Phase 3 clinical trial
Needham, MA January 6, 2021 Chiasma, Inc. (NASDAQ: CHMA), a commercial-stage biopharmaceutical company utilizing its delivery platform technology to develop and commercialize oral therapies to improve the lives of patients with rare diseases currently treated with burdensome and painful injections, as evidenced by its recent phased launch of MYCAPSSA® as the first oral therapy for the treatment of acromegaly, today reviewed its 2020 accomplishments and previewed its anticipated 2021 corporate milestones.
2020 Key Highlights:
MYCAPSSA Approved in the U.S. as the first and only oral somatostatin analog (SSA) for the long-term maintenance treatment of patients with acromegaly who responded to and tolerated treatment with octreotide or lanreotide.
Approval was based on the positive CHIASMA OPTIMAL Phase 3 trial results which were published in the Journal of Clinical Endocrinology & Metabolism and presented at the 2020 Endocrine Society Annual Meeting (ENDO) along with six other scientific posters relevant to MYCAPSSA.
Commercial Launch of MYCAPSSA in the U.S. Late in the third quarter of 2020, Chiasma commenced the initial phase of the MYCAPSSA U.S. commercial launch with a focused salesforce calling on approximately one-third of key prescribing accounts. Chiasma progressed to the second phase of the launch at the beginning of 2021 with an expanded salesforce hired during the fourth quarter of 2020. Progression to the final phase of the launch with a full-strength salesforce is expected after market conditions improve beyond the COVID era.
Early launch insights and progress:
Coverage of MYCAPSSA by multiple payers insuring approximately 150 million lives provides a solid foundation for MYCAPSSA uptake in 2021.
Positive and encouraging feedback on MYCAPSSA from endocrinologists and patients supports the companys goal of ultimately becoming the standard of pharmacological care in acromegaly.
Preliminary (unaudited) net revenues for 2020 are expected to be between $0.9 and $1.1 million.
Reported Positive Data from CHIASMA OPTIMAL Open Label Extension. The efficacy and safety (tolerability) seen in the 1-year extension phase of the study (48 weeks) was similar to that seen in the 36-week core study period. The mean of the insulin-like growth factor 1 (IGF-1) levels for the population of all MYCAPSSA treated patients that completed the 36-week core CHIASMA OPTIMAL trial and continued into the open-label extension (OLE) (n=19) was maintained within normal limits at the end of the 48-week OLE period.
Reported Positive MPOWERED Phase 3 Topline Data. The MPOWERED study met its primary non-inferiority endpoint. 91% of patients on MYCAPSSA maintained IGF-1 response (95% CI = 80%, 97%) compared to 100% on injectable SSAs (95% CI = 91%, 100%). The positive clinical trial results provide Chiasma a pathway to pursue regulatory approval of MYCAPSSA in the European Union (EU), further strengthen the available robust clinical data set for MYCAPSSA and provide additional meaningful information for healthcare providers in treating patients with acromegaly.
Strengthened Balance Sheet for U.S. Commercial Launch. In April, Chiasma entered into a revenue interest financing agreement with HealthCare Royalty Partners (HCR) for up to $75 million. Also, in July, Chiasma raised approximately $75 million of net proceeds through an underwritten public offering of common stock and pre-funded warrants. Chiasma ended the year with approximately $135 million in cash, cash equivalents and marketable securities (exclusive of approximately $20 million of restricted cash), which is expected to fund its operations as currently planned through at least the end of 2021, including the execution of Chiasmas planned 2021 U.S. MYCAPSSA commercial launch strategy.
2020 was the beginning of an important transformation for Chiasma from a company with a strong research and development foundation to a commercial-stage company, stated Raj Kannan, chief executive officer. We are pleased with the progress of the MYCAPSSA launch to date and the encouraging feedback we have received from physicians, patients and payers. In 2021, we continue to build upon the MYCAPSSA launch in the United States while pursuing MYCAPSSA approval in the European Union. The recently reported positive MPOWERED clinical trial results further support our goal to make MYCAPSSA the new standard of pharmacological care in the long-term maintenance treatment of people with acromegaly.
