UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): August 27, 2018
Ultragenyx Pharmaceutical Inc.
(Exact name of registrant as specified in its charter)
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Delaware |
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001-36276 |
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27-2546083 |
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(State or other jurisdiction of |
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(Commission File Number) |
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(I.R.S. Employer |
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60 Leveroni Court, Novato, California |
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94949 |
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(Address of principal executive offices) |
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(Zip Code) |
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Registrant’s telephone number, including area code: (415) 483-8800
Not Applicable
Former name or former address, if changed since last report
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
On August 27, 2018, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release (the “MPS Release”) announcing that the European Commission has approved the Marketing Authorization Application for Mepsevii™ (vestronidase alfa), for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII.
On August 29, 2018, the Company issued a press release (the “FAOD Release”) announcing that the U.S. Food and Drug Administration has accepted the Company’s most recent proposal to submit a New Drug Application for UX007 for the treatment of long-chain fatty acid oxidation disorders based on existing data.
Copies of the MPS Release and the FAOD Release are filed herewith as Exhibits 99.1 and 99.2, respectively.
Item 9.01Financial Statements and Exhibits.
(d) Exhibits
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Exhibit No. |
Description |
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99.1 |
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99.2 |
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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Date: August 29, 2018 |
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Ultragenyx Pharmaceutical Inc. |
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By: |
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/s/ Shalini Sharp |
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Shalini Sharp |
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Executive Vice President, Chief Financial Officer |
Exhibit 99.1
Ultragenyx Announces Approval of Mepsevii™ (vestronidase alfa) in Europe for the Treatment of Mucopolysaccharidosis VII
Mepsevii, an Enzyme Replacement Therapy, is the First Treatment Approved in the EU for Mucopolysaccharidosis VII
Novato, Calif. — August 27, 2018 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the European Commission (EC) has approved the Marketing Authorization Application (MAA) for Mepsevii™ (vestronidase alfa), for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). Mepsevii is now approved for use in all 28 EU countries and in Iceland, Liechtenstein and Norway.
“Dr William Sly’s science for this treatment has been around for over 25 years and we are honored to be able to develop and finally make this medicine available to MPS VII patients and families in Europe, people who did not know whether a therapy would ever be available to them,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “This would not have been possible without the commitment and dedication of the patients, their families, and the physicians who participated in our clinical program, and I would like to thank them.”
The approval follows a positive opinion adopted on June 28, 2018 by the European Committee for Medicinal Products for Human Use (CHMP) to recommend approval of Mepsevii under exceptional circumstances. The European Medicines Agency (EMA) granted Orphan Drug designation to Mepsevii in March 2012. Mepsevii was approved by the U.S. Food and Drug Administration (FDA) for the treatment of pediatric and adult patients with MPS VII in November 2017.
About MPS VII
MPS VII is a progressive, rare, genetic, metabolic lysosomal storage disorder (LSD) caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulphate, chondroitin sulphate and heparan sulphate. These complex GAG carbohydrates are a critical component of many tissues. The inability to properly break down GAGs leads to a progressive accumulation in many tissues and results in multisystem tissue and organ damage. MPS VII symptoms can include an abnormally coarsened face, pulmonary disease, cardiovascular complications, hepatosplenomegaly (in which the liver and spleen swell beyond their normal size), joint stiffness, short stature, cognitive impairment and the skeletal disease known as dysostosis multiplex.
MPS VII is one of the rarest MPS disorders, affecting an estimated 200 patients in the developed world.
About Mepsevii™ (vestronidase alfa)
Mepsevii will be available as 2 mg/ml concentrate for solution for infusion. The active substance of Mepsevii is vestronidase alfa, a recombinant form of human beta-glucuronidase (ATC code: A16AB18). Mepsevii is an enzyme replacement therapy intended to provide or supplement beta glucuronidase, an enzyme that helps with the degradation of glycosaminoglycans and thus prevents their accumulation in various tissues in the body.
INDICATION (IN THE E.U.)
Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).
INDICATION (IN THE U.S.)
Mepsevii is indicated in pediatric and adult patients for the treatment of mucopolysaccharidosis VII (MPS VII, Sly syndrome).
Limitations of Use
The effect of Mepsevii on the central nervous system manifestations of MPS VII has not been determined.
U.S. IMPORTANT SAFETY INFORMATION
What is the most important information I should know about Mepsevii?
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A severe allergic reaction called anaphylaxis has occurred with Mepsevii treatment, as early as the first dose. |
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Your doctor will monitor you closely for symptoms of an allergic reaction while you are receiving Mepsevii and for 60 minutes after your injection. |
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Your doctor will immediately discontinue the Mepsevii infusion if you experience anaphylaxis. |
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Your doctor should talk to you about the signs and symptoms of anaphylaxis and about getting medical treatment if you have symptoms after leaving the doctor's office or treatment center. |
What are the possible side effects of Mepsevii?
