CATB Catabasis Pharmaceuticals Inc

-- Enrollment Complete for MoveDMD® Phase 2 Trial of Edasalonexent, a Potential Disease-Modifying Treatment for Duchenne Muscular Dystrophy (DMD) --

-- Expanding Rare Disease Pipeline with CAT-5571, a Potential Treatment for Cystic Fibrosis --

-- Investor Day Scheduled for November 17 --

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced financial results for the third quarter ended September 30, 2016 and corporate highlights.

“Catabasis is executing on its mission to bring hope and life-changing therapies to patients and their families suffering from rare diseases,” said Jill C. Milne, Chief Executive Officer of Catabasis. “We completed enrollment in the Phase 2 portion of the MoveDMD® clinical trial in October and expect to report top-line safety and efficacy results from the placebo-controlled portion of the trial in the first half of Q1 2017; we expect to generate a rich and informative data set in DMD based on the design of the MoveDMD trial including the 36-week open-label extension. We believe in the potential of edasalonexent as monotherapy for the treatment of DMD and also believe that a combination drug approach may be able to offer additional benefits to patients. In addition to our work in DMD, we are evaluating other diseases where the inhibition of NF-kB may be beneficial. There are a number of other rare diseases where NF-kB plays an important role and edasalonexent could have the potential to positively impact patients affected by these diseases. Further, Catabasis recently expanded its rare disease pipeline by adding CAT-5571, an activator of autophagy, as a potential treatment of cystic fibrosis.”

Recent and Upcoming Corporate Highlights

Enrollment Complete for MoveDMD Phase 2 Trial of Edasalonexent (CAT-1004)

• 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.

Additional Rare Disease Programs

• 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.

Corporate

• 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.

Third Quarter 2016 Financial Results

Cash Position: At September 30, 2016, Catabasis had cash, cash equivalents and marketable securities of $47.3 million, compared to $42.8 million as of June 30, 2016. We expect that our cash, cash equivalents and marketable securities at September 30, 2016 will enable us to fund our operating expenses and capital expenditure requirements through at least September 30, 2017. Net cash used in operating activities for the three months ended September 30, 2016 was $6.9 million, compared to $7.8 million for the three months ended September 30, 2015. Net cash used in operating activities for the nine months ended September 30, 2016 was $24.9 million, compared to $21.1 million for the nine months ended September 30, 2015.

R&D Expenses: Research and development expenses were $5.9 million for the three months ended September 30, 2016, compared to $5.8 million for the three months ended September 30, 2015 and $19.2 million for the nine months ended September 30, 2016, compared to $16.4 million for the nine months ended September 30, 2015. The increase in research and development expenses for the 2016 periods relative to the 2015 periods was primarily attributable to increased direct program costs related to the edasalonexent MoveDMD trial.

G&A Expenses: General and administrative expenses were $2.3 million for the three months ended September 30, 2016, compared to $2.4 million for the three months ended September 30, 2015 and $7.7 million for the nine months ended September 30, 2016, compared to $6.0 million for the nine months ended September 30, 2015. The increase in general and administrative expenses for the first three quarters of 2016 relative to the prior year period was primarily attributable to increased employee compensation costs and increased consulting and professional expenses to support our R&D pipeline and overall growth.

Operating Loss: Loss from operations was $8.3 million for the three months ended September 30, 2016, compared to $8.2 million for the three months ended September 30, 2015, and $26.9 million for the nine months ended September 30, 2016, compared to $22.3 million for the nine months ended September 30, 2015.

Net Loss: Net loss was $8.4 million, or $0.54 per share, for the three months ended September 30, 2016, compared to a net loss of $8.5 million for the three months ended September 30, 2015. Net loss for the nine months ended September 30, 2016 was $27.3 million, compared to $23.0 million for the nine months ended September 30, 2015.

Conference Call and WebcastCatabasis will host a conference call and webcast at 4:30 pm ET today to provide an update on corporate developments and to discuss third quarter 2016 financial results.

Participant Toll-Free Dial-In Number:       (877) 388-2733 Participant International Dial-In Number: (541) 797-2984 Pass Code: 99833659

Please specify to the operator that you would like to join the “Catabasis Third Quarter 2016 Results Call.”

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,   2016     2015     2016     2015     Operating expenses: 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 expense (199 ) (282 ) (662 ) (709 ) Interest and investment income 50 - 183 - Other income, net   13     (2 )   82     11   Total other expense, net   (136 )   (284 )   (397 )   (698 ) Net loss $ (8,419 ) $ (8,485 ) $ (27,282 ) $ (23,024 ) 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, 2016 2015 Assets Cash and cash equivalents $ 26,464 $ 62,780 Available-for-sale securities 20,803 - Total assets 48,940 64,169

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 liabilities 12,079 13,676 Total stockholders’ equity $ 36,861 $ 50,493    

Catabasis Pharmaceuticals, Inc.

Condensed Consolidated Statements of Cash Flows

(In thousands)

(Unaudited)

  Nine Months Ended September 30,   2016           2015   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    

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Catabasis Pharmaceuticals, Inc.Andrea Matthews, 617-349-1971amatthews@catabasis.com