Anticipated 2021 Milestones and Related Guidance:
Grow MYCAPSSA revenues in the U.S. by building on the progress made with the initial, focused selling effort in the first four months since launch: Chiasma plans to drive adoption for MYCAPSSA with a broader group of physicians by expanding from a 36-person customer-facing team today to an approximately 45-person customer-facing team as market challenges associated with COVID-19 recede and payer coverage continues to expand.
MYCAPSSA EMA Submission. In mid-2021, Chiasma plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking regulatory approval to market MYCAPSSA to patients with acromegaly in the EU.
CHIASMA OPTIMAL Open Label Extension Data Oral Presentation at Virtual ENDO 2021. Chiasma plans to present data on longer-term efficacy and safety of MYCAPSSA from the CHIASMA OPTIMAL trials open-label extension at the virtual 2021 Endocrine Society Annual Meeting (ENDO), which is being held March 20-23.
Presentation of MPOWERED Phase 3 Data at Medical Conferences and Publication in a Peer Reviewed Medical Journal. Chiasma expects to present MPOWERED Phase 3 data analyses at upcoming endocrinology scientific meetings in spring and in fall 2021 and submit the results of the MPOWERED trial to a peer-reviewed journal for expected publication in 2021.
2021 Financial Guidance. Operating expense for the full year 2021 is expected to be in a range of $80 million to $90 million, including estimated stock-based compensation expense in a range of $5 million to $6 million. This guidance is based on the companys current U.S. commercial plans and excludes expenditure for potential EU launch preparations of MYCAPSSA and potential additional new product development programs.
2021 LifeSci Advisors Corporate Access Event
Chiasma management will be available to meet with institutional investors and analysts virtually at the 2021 LifeSci Advisors Corporate Access Event on January 7-8 and 11-14.
INDICATION AND IMPORTANT SAFETY INFORMATION
INDICATION AND USAGE
MYCAPSSA (octreotide) delayed-release capsules, for oral use, is a somatostatin analog indicated for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.
Hypersensitivity to octreotide or any of the components of MYCAPSSA. Anaphylactoid reactions, including anaphylactic shock, have been reported in patients receiving octreotide.
IMPORTANT SAFETY INFORMATION
Warnings and Precautions
MYCAPSSA can cause problems with the gallbladder. Monitor patients periodically. Discontinue if complications of cholelithiasis are suspected.
Blood sugar, thyroid levels, and vitamin B12 levels should be monitored and treated accordingly.
Bradycardia, arrhythmia, or conduction abnormalities may occur. Treatment with drugs that have bradycardia effects may need to be adjusted.
The most common adverse reactions (incidence >10%) are nausea, diarrhea, headache, arthralgia, asthenia, hyperhidrosis, peripheral swelling, blood glucose increased, vomiting, abdominal discomfort, dyspepsia, sinusitis, and osteoarthritis.
The following drugs require monitoring and possible dose adjustment when used with MYCAPSSA: cyclosporine, insulin, antidiabetic drugs, calcium channel blockers, beta blockers, lisinopril, digoxin, bromocriptine, and drugs mainly metabolized by CYP3A4. Counsel women to use an alternative non-hormonal method of contraception or a back-up method when MYCAPSSA is used with combined oral contraceptives.
Patients taking proton pump inhibitors, H2-receptor antagonists, or antacids concomitantly with MYCAPSSA may require increased dosages of MYCAPSSA.
Advise premenopausal females of the potential for an unintended pregnancy.
To report SUSPECTED ADVERSE REACTIONS, contact the product information department at 1-844-312-2462 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch
The full Prescribing Information for MYCAPSSA is available at www.MYCAPSSA.com.
