On November 5, 2018 INSYS Therapeutics, Inc. (NASDAQ: INSY), a leader in the development, manufacture and commercialization of pharmaceutical cannabinoids and spray technology, reported financial results for its third quarter ended Sept. 30, 2018 (Press release, Insys Therapeutics, NOV 5, 2018, View Source [SID1234530857]).

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OVERALL HIGHLIGHTS

Commenced a strategic alternatives review process for the company’s opioid-related assets

Expanded collaborative partnership with University of California San Diego’s Center for Medicinal Cannabis Research (CMCR) to study the company’s cannabidiol (CBD) oral solution in two additional disease states (early psychosis and anxiety in anorexia nervosa)

Achieved net revenue of $18.3 million in the third quarter of 2018

Advanced R&D programs with a $14.5 million investment in the third quarter of 2018:

Received "Fast Track" designation from the FDA for epinephrine nasal spray as an investigational treatment for anaphylaxis

Continued enrollment of three company-sponsored CBD clinical studies:

childhood absence epilepsy (Phase 2)

Prader-Willi syndrome (Phase 2)

infantile spasms (Phase 3)

Completed pharmacokinetic study of dronabinol inhalation

Signed definitive licensing agreement with Lunatus to commercialize SUBSYS in the Middle East

Received FDA approval of supplemental NDA for SYNDROS to expand label, enabling use of the product with feeding tubes for patients with cancer and AIDS

Appointed Elizabeth Bohlen to the Board of Directors and added Mark Nance to the management team as chief legal officer and general counsel

Announced settlement agreement in principle with Department of Justice consistent with previous public statement and disclosures

Optimized commercial organization and related support functions to control operating costs in line with lower revenue

Received confirmation from the SEC that it concluded its investigation of the company, and does not intend to recommend an enforcement action

"Our commitment to further establish our position as a leader in pharmaceutical cannabinoids and spray technology was exemplified by the achievement of several milestones in the third quarter," said Saeed Motahari, president and chief executive officer of INSYS Therapeutics. "We continued to make progress on our pipeline and expanded our collaborative partnerships with leading research institutions, increasing the number of CBD clinical studies in which we’re the sole sponsor or a key collaborator to seven. Furthermore, our 99.5 percent pure pharmaceutical-grade CBD oral solution allows INSYS to meet the needs of clinical study patients and become a strategic partner across our industry and in the medical community."

Motahari concluded, "The decline in the overall TIRF market continues to impact sales of our primary commercial product, SUBSYS. Our proactive efforts to expand managed care access and educate appropriate HCPs have enabled us to maintain our leading share of the branded TIRF market. However, this decline in revenue has required us to contain costs and as a result, we have taken actions to adjust our commercial organization to align with the realities of the market. We will continue to be disciplined with our cost structure, while appropriately investing in our pipeline, which is the key to transforming INSYS into a leader in pharmaceutical cannabinoids and spray technology."

Financial & Operating Highlights

During the preparation of the consolidated financial statements as of and for the quarter ended Sept. 30, 2018, the company identified errors that impacted the 2017 financial information, which have been revised for the correction of this error.

The company had $113.0 million in cash, cash equivalents and short-term and long-term investments with no debt outstanding as of Sept. 30, 2018

Net revenue for the third quarter of 2018 was $18.3 million, compared to $30.7 million for the third quarter of 2017, driven primarily by declines in the TIRF market

Gross margin was 87.0 percent for the third quarter of 2018, compared to 75.6 percent in the same period of 2017

Sales and marketing investment was $7.4 million for the third quarter of 2018, compared to $12.8 million for the third quarter of 2017, as the company took action to better align its commercial organization to the lower revenue base

Research and development investment decreased to $14.5 million for the third quarter of 2018, compared to $19.6 million for the third quarter of 2017, primarily as a result of the timing of clinical trials

General and administrative expense of $8.9 million for the third quarter of 2018 declined compared to $11.3 million in the third quarter of 2017, as the company continued to look for opportunities to level-set its cost base

Legal expense increased to $16.0 million for the third quarter of 2018, compared to $4.4 million in the third quarter of 2017, as a result of the company’s legal proceedings, including

expenses associated with indemnification of former executives in connection with their pending trial, which constitutes approximately 60 percent of the total Q3 2018 expense. Management is disputing the reasonableness of certain of these indemnification-related expenses

Income tax expense was $240 thousand for the third quarter of 2018 compared to a benefit of ($9.0 million) during the revised third quarter of 2017

