- Completed enrollment in Phase 2 (Part B) of the MoveDMD trial of edasalonexent in October. Top-line safety and efficacy results from the placebo-controlled portion of the MoveDMD trial are expected in the first half of Q1 2017.
- Presented positive biomarker gene expression and proteomic data from Part A of the MoveDMD trial of edasalonexent at the World Muscle Society Congress.
- Announced the initiation of an open-label extension for the MoveDMD trial, in which patients will continue on open-label edasalonexent for 36 weeks following completion of the 12-week, placebo-controlled portion of the trial. During the open-label extension, safety will be monitored and additional assessments including MRI, timed function tests, muscle strength measures, the North Star Ambulatory Assessment and the pediatric outcomes data collection instrument (PODCI) will be performed.
- Announced a joint research collaboration with Sarepta Therapeutics to explore a combination drug treatment approach for DMD. The two companies will contribute their respective expertise to study edasalonexent, an oral NF-kB inhibitor, developed by Catabasis, together with an exon skipping treatment developed by Sarepta in a mouse model of DMD.
- Presented positive preclinical data for CAT-5571, a potential therapy to treat cystic fibrosis (CF), at the North American Cystic Fibrosis Conference. CAT-5571, an activator of autophagy, in combination with lumacaftor/ivacaftor, enhanced cell-surface trafficking and function of cystic fibrosis transmembrane conductance regulator (CFTR) in bronchial epithelial cells from CF patients with the F508del mutation. Catabasis also demonstrated that CAT-5571 enhanced the clearance of Pseudomonas aeruginosa infection in preclinical models of CF, irrespective of CFTR mutation status.
- Continued CAT-4001 preclinical program research in rare neurodegenerative diseases. Preclinical activities exploring the potential of CAT-4001 in diseases such as amyotrophic lateral sclerosis (ALS) and Friedreich's ataxia are ongoing.
- Catabasis will host its first Investor Day in New York on November 17, 2016, focused on its strategy in rare diseases and the pipeline, including edasalonexent and other programs. The Investor Day is open to members of the professional investment community. For more information or to register, please email CATB@macbiocom.com.
- In September 2016, Catabasis sold 2,875,000 shares of common stock in an underwritten registered direct offering at a price of $4.00 per share and generated net proceeds of $10.6 million. During third quarter 2016, Catabasis sold 368,015 shares under an at-the-market offering program for net proceeds of $1.3 million.
|Participant Toll-Free Dial-In Number:||(877) 388-2733|
|Participant International Dial-In Number:||(541) 797-2984|
Interested parties may access a live audio webcast of the conference call via the investor section of the Catabasis website, www.catabasis.com. Please connect to the Catabasis website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The webcast will be archived for 90 days.About Edasalonexent (CAT-1004)Edasalonexent (CAT-1004) is an oral small molecule that has the potential to be a disease-modifying therapy for all patients affected by Duchenne muscular dystrophy (DMD or Duchenne), regardless of their underlying mutation. Edasalonexent inhibits NF-kB, a protein that is activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration. In animal models of DMD, edasalonexent produced beneficial effects in skeletal, diaphragm and cardiac muscle and improved function. The FDA has granted orphan drug, fast track and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. We have previously reported safety, tolerability and reduction in NF-kB activity in Phase 1 trials in adults. We are currently conducting the MoveDMD ® trial of edasalonexent in 4-7 year-old boys affected by Duchenne. From Part A of the MoveDMD trial, we have reported that edasalonexent was generally well tolerated with no safety signals observed and we observed NF-kB target engagement. Pharmacokinetic results demonstrated edasalonexent plasma exposure levels consistent with those previously observed in adults, at which inhibition of NF-kB was observed. About CAT-4001Catabasis is developing CAT-4001 as a potential treatment for neurodegenerative diseases such as Friedreich's ataxia (FA) and amyotrophic lateral sclerosis (ALS). CAT-4001 is a small molecule that activates Nrf2 and inhibits NF-kB, two pathways that have been implicated in FA and ALS. Catabasis has shown that CAT-4001 modulates the Nrf2 and NF-kB pathways in both cellular assays and animal models. About CAT-5571Catabasis is developing CAT-5571 as a potential oral treatment for cystic fibrosis (CF) with potential effects on both the cystic fibrosis transmembrane conductance regulator (CFTR) and on the clearance of Pseudomonas aeruginosa. CAT-5571 is a small molecule that activates autophagy, a process that maintains cellular homeostasis and host defense mechanisms, and is known to be impaired in CF. Catabasis has shown in preclinical studies that CAT-5571, in combination with lumacaftor/ivacaftor, enhances cell-surface trafficking and function of CFTR with the F508del mutation. Catabasis has also shown that CAT-5571 enhances the clearance of P. aeruginosa infection in preclinical models of CF, irrespective of CFTR mutation status. About CatabasisAt Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our SMART (Safely Metabolized And Rationally Targeted) linker drug discovery platform enables us to engineer molecules that simultaneously modulate multiple targets in a disease. We are applying our SMART linker platform to build an internal pipeline of product candidates for rare diseases and plan to pursue partnerships to develop additional product candidates. For more information on the Company's drug discovery platform and pipeline of drug candidates, please visit www.catabasis.com. Forward Looking StatementsAny statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2016, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.
|Catabasis Pharmaceuticals, Inc. Condensed Consolidated Statements of Operations(In thousands, except share and per share data) (Unaudited)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Research and development||$||5,936||$||5,813||$||19,190||$||16,360|
|General and administrative||2,347||2,388||7,695||5,966|
|Total operating expenses||8,283||8,201||26,885||22,326|
|Loss from operations||(8,283||)||(8,201||)||(26,885||)||(22,326||)|
|Other (expense) income:|
|Interest and investment income||50||-||183||-|
|Other income, net||13||(2||)||82||11|
|Total other expense, net||(136||)||(284||)||(397||)||(698||)|
|Net loss per share - basic and diluted||$||(0.54||)||$||(0.55||)||$||(1.77||)||$||(4.11||)|
|Weighted-average common shares outstanding used innet loss per share - basic and diluted||15,512,608||15,297,794||15,407,747||5,596,412|
|Catabasis Pharmaceuticals, Inc. Condensed Consolidated Balance Sheets (In thousands) (Unaudited)|
|September 30,||December 31,|
|Cash and cash equivalents||$||26,464||$||62,780|
|Liabilities and stockholders' equity|
|Current portion of notes payable, net of discount||3,225||3,173|
|Notes payable, net of current portion and discount||3,296||5,720|
|Total stockholders' equity||$||36,861||$||50,493|
|Catabasis Pharmaceuticals, Inc. Condensed Consolidated Statements of Cash Flows (In thousands) (Unaudited)|
|Nine Months Ended September 30,|
|Net cash used in operating activities||$||(24,874||)||$||(21,050||)|
|Net cash used in investing activities||(21,300||)||(60||)|
|Net cash provided by financing activities||9,858||79,151|
|Net (decrease) increase in cash and cash equivalents||$||(36,316||)||$||58,041|