Our new profile is- Sophiris Bio, Inc. (NASDAQ:SPHS)

 

Earnings due out possibly today, although the potential for their drug pipeline out weighs current earnings

Recent news

Sophiris Bio Reports Encouraging Preliminary Data from Phase 2a Proof of Concept Study in Localized Prostate Cancer

http://www.nasdaq.com/press-release/sophiris-bio-reports-encouraging-preliminary-data-from-phase-2a-proof-of-concept-study-in-localize-20160128-01088

 

Analyst strong Buy-  http://www.nasdaq.com/symbol/sphs/recommendations

Wall Street analysts covering shares of Sophiris Bio, Inc. (NASDAQ:SPHS) have recently chimed in on the stock. Compiling all of the price targets issued by equity research firms have yielded a consensus target of $5.00/share. This is according to First Call data compiled by Thomson Reuters.

Past and future earnings growth are also important indicators in determining where the stock might be headed. Analysts are predicting $0.0000 per share in earnings next quarter and $-0.9500 for the current year. In comparing the current price level of the equity to their moving averages, the shares are trading -0.0913 off of the 50-day average of $1.8006 and 0.1963 away from the 200-day moving average of $1.5130.

Another important factor to consider when evaluating a stock’s current and future value are the 52 week high and low levels. As of the most recent bid at the time of writing, Sophiris Bio, Inc. shares are trading -55.6026% away from the 52-week high mark of 3.8500 and +256.1042% off the 52-week bottom of 0.4800.

Taking a step further, investors might also consider evaluating a firm’s PEG ratio, which illustrates the relationship between stock price, earning per share, and the company’s growth rate. A firm with a PEG ratio below 1 is considered undervalued. A company with a PEG ratio around 1 is considered fairly valued. A company with a PEG ratio greater than 1 is considered overvalued. The stock has a current PEG of 0.0000.

 

Consensus from NASDAQ.com

http://www.nasdaq.com/symbol/sphs/analyst-research

 

 

 

 

Chart- http://stockcharts.com/h-sc/ui?s=sphs

 

Sources: wall street analyst, uptick analyst, Broad street Alerts, Nasdaq.com

 

 

 

Disclaimer

SCS LLC and broad street alerts have not been compensated for the mention of SPHS and we do not hold any positions.

– Builds out infrastructure in advance of April 15, 2016 PDUFA target date –

– EMA accepts Phase 3 trial design –

– Conference call scheduled for 5 p.m. ET today –

NEWTON, Mass., Nov. 12, 2015 (GLOBE NEWSWIRE) — Chiasma, Inc. (NASDAQ:CHMA), a late-stage biopharmaceutical company developing Mycapssa™ (octreotide capsules), an investigational oral drug for the maintenance therapy of adult patients with the orphan disease acromegaly, today provided a corporate update and reported financial results for the third quarter ended September 30, 2015.

“Chiasma achieved a series of important milestones in the third quarter that strengthened our foundation for the potential 2016 U.S. launch of Mycapssa™, which, if approved, would be the first oral somatostatin analog available to acromegaly patients,” said Mark Leuchtenberger, Chief Executive Officer of Chiasma. “We began the quarter by completing an upsized initial public offering, which significantly bolstered our balance sheet, and we also continued to build out our infrastructure by making a number of strategic hires in our commercial, medical and development groups.

“We made significant progress with our lead program in both the U.S. and European Union. Our first New Drug Application (NDA) for Mycapssa™ in acromegaly was accepted for filing by the FDA in August, and our Prescription Drug User Fee Act (PDUFA) target date was set for April 15, 2016,” Mr. Leuchtenberger continued. “The European Medicines Agency (EMA) also just recently accepted the protocol for our Phase 3 acromegaly trial of octreotide capsules, which we now expect to initiate by early in the second quarter of 2016. In addition, we have continued to explore additional indications that we might address utilizing Mycapssa™ and our Transient Permeability Enhancer (TPE^®) platform.”

Third Quarter and Recent Highlights:

• Finalized EU Phase 3 trial design: In October, the EMA accepted the design, enrollment criteria and required duration of Chiasma’s Phase 3 trial to evaluate the non-inferiority of oral octreotide to injectable somatostatin analogs. This is an open-label, randomized, active-controlled study that is anticipated to include approximately 150 acromegaly patients in the European Union, the United States and certain other countries.
• Announced FDA acceptance of NDA filing for Mycapssa™: In August 2015, the FDA accepted for filing Chiasma’s NDA for Mycapssa™ for the proposed maintenance therapy of adult patients with acromegaly, an orphan indication. The application is supported by the results from a multicenter Phase 3 study, which evaluated patients for biochemical and symptomatic disease control over a period of up to 13 months during treatment with Mycapssa™.
• Successfully completed initial public offering (IPO): In July 2015, Chiasma completed its IPO of common stock at a price of $16.00 per share. Including the underwriters’ full exercise of their overallotment option, Chiasma issued 7,319,750 shares of its common stock and netted proceeds of approximately $106.5 million.
• Continued to expand and strengthen the leadership team: Chiasma made several key hires during the third quarter as it prepares for the potential commercialization of Mycapssa™. New additions to the team include Anand Varadan as Chief Commercial Officer and William H. Ludlam, M.D., Ph.D., as Vice President of Medical Affairs. Mr. Varadan joined Chiasma from Amgen Inc., where he most recently served as Vice President of Marketing for their U.S. Inflammation and Nephrology Business Unit. Dr. Ludlam came to Chiasma from Novartis Pharmaceuticals Corporation, where he served most recently as Senior Medical Director for U.S. Clinical Development and Medical Affairs, Pituitary Disorders, Rare Diseases Franchise.

