On May 7, 2018 Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, reported its financial results and corporate update for the quarter ended March 31, 2018 (Press release, Ultragenyx Pharmaceutical, MAY 7, 2018, View Source [SID1234526172]).

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"With the recent approvals and launches of Crysvita in the United States and Europe, we have transformed into a commercial stage company with two medicines now available for patients," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We continue to advance our clinical and preclinical programs and expect significant progress across our two clinical-stage gene therapy programs as well as our small molecule and biologics programs this year."

First Quarter 2018 Financial Results

For the first quarter of 2018, Ultragenyx reported net income of $30.3 million, or $0.63 per basic share and $0.62 per diluted share, compared with a net loss for the first quarter of 2017 of $68.3 million, or $1.63 per share, basic and diluted. The income for the first quarter of 2018 includes the $130 million gain from the sale of the priority review voucher (PRV). The net income for the first quarter of 2018 reflected cash used in operations of $89.5 million compared to $61.2 million reflected in the net loss for the same period in 2017.

For the first quarter of 2018, Ultragenyx reported $10.7 million in total revenue, which includes $1.3 million in product revenue from Mepsevii (vestronidase alfa) and UX007, and $9.4 million in collaboration and license revenue, primarily from our research agreement with Bayer. Total operating expenses for the first quarter of 2018 were $107.2 million compared with $70.0 million for the same period in 2017, including non-cash stock-based compensation of $18.8 million and $14.5 million in the first quarter of 2018 and 2017, respectively. The increase in total operating expenses is due to the increase in development, commercial, and general and administrative costs as the company commercializes, grows and advances its pipeline.

Cash, cash equivalents, and investments were $571.3 million as of March 31, 2018.

Recent Highlights

Crysvita (burosumab) in X-Linked Hypophosphatemia (XLH)

In the U.S., Crysvita was approved on April 17, 2018 and is now commercially available to adults and children with X-linked hypophosphatemia (XLH). In April, the U.S. Food and Drug Administration (FDA) approved Crysvita for the treatment of XLH in adult and pediatric patients one year of age and older. The first patient has now received commercial treatment with Crysvita.

In Europe, burosumab received conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.
DTX301 Gene Therapy in ornithine transcarbamylase (OTC) Deficiency

Data from Phase 1/2 study of DTX301, our AAV8 vector in patients with OTC, showed positive topline results including normalization of ureagenesis in one patient in the first, lowest-dose cohort. The first patient’s rate of ureagenesis was normalized, maintained and then substantially increased over 24 weeks. The second and third patients did not show a clinically meaningful change in rate of ureagenesis over 20 weeks and 12 weeks, respectively. There have been no infusion-related adverse events and no serious adverse events reported. All adverse events have been Grade 1 or 2 and have resolved. Two patients have been enrolled in the higher dose Cohort 2 portion of the study, and data from the second cohort are expected in the second half of 2018.
DTX401 Gene Therapy in glycogen storage disease type Ia (GSDIa)

The U.S. FDA cleared the Investigational New Drug (IND) application for DTX401 for the treatment of patients with GSDIa. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2018, with data from the first cohort in the second half of 2018.
Corporate

PRV sold for $130 million: In January 2018, we completed the sale of the PRV that we received at the time of the approval of Mepsevii.

Equity financing of approximately $271.0 million: In January 2018, we completed an underwritten public offering, with net proceeds of approximately $271.0 million.
Upcoming Key Milestones

Crysvita (burosumab) in XLH

Data from the Phase 3 study in pediatric patients expected in the second half of 2018. The ongoing Phase 3 randomized open-label clinical study is comparing the efficacy and safety of burosumab to oral phosphate and active vitamin D therapy in pediatric patients with XLH. This study will serve as a confirmatory study in Europe.
Crysvita (burosumab) in tumor-induced osteomalacia (TIO)

Data from all patients in Phase 2 study in TIO expected in mid-2018. This is an open label Phase 2 study evaluating the safety and efficacy of burosumab in 17 adult patients with TIO.
Mepsevii (vestronidase alfa) in mucopolysaccharidosis VII (MPS VII)

