Galectin Therapeutics Reports Fiscal 2019 Financial Results and Provides Business Update
NASH-RX Trial Update
The NASH-RX trial is planned to use an adaptive design, confirm dose selection and reaffirm the efficacy data observed in the NASH-CX trial and, with pre-planned adaptations, inform the larger Phase 3 trial component. The adaptive design being considered allows pre-planned adjustments of the trial that may include, amongst other factors, optimization of dose selection, confirmation of efficacy and proof of concept observed in the NASH-CX trial, optimized sizing and statistical powering of the Phase 3 component, and possible inclusion of more advanced cirrhotic patients. We believe that these adaptations taken together should optimize conduct of the NASH-RX trial giving belapectin (GR-MD-02) the best opportunity to show a positive therapeutic effect. If the results of the NASH-RX trial are compelling, there could be the potential for accelerated FDA approval and/or partnership opportunity with a large pharmaceutical company.
The trial protocol is based on feedback from several interactions with the FDA during the last few months of 2019, including the
We believe the study design potentially could improve the likelihood of showing drug efficacy because:
- It clarifies and reaffirms NASH-CX efficacy and safety at two distinct drug doses supported by robust pharmacokinetic analysis
- It provides for appropriate selection of optimal dose – e.g., single dose (2 or 4 mg/kg) for Phase 3 component
- A separate Hepatic Impairment study may allow inclusion of more severe patients who are believed to have a much higher rate of esophageal varices progression and bleeding and other decompensating events
- Reduced frequency of esophagogastroduodenoscopys (EGD), elimination of biopsy endpoints and elimination of hepatic venous pressure gradient (HVPG) testing may make it easier to enroll trial patients and retain these patients during the duration of the trial
- Adaptation to size and power calculations based on sample size re-estimation and the interim analysis will allow better estimates of Phase 3 cohort sizing and of statistical power
- A planned interim analysis after 18 months of completed treatment will assess affirmation of Phase 2 efficacy and safety results, help select a single optimal dosage, and inform the Phase 3 stage of the study, including its size
In the Phase 3 component of this trial, the primary endpoint is development of new esophageal varices. Patients already enrolled for the Phase 2b component of the trial will continue on the selected single dose into the Phase 3 component of the trial. Patient selection for both Phase 2b and 3 components will be based on routine clinical signs of portal hypertension, including, amongst others, the presence or absence of varices, depressed platelet count (thrombocytopenia), enlargement of the spleen size and evidence of collateral blood vessels by imaging. The current study design and protocol are subject to modification after review by FDA.
The focus and goal of the therapeutic program is to prevent the development of large esophageal varices, which are strongly correlated with patient mortality due to sudden and severe bleeding. Based on the results of the NASH-CX trial, the clinical program will focus on patients who are at increased risk of developing varices, i.e. patients who have clinical signs of portal hypertension, such as low platelet counts or increased spleen size (splenomegaly).
The key milestones and associated target dates for the NASH-RX trial will be announced as elements of design of the trial are finalized based on the recent FDA feedback. However, we currently expect the first patient to be initiated in the second quarter of 2020. The study overall will likely involve approximately 130 sites in 11 countries in
- Announced that
Pol F. Boudes, M.D. has been appointed Chief Medical Officer - a key development for the company as it nears launch of its NASH-RX trial, an adaptively-designed Phase 3 trial in NASH cirrhosis. Dr. Boudes’ diverse background in drug development, especially his experience in NASH and other liver diseases, bolsters Galectin's global advanced clinical development of belapectin for NASH cirrhosis.
Peer-reviewed publication, Scientific Presentations and Conferences
- Gastroenterology, a prominent journal in the field of gastrointestinal disease, published a peer reviewed paper titled, “Effects of Belapectin, an Inhibitor of Galectin-3, in Patients with Nonalcoholic Steatohepatitis with Cirrhosis and Portal Hypertension,” highlighting the potential prevention of esophageal varices of its NASH-CX Phase 2 clinical trial in NASH cirrhosis. We were greatly honored that such a prestigious, peer-reviewed publication felt the quality of our science merited the industry-wide attention they provide.
