On August 1, 2019 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the second quarter ended June 30, 2019 and provided a business update (Press release, Insmed, AUG 1, 2019, View Source [SID1234538012]).

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"We are very excited about the continued strength of the U.S. launch of ARIKAYCE (amikacin liposome inhalation suspension), the first and only FDA-approved treatment for patients with refractory MAC lung disease, including the breadth and depth of prescribing, addition of new patients, and positive reception from the payer community throughout the first three quarters of launch," commented Will Lewis, Chairman and Chief Executive Officer of Insmed. "While the U.S. launch remains our greatest area of focus, we are pleased to have made significant progress on our other strategic priorities this quarter, including filing for regulatory approval of ARIKAYCE in the EU, advancing our planned regulatory filings in Japan, and completing enrollment in the Phase 2 WILLOW study of INS1007. As we move into the second half of the year as a fully operational commercial-stage company, we plan to execute against these priorities with a disciplined approach to investment."

Second Quarter 2019 Financial Results

· Total revenue for the second quarter ended June 30, 2019 was $30.0 million, comprising U.S. net sales of $29.0 million and ex-U.S. net sales of $1.0 million. The ex-U.S. net product sales include $0.9 million from the Temporary Authorization for Use (Autorisation Temporaire d’Utilisation or ATU) program in France and $0.1 million from the named patient program in Germany, both compassionate use programs.

· Cost of product revenues (excluding amortization of intangible assets) was $4.9 million for the second quarter of 2019.

·Research and development expenses were $33.5 million for the second quarter of 2019, compared with $35.7 million for the second quarter of 2018.

·Selling, general and administrative expenses for the second quarter of 2019 were $52.4 million, compared with $37.2 million for the second quarter of 2018. The increase was primarily due to higher expenses related to commercial activities for ARIKAYCE, including disease awareness, patient support activities, and field operations and, to a lesser extent, an increase in headcount, including non-cash stock-based compensation.

·For the second quarter of 2019, Insmed reported a GAAP net loss of $66.5 million, or $0.81 per share, compared with a GAAP net loss of $76.4 million, or $1.00 per share, for the second quarter of 2018.

· During the second quarter of 2019, Insmed completed a public offering of 10.7 million new shares of common stock that resulted in net cash proceeds of $261.2 million, after deducting underwriting discounts and commissions and other offering-related expenses.

Recent Corporate Developments & Program Highlights

Global Expansion Efforts Advance with Submission of MAA for ARIKAYCE to EMA

In July, Insmed submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ARIKAYCE for the treatment of persistent MAC lung infection as part of a combination antibacterial drug regimen in adults. The MAA was subsequently validated by the EMA. The proposed indication reflects the same population of refractory MAC lung disease patients for which ARIKAYCE is approved in the U.S.

ARIKAYCE previously received Orphan Drug Designation in the European Union (EU) for the treatment of nontuberculous mycobacterial (NTM) lung disease. MAC is the predominant pathogenic species in NTM lung disease in the United States, Europe, and Japan. The Company anticipates a 12-month review cycle with a potential launch of ARIKAYCE in certain European countries as early as the second half of 2020 if the MAA is approved.

In addition, the Company remains on track to submit a new drug application for ARIKAYCE in Japan in the first half of 2020.

Additional EU Patent Granted for ARIKAYCE

In July, the European Patent Office issued an additional patent to Insmed for ARIKAYCE in MAC lung disease. The claims of the patent relate in part to pharmaceutical compositions of amikacin encapsulated in liposomes, including ARIKAYCE, for treating or providing prophylaxis against MAC lung disease, where the composition is administered via aerosolization to the lungs of a patient once daily in a single dosing session for at least three months. The patent extends exclusivity of ARIKAYCE in Europe to May 15, 2035.

ARIKAYCE Launch and Lifecycle Management

Insmed continues to advance the U.S. launch of ARIKAYCE, which was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in September 2018 for the treatment of refractory MAC lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options.

As a next step toward advancing a post-approval confirmatory clinical trial for ARIKAYCE, the Company has initiated efforts to develop an appropriate patient reported outcome (PRO) tool that will enable the assessment of therapies for the treatment of NTM lung disease. If the PRO tool is verified, Insmed plans to conduct the confirmatory study of ARIKAYCE in a frontline setting of patients with MAC lung disease as well as a separate study in patients with NTM lung disease caused by Mycobacterium abscessus.