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The most common side effects of Mepsevii are: |
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Leakage of Mepsevii into the surrounding tissue during infusion |
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Diarrhea |
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Rash |
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Severe allergic reaction (anaphylaxis) |
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Infusion site swelling |
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Severe itching of the skin |
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One patient experienced a seizure during a fever while taking MEPSEVII. |
Before receiving Mepsevii, tell your doctor about all of your medical conditions, including if you:
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are pregnant, think you may be pregnant, or plan to become pregnant. There is not enough experience to know if Mepsevii may harm your unborn baby. |
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are breastfeeding or plan to breastfeed. There is not enough experience to know if Mepsevii passes into your breast milk. Talk with your doctor about the best way to feed your baby while you receive Mepsevii. |
These are not all the possible side effects of Mepsevii. Call your doctor for medical advice about side effects.
FOR U.S. PATIENTS
You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Ultragenyx at 1-888-756-8657.
Please see full U.S. Prescribing Information for additional Important Safety Information including serious side effects.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are no approved therapies.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
Forward-Looking Statements
Except for the historical information contained herein, the matters set forth in this press release, including statements relating to plans or expectations regarding the availability of Mepsevii, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking
statements 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 clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx 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 Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 3, 2018, and its subsequent periodic reports filed with the Securities and Exchange Commission.
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Contact:
Ultragenyx Pharmaceutical Inc.
Investors & Media
Danielle Keatley
+1-415-475-6876
dkeatley@ultragenyx.com
Exhibit 99.2
Contact:
Ultragenyx Pharmaceutical Inc.
Investors & Media
Danielle Keatley
415-475-6876
dkeatley@ultragenyx.com
Ultragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders
Novato, Calif. — August 29, 2018 — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted Ultragenyx’s most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data. Further details regarding timing will be forthcoming following a pre-NDA meeting, which is being scheduled for the second half of 2018.
“We appreciate FDA’s review of multiple data submissions and collaboration with us to develop a path for an early filing, and it is our commitment to get this important potential treatment to patients with this serious disease as quickly as possible,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We will meet with the FDA to discuss the details of the NDA submission and, if approved, appropriate post-approval commitments to further evaluate long-term outcomes of UX007 in patients with LC-FAOD.”
The data submitted to the FDA for evaluation included the recently published 78-week Phase 2 study results in 29 patients, a now published retrospective medical record review of 20 patients, and 56 emergency IND cardiomyopathy and other patients. In addition, these data were supported by results of a published randomized controlled investigator study of 32 patients showing an effect of triheptanoin on cardiac function. In the 78-week sponsored Phase 2 study, the data showed a 48.1 percent reduction in the mean annualized rate of major clinical events (MCEs) and a 50.6 percent reduction in the median annualized rate of MCEs after 78 weeks of treatment with UX007 compared to an annualized rate of MCEs in the 18 months prior to treatment with UX007. There was also a 50.3 percent reduction in the mean annualized duration of MCEs and a 76.7 percent reduction in the median annualized duration of MCEs following 78 weeks of UX007 treatment. The safety profile was consistent with what has been previously observed with UX007.
In the EU, Ultragenyx will discuss these data with the European Medicines Agency (EMA) and expects to have an update in the second half of 2018.
About LC-FAOD and UX007
LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to severe depletion of glucose in the body, and serious liver, muscle and heart disease, which can lead to hospitalizations or early death. LC-FAOD are included in newborn screening panels across the U.S. and in certain European countries. Patients with LC-FAOD are currently treated with the avoidance of fasting, low-fat/high carbohydrate diets, carnitine, and medium-chain triglyceride (MCT) oil, a medical food product. Despite current therapy, many patients have significant metabolic events including hospitalizations and mortality due to LC-FAOD.
UX007 is a highly purified, pharmaceutical-grade, synthetic, seven-carbon fatty acid triglyceride created via a multi-step chemical process. It is an investigational medicine intended to provide patients with medium-length, odd-chain fatty acids that can be metabolized to increase intermediate substrates in the Krebs cycle, a key energy-generating process. Unlike typical even-chain fatty acids, UX007 can be converted to new glucose through the Krebs cycle, potentially providing an important added therapeutic effect, particularly when glucose levels are too low.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The Company has rapidly built and advanced a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
The Company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.
Forward Looking Statements
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and future regulatory interactions, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements 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 clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx 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 Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 3, 2018, and its subsequent periodic reports filed with the Securities and Exchange Commission.
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