Net loss for the third quarter of 2018 was ($30.6 million), or ($0.41) per basic and diluted share, compared to a net loss of ($166.3 million), or ($2.28) per basic and diluted share, for the revised third quarter of 2017. Adjusted net loss for the third quarter of 2018 was ($0.37) per basic and diluted share

Adjusted EBITDA loss for the third quarter of 2018 was ($26.0 million), compared to Adjusted EBITDA loss of ($18.4 million) in the prior-year quarter. The reconciliation of net income to Adjusted EBITDA is included at the end of this news release

During the preparation of the consolidated financial statements as of and for the quarter ended Sept. 30, 2018, the company identified errors within the company’s consolidated financial statements for the year ended Dec. 31, 2017, resulting from the company’s adoption of ASU 2016-09 "Compensation – Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting" on Jan. 1, 2017, which impacted the previously filed financial statements for the quarters ended March 31, June 30, and Sept. 30 of 2017 as well as the audited financial statements for the year ended Dec. 31, 2017. In addition, we identified an error in the calculation of our weighted average shares outstanding, which impacted the previously filed financial statements for the three and nine months ended Sept. 30, 2017, and the year ended Dec. 31, 2017. The company evaluated the errors and concluded that they were not material to the financial statements previously issued. Accordingly, the Dec. 31, 2017 financial information has been revised to include a cumulative adjustment of $2.9 million to correctly present uncertain tax position liabilities and accumulated deficit, resulting in a reduction to total liabilities and an increase in stockholders’ equity. These revisions will be reflected and disclosed in the company’s Form 10-Q for the period ended Sept. 30, 2018 to be filed with the SEC on or before Nov. 9, 2018. Additionally, in order to correctly present income tax benefit and net loss in the appropriate periods for comparative purposes, the financial results for the three and nine months ended Sept. 30, 2017, have been revised to include adjustments of $40,000 and $120,000, respectively. These revisions resulted in net loss per share for the three and nine months ended Sept. 30, 2017, and the year ended Dec. 31, 2017, changing from $2.30 to $2.28, $2.51 to $2.50 and $3.16 to $3.12, respectively.

Webcast Information

A conference call is scheduled for 5:00 p.m. Eastern Standard Time on Nov. 5, 2018, to discuss the financial and operational results for the third quarter 2018. Interested parties can listen to the call live as it occurs via the company’s website, View Source, on the Investors section’s Presentations & Events page; or by dialing 844-263-8304 (from inside the U.S.) or 213-358-0958 (from outside the U.S.), and using the Conference ID 6149699. A webcasted replay of the call will be available on the site a few hours after the event.

On November 5, 2018 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the quarter ended September 30, 2018, and provided an update on the launch of INGREZZA (valbenazine) and its clinical development programs (Press release, Neurocrine Biosciences, NOV 5, 2018, View Source [SID1234530824]).

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"We remain committed to delivering hope for patients with serious unmet medical needs as evidenced by the growing adoption of INGREZZA and the recent commercial launch of ORILISSA – both treatments were discovered at Neurocrine Biosciences," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With the recently completed salesforce expansion, we continue to focus on educating healthcare providers on the benefits of INGREZZA to treat patients with tardive dyskinesia. We have also made great progress in our research and development programs with the advancement of two new internally discovered compounds into the clinic and our research collaboration with Jnana Therapeutics to develop new medicines to treat neurological diseases."

Financial Results

Total revenue for the three and nine months ended September 30, 2018 was $151.8 million and $319.7 million, respectively, compared to $60.8 million and $67.1 million for the same periods in 2017. Total revenues were comprised of the following (in thousands):

INGREZZA was made available for commercial distribution on May 1, 2017 and ORILISSA (elagolix) was approved by the U.S. Food and Drug Administration (FDA) during the third quarter of 2018 with AbbVie sales beginning in August 2018. With the FDA approval of ORILISSA, the Company recognized a $40 million event-based milestone as revenue under the Company’s collaboration agreement with AbbVie during the third quarter of 2018. During the third quarter of 2017, Mitsubishi Tanabe initiated a pivotal trial of INGREZZA in Asia for the treatment of tardive dyskinesia which generated a $15 million milestone.