Data from Phase 3 CONVERT Study Presented as Late-Breaker at ATS

In May, longer-term data on the sustainability and durability of culture conversion from the Phase 3 CONVERT Study of ARIKAYCE were presented in a late-breaking oral session at the American Thoracic Society International Conference. The data showed that among patients who achieved culture conversion by Month 6, 80.0% (52/65) of those receiving ARIKAYCE plus guideline-based therapy (GBT) sustained culture conversion for up to 12 months of treatment after the first dose that defined culture conversion compared to 30.0% (3/10) of patients receiving GBT alone (p=0.0014). Three months after the completion of treatment, 63.1% (41/65) of patients receiving ARIKAYCE plus GBT maintained durable culture conversion, compared to 0.0% (0/10) of patients receiving GBT alone (p=0.0002).

Enrollment Completed in WILLOW Study

Insmed has completed enrollment in the six-month Phase 2 WILLOW study of INS1007 for patients with non-cystic fibrosis (CF) bronchiectasis. Top-line data are expected in early 2020.

Financial Guidance and Balance Sheet

As of June 30, 2019, Insmed had cash and cash equivalents of $601.3 million. The Company’s total costs and expenses for the second quarter of 2019 were $92.1 million, compared with total costs and expenses for the second quarter of 2018 of $72.9 million. Cash-based operating expenses for the second quarter of 2019 were $77.4 million, compared with cash-based operating expenses for the second quarter of 2018 of $65.3 million.

The Company now expects full-year 2019 revenues for ARIKAYCE to be in the range of $110 million to $120 million.

The Company plans to continue to invest in the following key activities in 2019:

(i) support of the U.S. launch and commercialization of ARIKAYCE;

(ii) clinical trials including (a) the development and verification of a PRO for NTM lung disease as a pivotal step toward initiating a confirmatory clinical study of ARIKAYCE, (b) the six-month Phase 2 WILLOW study of INS1007 in patients with non-CF bronchiectasis, and (c) the advancement of other pipeline programs including INS1009 and our earlier-stage research pipeline;

(iii) global expansion in Europe and Japan to support pre-commercial activities in those regions and potential regulatory filings in Japan; and

(iv) buildout of an additional third-party manufacturing facility to increase long-term production capacity for ARIKAYCE and a new corporate headquarters facility.

Insmed expects cash-based operating expenses to be in the range of $140 million to $155 million for the second half of 2019. In addition, the Company expects capital expenditures, including those related to the buildout of a new corporate headquarters facility as well as payments classified within other assets for the future right-of-use asset related to the buildout of an additional third-party manufacturing facility, to be in the range of $20 million to $30 million for the second half of 2019.

Conference Call

Insmed will host a conference call beginning today at 8:30 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing 1-888-317-6003 (domestic) or 1-412-317-6061 (international) and referencing conference ID number 5579948. 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 one hour after its completion through August 8, 2019 by dialing 1-877-344-7529 (domestic) or 1-412-317-0088 (international) and referencing replay access code 10133256. 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.

Non-GAAP Financial Measures

In addition to the U.S. generally accepted accounting principles (GAAP) results, this earnings release includes cash-based operating expenses, a non-GAAP financial measure, which Insmed defines as total costs and expenses excluding cost of product revenues, stock-based compensation expense, depreciation and amortization of intangibles. A reconciliation of this non-GAAP financial measure to its most directly comparable GAAP financial measure is presented in the table attached to this press release.

Management believes that this non-GAAP financial measure is useful to both management and investors in analyzing our ongoing business and operating performance. Management believes that providing this non-GAAP information to investors, in addition to the GAAP results, allows investors to view our financial results in the way that management views financial results. Management does not intend the presentation of this non-GAAP financial measure to be considered in isolation or as a substitute for results prepared in accordance with GAAP. In addition, this non-GAAP financial measure may differ from similarly named measures used by other companies.

About MAC Lung Disease

Mycobacterium avium complex (MAC) lung disease is a rare and serious disorder that can significantly increase morbidity and mortality. Patients with MAC lung disease can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, MAC lung disease can cause severe, even permanent damage to the lungs, and can be fatal.