Third Quarter 2015 Financial Results:

• R&D Expenses: Research and development expenses were $4.4 million for the quarter ended September 30, 2015, compared to $1.7 million for the same period of 2014. The increase was primarily due to the filing of an NDA for Mycapssa™ in acromegaly in the United States and related activities, expenses associated with the manufacturing process validation, activities related to our planned additional Phase 3 clinical trial for the treatment of acromegaly using Mycapssa™ in Europe and an increase in research and development headcount.
• Marketing, G&A Expenses: Marketing, general and administrative expenses were $4.8 million for the quarter ended September 30, 2015, compared to $13,000 for the same period of 2014. The increase was largely due to the initiation of pre-commercial activities related to Mycapssa™, increased consulting costs, compensation-related expenses associated with the hiring of senior executives and other administrative employees, as well as increased legal fees related to intellectual property protection.
• Net Loss: For the quarter ended September 30, 2015, Chiasma’s net loss attributable to common stockholders was ($9.4) million, or ($0.46) per basic share, compared to net income attributable to common stockholders of $0.6 million, or $12.47 per basic share, for the same period of 2014. The basic share amounts outstanding for the quarter ended September 30, 2015 included the shares of common stock that were issued upon conversion of Chiasma’s redeemable preferred stock in connection with theJuly 2015 IPO; redeemable preferred stock outstanding as of September 30, 2014 was excluded from this calculation in periods before the IPO.
• Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2015 were $162.8 million, compared to $63.3 million as of June 30, 2015. This increase is the result of Chiasma’s successful IPO in July 2015.

2015 Financial Guidance

Chiasma expects its total cash, cash equivalents and marketable securities to be between $145 million and $150 million at December 31, 2015.

Conference Call Reminder

At 5:00 p.m. Eastern Time today, Chiasma’s senior management team will host a conference call to discuss the company’s financial results and other business updates. Investors can access a live and archived webcast of this call via the News & Investors section of the company’s website. Individuals may also participate in the live call via telephone by dialing (877) 317-6789 (domestic) or (412) 317-6789 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10075383.

About Acromegaly

Acromegaly typically develops when a benign tumor of the pituitary gland produces too much growth hormone (GH), ultimately leading to significant health problems and early death if untreated. Common features of acromegaly are facial changes, intense headaches, joint pain, impaired vision and enlargement of the hands, feet, tongue and internal organs. Serious health conditions associated with the progression of acromegaly include type 2 diabetes, hypertension, respiratory disorders and cardiac and cerebrovascular disease.

Current treatment options include surgery to remove the pituitary tumor, radiation therapy which destroys any lingering tumor cells and/or medical treatment in cases where these approaches are not possible or fully effective. Today’s medical treatments include dopamine agonists, GH antagonists and injectable somatostatin analogs, the current standard of care. Currently available somatostatin analogs require large needles for injections into the muscle (octreotide) or deep into the tissue underlying the skin (lanreotide).

According to the U.S. National Institutes of Health, acromegaly occurs in approximately 60 people per million in the U.S. In addition, researchers estimate that three to four out of every million people develop acromegaly each year. Because symptoms often develop slowly, diagnosis may be delayed by years or decades, making it difficult to determine the total number of people with the disease.

About Mycapssa™ (octreotide capsules)

Mycapssa™ (octreotide capsules) is an oral drug proposed for the maintenance therapy of adult patients with acromegaly. If approved, octreotide capsules would be the first oral somatostatin analog approved for acromegaly. Chiasma filed a NDA on June 15, 2015 for this product candidate. The FDA has accepted the NDA for filing and the PDUFA date is April 15, 2016. The PDUFA date is the target date for the FDA to complete its review of the NDA. Octreotide capsules have been granted orphan designation in the United States and the European Union for the treatment of acromegaly.

Octreotide capsules are an investigational drug that have not been approved for use in any jurisdiction. The trade name Mycapssa™ has been conditionally accepted by the FDA as the proprietary name for Chiasma’s octreotide capsules product candidate.