For the third quarter of 2018, the Company reported net income of $50.8 million, or $0.52 diluted net income per share, compared to a net loss of $11.1 million, or $0.13 net loss per share, for the same period in 2017. For the nine months ended September 30, 2018, the Company reported net income of $3.0 million, or $0.03 diluted net income per share, compared to a net loss of $149.4 million, or $1.70 net loss per share, for the same period in 2017. The increase in income across both periods is due to increased INGREZZA net product sales and the $40 million event-based milestone from AbbVie.

Research and development (R&D) expenses were $35.5 million during the third quarter of 2018, compared to $22.5 million for the third quarter of 2017. For the nine months ended September 30, 2018, R&D expenses were $121.4 million, compared to $96.2 million for the same period last year. The increase in R&D expenses across both periods is principally due to expanded research efforts, the advancement of the Company’s clinical programs including Tourette syndrome, congenital adrenal hyperplasia (CAH), and the two new programs announced today, as well as activities to prepare for the Company’s intended opicapone New Drug Application (NDA) submission in the second quarter of 2019.

Sales, general and administrative (SG&A) expenses increased to $60.4 million for the third quarter of 2018 from $43.9 million for the third quarter of 2017. For the nine months ended September 30, 2018, SG&A expenses were $180.0 million, compared to $113.6 million for the same period last year. The increase in SG&A expense for both periods is primarily due to the hiring of the Company’s sales force, including the third quarter 2018 sales force expansion, and commercialization activities for INGREZZA, which launched in the third quarter of 2017.

The Company’s balance sheet at September 30, 2018 reflected total assets of $933.7 million, including cash and investments of $820.6 million, compared with total assets at December 31, 2017 of $817.6 million.

Updated 2018 Expense Guidance

Ongoing operating expenses for 2018 are now expected to approximate $410 million to $420 million, which compares to the prior operating expense guidance of $395 million to $420 million. The operating expense guidance range includes the clinical pipeline expansion and the recently announced research collaboration with Jnana Therapeutics.

Pipeline Highlights

INGREZZA (valbenazine) Update

INGREZZA received FDA approval on April 11, 2017, becoming the first medicine approved in the United States for the treatment of adults with tardive dyskinesia.

Valbenazine is being investigated in Tourette syndrome and has been granted Orphan Drug Designation by the FDA for the treatment of pediatric patients with Tourette syndrome. Orphan Drug Designation is granted by the FDA to drugs that are intended to treat rare diseases or conditions in the United States.

In the fourth quarter of 2017, the Company initiated T-Force GOLD, a Phase IIb study of valbenazine in pediatric patients with Tourette syndrome. This study is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, which will evaluate the safety, tolerability, efficacy and optimized dosing of once-daily valbenazine in up to 120 pediatric patients with moderate to severe Tourette syndrome over 12 weeks of treatment. The primary endpoint of this study is the comparison of the change from baseline of the Yale Global Tic Severity Scale between placebo and active treatment groups at the end of week 12. Top-line data are expected in December 2018.

In the second quarter of 2018, the Company commenced enrollment into the open label extension study, T-Force GOLD Plus, for pediatric patients with Tourette syndrome. Patients who complete participation in the T-Force GOLD study are eligible to roll over into participation in this open label extension study for an additional six months of treatment with optimized doses of valbenazine. The study will collect longer-term safety and tolerability data in children and adolescents as well as providing useful information about the maintenance of efficacy in these patients over the six months period of dosing.

In the second quarter of 2018, the Company started T-Force PLATINUM, a double-blind, placebo-controlled, randomized withdrawal study of valbenazine in pediatric patients with Tourette syndrome. This study is designed to evaluate longer term efficacy and safety in patients who initially responded to open-label therapy with optimized doses of valbenazine. Approximately 180 patients will participate in the study with top-line data expected in late 2019.

In March 2015, the Company announced that it had entered into an exclusive collaboration and licensing agreement for the development and commercialization of INGREZZA in Japan and other select Asian markets with Mitsubishi Tanabe Pharma Corporation (MTPC). In 2017, MTPC initiated a pivotal trial of INGREZZA in Japan for the treatment of tardive dyskinesia.

ORILISSA (elagolix) Update

On July 24, 2018, AbbVie, in collaboration with Neurocrine, announced FDA approval and in October 2018 Health Canada approval for ORILISSA for the management of endometriosis with associated moderate to severe pain. The FDA granted priority review to ORILISSA. The FDA grants priority review designation to medicines that, if approved, would provide a significant improvement in the safety or effectiveness of treatment of a serious condition. AbbVie began commercialization of ORILISSA in the United States in August 2018.