MAC lung disease is an emerging public health concern worldwide with significant unmet needs. Current guideline-based treatment involves the use of multi-drug regimens that are not specifically approved for MAC lung disease. The course of treatment is often two years or more and is inadequate in treating the disease in many patients.

About ARIKAYCE (amikacin liposome inhalation suspension)

ARIKAYCE is the first and only FDA-approved therapy indicated for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. 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. This approach prolongs the release of amikacin in the lungs 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 (amikacin liposome inhalation suspension) 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 liquid medications, including liposomal formulations such as 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 and new pharmaceutical formulations that work together to improve patient care.

IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.

WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS

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

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

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

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

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

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.

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.

On August 1, 2019 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the quarter ended June 30, 2019 and highlighted recent business achievements news. Quarter-end cash and cash equivalents were $184.2 million.

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"In the second quarter of 2019, Pfizer completed enrollment in the pivotal Phase 3 trial of rivipansel and has recently given public guidance that it expects to report topline results in the third quarter of the year. We eagerly await those results, which will mark an important milestone for GlycoMimetics. In parallel, we continued to progress the late-stage clinical development of our uproleselan product candidate. Our Phase 3 trial in relapsed or refractory AML patients and the NCI-sponsored Phase 3 trial for newly diagnosed patients with AML both advanced during the quarter, with initiation of new sites and patient enrollment underway. During the same period, with our clinical collaborators at Duke Cancer Institute, we progressed our plans for a single center, proof-of-concept Phase 1b trial for GMI-1359, our dual antagonist of E-selectin and CXCR-4, in breast cancer patients with bone metastases," said Rachel King, GlycoMimetics’ Chief Executive Officer.

Key Second-Quarter 2019 and Recent Operational Highlights:

Pfizer completed enrollment in the Phase 3 trial of rivipansel in individuals with SCD experiencing vaso-occlusive crisis (VOC).
GlycoMimetics’ pivotal Phase 3 trial of uproleselan in relapsed/refractory AML continued to initiate and activate clinical sites and to enroll patients in the US, Australia and now in Europe.
Investigators initiated enrollment in the NCI-sponsored Phase 3 clinical trial designed to evaluate uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy.
Start-up activities continued for the collaborative Haemato Oncology Foundation for Adults in the Netherlands (HOVON) European Phase 2 trial of uproleselan in newly diagnosed patients unfit for chemotherapy.
The Company announced plans to initiate a Phase 1b proof-of-concept clinical trial of GMI-1359 in individuals with breast cancer whose tumors have spread to bone. The trial will evaluate safety and biomarkers of cancer cell mobilization in individuals with hormone receptor positive metastatic breast cancer. The trial will be conducted at Duke University.
Data were published in Nature Cell Biology that strongly suggest E-selectin is key to tumor growth and metastasis to bone and provide further support for the planned clinical trial of GMI-1359 in individuals with metastatic breast cancer.
Eric Feldman, M.D., joined the GlycoMimetics executive team as Vice President, Clinical Development, and Christian Dinneen-Long, J.D., joined as Vice President, Corporate Counsel.
Second Quarter 2019 Financial Results:

Cash position: As of June 30, 2019, GlycoMimetics had cash and cash equivalents of $184.2 million as compared to $209.9 million as of December 31, 2018.
R&D Expenses: The Company’s research and development expenses increased to $13.1 million for the quarter ended June 30, 2019 as compared to $9.3 million for the second quarter of 2018. This increase was primarily the result of expenses relating to the Company’s Phase 3 clinical trial of uproleselan in relapsed or refractory AML patients and supporting the clinical trials of uproleselan conducted by or in collaboration with third parties.
G&A Expenses: The Company’s general and administrative expenses increased to $3.8 million for the quarter ended June 30, 2019 as compared to $2.8 million for the quarter ended June 30, 2018. The increase was due to higher patent, legal and non-cash stock-based compensation expenses.
Shares Outstanding: Shares outstanding as of June 30, 2019 were 43,193,190.
The Company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 (U.S. and Canada) or (216) 562-0466 (international) with passcode 8268638. To access the live audio webcast, or the subsequent archived recording, visit the "Investors – Events & Presentations" section of the GlycoMimetics website at www.glycomimetics.com. The webcast will be recorded and available for replay on the GlycoMimetics website for 30 days following the call.

