RENEO PHARMACEUTICALS, INC. “The second quarter of 2021 was highly productive for the Reneo team as we launched the international STRIDE study, completed enrollment of the McArdle disease study, and made substantial progress in our long-chain fatty acid oxidation disorders studies,” said
Key Operational Highlights
- Dosed the first patient in the STRIDE study, a global, randomized, double-blind, placebo-controlled Phase 2b clinical trial of REN001 in patients with primary mitochondrial myopathies (PMM)
- Completed enrollment of study REN001-103, an open-label Phase 1b clinical trial in patients with glycogen storage disease type V (McArdle disease)
- Dosed 20 patients to date in study REN001-102, an open-label Phase 1b clinical trial in patients with long-chain fatty acid oxidation disorder (LC-FAOD)
- Enrolled 18 patients to date in the FORWARD study, an observational clinical trial in patients with LC-FAOD
- The company was added to the Russell 2000® and Russell 3000® Indices
Financial Results for the Three Months Ended
The company reported a net loss of
Research and development expenses for the three months ended
General and administrative expenses for the three months ended
Anticipated Upcoming Milestones
- Results from the Phase 1b clinical trial in McArdle disease (1Q 2022)
- Results from the Phase 1b clinical trial in LC-FAOD (1H 2022)
- Results from the FORWARD study (2H 2022)
Corporate Access Events
- Citi’s 16th Annual
BioPharma Conference ,September 8-10, 2021 H.C. Wainwright 23rd AnnualGlobal Investment Conference ,September 13-15, 2021 Baird Global Healthcare Conference ,September 14-15, 2021 - SVB Leerink Neuromuscular, Rare Diseases & Genetic Medicines Event,
September 22-23, 2021 Cantor Global Healthcare Conference ,September 27-30, 2021
About Reneo Pharmaceuticals
Reneo is a clinical stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, which are often associated with the inability of mitochondria to produce adenosine triphosphate (ATP). Reneo is developing REN001 to modulate genes critical to metabolism and generation of ATP, which is the primary source of energy for cellular processes. REN001 has been shown to increase transcription of genes involved in mitochondrial function and increase fatty acid oxidation, and may increase production of new mitochondria.
About REN001
REN001 is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in clinical development for three rare genetic mitochondrial diseases that typically present with myopathy and have high unmet medical needs: primary mitochondrial myopathies (PMM), long-chain fatty acid oxidation disorders (LC-FAOD), and glycogen storage disorder type V (McArdle disease). For more information on REN001 clinical trials, please see clinicaltrials.gov.
About PMM
PMM are a group of disorders that affect roughly 1 in 5,000 people worldwide. PMM are caused by genetic mutations in the mitochondrial or nuclear DNA that reduce the ability of mitochondria to produce energy from nutrient sources. This energy deficit particularly affects tissues and organs with high energy demand such as muscle, brain, and heart. The symptoms of PMM include muscle weakness or exercise intolerance, movement disorder, deafness, blindness, and droopy eyelids among others. The prognosis for these disorders ranges in severity from progressive weakness to death.
About STRIDE
STRIDE is a global, randomized, double-blind, placebo-controlled Phase 2b clinical trial designed to assess the efficacy and safety of 100 mg REN001 administered orally once daily to patients with PMM. Approximately 200 adult PMM patients with alterations in mitochondrial DNA and a history of myopathy are expected to be enrolled into STRIDE. The primary efficacy endpoint of the trial is the change from baseline in the distance walked during a 12-minute walk test after 24 weeks of treatment. Secondary efficacy endpoints include changes from baseline in the Modified Fatigue Impact Scale (MFIS), Patient Global Impression of Change scale (PGIC), and other patient-reported outcomes.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the results, conduct, progress and timing of Reneo’s clinical trials, the regulatory approval path for REN001 and uses of capital. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “plans,” “will,” “believes,” “anticipates,” “expects,” “intends,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Reneo’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Reneo’s business in general, and the other risks described in Reneo’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Reneo undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands, except share and par value data)
| 2021 | 2020 | |||||||