AbbVie provided positive top-line efficacy data from two Phase III studies in women with uterine fibroids in the first quarter of 2018 and from the associated six-month safety extension during the third quarter of 2018. The ELARIS UF-I and UF-II studies of elagolix met all primary and ranked secondary endpoints at month six. These replicate Phase III studies were randomized, parallel, double-blind, placebo-controlled clinical trials evaluating elagolix alone or in combination with low-dose hormone (add-back) therapy in women with heavy uterine bleeding associated with uterine fibroids. The studies enrolled approximately 400 patients each for an initial six-month placebo-controlled dosing period. At the end of the six months of placebo-controlled evaluation, patients were eligible to enter an additional six-month safety extension study. The primary efficacy endpoint of the study was an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints were evaluated including the change in fibroid volume and hemoglobin. Bone mineral density was assessed via dual-energy x-ray absorptiometry (DEXA) scan at baseline, at the conclusion of dosing, and at six months post-dosing. Results from these studies will form the basis for an anticipated 2019 NDA submission to the FDA for the approval of elagolix in the treatment of uterine fibroids.

Opicapone Update

In February 2017, the Company entered into an exclusive licensing agreement with BIAL – Portela & CA, S.A. (BIAL) for the development and commercialization of opicapone in the United States and Canada. Opicapone is a once-daily, peripherally-acting, highly-selective catechol-O-methyltransferase inhibitor, being developed as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors for adult patients with Parkinson’s disease and motor fluctuations. The Company met with the FDA in January and based upon the BIPARK-I and BIPARK-II pivotal Phase III studies conducted by BIAL, the FDA did not require additional Phase III trials to form an NDA submission. The Company is in the process of preparing for an NDA submission which it anticipates will occur during the second quarter of 2019.

Congenital Adrenal Hyperplasia (CAH) Program (NBI-74788) Update

In the second quarter of 2017, the Company successfully completed the Phase I, Investigational New Drug (IND)-opening study of NBI-74788 in healthy volunteer participants. The study was a randomized, open-label, two-period crossover study to evaluate the pharmacokinetics, the effect of food on pharmacokinetics, and the safety of NBI-74788 in a total of 16 healthy adults.

The Company began recruitment for a Phase II, proof-of-concept study examining the pharmacokinetics, pharmacodynamics, and safety of NBI-74788 in adult males and females with classic, 21-hydroxylase deficiency CAH in November of 2017. The study will evaluate the relationship between NBI-74788 exposures and specific steroid hormone levels in these patients. The Company recently expanded this study to include additional patients to further optimize dosing flexibility and convenience. Data are expected during the first quarter of 2019.

New VMAT2 Inhibitor

The Company has filed an IND and initiated a Phase I study for a novel, internally discovered vesicular monoamine transporter 2 (VMAT2) inhibitor with potential use in the treatment of several neurology and/or psychiatry disorders. The initial randomized, double-blind, single ascending dose study to evaluate the safety, tolerability, and pharmacokinetic profile of the compound in healthy participants is anticipated to be completed during the fourth quarter of 2018.

New CNS Compound

The Company has filed an IND and completed dosing in a Phase I single ascending dose study for an internally discovered first-in-class central nervous system (CNS) compound with potential use in the treatment of several neurology and/or psychiatry disorders. This study is a randomized, double-blind, single ascending dose study to evaluate the safety, tolerability, and pharmacokinetic profile of the compound in healthy participants. The Company is currently analyzing the data from this study to inform the design of future clinical studies for the program.

Conference Call and Webcast Today at 4:30 PM Eastern Time

Neurocrine will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 877-876-9176 (US) or 785-424-1667 (International) using the conference ID: NBIX. The webcast and supplemental information in the form of a slide presentation can also be accessed on Neurocrine’s website under Investors at www.neurocrine.com. The webcast and slide presentation will be archived for approximately one month.

About INGREZZA (valbenazine) Capsules

INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release from presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity or functional inhibition for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information

Contraindications

INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

Warnings & Precautions

Somnolence

INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation

INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Adverse Reactions

The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.

On November 5, 2018 Cambrex Corporation (NYSE: CBM), the leading manufacturer of small molecule innovator and generic Active Pharmaceutical Ingredients (APIs), and finished dosage forms, reported that third quarter 2018 financial results will be released on Thursday, November 8, 2018 before the market opens (Press release, Cambrex, NOV 5, 2018, View Source [SID1234530775]).