About Rivipansel

Rivipansel, the most advanced drug candidate in the GlycoMimetics pipeline, is a glycomimetic drug candidate that acts as a pan-selectin antagonist, meaning it binds to all three members of the selectin family: E-, P- and L-selectin. The first potential indication for rivipansel is VOC, one of the most severe complications of SCD that can result in acute ischemic organ injury at one or more sites. By reducing cell adhesion, activation and inflammation that are believed to contribute to reduced blood flow through the microvasculature during VOC, GlycoMimetics believes that rivipansel could be the first drug to interrupt the underlying cause of VOC, thereby potentially enabling patients to leave the hospital more quickly. In June 2019, Pfizer Inc., the exclusive licensee of rivipansel for clinical development and worldwide commercialization, completed its Phase 3 trial to evaluate the efficacy and safety of rivipansel in the treatment of VOC in hospitalized patients with SCD. Topline results from this clinical trial are expected in the third quarter of 2019.

About Uproleselan (GMI-1271)

Uproleselan (yoo’ pro le’ sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed or refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better-than-expected remission rates and overall survival compared to historical controls, which have been derived from results from third-party clinical trials evaluating standard chemotherapy, as well as lower-than-expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. In the second half of 2019, the Company plans to initiate an exploratory clinical trial in individuals with breast cancer whose tumors have spread to bone.

On August 1, 2019 Geron Corporation (Nasdaq: GERN) reported financial results for the second quarter ended June 30, 2019, and recent events (Press release, Geron, AUG 1, 2019, View Source [SID1234538010]). The Company ended the second quarter with $162.3 million in cash and marketable securities.

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During the second quarter, data from the Phase 2 portion of IMerge was reported at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress that further support Geron’s late-stage development plans for the imetelstat program. Geron continues to plan to open the Phase 3 portion of IMerge in lower risk myelodysplastic syndromes (MDS) for screening and enrollment in August 2019.

"This past quarter, data presented at EHA (Free EHA Whitepaper) for the Phase 2 portion of IMerge reported higher efficacy responses from prior reported data for both 8-week and 24-week RBC-TI rates, highlighting the meaningful and durable transfusion independence potentially achievable with imetelstat treatment in heavily transfusion dependent lower risk MDS patients. Accordingly, we’re very much looking forward to the Phase 3 portion of IMerge opening for screening and enrollment later this month," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "In addition, a second EHA (Free EHA Whitepaper) presentation reported results of statistical analyses in which the months of median overall survival were more than double for imetelstat-treated relapsed/refractory MF patients in IMbark, compared to real-world data from closely matched patients treated with best available therapy. This provides additional support for our ongoing strategic evaluation of potential late-stage development approaches and regulatory scenarios in relapsed/refractory MF as we prepare for an End of Phase 2 meeting with the FDA, which we’re planning to conduct by the end of the first quarter of 2020."

Phase 3 Portion of IMerge Phase 2/3 Clinical Trial – Initial Site Initiations Complete and Trial to Open for Patient Screening and Enrollment in August 2019

In May, Geron completed the transfer of the imetelstat investigational new drug (IND) sponsorship from Janssen Biotech, Inc. (Janssen), and assumed complete development responsibility for imetelstat. Site initiations for the Phase 3 portion of IMerge were completed for several clinical sites in July 2019. Geron is planning for the trial to open for patient screening and enrollment later this month.

IMerge is a two-part Phase 2/3 clinical trial of imetelstat in transfusion dependent patients with lower risk MDS who are relapsed/refractory to erythroid stimulating agents (ESAs). The primary efficacy endpoint is the rate of red blood cell transfusion independence (RBC-TI) lasting at least eight weeks, or 8-week RBC-TI rate, which is defined as the proportion of patients achieving RBC-TI during any consecutive eight weeks since entry into the trial. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, durability of transfusion independence and the amount and relative change in RBC transfusions.

The Phase 3 portion of IMerge is designed as a randomized, double-blind, placebo-controlled clinical trial to test the hypothesis that imetelstat improves the rate of RBC-TI compared to placebo.