Acromegaly typically develops when a benign tumor of the pituitary gland produces too much growth hormone, ultimately leading to significant health problems. Common features of acromegaly are facial changes, intense headaches, joint pain, impaired vision and enlargement of the hands, feet, tongue and internal organs. Serious health conditions associated with the progression of acromegaly include type 2 diabetes, hypertension, respiratory disorders and cardiac and cerebrovascular disease. Chiasma estimates that approximately 8,000 adult acromegaly patients are chronically treated with somatostatin analog injections in the United States.
Chiasma is a commercial-stage biopharmaceutical company focused on developing and commercializing oral therapies to improve the lives of patients who face challenges associated with their existing treatments for rare and serious chronic diseases. Employing its Transient Permeability Enhancer (TPE®) technology platform, Chiasma seeks to develop oral medications that are currently available only as injections. In June 2020, Chiasma received FDA approval of MYCAPSSA for long-term maintenance therapy in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide. MYCAPSSA, the first and only oral somatostatin analog approved by the FDA, is available for commercial sale. Chiasma is headquartered in Needham, MA with a wholly owned subsidiary in Israel. MYCAPSSA, TPE and CHIASMA are registered trademarks of Chiasma. For more information, please visit the companys website at www.chiasma.com.
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the companys expectations relating to MYCAPSSA for the long-term maintenance therapy in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide, the commercialization of MYCAPSSA, including efficiency improvements in processes for prescriptions and delivery of commercial product, the commercial or therapeutic potential of MYCAPSSA, including its ability to become a standard of care and the anticipated market acceptance and third-party reimbursement of and access to MYCAPSSA, the expansion of the customer-facing team, the data from the MPOWERED trial and whether the data will support the submission of a marketing authorization application (MAA) to the European Medicines Agency (EMA) for MYCAPSSA in the European Union and ultimately regulatory approval, the presentation
and publication plans for the data from the MPOWERED trial, and the companys financial guidance, including operating expense and cash runway guidance. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond the companys control, that may cause actual events or results to differ materially from the companys current expectations. Managements expectations and, therefore, any forward-looking statements in this press release could be affected by risks and uncertainties relating to a number of factors, including the following: the content and timing of decisions made by the FDA or EMA, the sufficiency of the data collected from the companys clinical trials to obtain regulatory approval in the European Union or elsewhere, the companys ability to retain requisite regulatory approvals for the commercial sale of MYCAPSSA in the United States, the timing and costs involved in establishing and maintaining a commercial organization and launching the sale of MYCAPSSA, and the impact the ongoing COVID-19 pandemic may have on the companys business, including its expected development, manufacturing, regulatory and commercialization timelines for MYCAPSSA. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause the companys actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Chiasmas Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, and in subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Chiasma undertakes no duty to update this information unless required by law.
Investor Relations and Corporate Communications:
LifeSci Advisors, LLC
Chiasma Overview January 2021 | NASDAQ: CHMA Exhibit 99.2
These slides and the accompanying presentation contain forward-looking statements and information. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements. These statements include, without limitation, those statements regarding the company’s expectations relating to MYCAPSSA for the long-term maintenance treatment in patients with acromegaly who have responded to and tolerated treatment with octreotide or lanreotide, statements regarding the commercialization of MYCAPSSA, including its pricing, reimbursement, payer mix and market adoption, statements regarding the data from the open label extension of the CHIASMA OPTIMAL trial, statements regarding the size and composition of the U.S. market for MYCAPSSA, the commercial or therapeutic potential of MYCAPSSA, including its ability to become a standard of care, and anticipated market acceptance of and access to MYCAPSSA, statements regarding the expansion of its customer-facing team, statements regarding the success of commercial launch of MYCAPSSA in the United States, statements regarding the company’s expectations regarding formulary coverage for MYCAPSSA, statements regarding the data from the MPOWERED trial and whether the data will support the submission of a marketing authorization application (MAA) to the European Medicines Agency (EMA) for MYCAPSSA in the European Union and ultimately regulatory