In Europe, an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in mid-2018.
UX007 in long-chain fatty acid oxidation disorders (FAOD) and glucose transporter type-1 deficiency syndrome (Glut1 DS)

Completing study design of Phase 3 study in patients with FAOD; providing additional data to FDA for consideration of early filing based on Phase 2 data. Following an end-of-phase 2 meeting, we are providing additional information to submit to FDA for consideration of an early filing based on the results from the Phase 2 study. While the FDA still prefers that a randomized controlled trial be completed before filing, it left open the possibility of filing on the current data. We are simultaneously completing the design of a Phase 3 study that could be used for registration or confirmatory purposes. We expect that a decision on a potential filing for approval based on Phase 2 data will be made in mid-2018.

Data from the Phase 3 movement disorder study in patients with Glut1 DS. Enrollment is complete and data are expected in the second half of 2018.
Conference Call & Webcast Information

Ultragenyx will host a conference call today, Monday, May 7, 2018 at 5pm ET to discuss first quarter 2018 financial results and to 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 3748439. The replay of the call will be available for one year.

On May 7, 2018 Omeros Corporation (NASDAQ: OMER) reported that the company will issue its first quarter 2018 financial results for the period ended March 31, 2018, on Thursday, May 10, 2018, after the market closes (Press release, Omeros, MAY 7, 2018, View Source;p=RssLanding&cat=news&id=2347354 [SID1234526171]). Omeros management will host a conference call and webcast that day at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss the financial results.

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Conference Call Details

To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 8579459. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 8579459.

To access the live and subsequently archived webcast of the conference call, go to Omeros’ website at www.omeros.com and go to "Events" under the Investors section of the website. Please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

On May 7, 2018 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage oncology company, reported financial results for the first quarter ended March 31, 2018 (Press release, Mirati, MAY 7, 2018, View Source [SID1234526170]).

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"We have made significant progress in all of our programs and continue to be encouraged by positive clinical results for sitravatinib and mocetinostat with planned data presentations at a fall oncology conference," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer. "Additionally, we are on track to file our planned Investigational New Drug application (IND) for MRTX849, a potent and selective inhibitor for KRAS, in the fourth quarter of 2018."

Financial Results for the First Quarter 2018

Cash, cash equivalents, and short-term investments were $148.7 million at March 31, 2018, compared to $150.8 million at December 31, 2017.

License and collaboration revenues for the first quarter of 2018 were $9.5 million, compared to none in the same period in 2017. License and collaboration revenues relate to the Collaboration and License Agreement between the Company and BeiGene, Ltd. ("BeiGene"), which became effective January 7, 2018, under which the Company granted BeiGene an exclusive license to develop, manufacture and commercialize sitravatinib in Asia (excluding Japan and certain other countries).

Research and development expenses for the first quarter of 2018 were $19.7 million, compared to $14.4 million for the same period in 2017. The increase in research and development expenses is primarily due to an increase in third party research and development expense for sitravatinib due to the continuation and expansion of ongoing clinical trials. The increase is also related to continued development of our KRAS inhibitor program for costs associated with preparing to file a planned IND application for our selected lead clinical compound, MRTX849. These increases are partially offset by a decrease in glesatinib expenses.

General and administrative expenses for the first quarter of 2018 were $5.2 million, compared to $3.7 million for the same period in 2017. The increase is primarily due to an increase in share-based compensation expense due to an increase in the fair value of stock options granted during the three months ended March 31, 2018 compared to the same period in 2017.

Net loss for the first quarter of 2018 was $14.7 million, or $0.51 per share basic and diluted, compared to net loss of $17.8 million, or $0.73 per share basic and diluted for the same period in 2017.

On May 7, 2018 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer,reported financial results for the quarter ended March 31, 2018 (Press release, MacroGenics, MAY 7, 2018, View Source [SID1234526169]).