Approximately 170 patients are planned to be enrolled and randomized in a 2:1 ratio to receive either imetelstat or placebo. Many key aspects from the Phase 2 portion of IMerge remain the same for the Phase 3 portion. A target patient population of non-del(5q) lower risk MDS patients who were naïve to treatment with hypomethylating agents (HMAs) and lenalidomide was identified from the Phase 2 portion and will be enrolled in the Phase 3. In addition, the primary and secondary endpoints, the dose and schedule of imetelstat administration and many of the clinical sites remain the same as in the Phase 2. The Company is planning for the trial to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia.

Second Quarter and Year-to-Date 2019 Results

For the second quarter of 2019, the Company reported a net loss of $14.2 million, or $0.08 per share, compared to $6.9 million, or $0.04 per share, for the comparable 2018 period. Net loss for the first six months of 2019 was $24.3 million, or $0.13 per share, compared to $14.1 million, or $0.08 per share, for the comparable 2018 period.

Revenues for the three and six months ended June 30, 2019 were $101,000 and $158,000, respectively, compared to $208,000 and $526,000 for the comparable 2018 periods. Revenues for the three and six months ended June 30, 2019 and 2018 included royalty and license fee revenues under various non-imetelstat license agreements. The decline in revenues reflects a reduction in the number of active research license agreements in 2019 related to the Company’s human telomerase reverse transcriptase, or hTERT, technology as a result of patent expirations on the underlying technology.

Total operating expenses for the three and six months ended June 30, 2019 were $15.3 million and $26.7 million, respectively, compared to $7.5 million and $15.2 million for the comparable 2018 periods. Research and development expenses for the three and six months ended June 30, 2019 were $10.1 million and $16.0 million, respectively, compared to $3.2 million and $5.6 million for the comparable 2018 periods. The increase in research and development expenses, compared to the same periods in 2018, primarily reflects costs for the transition of the imetelstat program, including resuming sponsorship of the ongoing imetelstat clinical trials; expenses for start-up activities for the Phase 3 portion of IMerge; and higher personnel-related costs for the expanding development team. General and administrative expenses for the three and six months ended June 30, 2019 were $5.2 million and $10.6 million, respectively, compared to $4.2 million and $9.6 million for the comparable 2018 periods. The increase in general and administrative expenses, compared to the same periods in 2018, primarily reflects higher corporate and patent legal costs and increased personnel-related expenses for additional headcount to support the development organization.

Interest and other income for the three and six months ended June 30, 2019 was $1.1 million and $2.3 million, respectively, compared to $717,000 and $1.1 million for the comparable 2018 periods. The increase in interest and other income, compared to the same periods in 2018, primarily reflects higher yields on the Company’s increased marketable securities portfolio.

The Company ended the second quarter of 2019 with $162.3 million in cash and marketable securities. Since May 2019, the Company has raised cumulative net cash proceeds of approximately $2.6 million from the sales of an aggregate of 1,893,091 shares of common stock under an At Market Issuance Sales Agreement, after deducting sales commissions and other offering expenses payable by Geron. The Company expects these net cash proceeds to provide additional financial flexibility as it advances the imetelstat development program. The funds will support future development costs, including conducting the Phase 3 portion of IMerge.

2019 Financial Guidance Reaffirmed

For fiscal year 2019, the Company expects total operating expenses to range from $80 to $85 million, of which approximately $20 to $25 million represents one-time costs that include imetelstat program transition activities from Janssen to Geron and purchase of drug product, drug substance and raw materials from Janssen to supply the Phase 3 portion of IMerge and prepare for new drug manufacturing. The Company continues to expect transition of the imetelstat program from Janssen to be completed by the end of the third quarter of 2019.

As of July 31, 2019, the Company had 38 employees, and plans to grow to a total of approximately 45 to 50 employees by year-end 2019, of whom half will be research and development personnel.

Conference Call

Geron will host a conference call to discuss second quarter and year-to-date financial results and recent events at 4:30 p.m. ET on Thursday, August 1, 2019.

Participants may access the conference call live via telephone by dialing domestically +1 (877) 303-9139 or internationally +1 (760) 536-5195. The conference ID is 2379646. A live, listen-only webcast will also be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS) and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for the treatment of patients with transfusion dependent anemia due to non-del(5q) lower risk MDS who are refractory or resistant to an erythroid stimulating agent.