- Initial results of the efforts at
Galectin Sciences LLC(our majority-owned subsidiary) were presented by Dr. E. Zomer, Ph.D., Vice President, Discovery Research and Product Development, at the 3rd Annual Anti-Fibrotic Drug Development (AFDD) Summit regarding Galectin’s discovery program of its next generation of oral galectin-3 inhibitors. The presentation entitled “Therapeutic Integrin Inhibition,” discussed the next generation of galectin-3 inhibitors, as well as the discovery of functional allosteric inhibitors.
For the year ended December 31, 2019, the Company reported a net loss applicable to common stockholders of
Research and development expense for 2019 was
As of December 31, 2019, the Company had $47.5 million of cash and cash equivalents. During 2019, the company effected a Rights Offering which, together with other common stock and warrants issued, raised
The Company expects that it will require more cash to fund operations after
About Galectin Therapeutics
Galectin Therapeutics is dedicated to developing novel therapies to improve the lives of patients with chronic liver disease and cancer. Galectin’s lead drug belapectin (formerly known as GR-MD-02) is a carbohydrate-based drug that inhibits the galectin-3 protein which is directly involved in multiple inflammatory, fibrotic, and malignant diseases, for which it has Fast Track designation by the
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements relate to future events or future financial performance, and use words such as “may,” “estimate,” “could,” “expect” and others. They are based on management’s current expectations and are subject to factors and uncertainties that could cause actual results to differ materially from those described in the statements. These statements include those regarding the hope that Galectin’s development program for belapectin will lead to the first therapy for the treatment of fatty liver disease with cirrhosis and those regarding the hope that our lead compounds will be successful in cancer immunotherapy and in other therapeutic indications. Factors that could cause actual performance to differ materially from those discussed in the forward-looking statements include, among others, that trial endpoints required by the FDA may not be achieved; Galectin may not be successful in developing effective treatments and/or obtaining the requisite approvals for the use of belapectin or any of its other drugs in development; the Company may not be successful in scaling up manufacturing and meeting requirements related to chemistry, manufacturing and control matters; the Company’s currently planned clinical trial and any future clinical studies as modified to meet the requirements of the FDA may not produce positive results in a timely fashion, if at all, and could require larger and longer trials, which would be time consuming and costly; plans regarding development, approval and marketing of any of Galectin’s drugs are subject to change at any time based on the changing needs of the Company as determined by management and regulatory agencies; regardless of the results of any of its development programs, Galectin may be unsuccessful in developing partnerships with other companies or raising additional capital that would allow it to further develop and/or fund any studies or trials. Galectin has incurred operating losses since inception, and its ability to successfully develop and market drugs may be impacted by its ability to manage costs and finance continuing operations. Global factors such as coronavirus may limit access to NASH patient populations around the globe and slow trial enrollment and prolong the duration of the trial and significantly impact associated costs. For a discussion of additional factors impacting Galectin’s business, see the Company’s Annual Report on Form 10-K for the year ended
Condensed Consolidated Statements of Operations
|Research and development||$||7,467||$||6,471|
|General and administrative||5,971||7,131|
|Total operating expenses||13,438||13,602|
|Total operating loss||(13,438)||(13,602)|
|Other income (expense):|
|Total other income||144||(298)|
|Preferred stock dividends||(263)||(1,147)|
|Net loss applicable to common stock||$||(20,179)||$||(15,047)|
|Basic and diluted net loss per share||$||(0.39)||$||(0.38)|
|Shares used in computing basic and diluted net loss per share||52,238||39,414|
Condensed Consolidated Balance Sheet Data
|Cash and cash equivalents||$||47,480||$||8,253|
|Total current liabilities||2,820||2,108|
|Total redeemable, convertible preferred stock||1,723||1,723|
|Total stockholders’ equity||$||43,872||$||5,175|
Source: Galectin Therapeutics Inc.