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"Our momentum continues to build in 2018, as our multiple product candidates advance toward data read-outs," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "During the first quarter, margetuximab passed an interim futility analysis for the SOPHIA Phase 3 metastatic breast cancer study. We also presented clinical data for the combination of margetuximab with an anti-PD-1 agent showing encouraging activity in the treatment of gastric cancer patients in a Phase 2 study. We plan to provide an update on this study, including presentation of biomarker data, at the upcoming ASCO (Free ASCO Whitepaper) meeting. During the second half of the year, we expect to provide clinical updates on both flotetuzumab in patients with relapsed/refractory acute myeloid leukemia (AML), and on the combination of enoblituzumab with an anti-PD-1 agent. In addition, we anticipate that two of our oncology product candidates will move into our clinical pipeline this year: MGD019 (PD-1 x CTLA-4 DART molecule) and MGC018 (B7-H3 ADC), the planned investigational new drug (IND) application submissions for which are both on track."

Key Pipeline Updates

Margetuximab. Recent highlights related to the Company’s Fc-optimized monoclonal antibody (mAb) that targets the human epidermal growth factor receptor 2, or HER2, include:

Phase 3 Metastatic Breast Cancer Study. The pivotal SOPHIA study is evaluating the efficacy of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in approximately 530 relapsed/refractory HER2-positive metastatic breast cancer patients. In January 2018, the Company announced the completion of a pre-planned interim futility analysis with the recommendation of an independent data safety monitoring committee to continue SOPHIA as planned without modification. This analysis was based on a pre-specified assessment of progression-free survival as determined by independent central review. The Company also announced that the U.S. FDA had granted Fast Track designation for the investigation of margetuximab for treatment of patients with metastatic or locally advanced HER2 positive breast cancer who have previously been treated with anti-HER2-targeted therapy. MacroGenics remains on track to complete enrollment of the study in the fourth quarter of 2018, with anticipated disclosure of topline progression-free survival data in the first half of 2019.

Phase 2 Gastric Cancer Study. In January 2018, MacroGenics presented interim clinical data from a Phase 2 study of margetuximab plus an anti-PD-1 agent in patients with gastric and gastroesophageal junction (GEJ) cancer. These results included encouraging tolerability and anti-tumor activity in a subpopulation of 25 gastric cancer patients. Based on these results, MacroGenics expanded the study and is enrolling 25 additional gastric cancer patients. The Company will present updated clinical and biomarker data at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting in June.

Exhibit 99.1

Flotetuzumab. Recent highlights of the Company’s bispecific, humanized DART molecule that recognizes both CD123 and CD3, include:

Monotherapy Study. MacroGenics has completed the enrollment of its AML dose expansion cohort. The Company anticipates presenting updated clinical data and defining a potential registration path during the second half of 2018. The Company’s collaborator, Servier, has development and commercialization rights outside North America, Japan, Korea and India for flotetuzumab, also known as S80880.

Planned Combination Study with an anti-PD-1. MacroGenics has previously presented data supporting the rationale for using checkpoint blockade as an approach to potentially enhance the anti-leukemic activity of flotetuzumab. MacroGenics intends to initiate a combination study with INCMGA0012, an anti-PD-1 mAb also known as MGA012, during the third quarter of 2018.
Other Pipeline Assets Update
Additional programs that the Company is advancing include the following:
PD-1-Directed Immuno-Oncology Franchise. MacroGenics is advancing multiple PD-1-directed programs to enable both a broad set of combination opportunities across the Company’s portfolio and provide further differentiation from existing PD-1-based treatment options. These programs include:

INCMGA0012. INCMGA0012 is a humanized, proprietary anti-PD-1 mAb being developed for use as monotherapy as well as in combination with other potential cancer therapeutics. INCMGA0012 was licensed to Incyte Corporation in 2017 under a global collaboration and license agreement. MacroGenics transferred the INCMGA0012 U.S. IND to Incyte during the first quarter of 2018.