On August 1, 2019 Corvus Pharmaceuticals, Inc. (Nasdaq: CRVS), a clinical-stage biopharmaceutical company focused on the development and commercialization of precisely targeted oncology therapies, reported financial results for the second quarter ended June 30, 2019 (Press release, Corvus Pharmaceuticals, AUG 1, 2019, View Source [SID1234538009]).

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"Corvus continues to be a leader in the field of drug development targeting the adenosine pathway, with complementary lead programs – ciforadenant and CPI-006 – that have shown promising activity in a range of solid tumors. We also have a third program in the clinic, CPI-818, which is an ITK inhibitor for the treatment of patients with advanced T-cell lymphomas. Together, I believe these three programs position Corvus as a leader in the development of second-generation immuno-oncology agents," said Richard A. Miller, M.D., president and chief executive officer of Corvus. "At ASCO (Free ASCO Whitepaper) this year, initial clinical data on CPI-006 was presented in an oral session and subsequently selected for the Highlight of the Day session. The data demonstrated activity as a monotherapy and in combination with ciforadenant and that CPI-006 represents a potential novel immuno-oncology approach via activation of immune cells and the inhibition of adenosine production. We plan to continue presenting new data on our programs at the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting and other meetings over the remainder of the year."

Recent Achievements
Ciforadenant (CPI-444): A2A Receptor Antagonist of Adenosine

Continued enrollment of patients with renal cell cancer (RCC) in an amended Phase 1b/2 clinical trial evaluating ciforadenant in combination with Genentech’s Tecentriq (atezolizumab), an anti-PD-L1 antibody. The RCC patients in the trial have failed treatments with anti-PD-(L)1 antibodies and tyrosine kinase inhibitors. This trial is also evaluating the use of a novel gene expression biomarker known as the Adenosine Signature, that may have the potential to predict patients most likely to respond to therapy.
Amending our ongoing Phase 1b/2 study to include an arm evaluating ciforadenant in combination with Tecentriq in patients with prostate cancer. This arm will also evaluate the use of the Adenosine Signature.
Continued follow-up in a Phase 1b/2 trial of ciforadenant and Tecentriq being conducted by Genentech as part of their MORPHEUS platform. The study is evaluating this combination in patients who have failed no more than two prior regimens.
CPI-006: Immunomodulatory Anti-CD73 Antibody

Continued enrollment of up to 350 patients with advanced cancer in a Phase 1/1b clinical trial evaluating CPI-006 as a single agent and in combination with either ciforadenant or pembrolizumab. The trial is currently enrolling patients in the dose escalation phase for CPI-006 administered as a monotherapy and in combination with ciforadenant.
Initial clinical data covering 20 patients with advanced, refractory cancer from the Phase 1/1b trial was delivered in an oral presentation at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2019. The initial data showed a trend toward longer disease control with higher doses of CPI-006, and enhanced disease control with CPI-006 in combination with ciforadenant compared with monotherapy. Additional highlights from the presentation:
Clinical and laboratory results demonstrated potential for a new immuno-oncology approach with CPI-006 via activation of immune cells and the inhibition of adenosine production.
Dose-dependent disease control when administered as a monotherapy and in combination with ciforadenant.
CPI-006 was well tolerated at all dose levels, with no dose-limiting toxicities. Grade 1 infusion reactions were detected (N=3 patients) and mitigated with premedication with acetaminophen and antihistamine. Grade 3 or 4 toxicities included a grade 3 anemia (N=1).
CPI-818: A small molecule ITK inhibitor

Continued enrolling patients with T-cell lymphomas, including peripheral T-cell lymphoma (PTCL), cutaneous T-cell lymphoma (CTCL) and others, in a Phase 1/1b study with CPI-818, an ITK inhibitor.
Anticipated Events in 2H 2019

Updated clinical and immunologic data from the Phase 1/1b clinical trial of CPI-006 monotherapy and combination with ciforadenant is expected to be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in November 2019.
Presentation on identification and role of biomarkers in adenosine pathway therapies is expected to be presented at SITC (Free SITC Whitepaper).
Preclinical and early clinical data regarding CPI-818 is expected to be presented in late 2019 or early 2020.
Financial Results

At June 30, 2019, Corvus had cash, cash equivalents and marketable securities totaling $96.8 million, as compared to cash, cash equivalents and marketable securities of $114.6 million at December 31, 2018. Corvus expects full-year 2019 net cash used in operating activities to be between $38 and $42 million and estimates ending 2019 with cash, cash equivalents and marketable securities of between $73 and $77 million.