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The Company will host a conference call to discuss the financial results.

Third Quarter 2018 Earnings Conference Call
When: Thursday, November 8, 2018 at 8:30 a.m. Eastern Time
Dial-in: 1-877-830-2649 for U.S.
+1-785-424-1824 for International
Passcode: 3109072
Dial-in Replay: 1-888-203-1112 for U.S.
+1-719-457-0820 for International
Passcode: 3109072
Available through Thursday, November 15, 2018
Webcast: www.cambrex.com

On November 5, 2018 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) reported financial results for the third quarter and first nine months of 2018 (Press release, MorphoSys, NOV 5, 2018, View Source [SID1234530766]).

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"The third quarter of 2018 was a productive one for MorphoSys, highlighted by very encouraging progress in therapeutic programs within both our proprietary development and partnered portfolios. Our primary focus is on our lead program MOR208, and here we look forward to releasing latest data from the ongoing L-MIND trial in an oral presentation at the forthcoming ASH (Free ASH Whitepaper) 2018 meeting. This program is proceeding according to plan, and we are committed to completing L-MIND and seeking U.S. approval based on this trial," commented Dr. Simon Moroney, CEO of MorphoSys AG. "Meanwhile, we continue to prepare our commercial organization in the U.S., with the goal of launching MOR208 there, subject, of course, to FDA approval of this investigational drug."

"We are very pleased with MorphoSys’s business performance in 2018 to date. Driven by an attractive licensing deal with Novartis for MOR106 and increasing royalty income from Tremfya(R), we saw strong revenue development in the third quarter. The very good business performance enabled us to increase our financial guidance for the year. Based on a solid cash position, which we further strengthened with our Nasdaq IPO earlier in the year, we are well positioned to continue the advancement of our pipeline products, in particular to drive our lead program MOR208 towards the market," said Jens Holstein, CFO of MorphoSys AG.

Financial review for the third quarter of 2018 (IFRS; all figures rounded)

In Q3 2018, MorphoSys continued to focus on the research and development of drug candidates in its proprietary portfolio, while also supporting the activities of its partners. Group revenues in Q3 2018 amounted to EUR 55.0 million (Q3 2017: EUR 15.0 million). The revenue increase was mainly driven by the up-front payment of EUR 47.5 million for the license agreement for MOR106 with Novartis. As the contractual royalty reporting from Janssen for Q3 2018 has not yet been received due to the reporting schedules of Janssen and MorphoSys, Tremfya(R) royalties booked for Q3 2018 were estimated based on public announcements made by Janssen/J&J on Tremfya(R) sales in Q3 2018.

In the Proprietary Development segment, MorphoSys focuses on the research and clinical development of its own drug candidates in the fields of cancer and inflammation. In Q3 2018, this segment recorded revenues of EUR 48.8 million (Q3 2017: EUR 0.2 million). In the Partnered Discovery segment, MorphoSys applies its proprietary technology to discover new antibodies for pharmaceutical companies, benefiting from its partners’ development advances through R&D funding, licensing fees, success-based milestone payments and royalties. In Q3 2018, revenues in this segment amounted to EUR 6.2 million (Q3 2017: EUR 14.8 million).

Total operating expenses reached EUR 25.3 million in the third quarter of 2018 (Q3 2017: EUR 38.2 million). R&D expenses for proprietary development and technology development amounted to EUR 15.9 million (Q3 2017: EUR 29.8 million).

Earnings before interest and taxes (EBIT) in Q3 2018 amounted to EUR 30.1 million (Q3 2017: EUR -23.5 million), reflecting in particular the up-front payment made by Novartis under the MOR106 license agreement. The Proprietary Development segment reported an EBIT of EUR 30.3 million (Q3 2017: EUR -29.8 million), while the Partnered Discovery segment recorded an EBIT of EUR 3.8 million (Q3 2017: EUR 10.4 million). In Q3 2018, the consolidated net result amounted to EUR 30.2 million (Q3 2017: EUR -24.0 million). Basic earnings per share for Q3 2018 reached EUR 0.96 (Q3 2017: EUR -0.83).

At the end of Q3 2018, the Company had a cash position of EUR 481.2 million, compared to EUR 312.2 million on December 31, 2017. On the balance sheet, this cash position is reported under the following items: cash and cash equivalents; financial assets at fair value through profit or loss; and current and non-current other financial assets at amortized cost. The increase in funds resulted mainly from the capital increase in conjunction with the successful Nasdaq listing completed in April 2018 with gross proceeds of USD 239 million and the upfront payment made by Novartis in the third quarter 2018 in the amount of EUR 47.5 million in connection with the license agreement for MOR106. This was partially offset by the use of cash for operating activities.