About Chiasma

Chiasma is a late-stage biopharmaceutical company focused on improving the lives of patients suffering from orphan diseases by developing and commercializing novel oral forms of therapies that are available today only by injection. The company’s lead product candidate is Mycapssa™ (octreotide capsules), a new investigational drug for the orphan condition acromegaly, developed with Chiasma’s Transient Permeability Enhancer (TPE^®) technology to facilitate gastrointestinal absorption of unmodified drug into the bloodstream safely. Mycapssa™ has not been approved for use in any jurisdiction. Using TPE^® technology, Chiasma is evaluating additional proteins, peptides and small molecule drugs that are currently only available by injection, but could potentially be converted to oral delivery. TPE^® technology is potentially well suited for drugs with chronic indications, where frequent dosing is required and the need for an oral alternative is greatest. Chiasma is a Delaware corporation with a wholly owned Israeli subsidiary.

 

Forward-Looking Statements:

This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the safety, efficacy and potential clinical benefits ofMycapssa™, the timing of the FDA’s review of Chiasma’s NDA, the timing of the potential commercial launch ofMycapssa™, the timing of initiation of Chiasma’s second Phase 3 trial to support approval by the EMA and the financial guidance regarding cash, cash equivalents and marketable securities at December 31, 2015. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks associated with the regulatory review process generally; the risk that the FDA may not make a decision on Chiasma’s NDA by the PDUFA target date; the risk that the FDAmay determine that that the data included in the NDA are insufficient for approval and that we must conduct additional clinical trials, or nonclinical or other studies before oral octreotide can be approved; the risk that the results of previously conducted studies involving oral octreotide or other product candidates will not be repeated or observed in ongoing or future studies or following commercial launch, if such product candidates are approved; risks associated with obtaining, maintaining and protecting intellectual property; risks associated with Chiasma’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties; the risk that oral octreotide, if approved, will not be successfully commercialized; the risk of competition from currently approved therapies and from other companies developing products for similar uses; risk associated with Chiasma’s ability to manage operating expenses and/or obtain additional funding to support its business activities; and risks associated with Chiasma’s dependence on third parties, including with respect to the manufacture of commercial supply in anticipation of commercial launch, if oral octreotide is approved. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in the final prospectus related to Chiasma’s initial public offering filed with the Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act of 1933, as amended, as well as discussions of potential risks, uncertainties and other important factors in Chiasma’s subsequent filings with the Securities and Exchange Commission, including our most recent Quarterly Report on Form 10-Q filed on August 31, 2015. All information in this press release is as of the date of the release, and Chiasma undertakes no duty to update this information unless required by law.

Chiasma, Inc.
Condensed Consolidated Statements of Operations
(unaudited)
	Three Months Ended September 30,		Nine Months Ended September 30,
	2015	2014	2015	2014
Revenue from license agreement	$ —	$ 2,604,469	$ —	$ 13,165,795
Operating expenses:
Research and development	4,367,992	1,723,803	10,745,527	4,573,881
Marketing, general and administrative	4,813,658	13,013	10,181,411	1,514,708
Total operating expenses	9,181,650	1,736,816	20,926,938	6,088,589
Income (loss) from operations	(9,181,650)	867,653	(20,926,938)	7,077,206
Other expense (income), net	105,395	(61,933)	303,673	2,701
Income (loss) before provision for income taxes	(9,287,045)	929,586	(21,230,611)	7,074,505
Provision for income taxes	71,540	168,237	145,011	233,339
Net income (loss)	(9,358,585)	761,349	(21,375,622)	6,841,166
Accretion of redeemable convertible preferred stock	(31,226)	(208,741)	(317,801)	(896,559)
Net income (loss) attributable to common stockholders	$ (9,389,811)	$ 552,608	$ (21,693,423)	$ 5,944,607
Net income (loss) per share attributable to common stockholders – basic	$ (0.46)	$ 12.47	$ (3.17)	$ 135.37
Weighted-average common shares outstanding – basic	20,280,385	44,326	6,837,272	43,912
Net income (loss) per share attributable to common stockholders – diluted	$ (0.46)	$ 0.07	$ (3.17)	$ 0.61
Weighted-average common shares outstanding – diluted	20,280,385	11,082,922	6,837,272	11,146,273

Chiasma, Inc.
Condensed Consolidated Balance Sheets Information
(unaudited)
	September 30, 2015	December 31, 2014
Cash and cash equivalents	$ 140,869,221	$ 40,160,435
Marketable securities	21,976,790	—
Prepaid expenses and other current assets	1,400,836	311,299
Property and equipment, net	573,539	615,242
Other assets	404,476	311,730
Total assets	$ 165,224,862	$ 41,398,706
Accounts payable and accrued liabilities	$ 4,531,045	$ 4,318,288
Current maturities of long-term liabilities	1,700,000	—
Long-term liabilities	3,652,211	4,612,450
Total liabilities	9,883,256	8,930,738
Total redeemable convertible preferred stock	—	104,485,972
Total stockholders’ equity (deficit)	155,341,606	(72,018,004)
Total liabilities, redeemable convertible preferred stock and stockholders’ equity (deficit)	$ 165,224,862	$ 41,398,706

CONTACT: Media:
         Carolyn Sobczyk
         JPA Health Communications
         (202) 591-4052
         

         Investors:
         Jason Fredette
         Chiasma, Inc.
         (617) 928-5306

Chiasma, Inc.