Research and development expenses for the three months ended June 30, 2019 totaled $10.6 million, as compared to $9.7 million for the same period in 2018. The increase of $0.9 million was primarily due to an increase in CPI-006 program costs.

The net loss for the three months ended June 30, 2019 was $13.0 million, compared to a net loss of $11.6 million for the same period in 2018. Total stock compensation expense for the three months ended June 30, 2019 was $1.9 million, as compared to $1.7 million for the same period in 2018.

Conference Call Details
Corvus will host a conference call and webcast today, Thursday, August 1, 2019, at 4:30 p.m. ET (1:30 p.m. PT), during which time management will provide a business update and discuss the second quarter 2019 financial results. The conference call can be accessed by dialing 1-888-599-8686 (toll-free domestic) or 1-720-543-0302 (international) and using the conference ID 2091306. The live webcast may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days following the call.

On August 1, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its results for the quarter ended June 30, 2019 (Press release, Corcept Therapeutics, AUG 1, 2019, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-second-quarter-2019-financial [SID1234538008]).

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Financial Highlights

Revenue of $72.3 million, a 16 percent increase from second quarter 2018
GAAP net income of $0.17 per share, compared to $0.14 per share in second quarter 2018
Non-GAAP net income of $0.25 per share, compared to $0.20 per share in second quarter 2018
Cash and investments of $225.7 million, compared to $215.7 million at March 31, 2019
Reaffirmed 2019 revenue guidance of $285 – $315 million
Corcept reported quarterly revenue of $72.3 million, compared to $62.3 million in the second quarter of 2018. Second quarter GAAP net income was $20.2 million, compared to $18.2 million in the same period last year. Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the second quarter was $31.0 million, compared to $25.4 million in the second quarter of 2018. A reconciliation of GAAP to non-GAAP net income is included below.

The company reaffirmed its 2019 revenue guidance of $285 – $315 million.

Second quarter operating expenses were $47.6 million, compared to $41.7 million in the second quarter of 2018, primarily due to growth in the number of research and development personnel, increased spending to advance new compounds, and increased spending to formulate and manufacture the selective cortisol modulators relacorilant, exicorilant and miricorilant.

Cash and investments were $225.7 million at June 30, 2019, an increase of $10.0 million from March 31, 2019. This increase was after the expenditure of $17.4 million in the second quarter to repurchase 1.6 million shares of common stock pursuant to Corcept’s stock repurchase program.

"Our Cushing’s franchise had a strong quarter," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "As they have every year, patients taking Korlym successfully overcame the insurance reauthorization hurdles many of them face in the first quarter. These obstacles do not interrupt their care, because we provide them with free Korlym. They do, however, temporarily reduce our revenue. Meanwhile, our business grew as the number of patients receiving Korlym and the number of physicians prescribing the medication continued to increase."

Clinical Highlights

"It is gratifying to work on development programs that may benefit so many patients," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "We are advancing our proprietary, selective cortisol modulators in Cushing’s syndrome, solid tumors and metabolic diseases. Our clinical trials in these areas are progressing and we plan to start important new trials later this year and in 2020."

Cushing’s Syndrome

Dosing continues in Phase 3 trial ("GRACE") of relacorilant in patients with Cushing’s syndrome
Placebo-controlled trial of relacorilant to treat patients with less severe Cushing’s syndrome
to begin late this year
GRACE seeks to confirm relacorilant’s positive Phase 2 results and to provide the basis for its approval in the United States and Europe as a treatment for Cushing’s syndrome.1 Patients in relacorilant’s Phase 2 trial exhibited meaningful improvements in glucose control and hypertension – two of Cushing’s syndrome’s most common and pernicious manifestations. The trial also met a wide range of secondary endpoints, including weight loss, liver function, coagulopathy, insulin resistance, cognitive function, mood and quality of life. These results were achieved without instances of Korlym’s significant off-target effects – vaginal bleeding, endometrial thickening and low potassium.2

In the fourth quarter, Corcept plans to start a double-blind, placebo-controlled trial of relacorilant in patients whose Cushing’s syndrome is caused by an adrenal adenoma – an etiology where the effect of medical treatment has not been extensively studied. A controlled study is possible in these patients because their symptoms, while serious, are usually less severe than those experienced by patients with other etiologies of the disorder. The trial will enroll approximately the same number of patients as the GRACE trial at sites in the United States and Europe. Most of these sites are also participating in GRACE.

