Soleno Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full-Year 2019 Financial Results
“The recent completion of enrollment in our ongoing Phase III clinical trial, DESTINY PWS, evaluating once-daily Diazoxide Choline Controlled-Release (DCCR) tablets for patients with Prader Willi Syndrome (PWS), represents a significant achievement for our company,” said
Fourth Quarter 2019 and Recent Corporate Highlights
- Completed enrollment for Phase III DESTINY PWS study of DCCR
- Top-line data are anticipated in the first half of 2020.
- A total of 127 subjects were randomized at 29 sites in the US and the
UK . This includes additional patients who were in screening at the time target enrollment was met. - This final sample size is powered to detect a difference in change from baseline in HQ-CT score between DCCR and placebo of 4 points. The HQ-CT is a validated questionnaire to measure hyperphagia, the primary outcome measure for the Phase III study. The range of scores is 0-36, with scores above 13 typically considered to be moderate to severe hyperphagia.
- A blinded review of preliminary data found that the median baseline HQ-CT score for all randomized subjects is approximately 22. Treatment assignments (DCCR or Placebo) for all patients remain blinded.
- The duration of the open-label extension study (C602) has been increased from 12 months to up to 36 months. All subjects who have completed 12 months of open-label treatment in C602 have elected to continue to receive DCCR.
- No new safety signals associated with DCCR have been identified to date and no serious, unexpected adverse events related to DCCR have been reported.
- The Data Safety Monitoring Board (DSMB) for the Phase III DESTINY PWS recommended, for the second time, the continuation of the trial without modification
- The outcome of this second planned meeting was based on the review of data from more than 50% of subjects enrolled and treated.
- Closed an underwritten public offering of 12,841,667 shares of common stock, including 1,675,000 shares sold upon full exercise of the underwriters’ option to purchase additional shares
- Net proceeds from the offering was approximately
$14.5 million .
- Net proceeds from the offering was approximately
Financial Results
Soleno’s current research and development efforts are primarily focused on advancing its lead product candidate, DCCR for the treatment of PWS, through late-stage clinical development.
Fourth Quarter Ended
Research and development expenses were
General and administrative expense was
Total other income (expense) of (
Net loss for the quarter ended
Year Ended
Research and development expenses were
General and administrative expense was
The fair value of contingent consideration results from Soleno’s obligation to make cash payments to
Total other income (expense) of (
Net loss for the year ended
As of
About PWS
About the DESTINY PWS Trial
DESTINY PWS is a randomized, double-blind, placebo-controlled study of once-daily oral administration of DCCR versus placebo in 127 randomized subjects. Patients who complete DESTINY PWS have the option to enroll into an open-label extension study (C602) and continue treatment with DCCR.
For further information about DESTINY PWS (NCT03440814), please visit: www.clinicaltrials.gov.
About Diazoxide Choline Controlled-Release (DCCR) Tablet
Diazoxide Choline Controlled-Release tablet is a novel, proprietary extended-release, crystalline salt formulation of diazoxide, which is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of patients in a few rare diseases in neonates, infants, children and adults, but has not been approved for use in PWS. Soleno conceived of and established extensive patent protection on the therapeutic use of diazoxide and DCCR in patients with PWS. The DCCR development program is supported by data from five completed Phase I clinical studies in healthy volunteers and three completed Phase II clinical studies, one of which was in PWS patients. In the PWS Phase II study, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and abnormal lipid profiles.