CUPERTINO, Calif., Feb. 29, 2016 /PRNewswire/ — DURECT Corporation (Nasdaq: DRRX) announced today financial results for the three months ended December 31, 2015. Total revenues were $5.2 million for the three months ended December 31, 2015 and $4.3 million for the three months ended December 31, 2014.  Net loss was$5.8 million for the three months ended December 31, 2015, compared to a net loss of $5.9 million for the same period in 2014.

Total revenues were $19.1 million and net loss was $22.7 million for the year endedDecember 31, 2015, compared to total revenues of $19.4 million and net loss of $22.1 million for the year ended December 31, 2014.

At December 31, 2015, we had cash and investments of $29.3 million, compared to cash and investments of $34.9 million at December 31, 2014.  At December 31, 2015, we had $19.7 million in long term debt.

“2015 was a transformative year for DURECT as our most mature programs advanced in meaningful ways and we announced and made rapid progress with our Epigenomics Regulator Program,” stated James E. Brown, D.V.M., President and CEO of DURECT.  “For DUR-928, in 2015 we completed five Phase 1 clinical trials, dosed over 75 healthy volunteers with either our oral or injectable formulations, and saw no serious or treatment-related adverse events, enabling us to start 2016 by initiating our first DUR-928 patient trial. In November 2015, we began recruiting patients in PERSIST, our POSIMIR^™ pivotal Phase 3 clinical trial which is designed to generate the data required for a resubmission of the NDA.  In addition, we understand that Pain Therapeutics is in the later stages of completing the resubmission of the REMOXY^® NDA, which should lead to a 6 month review by the FDA.”

In 2016, we look forward to:

• Completing a Phase 1b patient study with DUR-928 for patients with nonalcoholic steatohepatitis (NASH) with our oral formulation, enabling and informing subsequent patient studies
• Initiating a multi-dose study with our oral formulation of DUR-928 in patients with NASH or other liver function impairment
• Completing a Phase 1b patient study with DUR-928 for patients with renal impaired kidney function with our injectable formulation, enabling and informing subsequent patient studies
• Initiating at least one study designed to evaluate an initial indication of potential efficacy in one or more medical conditions
• Completing enrollment in the Phase 3 POSIMIR clinical trial in patients undergoing laparoscopic gall bladder removal
• Potential FDA approval for REMOXY
• Completion of the Phase 3 trial with ORADUR^®-Methylphenidate in Taiwan by our partner Orient Pharma
• Supporting Zogenix as they seek a development and commercialization partner for Relday^™
• Advancing existing feasibility projects and potentially entering into additional feasibility studies and collaborations

Highlights for DURECT in Fiscal Year 2015 and Major Potential Milestones over the Next 12-18 Months:

• Epigenomic Regulator Program. DUR-928, our Epigenomic Regulator Program’s lead product candidate, is an endogenous, small molecule, new chemical entity (NCE), which may have broad applicability in several metabolic diseases such as nonalcoholic fatty liver disease (NAFLD) and NASH, and in acute organ injuries such as acute kidney injury.

  During 2015 more than 75 healthy volunteers in our five Phase 1 studies received DUR-928 given either orally or through injection at varying doses substantially in excess of endogenous levels, with no serious treatment-related adverse events reported.

  Building on our learnings from 8 animal models that were previously reported and multiple Phase 1 trials, in January 2016 we began our first patient trial utilizing DUR-928. This study is a single-ascending-dose safety and pharmacokinetic Phase 1b trial of DUR-928 in NASH patients and matched control subjects. This study will be conducted in three successive cohorts evaluating three single-dose levels of oral DUR-928. After a PK/safety review at each dose, the study can proceed to the next higher dose. Assuming all three cohorts are dosed, the study will comprise approximately 48 subjects, of which approximately 30 will have received DUR-928. The study is being conducted inAustralia, and we anticipate that we will obtain results from this trial in the first half of 2016. This study is designed to enable and inform a subsequent multi-dose study in NASH or other chronic metabolic disease.

  We are planning to shortly commence a second study in patients with DUR-928, also to be conducted in Australia. This Phase 1b trial of DUR-928 will be a single-ascending-dose safety and pharmacokinetic study of DUR-928 in patients with impaired kidney function and matched control subjects. This study will be conducted in three successive cohorts evaluating three single-dose levels of DUR-928 administered by injection. After a PK/safety review at each dose, the study can proceed to the next higher dose. Assuming all three cohorts are dosed, the study will comprise approximately 45 subjects, of which approximately 30 will have received DUR-928. We anticipate that this study will be completed in 2016, and that this study will enable and inform subsequent trials for patients with either acute kidney injury or other kidney function impairment.