approval, statements regarding the timing of an MAA submission and regulatory review, statements regarding the company’s plans for the presentation of the full trial results, statements regarding the potential commercialization of MYCAPSSA in the European Union and in other jurisdictions, statements concerning the utilization of TPE platform to develop new therapeutic agents, and statements regarding Chiasma’s commercial organization and efforts and potential sales and revenue growth. All forward-looking statements are based on estimates and assumptions by Chiasma’s management that, although Chiasma believes them to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Chiasma expects. Management’s expectations and, therefore, any forward-looking statements in these slides and the accompanying presentation could be affected by risks and uncertainties relating to a number of factors, including the following: the content and timing of decisions made by the FDA, including with respect to the manufacturing supplements to the company’s approved new drug application, the results of any inspections of the company’s third-party manufacturers, the company’s reliance on third parties to manufacture active pharmaceutical ingredient and commercial octreotide capsules, the company’s ability to retain requisite regulatory approvals for the commercial sale of octreotide capsules in the United States, the content and timing of decisions made by the EMA, the sufficiency of the data collected from the company’s clinical trials to obtain regulatory approval in the European Union or elsewhere, and the timing and costs involved in commercializing MYCAPSSA, and the impact the ongoing COVID-19 pandemic may have on the company’s business, including its expected development, manufacturing, regulatory and commercialization timelines for MYCAPSSA. These and other potential risks, uncertainties and other important factors are described under the heading “Risk Factors” in our Form 10-Q for the quarter ended September 30, 2020 filed with the Securities and Exchange Commission, or SEC, as well as in Chiasma’s subsequent filings with the SEC. Undue reliance should not be placed on any forward-looking statement, which speak only as of the date on which it was made. Chiasma undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. Unless otherwise noted, all references to acromegaly market sizes are Chiasma internal estimates. This presentation is intended only for communications with investors. MYCAPSSA has been approved by the FDA for the long-term maintenance treatment in patients with acromegaly who have responded to and tolerated treatment with octreotide or lanreotide, but remains an investigational drug outside the U.S. Forward-Looking Statements
Chiasma Overview MYCAPSSA® was FDA approved in June 2020 and launched in September 2020 as the first and only oral somatostatin analog (SSA) for patients with acromegaly Acromegaly patients on SSA injectables face significant treatment challenges Attractive U.S. commercial opportunity ~8,000 patients* on first line SSA injectables MYCAPSSA has the potential to become the new standard of pharmacological care** Validated technology delivery platform with approval of MYCAPSSA in acromegaly Novel technology platform that can enable oral delivery of select therapeutic agents Commercial biopharma company Focused on oral treatment options for patients facing significant challenges with injectables Strong cash position as of Dec. 31, 2020; ~$135M in cash, cash equivalents, and marketable securities (unaudited) Well-capitalized to support U.S. commercial launch execution Financial Position * Company estimate based on available data. ** Full prescribing information available at www.MYCAPSSA.com
z Acromegaly U.S. Market Overview Octreotide and lanreotide injections are broadly used as first-line pharmacological treatments ~90% of these patients are treated at fewer than 1,000 medical centers Potential addressable patient population of ~8,000 patients in the U.S.* Acromegaly is a rare disease most often caused by a benign pituitary tumor and characterized by an excess of growth hormone and insulin-like growth factor-1 hormone. Treatment options include surgery, medication and radiation or a combination of these. If untreated, acromegaly may cause *Company estimate based on available data. Enlarged organs Intense headaches Altered facial appearance Enlargement of the hands and feet Type 2 diabetes Respiratory disorders Cardiac disease Joint pain Cerebrovascular disease The global market for SSAs in the treatment of acromegaly is estimated at ~$800 million with U.S. estimated at ~$400 million *
experience pain during injection; half of these experienced continuing pain days later Injections Carry Significant Treatment Burdens1 Pain Suboptimal Symptom Control Lost Workdays Emotional Impact Injection Site Reactions Hardness (48%), nodules (38%), swelling (28%), bruising (16%) and inflammation (7%) report symptoms worsen toward the end of the monthly dosing interval feel loss of independence due to chronic injections regularly miss work for injections (averages 11 days/year) 70% 52% 36% 16% 1Strasburger et al. Patient reported outcomes of parenteral somatostatin analogue injections in 195 patients with acromegaly. Eur J Endocrinol. 2016 Mar;174(3):355-62.