The number of shares issued totaled 31,839,572 at the end of Q3 2018 (year-end 2017: 29,420,785). The increase was mainly driven by the capital increase in April 2018.

Results for the first nine months 2018

For the first nine months of 2018, group revenues amounted to EUR 66.0 million (Q1-Q3 2017: EUR 38.6 million). Expenditure for proprietary development and technology development amounted to EUR 55.1 million in the first nine months of 2018 (Q1-Q3 2017: EUR 67.1 million). Consequently, EBIT in the first nine months of 2018 amounted to EUR -13.0 million, compared to EUR -53.8 million in the first nine months of 2017.

Financial guidance and operational outlook for 2018

MorphoSys confirms its 2018 financial guidance which had been increased after signing an agreement with Novartis for MOR106 in July 2018. In the light of the recent positive development of Tremfya(R) royalties, MorphoSys expects revenues on the upper end of the guided range from EUR 67 million to EUR 72 million for 2018. Earnings before interest and taxes (EBIT) are expected to be EUR -55 million to EUR -65 million. R&D expenses for proprietary programs and technology development are expected to be in a range of EUR 87 million to EUR 97 million. This guidance does not include additional revenues from potential future collaborations and/or license agreements nor any effects from possible in-licensing or development partnerships for new drug candidates.

MorphoSys expects the following events and activities in the Proprietary Development segment during the remainder of the year:

MOR208

– L-MIND:

– Continue discussions with the FDA to evaluate possible paths to market, including the possibility of an expedited regulatory submission and potential approval based primarily on the L-MIND study.

– Presentation of updated interim results on all 81 patients enrolled in the study evaluating MOR208 plus lenalidomide in r/r DLBCL at the ASH (Free ASH Whitepaper) (American Society of Hematology) 2018 Annual Meeting, which will be held in San Diego, California, in early December.

– B-MIND: Continue the enrollment in the phase 3 part of the study evaluating MOR208 plus bendamustine versus rituximab plus bendamustine in r/r DLBCL.

– COSMOS: Continue the phase 2 trial of MOR208 plus idelalisib or venetoclax in chronic lymphocytic leukemia (CLL/SLL) and present data from cohort B (MOR208 plus venetoclax) at the upcoming ASH (Free ASH Whitepaper) 2018 Annual Meeting.

– Commercial and CMC activities: Secure commercial supply of MOR208 and continue to build commercial capabilities for MOR208 in the U.S. under the newly established MorphoSys US Inc., in preparation for a potential market launch, currently anticipated in 2020 pending FDA approval.

MOR202

– Multiple myeloma (MM): Study results from the ongoing phase 1/2a study will also be presented at the upcoming ASH (Free ASH Whitepaper) 2018 Annual Meeting. As previously communicated, MorphoSys will not continue development in MM beyond completion of the current trial without an additional partner for Ex-China; MorphoSys expects its partner I-Mab Biopharma to continue preparations for clinical development in MM and to start a pivotal trial in early 2019 in China.

– Other indications: MorphoSys continues to evaluate the development of MOR202 in other indications including autoimmune disorders.

MOR106: Continue ongoing development activities with partner Galapagos under the new global licensing agreement with Novartis.

– Continue the ongoing phase 2 trial IGUANA in atopic dermatitis.

– Continue the phase 1 bridging study initiated in September 2018 to evaluate a subcutaneous formulation of MOR106.

– All future costs related to MOR106 development to be borne by Novartis.

MOR107: Continue preclinical investigations of MOR107 with a focus on oncology indications.

MOR103/GSK3196165: Following presentation of data from several phase 2 trials in inflammatory diseases including rheumatoid arthritis (RA) run by partner GlaxoSmithKline (GSK) at the American College of Rheumatology (ACR) meeting in October 2018, MorphoSys expects GSK to further continue clinical development activities in RA.

In its Partnered Discovery segment, MorphoSys expects the following events in the fourth quarter of 2018:

Tremfya(R) (guselkumab): Several phase 3 trials in psoriasis are scheduled for primary completion in 2018 according to clinicaltrials.gov. This includes a head-to-head trial comparing Tremfya(R) to Cosentyx(R) in adults with plaque psoriasis, results of which are expected to be communicated in early 2019. MorphoSys expects Janssen to continue its current development program with Tremfya(R) including two phase 3 trials in psoriatic arthritis, the phase 2/3 GALAXI program in Crohn’s disease as well as the clinical phase 3 PROTOSTAR trial in pediatric psoriasis patients.