• POSIMIR (SABER^®-Bupivacaine) Post-Operative Pain Relief Depot. InNovember 2015, we began enrolling patients for PERSIST, the POSIMIR pivotal Phase 3 clinical trial. PERSIST is planned to involve slightly over 300 patients undergoing laparoscopic cholecystectomy (gallbladder removal) surgery. These patients will be randomized on a one-to-one basis to receive either POSIMIR or placebo as a one-time intra-incisional instillation at the time of surgery. In a previous clinical trial of 50 patients undergoing laparoscopic cholecystectomy, POSIMIR was compared with the active control bupivacaine hydrochloride, against which POSIMIR demonstrated an approximately 25% reduction in pain intensity on movement for the first 3 days after surgery (p=0.024), using the same statistical methodology specified for the current trial. We believe that PERSIST represents the first pivotal efficacy trial in this category in a laparoscopic procedure. We expect that it will take approximately one year to complete patient enrollment in PERSIST. This clinical trial is designed to generate additional data necessary to support an NDA resubmission.

  POSIMIR is our investigational post-operative pain relief depot that utilizes our patented SABER technology and is intended to deliver bupivacaine to provide 3 days of pain relief after surgery. We are in discussions with potential partners regarding licensing development and commercialization rights to POSIMIR, for which we hold worldwide rights. We are also continuing to evaluate the requirements for commercializing POSIMIR on our own in the U.S., in the event that we determine that to be the preferred route of commercialization.

• REMOXY (oxycodone) Extended-Release Capsules CII. Based on our ORADUR technology, REMOXY is a unique long-acting formulation of oxycodone designed to discourage common methods of tampering associated with opioid misuse and abuse. In July 2015, Pain Therapeutics (our licensee) has stated that they expect to resubmit the NDA in the first quarter of 2016. The extended release oxycodone market is ~$2.4 billion in the U.S. alone, and we are eligible for a potential royalty on REMOXY between 6.0% to 11.5% of net sales depending on sales volumes.
• Relday (Risperidone Program). Relday is a proprietary, long-acting, once-monthly subcutaneous injectable formulation of risperidone for the treatment of schizophrenia. In September 2015, Zogenix announced positive top-line results from a Phase 1b multi-dose parallel group clinical trial that enrolled 60 subjects. According to Zogenix, the trial results for Relday demonstrated that risperidone plasma concentrations in the therapeutic range were achieved on the first day of dosing, reached steady state levels following the second dose and consistently maintained therapeutic levels throughout the four-month period. Also according to Zogenix, Relday was generally safe and well-tolerated, with results consistent with the profile of risperidone and the previous Phase 1 single-dose clinical trial. Zogenix further stated that it has initiated efforts to secure a development and commercialization partner for Relday, and that Relday is well-positioned to begin a Phase 3 program once a partner is secured.
• ORADUR-ADHD Program. In 2013, we selected a formulation for the lead program in our ORADUR-ADHD (Attention Deficit Hyperactivity Disorder) program, ORADUR-Methylphenidate. This formulation was chosen based on its potential for rapid onset of action, long duration with once-a-day dosing and target pharmacokinetic profile as demonstrated in a Phase 1 trial. In addition, this product candidate utilizes a small capsule size relative to the leading existing long acting products on the market and incorporates our ORADUR anti-tampering technology. Orient Pharma, our licensee in defined Asian and South Pacific countries, has initiated a Phase 3 study in Taiwan and anticipates completing it in 2016. We retain rights to all other markets in the world, notably including the U.S., Europe and Japan, and are engaged in licensing discussions with other companies.
• Feasibility Projects and Other Activities. During the fourth quarter of 2015, we continued work on several feasibility projects and have multiple discussions underway with other parties about new feasibility projects which are designed to demonstrate that our technologies can achieve the drug delivery objectives set forth by our collaborators and are worthy of further development. The Relday program and the Santen ophthalmic program are two such projects which have matured into development and license agreements.
• Business Development Activities. We have multiple programs that may potentially be licensed over the next 12-18 months. These include POSIMIR, DUR-928, ORADUR-ADHD (territories outside certain Asian and South Pacific markets), as well as various other programs which we have not described publicly in detail.

 

About DURECT Corporation

DURECT is a biopharmaceutical company actively developing new therapeutics based on its Epigenomic Regulator Program and proprietary drug delivery platforms.  DUR‑928, a new chemical entity in Phase 1 development, is the lead candidate inDURECT’s Epigenomic Regulator Program.  An endogenous, orally bioavailable small molecule, DUR-928 plays an important regulatory role in lipid homeostasis, inflammation, and cell survival, and may have applications related to acute organ injury and chronic metabolic disease, notably nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH).  DURECT’s advanced oral, injectable, and transdermal delivery technologies enable new indications and enhanced attributes, such as abuse deterrence, extended dosing intervals, and superior safety and efficacy, for small-molecule and biologic drugs. Late-stage development programs in this category include POSIMIR^™ and REMOXY^®, addressing key unmet needs in pain management.

NOTE: POSIMIR^™, SABER^®, ORADUR^®, and TRANSDUR^® are trademarks ofDURECT Corporation. Other referenced trademarks belong to their respective owners.  REMOXY, POSIMIR, ELADUR, ORADUR-Methylphenidate, Relday and DUR-928 are drug candidates under development and have not been approved for commercialization by the U.S. Food and Drug Administration or other health authorities.