Metabolic Disease

Phase 1b trial underway in reduction of antipsychotic-induced weight gain; results expected late this year
Phase 2 trial in reversal of recent antipsychotic-induced weight gain planned to start late this year
Corcept’s Phase 1b trial in the reduction of weight-gain caused by antipsychotic medication is on track to produce results late this year. Approximately 60 healthy subjects will receive olanzapine (Eli Lily’s Zyprexa) and either miricorilant or placebo for two weeks, with the primary endpoint being change in weight. "We modeled this trial on the successful placebo-controlled studies3 we conducted with Korlym," said Dr. Grauer. "Unfortunately, Korlym’s off-target effects preclude its development for such a common disorder. Miricorilant is a better potential medication because it does not bind to the progesterone receptor. Activity at the progesterone receptor is what causes Korlym’s off-target effects. We look forward to completing this trial and starting one double-blind, placebo-controlled Phase 2 trial in patients later this year and another in 2020."

Corcept is also advancing miricorilant as a treatment for NASH, a serious and widespread liver disorder, and plans to start a double-blind, placebo-controlled Phase 2 trial in 2020.

Solid Tumors

Phase 3 trial of relacorilant plus nab-paclitaxel in patients with metastatic pancreatic cancer planned
to start late this year
European Medicines Agency Committee for Orphan Medicinal Products (COMP) recommends orphan drug designation for relacorilant to treat metastatic pancreatic cancer
"At the American Society of Clinical Oncologists (ASCO) (Free ASCO Whitepaper) meeting this June, we presented data from our Phase 1/2 study of relacorilant plus the taxane nab-paclitaxel (Celgene’s Abraxane)," said Dr. Grauer. "Seven of 25 patients with metastatic, pancreatic cancer and five of 11 patients with advanced ovarian cancer achieved "durable disease control," meaning their tumors either shrank or ceased growing for a period of 16 weeks or longer. Tumor response in two patients with pancreatic disease persisted for more than 50 weeks. One patient’s ovarian tumor disappeared completely; another’s responded for 65 weeks.4Any response to treatment in patients with such advanced disease is surprising. That is especially true in these patients, whose tumors had progressed during multiple lines of prior therapy, including treatments with nab-paclitaxel or another taxane.

"We hope to confirm these findings in larger, more definitive studies. Our 180-patient, placebo-controlled Phase 2 trial in ovarian cancer is enrolling patients. We plan to start a Phase 3 trial in metastatic pancreatic cancer later this year and are seeking FDA guidance regarding the fastest path to approval in that indication. Relacorilant’s designation as an orphan drug for pancreatic cancer in the United States and the European Union will be helpful as our program advances."

On July 18, 2019, the COMP recommended orphan designation of relacorilant for the treatment of pancreatic cancer. The COMP’s letter stated that "relacorilant has the potential to restore tumour sensitivity to taxane therapy. This was demonstrated by non-clinical and clinical results, i.e. the achievement of durable partial responses or disease control in some patients, despite previously failed treatment regimens. The COMP considered that the preliminary clinical data submitted by the sponsor supported the claim [of] significant benefit for the purpose of an initial orphan designation."

The European Commission is expected to adopt the COMP’s recommendation later this year. The FDA designated relacorilant an orphan drug for the treatment of pancreatic cancer in September 2018.

Conference Call

We will hold a conference call on August 1, 2019, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, dial 1-888-394-8218 from the United States or 1-323-794-2590 internationally approximately ten minutes before the start of the call (passcode 9712194). A replay will be available through August 15, 2019 at 1-888-203-1112 in the United States and 1-719-457-0820 internationally (passcode 9712194).

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most people with Cushing’s syndrome experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.