MGD013. MacroGenics designed a DART molecule, MGD013, to provide co-blockade of two immune checkpoint molecules expressed on T cells, PD-1 and LAG-3, for the potential treatment of a range of solid tumors and hematological malignancies. MGD013 is currently being evaluated in a Phase 1 dose escalation study. MacroGenics expects to establish the dose and schedule for MGD013 administration as well as initiate dose expansion cohorts in the second half of 2018.

MGD019. This DART molecule is designed to provide co-blockade of both PD-1 and CTLA-4 on T cells. The Company is completing IND-enabling studies and anticipates submitting the IND application for MGD019 in the second half of 2018.
B7-H3 Franchise. MacroGenics is developing a portfolio of therapeutics that target B7-H3, a member of the B7 family of molecules involved in immune regulation. The Company is advancing multiple programs that target B7-H3 through complementary mechanisms of action that take advantage of this antigen’s broad expression across multiple solid tumor types. These molecules include:

Enoblituzumab: The Company completed the recruitment of patients with four solid tumor types in an ongoing study of this Fc-optimized mAb that targets B7-H3, in combination with an anti-PD-1 mAb and expects to present clinical data from this study in the second half of 2018.

MGD009: This DART molecule targeting B7-H3 and CD3 is being evaluated in a Phase 1 study across multiple solid tumor types. The Company expects to establish the dose and schedule for MGD009 administration as well as initiate monotherapy dose expansion cohorts in the second half of 2018. In addition, a combination study of MGD009 and INCMGA0012 was initiated during the first quarter of 2018.

Exhibit 99.1

MGC018: The Company is completing IND-enabling activities to support submission of an IND application for this anti-B7-H3 antibody drug conjugate (ADC) and anticipates initiation of a Phase 1 study in the second half of 2018.
Additional DART Clinical Programs. Additional DART molecules in Phase 1 clinical development being led by MacroGenics include the following:

MGD007. The Company recently completed a monotherapy study of MGD007, a DART molecule that recognizes gpA33 and CD3, and anticipates commencing a combination study with INCMGA0012 in the second quarter of 2018.

MGD014. MacroGenics’ first DART molecule designed to target an infectious agent, MGD014 recognizes the envelope protein of HIV-infected cells (Env) and the T cells’ CD3 component, to redirect the immune system’s T cells to kill HIV-infected cells. The Company expects to commence the Phase 1 study during the second quarter of 2018.

Corporate Update

Roche Collaboration. In January 2018, MacroGenics announced that it had entered into a research collaboration and license agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (Roche) to jointly discover and develop novel bispecific molecules to undisclosed targets. MacroGenics received an upfront payment of $10 million from Roche in January 2018 and is eligible to receive potential milestone payments and royalties on future sales.

GMP Manufacturing Suite Build-out: The Company is expanding its manufacturing capacity by completing the build-out of a GMP suite in its headquarters building in Rockville, Maryland to support larger-scale clinical and commercial manufacturing. MacroGenics expects to commence GMP production runs in this facility in the third quarter of 2018.

Common Stock Financing. On April 2, 2018, the Company closed its public offering of 5,175,000 shares of common stock. Net proceeds to MacroGenics, after deducting underwriting discounts and commissions and offering expenses, were $103 million.
First Quarter 2018 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2018, were $260.1 million, compared to $305.1 million as of December 31, 2017.

Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $4.7 million for the quarter ended March 31, 2018, compared to $2.1 million for the quarter ended March 31, 2017. Revenue from collaborative agreements includes the recognition of deferred revenue from payments received in previous periods as well as payments received during the year.

R&D Expenses: Research and development expenses were $45.7 million for the quarter ended March 31, 2018, compared to $32.8 million for the quarter ended March 31, 2017. This increase was primarily due to the continued enrollment in the Company’s two margetuximab studies and the INCMGA0012 monotherapy clinical trial.