About
Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company’s lead candidate, Diazoxide Choline Controlled Release (DCCR) tablets, a once-daily oral tablet for the treatment of Prader-Willi Syndrome (PWS), is currently being evaluated in a Phase III clinical development program. For more information, please visit www.soleno.life.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the Company’s expectations concerning, among other things, our ability to receive top-line data in the first half of 2020 from Phase III DESTINY PWS. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including the risks and uncertainties associated with market conditions, as well as risks and uncertainties inherent in Soleno’s business, including those described in the company's prior press releases and in the periodic reports it files with the
Corporate Contact:
212-915-2578
Condensed Consolidated Balance Sheets
(In thousands except share and per share data)
2019 |
2018 |
|||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 20,733 | $ | 23,099 | ||||
| Prepaid expenses and other current assets | 411 | 529 | ||||||
| Due from related party | — | 64 | ||||||
| Minority interest investment in former subsidiary | — | 978 | ||||||
| Total current assets | 21,144 | 24,670 | ||||||
| Long-term assets | ||||||||
| Property and equipment, net | 22 | 12 | ||||||
| Operating lease right-of-use assets | 398 | — | ||||||
| Finance lease right-of-use assets | 24 | — | ||||||
| Intangible assets, net | 16,525 | 18,469 | ||||||
| Other long-term assets | 59 | — | ||||||
| Total assets | $ | 38,172 | $ | 43,151 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 1,995 | $ | 934 | ||||
| Accrued compensation | 283 | 274 | ||||||
| Accrued clinical trial site costs | 1,999 | 320 | ||||||
| Operating lease liabilities | 305 | — | ||||||
| Other current liabilities | 382 | 349 | ||||||
| Total current liabilities | 4,964 | 1,877 | ||||||
| Long-term liabilities | ||||||||
| Series A warrant liability | — | 49 | ||||||
| 2017 PIPE Warrant liability | 10,822 | 4,563 | ||||||
| 2018 PIPE Warrant liability | 1,354 | 600 | ||||||
| Contingent liability for |
5,938 | 5,649 | ||||||
| Other long-term liabilities | 147 | — | ||||||
| Total liabilities | 23,225 | 12,738 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity | ||||||||
| Preferred Stock, |
||||||||
| Series B convertible preferred stock, 13,780 shares designated at and outstanding at Liquidation value of zero. |
— | — | ||||||
| Common stock, 44,658,054 and 31,755,169 shares issued and outstanding at |
45 | 32 | ||||||
| Additional paid-in-capital | 172,708 | 157,413 | ||||||
| Accumulated deficit | (157,806 | ) | (127,032 | ) | ||||
| Total stockholders’ equity | 14,947 | 30,413 | ||||||
| Total liabilities and stockholders’ equity | $ | 38,172 | $ | 43,151 | ||||
Condensed Consolidated Statements of Operations
(In thousands except share and per share data)
| For the Three Months Ended |
For the Years Ended |
|||||||||||||||||||
| 2019 | 2018 | 2019 | 2018 | |||||||||||||||||
| Operating expenses | ||||||||||||||||||||
| Research and development | $ | 5,272 | $ | 2,192 | $ | 16,267 | $ | 7,178 | ||||||||||||
| General and administrative | 1,608 | 1,365 | 6,930 | 6,556 | ||||||||||||||||
| Change in fair value of contingent consideration | (128 | ) | (22 | ) | 289 | 567 | ||||||||||||||
| Total operating expenses | 6,752 | 3,535 | 23,486 | 14,301 | ||||||||||||||||
| Operating loss | (6,752 | ) | (3,535 | ) | (23,486 | ) | (14,301 | ) | ||||||||||||
| Other (expense) income | ||||||||||||||||||||
| Change in fair value of warrants liabilities | (7,894 | ) | 2,071 | (6,964 | ) | 522 | ||||||||||||||
| Gain on deconsolidation of former subsidiary | — | 1,994 | — | 1,994 | ||||||||||||||||
| Loss from minority interest investment | — | — | (478 | ) | (160 | ) | ||||||||||||||
| Interest and other income | 21 | (137 | ) | 154 | 104 | |||||||||||||||
| Total other (expense) income | (7,873 | ) | 3,928 | (7,288 | ) | 2,460 | ||||||||||||||
| Loss from continuing operations | (14,625 | ) | 393 | (30,774 | ) | (11,841 | ) | |||||||||||||
| Loss from discontinued operations | — | (130 | ) | — | (1,494 | ) | ||||||||||||||
| Net loss | $ | (14,625 | ) | $ | 263 | $ | (30,774 | ) | $ | (13,335 | ) | |||||||||
| Loss per common share from continuing operations, basic and diluted | $ | (0.36 | ) | $ | 0.02 | $ | (0.90 | ) | $ | (0.56 | ) | |||||||||
| Loss per common share from discontinued operations, basic and diluted | — | (0.01 | ) | — | (0.07 | ) | ||||||||||||||
| Net loss per common share, basic and diluted | $ | (0.36 | ) | $ | 0.01 | $ | (0.90 | ) | $ | (0.64 | ) | |||||||||
| Weighted-average common shares outstanding used to calculate basic and diluted net loss per common share |
41,165,960 | 22,555,421 | 34,142,478 | 20,975,479 | ||||||||||||||||
Source: Soleno Therapeutics