MYCAPSSA® First and only FDA-approved oral SSA for acromegaly; developed using TPE
Commercial Launch Strategy Pituitary Centers Regional Referral Centers High Volume Community Endocrinologists Other Community Endocrinologists ~90% of patients receiving injectable SSA therapies are treated at fewer than 1,000 medical practices ~1,000 Target Accounts Acromegaly Market Overview Promotional Strategy Expand to ~ 45-person sales, market access and patient services team in phases as external conditions permit Focus on patients and nurses to facilitate switching to MYCAPSSA Hybrid go-to-market model with a mix of digital tools and customer facing personnel on the ground
MYCAPSSA Phased Launch Approach 2020 2021 SEP OCT NOV DEC JAN FEB MAR APR MAY JUN JUL AUG SEP OCT NOV DEC 10 Field Sales FTEs (Total 22 FTE Customer-Facing Team*) 22 Field Sales FTEs (Total 36 FTE Customer-Facing Team*) ~30 Field Sales FTEs Planned (Total ~45 FTE Customer-Facing Team*) Reduction of in-person patient office visits Many HCPs offices closed to in-person calls Smaller office staff working separately and remotely Note: Full Strength Phase contingent upon market conditions and launch insights *Includes Field Management, Market Access, and Patient Services. Significantly increased digital promotion and engagement Equipped and trained sales force to conduct remote calls Strengthened resources to support reimbursement COVID-19 Impact on Treatment Landscape MYCAPSSA Launch Strategy Adaptations OBJECTIVES Introduce Chiasma and MYCAPSSA Build relationships with top-tier accounts Generate initial prescriptions and payer coverage OBJECTIVES Expand reach to broader base of accounts Accelerate pull through of prescriptions and expedite payer documentation Continue expanding payer coverage OBJECTIVES Reach target customers with optimal frequency including in-person calls Achieve widespread payer coverage and reimbursement Drive towards goal of becoming pharmacological standard of care in acromegaly FULL STRENGTH PHASE 1 PHASE 2
SSA-treated acromegaly patients are readily identifiable and are managed by a small number of endocrinologists Priced competitively – significant advancement for payers, patients, and the healthcare system Octreotide is well known and has been used for ~30 years Strong and informed acromegaly patient community Expected Drivers for a Differentiated U.S. Launch Solution for patients who prefer oral treatment options
Pricing Designed to Support Broad Payer Coverage MYCAPSSA: Compelling Value for Payers Wholesale Acquisition Cost (WAC) of $5,384 for 28-day supply at 40 mg starting dose (linear pricing for 60 mg and 80 mg) Oral option addressing a critical unmet need SSAs already reimbursed and in payers' budgets MYCAPSSA pricing designed to facilitate broad access Payer Mix* Commercial 57% Medicaid / 18% *Company estimates based on 2019 claims data. Priced to deliver significant value to patients and healthcare system MYCAPSSA covered for ~150 million lives at year end 2020
Telehealth consultation with the clinician A prescription communicated over the phone or internet Mobile phlebotomy for labs Home delivery and oral administration MYCAPSSA Adapts Well to Telemedicine Reduces the need for office visits
Comprehensive Multichannel Digital Campaign MYCAPSSA is the first and only FDA-approved oral somatostatin analog (SSA) for appropriate patients with acromegaly, providing effective and consistent biochemical control while freeing patients from the burden of injections. Activate Patients Paid Search Patient Ambassadors Paid Social Programmatic Display Peer to Peer Paid Search Paid Social Programmatic Display Point of Decision Engage HCPs