DURECT Forward-Looking Statement

The statements in this press release regarding regulatory matters, including the anticipated NDA resubmission REMOXY and potential FDA approval of REMOXY and our other product candidates, anticipated clinical trials (including timing and results) for POSIMIR, DUR-928, ORADUR-Methylphenidate and our other drug candidates, potential royalties from Pain Therapeutics, the potential benefits and uses of our drug candidates, potential markets for our product candidates, the potential license  of POSIMIR, DUR-928, ORADUR-ADHD and other products, advancing and commencing new feasibility projects, collaborations with third parties, including Pain Therapeutics’ plans for REMOXY and Zogenix’s plans for Relday, and other potential business development activities are forward-looking statements involving risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. Potential risks and uncertainties include, but are not limited to, the risk that the clinical trial of POSIMIR will take longer to conduct than anticipated or result in data that will not support a successful resubmission, the risk that Pain Therapeutics or Zogenix will discontinue development of REMOXY or Relday, respectively, or be delayed in development or regulatory submissions, the risk of adverse decisions by regulatory agencies, including requests for additional information or product non-approval or non-acceptance of our POSIMIR, REMOXY or other NDA submissions, delays and additional costs due to requirements imposed by regulatory agencies, additional time and resources that may be required for development, testing and regulatory approval of DUR-928, potential adverse effects arising from the testing or use of our drug candidates, the risk that prior clinical trials will not be confirmed in subsequent trials, the potential failure of clinical trials to meet their intended endpoints, our potential failure to maintain our collaborative agreements with third parties or consummate new collaborations and risks related to our (and our third party collaborators where applicable) ability to design, enroll, conduct and complete clinical trials, complete the design, development, and manufacturing process development of product candidates, manufacture and commercialize product candidates, obtain marketplace acceptance of product candidates, avoid infringing patents held by other parties and secure and defend patents of our own, and manage and obtain capital to fund operations and expenses. Further information regarding these and other risks is included in DURECT’s Form 10-Q on November 3, 2015 under the heading “Risk Factors.”

 

BERKELEY, CA — (Marketwired) — 03/08/16 —  Dynavax Technologies Corporation (NASDAQ: DVAX) today reported financial results for the fourth quarter and year ended December 31, 2015.

The Company had $196.1 million in cash, cash equivalents and marketable securities as of December 31, 2015.

Total revenues for the year ended December 31, 2015, decreased by $7.0 million or 63 percent compared to the same period in 2014, primarily due to a $5.2 million decrease in collaboration revenue due to winding down of work performed for the AZD1419 program and expiration of our collaboration agreement with GSK in 2014.

Operating expenses increased by $6.8 million or seven percent during 2015 compared to 2014, primarily due to costs related to HBV-23, the Phase 3 clinical study of HEPLISAV-B™ completed in October 2015, preparation for the commercial launch of HEPLISAV-B in the United States and clinical trial expense for SD-101, Dynavax’s cancer immunotherapeutic product candidate.

The net loss allocable to common stockholders for the year ended December 31, 2015 was $106.8 million, or $3.25 per share, compared to $90.7 million, or $3.45 per share for the year ended December 31, 2014.

“During 2015, we completed HBV-23 and significantly strengthened the Company’s cash position. Earlier this year we reported that this third pivotal study had met both co-primary endpoints. We plan to resubmit the HEPLISAV-B BLA (Biologics License Application) to the FDA by the end of this month. Based on our expectation of a six-month review, if our application is approved we expect to launch this product in the fourth quarter of this year,” saidEddie Gray, chief executive officer of Dynavax.

About Dynavax

Dynavax, a clinical-stage biopharmaceutical company, uses TLR biology to discover and develop novel vaccines and therapeutics in the areas of infectious and inflammatory diseases and oncology. Dynavax’s lead product candidates are HEPLISAV-B, a Phase 3 investigational adult hepatitis B vaccine, and SD-101, an investigational cancer immunotherapeutic currently in several Phase 1/2 studies.

Forward Looking Statements

This release contains forward-looking statements, including statements regarding expected timing of the HEPLISAV-B BLA resubmission, possible approval, and expected launch. These statements are subject to a number of risks and uncertainties that could cause actual results to differ materially, including whether there will be the need for additional studies, further manufacturing enhancements or other activities, or other issues will arise that will delay the BLA resubmission and/or negatively impact the acceptance, review, duration of review and approval of the BLA by the FDA; whether we will successfully launch the product, possible claims against us, including enjoining sales of HEPLISAV-B based on the patent rights of others, and the potential size and value of approved indications addressable with HEPLISAV-B; initiation and completion of pre-clinical studies and clinical trials of our other product candidates, including SD-101, in a timely manner; the results of clinical trials and the impact of those results on the initiation or continuation of subsequent trials and issues arising in the regulatory process; achieving the objectives of and maintaining our collaborative and licensing agreements; our ability to execute on our commercial strategies; whether our financial resources will be adequate without the need to obtain additional financing and other risks detailed in the “Risk Factors” section of our most recent current periodic report filed with the SEC. These statements represent our estimates and assumptions only as of the date of this release. We do not undertake any obligation to update publicly any such forward-looking statements, even if new information becomes available.