Other partnered programs: The publication of clinical data and the achievement of regulatory milestones from other partnered programs may occur during the remainder of 2018.

MorphoSys will continue to support its proprietary development activities by evaluating potential in-licensing, co-development, and/or acquisition opportunities as well as by initiating new proprietary development programs with the goal of maintaining and expanding the Company’s position in its current therapeutic and technological fields of activities.

** Including MOR107, which concluded a phase 1 study in 2017 and is currently in preclinical investigation with a focus on oncology indications. Tremfya(R) is still considered as a clinical program due to ongoing studies in various indications.
*** Including MOR103/GSK3196165, which is fully out-licensed to GSK, and MOR106, for which MorphoSys and Galapagos have signed a global licensing agreement with Novartis.

The interim statement for the third quarter of 2018 (IFRS) is available online:

View Source

MorphoSys will hold a conference call and webcast on November 6, 2018, to present the third quarter 2018 financial results and provide an outlook for the remainder of 2018.

Dial-in number for the analyst conference call (in English) at 2:00pm CET; 1:00pm GMT; 8:00am EST (listen-only):

Germany: +49 (0) 69 201 744 210

For UK residents: +44 (0) 203 009 2470

For US residents: +1 (0) 877 423 0830

Participant PIN: 79499880#

Participants are asked to dial in 10 minutes before the beginning of the conference. A live webcast and slides will be made available at View Source After the conference call, a slide-synchronized audio replay of the conference and a transcript will be available at View Source

On November 5, 2018 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported its financial results and corporate update for the quarter ended September 30, 2018 (Press release, Ultragenyx Pharmaceutical, NOV 5, 2018, View Source [SID1234530762]).

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"We’re encouraged by the growing demand for Crysvita from both children and adults with XLH in the United States, and we are making significant progress reaching doctors and helping them get patients on commercially available therapy in this early launch phase," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "Next year, we expect to file for potential approval of our third therapy, UX007 in fatty acid oxidation disorders, and we continue to advance our two gene therapy clinical programs with additional data expected around the end of this year and in 2019."

Financial Results

For the third quarter of 2018, Ultragenyx reported a net loss of $87.3 million, or $1.74 per share, basic and diluted, compared with a net loss for the third quarter of 2017 of $79.2 million, or $1.87 per share, basic and diluted. For the nine months ended September 30, 2018, net loss was $109.8 million, or $2.22 per share, basic and diluted, compared with a net loss for the same period in 2017 of $220.4 million, or $5.22 per share, basic and diluted. The loss from the first nine months was reduced by the sale of the Mepsevii (vestronidase alfa) priority review voucher (PRV) in January 2018 for net proceeds of $130.0 million and a $40.3 million gain from Ultragenyx’s portion of the sales of the PRV received with the Crysvita (burosumab) approval. The net loss for the first nine months of 2018 reflected cash used in operations of $234.7 million compared to $172.0 million for the same period in 2017.

Net Revenues

For the third quarter of 2018, Ultragenyx reported $11.8 million in total revenue. For Crysvita, Ultragenyx recognized $5.4 million in profit sharing and royalty revenue from its collaboration and license agreement with Kyowa Hakko Kirin. This includes $4.4 million in collaboration revenue in the U.S. profit share territory and $1.0 million in royalty revenue in the European territory. Net product sales for Crysvita in other regions were nominal. Mepsevii product revenue for the third quarter of 2018 was $2.1 million, and UX007 named patient revenue was $0.4 million. Ultragenyx recognized $3.6 million in revenue from its research agreement with Bayer.

Operating Expenses

Total operating expenses for the third quarter of 2018 were $101.4 million compared with $83.9 million for the same period in 2017, including non-cash stock-based compensation of $20.7 million and $17.2 million in the third quarter of 2018 and 2017, respectively. Total operating expenses for the nine months ended September 30, 2018, were $316.3 million compared with $232.3 million for the same period in 2017, including non-cash stock-based compensation of $59.0 million and $48.5 million in the first nine months of 2018 and 2017, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows and advances its pipeline.