Chiasma Access and Patient Support Dedicated Patent Care Specialist Financial Assistance Benefits Investigation Specialty Pharmacy Interactions Coordination with Physician Offices Help patients with acromegaly get started on MYCAPSSA and support them throughout their treatment Chiasma case managers offer personalized patient support
Top Tier Commercial and Medical Leaders in Place Scott McConnell | Head of Medical Affairs Built and led multiple Medical Affairs organizations at Kaleido Biosciences, Alkermes, and Cubist Pharmaceuticals / Merck & Co. Derek Brown | Head of Marketing Led the global team responsible for the commercialization of Ultomiris® (Alexion) in two ultra-rare hematology diseases (PNH and aHUS) and held commercial leadership roles at Boehringer Ingelheim Anand Varadan | Chief Commercial Officer Built commercial organization and successfully launched orphan oncology drug for Karyopharm Therapeutics as CCO (2018 to 2019) General Management at Amgen in U.S. and internationally across numerous therapeutic areas (1999-2015) Jim Dion | Head of Sales Held Sales leadership roles at Tercica and Synageva; Ipsen; Head of US Patient Services at Akcea Launched Somatuline Depot at Ipsen Dan Thornton | Head of Market Access and Patient Services Held Market Access roles, including leadership roles, at Flexion Therapeutics, Shire, Targanta Therapeutics, Therion Biologics, Biogen Idec, and Johnson & Johnson
OUR SCIENCE TPE technology and MYCAPSSA clinical trials
Capsules with TPE technology have an enteric coating to protect against degradation in the stomach. Once in the small intestine, the capsule is designed to dissolve and release the TPE formulation. TPE technology induces the reversible expansion of tight junctions between intestinal epithelial cells, a natural process to absorb nutrients. Capsules containing TPE can allow drug therapies to enter systemic circulation while excluding toxins, bacteria and viruses. Validated TPE Delivery Technology Platform TPE enhances oral bioavailability, allowing for the development of oral formulations of injectable-only therapies
CHIASMA OPTIMAL Phase 3 trial, met primary and all secondary endpoints completed in 2019 MYCAPSSA® Phase 3 Trials for U.S. and Europe MPOWERED Phase 3 trial top-line data show Non-inferiority to leading SSA injectables Basis for U.S. approval Rigorous placebo-controlled trial Designed to support EMA application Comparative trial vs. injectable standard of care* CH-ACM-01 open-label Phase 3 trial completed in 2014 Robust safety database included in U.S. approved label Designed to reflect real-world clinical practice *octreotide and lanreotide
CHIASMA OPTIMAL Phase 3 Multinational, Randomized, Placebo-Controlled Study Double-blind placebo-controlled (DPC) (36 Weeks) Open Label Extension Primary Endpoint Proportion of patients who maintain biochemical response (average of week 34 and 36 IGF ≤ 1.0 x ULN ) Pre-defined Withdrawal Criteria (Both Arms) IGF-1 ≥1.3 x ULN for 2 consecutive visits on the highest dose of oral and exacerbation of clinical signs/symptoms Early terminated patients followed up to 36 weeks on injections, per protocol Eligibility Criteria: Average IGF-1 ≤ 1.0 x ULN Confirm active disease (IGF-1 ≥ 1.3 x ULN) post last surgery