Contact:
Michael Ostrach
Chief Financial Officer
510-665-7257
Email contact

Source: Dynavax Technologies

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RADNOR, Pa., Oct. 29, 2015 (GLOBE NEWSWIRE) — Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today provided a business update on its clinical development activities and reported its financial results for the third quarter ended September 30, 2015.

Recent Highlights:

• Successful End-of-Phase 2 meeting with the United States Food and Drug Administration (FDA) for ganaxolone in focal onset seizures
• Initiated clinical phase of ganaxolone intravenous (IV) clinical program in status epilepticus (SE)
• Robust efficacy in benzodiazepine-resistant SE animal models seen with Ganaxolone IV
• Preclinical data further supports clinical evidence that ganaxolone does not activate the human progesterone receptor
• Announced interim observations from exploratory Phase 2 proof-of-concept trial in PCDH19 female pediatric epilepsy

“Marinus has made significant progress advancing our ganaxolone programs,” commented Christopher M. Cashman, Chief Executive Officer of Marinus Pharmaceuticals. “Of particular note is the addition of our ganaxolone intravenous formulation to our development portfolio. The GLP safety and tolerability studies with a clean safety profile coupled with robust efficacy observed in benzodiazepine-resistant SE animal models support the advancement of ganaxolone IV into the clinic. We are excited about the potential for ganaxolone IV in the acute care setting which, together with our convenient oral capsule and liquid formulations, provides a continuity of care for patients suffering from drug-resistant seizures.”

Business Update

Ganaxolone is a CNS-selective GABA_A modulator being developed in three different dose forms (IV, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Ganaxolone acts on a well-characterized target in the brain known to have both anti-seizure and anti-anxiety effects.

Ganaxolone IV — Status Epilepticus (Orphan Indication):

Marinus recently announced the initiation of the clinical phase of its ganaxolone IV program and its initial target orphan indication in SE. SE is a life-threatening condition, which, if not arrested immediately, can result in marked neuronal damage, cognitive impairment and death.  Data from preclinical studies yielded positive results testing ganaxolone IV in benzodiazepine-resistant SE. Two separate laboratories using different measurements tested ganaxolone IV in clinically translatable rodent models of SE and in both studies ganaxolone IV promoted survival and showed better or comparable reversal of seizures than the endogenous neurosteroid allopregnanolone.

Marinus plans to commence a Phase 1 clinical trial designed to evaluate the safety, tolerability and pharmacokinetics of ganaxolone IV in the first half 2016 followed by initiation of a clinical trial in SE patients later next year.

Ganaxolone IV for the hospital setting offers a new mechanism of action for the treatment of SE that is complemented by its oral dose forms, providing the potential for IV-to-oral continuation therapy as patients transition from acute care to outpatient settings.

Ganaxolone Capsule — Refractory Focal Onset Seizures:

Marinus has focused most of its recent clinical development efforts on advancing its outpatient chronic epilepsy indications where oral administration is required.

The most advanced clinical study with ganaxolone is the ongoing Phase 3 trial assessing the efficacy and safety of ganaxolone for adjunctive treatment of refractory focal onset seizures.  Enrollment in this Phase 3 study is nearly complete with final enrollment in the registration component of the study expected to reach between 300-350 patients.  Top-line data are expected to be announced mid-2016.

A successful End-of-Phase 2 meeting took place during the third quarter, where the FDA was in agreement with the planned path to support registration of ganaxolone for adjunctive treatment of focal onset seizures, which, among other anticipated preclinical and clinical studies, includes a single additional Phase 3 registration study. Marinus and the FDA were in general agreement on the design, population and primary endpoint for both the ongoing and planned second Phase 3 clinical study, pending submission of the protocol of the second Phase 3 study for a Special Protocol Assessment.

As further evidence, and consistent with ganaxolone’s pre-clinical and clinical safety experience to date, a preclinical study was recently conducted to evaluate ganaxolone’s activity on the human progesterone receptor. Ganaxolone was designed to have the same neuronal activity as allopregnanolone without exposing patients to the potential hormonal side effects that could be seen with prolonged use of allopregnanolone. In the preclinical study, ganaxolone did not activate the human progesterone receptor, while allopregnanolone acted as a partial agonist to the receptor. The data support ganaxolone’s CNS-selectivity and lack of off-target activity at the human progesterone receptor.

Ganaxolone’s mechanism acts on GABA_A receptor targets known to have anti-anxiety benefits in addition to anti-seizure activity. With approximately 50% of epilepsy patients estimated to experience comorbid anxiety and/or depression, this added effect on mood could provide benefits to these epilepsy patients beyond seizure control.