G&A Expenses: General and administrative expenses were $9.2 million for the quarter ended March 31, 2018, compared to $7.5 million for the quarter ended March 31, 2017. This increase

Exhibit 99.1

was primarily due to consulting and other costs incurred related to the implementation of the Company’s new enterprise resource planning (ERP) system.
Net Loss: Net loss was $49.5 million for the quarter ended March 31, 2018, compared to net loss of $37.7 million for the quarter ended March 31, 2017.

Shares Outstanding: Shares outstanding as of March 31, 2018 were 37,024,623.

Conference Call Information

MacroGenics will host a conference call today at 4:30 pm (ET) to discuss financial results for the quarter ended March 31, 2018 and provide a corporate update. To participate in the conference call, please dial (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and provide the Conference ID: 1647389.
The recorded, listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

On May 7, 2018 Lipocine Inc. (NASDAQ: LPCN), a specialty pharmaceutical company, reported financial results for the first quarter ended March 31, 2018 (Press release, Lipocine, MAY 7, 2018, View Source [SID1234526168]).

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First Quarter and Recent Corporate Highlights

The Company received $10 million through a Loan and Security Agreement with Silicon Valley Bank ("SVB Loan").
·The Bone, Reproductive and Urologic Drugs Advisory Committee ("BRUDAC") of the U.S. Food and Drug Administration ("FDA") met to discuss the New Drug Application ("NDA") for TLANDO, Lipocine’s oral testosterone product candidate for the proposed indication of testosterone replacement therapy in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism.
BRUDAC voted six in favor and thirteen against the acceptability of the overall benefit/risk profile to support approval of TLANDO as a testosterone replacement therapy ("TRT").
·The Company and the other defendants entered into a memorandum of understanding to settle the purported securities class action litigation captioned In re Lipocine Inc. Securities Litigation.
·The Company initiated a phlebotomy clinical study under the TLANDO investigational new drug ("IND") Application to confirm no ex-vivo conversion of testosterone undecanoate to testosterone.
·The Company submitted a draft protocol for an ambulatory blood pressure monitoring ("ABPM") clinical study to the FDA for review under the TLANDO IND.
The FDA’s assigned Prescription Drug User Fee Act ("PDUFA") goal date for the TLANDO NDA is May 8, 2018.

"We look forward to learning the FDA outcome on our PDUFA goal date for TLANDO. We continue to believe that as an oral drug TLANDO offers significant benefits to patients compared to topical gels and injections. These benefits include overcoming the inadvertent testosterone transference risk to children and partners that exist with topical gels," said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine.

First Quarter 2018 Financial Results

Lipocine reported a net loss of $2.7 million, or ($0.13) per diluted share, for the quarter ended March 31, 2018, compared with a net loss of $4.9 million, or ($0.26) per diluted share, in the quarter ended March 31, 2017.

License revenues were $428,000 during the three months ended March 31, 2018, compared to no revenue being received during the three months ended March 31, 2017. License revenue relates to royalty payments received from Spriaso, LLC under a licensing agreement for the cough and cold field.

Research and development expenses were $1.4 million in the quarter ended March 31, 2018, compared with $3.1 million in the quarter ended March 31, 2017. The decrease in research and development expenses was primarily due to reduced contract research organization costs for TLANDO and lower personnel costs offset by increased outside service costs primarily related to the TLANDO BRUDAC meeting in January 2018 and increased contract manufacturing costs for LPCN 1107.

General and administrative expenses were $1.7 million in the quarter ended March 31, 2018, compared with $1.8 million in the quarter ended March 31, 2017. The decrease in general and administrative expenses was primarily due to decreased personnel costs and overhead costs offset by increased professional fees related to legal, intellectual property and commercial activities.

As of March 31, 2018, the Company had cash, cash equivalents, and marketable securities of $27.8 million, compared to cash, cash equivalents, and marketable securities of $21.5 million at December 31, 2017. In the event TLANDO is not approved by the FDA on or prior to May 31, 2018, the SVB loan requires $5.0 million of cash to be restricted and held as cash collateral until such time as TLANDO is approved by the FDA.