Endpoints Octreotide (N=28) Placebo (N=28) P-value MAINTENANCE of CONTROL Mean IGF-1 at end of oral treatment 0.97 x ULN 1.69 x ULN PRIMARY Proportion maintaining IGF-1 response 58% 19% 0.008 SECONDARY Proportion maintaining GH response 78% 30% 0.001 Time to IGF-1 > 1.0 x ULN Time to IGF-1 ≥ 1.3 x ULN Median >36 weeks Median = 16 weeks <0.001 Reversion to prior injectable 25% 68% 0.003 Primary & All Secondary Endpoints Met
Octreotide capsules Placebo Subjects with: n % n % At least one TEAE 28 100.0 27 96.4 Treatment-related TEAE 18 64.3 15 53.6 SAEs 2 7.1 1 3.6 Treatment-related SAEs 0 0.0 0 0.0 Severe TEAEs 3 10.7 7 25.0 TEAE leading to study drug discontinuation 2 7.1 1 3.6 TEAEs of special interest (acromegaly symptoms) 15 53.6 26 92.9 MYCAPSSA Demonstrated as Safe & Well-Tolerated TEAE: Treatment-emergent adverse event SAE: Serious adverse event TEAEs of special interest: e.g., headache, perspiration, joint pain, fatigue, soft tissue swelling
The mean of the IGF-1 levels for the population of all MYCAPSSA treated patients that completed the 36-week, double-blind placebo controlled (DPC) CHIASMA OPTIMAL trial and continued into the OLE (n=19) were maintained within normal limits at the end of the 48-week OLE period. 90% of patients enrolled into the OLE who were treated with MYCAPSSA during the DPC phase of the study (n=20) completed the 48-week OLE period. All patients who enrolled into the OLE as responders to MYCAPSSA (IGF-1 within normal limits, n=14) completed the 48-week OLE period and 93% maintained their response within the normal limits at the end of this period. The safety profile observed during OLE was generally consistent with the safety of MYCAPSSA noted in the 36-week CHIASMA OPTIMAL trial with no new patterns noted with the increased duration of exposure. CHIASMA OPTIMAL 48-Week Open Label Extension Data
MPOWERED Non-inferiority Study Design and Top-line Data Last Injection Day (-28) Screening First Dose 6-Month Run-in Phase Responders Randomized to 9-Month Controlled Phase (RCT) Open Label Extension Follow for safety and efficacy 60% (55 pts) 40% (37 pts) Non-Responder Sub-study or Non-Responders Screening phase up to 4 weeks Eligibility criteria: IGF-1 <1.3 x ULN and GH<2.5 ng/mL
Higher Baseline IGF-1 Levels in the MYCAPSSA Arm Compared to Injectable SSA Arm MYCAPSSA Injectable Long-acting SSAs Total (N=55) Total (N=37) Week 26 IGF-1 (categorical) (n [%]) ≤ 1 x ULN 37 (67%) 29 (78%) > 1 to < 1.3 x ULN 12 (22%) 8 (22%) ≥ 1.3 x ULN 6 (11%) 0 (0%) Week 26 IGF-1 (ULN) Mean (SD) 0.9 (0.35) 0.8 (0.21)
MYCAPSSA Non-inferior to Long-acting Injectable SSAs Achieved the pre-specified non-inferiority margin of -20% Primary Endpoint: IGF-1 Time Weighted Average (TWA)1 throughout the RCT phase < 1.3 x ULN 1 TWA response represents an integrated measure of efficacy across time. It is derived as the area under the curve (AUC) divided by the total amount of time under observation between the first and last IGF-1 values. MYCAPSSA (n=55) Injectable SSAs (n=37) 95% CI 95% CI Proportion of Acromegaly Patients Achieving Primary Endpoint 91% 100%
Mean IGF-1 Levels Were Maintained Within Normal Limits Baseline of RCT Baseline of RCT End of Treatment End of Treatment
MYCAPSSA Appeared to be Safe and Well-Tolerated MYCAPSSA Injectable Long-acting SSAs Subjects with: n % n % At least one TEAE 39 70.9 26 70.3 Treatment-Related TEAE 19 34.5 15 40.5 SAEs 3 5.5 3 8.1 Severe TEAEs 5 9.1 4 10.8 TEAE Leading to Study Drug Discontinuation(1) 1 1.8 0 0 SAEs - Serious Adverse Events; TEAE - Treatment-Emergent Adverse Events (1) Discontinuation was determined by the applicable investigator to be unrelated to trial-related treatment.