Ganaxolone Liquid — Pediatric Orphan (Anxiety and Seizure):

PCDH19 Female Pediatric Epilepsy:
Marinus recently announced initial observations from its ongoing Phase 2 clinical trial for PCDH19 female pediatric epilepsy. PCDH19 is a serious and rare epileptic syndrome characterized by highly variable early-onset cluster seizures, cognitive, and behavioral disturbances. Initial observations showed that 63% of patients at various durations of therapy experienced a greater than 50% reduction for at least one 28-day treatment period compared to baseline. Consistent with earlier studies, ganaxolone was shown to be generally safe and well-tolerated. Improvements in behavior were also reported by investigators and caretakers for some patients. Enrollment is continuing in the study with full data expected mid-2016.

Fragile X Syndrome:
As an initial proof-of-concept study to explore the mood effects of ganaxolone, an exploratory double-blind placebo-controlled Phase 2 study in Fragile X Syndrome is underway.  This study is fully enrolled and data from this grant-funded, investigator-sponsored clinical trial are expected to be announced in Q1 2016.

Upcoming Milestones:

• Initiation of Phase 1 clinical trial with ganaxolone IV in 1H 2016
• Top-line data from Phase 2 clinical trial in Fragile X Syndrome expected in Q1 2016
• Top-line data from Phase 3 clinical trial in focal onset seizures expected mid-2016
• Full data set from Phase 2 PCDH19 clinical trial expected mid-2016

Third Quarter Financial Update

At September 30, 2015, the Company had cash, cash equivalents and investments of $34.5 million, compared to $49.7 million at December 31, 2014.  The Company believes that its cash, cash equivalents and investments, as of September 30, 2015, are adequate to fund operations into the fourth quarter of 2016.

Research and development expenses increased to $3.5 million and $12.9 million for the three and nine months ended September 30, 2015, respectively, as compared to $1.6 million and $6.5 million for the same periods in the prior year. The increases in both periods resulted primarily from an increase in clinical, drug development and consulting costs related to our ongoing clinical trials for ganaxolone, as well as increases in compensation-related costs due to hiring additional clinical resources, including our Chief Medical Officer, who was hired in December 2014.  Most of our research and development expenses relate to our ongoing Phase 3 clinical trial of ganaxolone in adults with focal onset seizures.

General and administrative expenses increased to $1.4 million and $4.1 million for the three and nine months ended September 30, 2015, respectively, as compared to $0.9 million and $1.8 million for the same periods in the prior year. The increases in general and administrative expenses were primarily due to the hiring of new management and the upward scaling of our operations in connection with both our public company status as of July 31, 2014, and our ongoing Phase 3 clinical trial of ganaxolone in adults with focal onset seizures.

Marinus reported net losses of $5.0 million and $17.2 million for the three and nine months ended September 30, 2015, respectively.  Our cash used in operating activities was $15.1 million for the nine months ended September 30, 2015 compared to $6.6 million for the same period a year ago.

Readers are referred to, and encouraged to read in its entirety, the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2015 to be filed with the Securities and Exchange Commission, which includes further detail on the above-referenced transactions and the Company’s business plans and operations, financial condition and results of operations.

About Marinus Pharmaceuticals

Marinus Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, proven efficacy, safety and convenient dosing, to improve the lives of patients suffering from epilepsy and neuropsychiatric disorders.  Ganaxolone is a CNS-selective GABA_A modulator that acts on a well-characterized target in the brain known to have both anti-seizure and anti-anxiety effects. Ganaxolone is being developed in three different dose forms (IV, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings.  Ganaxolone IV is planned to enter the clinic in 2016 and is being developed to treat status epilepticus. Ganaxolone IV is complemented by its oral dose forms, providing the potential for IV-to-oral continuation therapy for patients transitioning from acute care to outpatient settings.  Ganaxolone capsule is being evaluated in a Phase 3 multi-national clinical trial as adjunctive treatment of focal onset seizures in adults.  Ganaxolone capsule and liquid are being studied in orphan pediatric indications with comorbidities in seizures and behavior disorders — PCDH19 epilepsy and Fragile X Syndrome. For additional information, please visit the Company’s website at www.marinuspharma.com.

Forward-Looking Statements

To the extent that statements contained in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.  Words such as “may”, “will”, “expect”, “anticipate”, “estimate”, “intend”, “believe”, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements.  Examples of forward looking statements contained in this press release include, among others, statements regarding our expectations regarding our development plans for our product candidate, including the development of dose forms, the clinical trial testing schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements.  Such risks and uncertainties include, among others, the uncertainties inherent in the conduct of future clinical trials, the timing of the clinical trials, enrollment in clinical trials, availability of data from ongoing clinical trials, expectations for regulatory approvals, and other matters, including the development of formulations of ganaxolone, that could affect the availability or commercial potential of our drug candidates.  Marinus undertakes no obligation to update or revise any forward-looking statements.  For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the Company in general, see filings Marinus has made with the Securities and Exchange Commission.

 

CONTACT:   
Company:        
Lisa M. Caperelli
Senior Director, Investor Relations & Corporate Communications
Marinus Pharmaceuticals, Inc.
484-801-4674
lcaperelli@marinuspharma.com

Media Contact:
Tiberend Strategic Advisors, Inc.
Amy S. Wheeler
646-362-5750
awheeler@tiberend.com