Cash, Cash Equivalents and Investments

Cash, cash equivalents and investments were $503.1 million as of September 30, 2018.

Recent Updates

Crysvita in X-Linked Hypophosphatemia (XLH)

Ultragenyx submitted regulatory filings in Canada, Brazil and Colombia. We anticipate regulatory decisions in these markets over the course of 2019. Reimbursed named patient treatment has already begun in Argentina in response to physician requests for multiple patients.
Crysvita in Tumor-Induced Osteomalacia (TIO)

Positive longer-term 48-week and 72-week data from the Phase 2 study of Crysvita in adults with TIO were presented at the American Society for Bone and Mineral Research (ASBMR) 2018 Annual Meeting in Montreal. In adults with TIO, Crysvita was associated with increases in serum phosphorus and serum 1,25 dihydroxy vitamin D levels; improvements in osteomalacia, mobility and vitality; and reductions in fatigue. Adverse events (AE) generally reflected the patients’ underlying diseases, and there were no serious treatment-related AEs during the study. Discussions with the U.S. Food and Drug Administration (FDA) regarding the regulatory pathway are ongoing.
Mepsevii in Mucopolysaccharidosis VII (MPS VII)

The European Commission (EC) approved the Marketing Authorization Application (MAA) for Mepsevii under exceptional circumstances for the treatment of non-neurological manifestations of MPS VII. Mepsevii is now approved for use in all 28 EU countries as well as in Iceland, Liechtenstein and Norway, and has launched in Germany.
Brazil’s National Health Surveillance Agency approved Mepsevii for the treatment of MPS VII for patients of all ages. Additional regulatory decisions for patients in Columbia and Chile are anticipated over the course of 2019.
UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

The FDA accepted Ultragenyx’s proposal to submit a New Drug Application (NDA) for UX007 for the treatment of LC-FAOD based on currently available data. Further details will be forthcoming following a pre-NDA meeting, which is scheduled to take place by the end of 2018. Additionally, discussions are progressing with the European Medicines Agency regarding a potential conditional marketing authorization in Europe.

In October 2018, Ultragenyx announced that the Phase 3 study evaluating UX007 in patients with Glut1 DS did not achieve its primary endpoint compared to placebo. The safety profile observed in this study was consistent with what has been previously reported with UX007. Ultragenyx plans to discontinue further Glut1 DS clinical development for UX007, and expects no impact on plans for the LC-FAOD indication.
DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

Positive topline data from the first two dose cohorts of the Phase 1/2 study demonstrate normalization of ureagenesis in two patients and further support proof-of-concept. The first responder (Cohort 1) showed a further increased level of ureagenesis at 52 weeks and has been clinically stable for more than eight months after discontinuing alternate pathway medications and also after liberalizing a protein-restricted diet near the 52 week time point. The second responder (Cohort 2) achieved normalization of ureagenesis at 24 weeks, and has been clinically stable for more than one month after also discontinuing all alternate pathway medications. The remaining four patients in Cohorts 1 and 2 continue to demonstrate no clinically meaningful change in rate of ureagenesis. All patients have remained clinically and metabolically stable, with only modest rises in ALT well treated by a reactive, tapering steroid regimen. The first patient in cohort 3 has been dosed. Data from higher dose Cohort 3 are expected in mid-2019.
DTX401 Gene Therapy in Glycogen Storage Disease Type Ia (GSDIa)

All three patients in the lowest-dose Cohort 1 of the Phase 1/2 study have been dosed. Data from this cohort are expected around the end of 2018.
CDKL5 Deficiency Disorder (CDD)

Ultragenyx exercised its option with REGENXBIO Inc. to develop a gene therapy to treat patients with CDD using REGENXBIO’s adeno-associated virus (AAV) vectors, including AAV9. CDD is a severe and debilitating neurological disorder that shares many features of Rett Syndrome. It was commonly identified as an atypical Rett Syndrome until improved genetic testing led to more accurate diagnosis and an increasing prevalence of diagnosed CDD-affected patients.
Corporate

Ultragenyx appointed a Chief Operating Officer, Wladimir (Vlad) Hogenhuis, M.D., on September 28, 2018. In this newly created role, Dr. Hogenhuis oversees the Global Commercial, Business Development, and Manufacturing organizations.
Conference Call and Webcast Information

Ultragenyx will host a conference call today, Monday, November 5, 2018, at 2 p.m. PT/ 5 p.m. ET to discuss third quarter 2018 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 1290627. The replay of the call will be available for one year.