MYCAPSSA Injectable Long Acting SSAs Total (N=55) Total (N=37) Gastrointestinal disorders Flatulence 14 (25.5) 8 (21.6) Nausea 11 (20.0) 3 (8.1) Diarrhea 6 (10.9) 5 (13.5) Abdominal pain 5 (9.1) 3 (8.1) Constipation 3 (5.5) 5 (13.5) Infections & infestations 5 (9.1) 0 Nasopharyngitis General disorders & admin. site conditions 0 5 (13.5) Injection site nodule Nervous system disorders 5 (9.1) 1 (2.7) Headache Musculoskeletal & connective tissue disorders 5 (9.1) 2 (5.4) Arthralgia MYCAPSSA Appeared Safe and Well-Tolerated Incidence of most common TEAEs (≥ 5%) in either treatment group by patient during the RCT TEAEs higher in the injectable SSAs arm compared to MYCAPSSA were constipation and injection site nodules TEAEs higher in the MYCAPSSA arm compared to injectable SSAs were nausea, nasopharyngitis, headache and arthralgia
MYCAPSSA® Patents Timeline Strong U.S. and EU Patent Position June 2020 U.S. Marketing Approval June 2027 Expiration of U.S. Orphan Exclusivity, assuming granted by FDA September 2029 Expiration of Formulation Patent (U.S. & EU) February 2036 Expiration of U.S. Issued Dosing Patent (EU patent approval pending) NOTES: Generics may enter the market at the end of the patent exclusivity and our patents may be challenged at any time; if a generic challenger wins a patent challenge, the generic can enter the market after expiration of regulatory and orphan exclusivity.
Timing (Est.) Anticipated Key Milestones Status Q1:2020 MYCAPSSA Octreotide Capsule NDA Acceptance from the FDA Mid-2020 MYCAPSSA CHIASMA OPTIMAL Phase 3 Study Data Presentation Mid-2020 MYCAPSSA PDUFA Approval (June 2020) Mid-2020 Publish CHIASMA OPTIMAL Phase 3 Study Data in Peer Reviewed Journal (July 2020) Q3:2020 MYCAPSSA API Manufacturing Supplement Acceptance/ Commercial Supply Availability (August 2020) Q3:2020 MYCAPSSA U.S. Launch (September 2020) Q4:2020 MPOWERED Phase 3 Study Top-Line Data (November 2020) Key 2020 Milestones Achieved
Timing (Est.) Anticipated Key Milestones 2021 Execute successful MYCAPSSA U.S. Launch Mid-2021 Publish MPOWERED Phase 3 Study Data in Peer Reviewed Journal Mid-2021 Planned MYCAPSSA EMA Regulatory Submission 2021 MYCAPSSA MPOWERED Virtual Data Presentation at Medical Conferences Planned 2021 Milestones
Financial Summary* * Unaudited cash, cash equivalents and marketable securities of ~$135 million at December 31, 2020. (In thousands, except per share data) Qtr. Ended Sept. 30, 2019 Qtr. Ended Sept. 30, 2020 Product Revenues, net $ - $142 Gross Profit $ - $139 Selling, General & Administrative $4,116 $13,048 Research & Development $4,110 $4,523 Net Loss $(7,683) $(18,457) Net Loss Per Basic Share $(0.20) $(0.30) Weighted Average Common Shares Outstanding – Basic 38,491 61,459 Dec. 31, 2019 Sept. 30, 2020 Cash, Cash Equivalents & Marketable Securities $92,375 $156,757** ** Excludes $20.3 million of restricted cash.
Raj Kannan Chief Executive Officer Management Team Mark J. Fitzpatrick President (Principal Financial Officer) William Ludlam, M.D., Ph.D. Clinical Development & Medical Affairs Anand Varadan Chief Commercial Officer Drew Enamait Finance & Administration Lee Giguere General Counsel Shoshie Katz VP, Regulatory & Quality; Israel Site Head Gary Patou, M.D. Strategic Clinical Advisor Roni Mamluk, Ph.D. Director Board of Directors Dave Stack Chairman of the Board Raj Kannan CEO & Director Todd Foley Director Bard Geesaman, M.D., Ph.D. Director John F. Thero Director Scott Minick Director John A. Scarlett, M.D. Director David Schubert SVP, Regulatory & Quality
Successful Launch with MYCAPSSA Enables a Bright Future Clinical Stage Company Commercial Stage Company with Validated TPE Platform Vision: Become a Successful Commercial Company with a Promising Pipeline
Thank You January 2021 | NASDAQ: CHMA