| Assets | (unaudited) | |||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 140,368 | $ | 53,613 | ||||
| Short-term investments | 26,963 | — | ||||||
| Prepaid expenses and other current assets | 3,810 | 1,412 | ||||||
| Total current assets | 171,141 | 55,025 | ||||||
| Property and equipment, net | 85 | 69 | ||||||
| Other non-current assets | 78 | 127 | ||||||
| Total assets | $ | 171,304 | $ | 55,221 | ||||
| Liabilities, convertible preferred stock and stockholders’ equity (deficit) | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 434 | $ | 908 | ||||
| Accrued expenses | 2,902 | 3,672 | ||||||
| Total current liabilities | 3,336 | 4,580 | ||||||
| Deferred rent | 54 | 36 | ||||||
| Performance Award | 363 | — | ||||||
| Total liabilities | 3,753 | 4,616 | ||||||
| Commitments and contingencies | ||||||||
| Series A convertible preferred stock, | — | 45,652 | ||||||
| Series B convertible preferred stock, | — | 47,068 | ||||||
| Stockholders’ equity (deficit): | ||||||||
| Preferred stock, | — | — | ||||||
| Common stock, | 3 | — | ||||||
| Additional paid-in capital | 228,929 | 2,843 | ||||||
| Accumulated deficit | (61,386 | ) | (44,958 | ) | ||||
| Accumulated other comprehensive income | 5 | — | ||||||
| Total stockholders’ equity (deficit) | 167,551 | (42,115 | ) | |||||
| Total liabilities, convertible preferred stock and stockholders’ equity (deficit) | $ | 171,304 | $ | 55,221 | ||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(Unaudited)
(in thousands, except share and per share data)
| THREE MONTHS ENDED | SIX MONTHS ENDED | ||||||||||||||||
| 2021 | 2020 | 2021 | 2020 | ||||||||||||||
| Operating expenses: | |||||||||||||||||
| Research and development | $ | 6,279 | $ | 2,817 | $ | 11,751 | $ | 6,395 | |||||||||
| General and administrative | 2,949 | 882 | 4,691 | 1,807 | |||||||||||||
| Total operating expenses | 9,228 | 3,699 | 16,442 | 8,202 | |||||||||||||
| Loss from operations | (9,228 | ) | (3,699 | ) | (16,442 | ) | (8,202 | ) | |||||||||
| Other income | 12 | 12 | 14 | 84 | |||||||||||||
| Net loss | (9,216 | ) | (3,687 | ) | (16,428 | ) | (8,118 | ) | |||||||||
| Unrealized gain (loss) on short-term investments | 5 | (6 | ) | 5 | 1 | ||||||||||||
| Comprehensive loss | $ | (9,211 | ) | $ | (3,693 | ) | $ | (16,423 | ) | $ | (8,117 | ) | |||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.43 | ) | $ | (1.82 | ) | $ | (1.40 | ) | $ | (4.02 | ) | |||||
| Weighted-average shares used in computing net loss per share, basic and diluted | 21,364,369 | 2,022,174 | 11,770,948 | 2,018,602 | |||||||||||||
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(Unaudited)
(in thousands)
| SIX MONTHS ENDED | ||||||||
| 2021 | 2020 | |||||||
| Cash flows from operating activities | ||||||||
| Net loss | $ | (16,428 | ) | $ | (8,118 | ) | ||
| Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||
| Depreciation and amortization | 20 | 18 | ||||||
| Amortization/accretion on short-term investments | 33 | (16 | ) | |||||
| Changes in the fair value of Performance Award | 363 | — | ||||||
| Stock-based compensation | 1,325 | 180 | ||||||
| Changes in operating assets and liabilities: | ||||||||
| Accounts payable, accrued expenses and other | (1,164 | ) | 934 | |||||
| Prepaid expenses and other assets | (2,457 | ) | (380 | ) | ||||
| Deferred rent | 18 | (2 | ) | |||||
| Net cash used in operating activities | (18,290 | ) | (7,384 | ) | ||||
| Cash flows from investing activities | ||||||||
| Purchases of property and equipment | (31 | ) | (1 | ) | ||||
| Purchase of available-for-sale short-term investments | (26,989 | ) | — | |||||
| Proceeds from maturities of available-for-sale short-term investments | — | 6,200 | ||||||
| Net cash (used in) provided by investing activities | (27,020 | ) | 6,199 | |||||
| Cash flows from financing activities | ||||||||
| Proceeds from issuance of Series B convertible preferred stock, net of issuance costs | 47,239 | — | ||||||
| Proceeds from initial public offering, net of offering costs | 84,639 | — | ||||||
| Proceeds from exercise of stock options | 187 | 26 | ||||||
| Net cash provided by financing activities | 132,065 | 26 | ||||||
| Net increase (decrease) in cash and cash equivalents | 86,755 | (1,159 | ) | |||||
| Cash and cash equivalents, beginning of period | 53,613 | 17,501 | ||||||
| Cash and cash equivalents, end of period | $ | 140,368 | $ | 16,342 | ||||
| Supplemental cash flow information: | ||||||||
| Property and equipment in accounts payable | $ | 10 | $ | — | ||||
| Costs incurred in connection with initial public offering included in accrued expenses | $ | 30 | $ | — | ||||
Contacts:
| Joyce Allaire Managing Director jallaire@lifesciadvisors.com | Chief Financial Officer vjindal@reneopharma.com |
2021 GlobeNewswire, Inc., source
