On February 28, 2018 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported that it will host a full-year 2017 investor call on Friday, March 9, 2018 at 8:00 AM (EST) to discuss financial results and provide a corporate update (Press release, Pieris Pharmaceuticals, FEB 28, 2018, View Source [SID1234524247]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To access the call, participants may dial 877-407-8920 (US & Canada) or 412-902-1010 (International) at least 10 minutes prior to the start of the call.

An archived replay of the call will be available for 30 days by dialing 877-660-6853 (US & Canada) or 201-612-7415 (International) and providing the Conference ID #: 13661472.

On February 28, 2018 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported its fourth quarter business update and full year results for 2017 (Press release, argenx, FEB 28, 2018, View Source;p=RssLanding&cat=news&id=2335501 [SID1234524272]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The full year results will be discussed during a conference call and webcast presentation today at 3 pm CET/ 9 am EST. To participate in the conference call, please select your dial in number from the list included below, and use the confirmation code 6683242. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

"2017 was a year of tremendous momentum for argenx as we made progress on our pipeline and increased value for our shareholders. As a result of this focused execution, we believe we are well-positioned to advance our pipeline through several key clinical milestones in the year ahead," commented Tim Van Hauwermeiren, CEO of argenx. "In December, we announced positive clinical data from our Phase 2 trial of ARGX-113 for the treatment of generalized myasthenia gravis and plan to advance ARGX-113 to Phase 3 in this indication. This also marked the first dataset from our Phase 2 program aimed at evaluating the role of ARGX-113 in indications where the reduction of pathogenic autoantibodies may mediate disease. We continue to evaluate the asset in two additional Phase 2 indications, immune thrombocytopenia and pemphigus vulgaris, and expect to report data from both in the second half of 2018. We also advanced our lead oncology asset and reported interim data from our Phase 1/2 trial of ARGX-110 in acute myeloid leukemia, which we expect to transition into a Phase 2 trial this year. We feel that our achievements in 2017 have further validated our disciplined and differentiated approach to antibody development and look forward to providing updates on several important catalysts from our wholly-owned pipeline and strategic collaborations in 2018."

FOURTH QUARTER 2017 AND RECENT HIGHLIGHTS

Reported positive topline results from Phase 2 proof-of-concept trial of ARGX-113 (efgartigimod) in generalized myasthenia gravis (MG) showing a strong clinical improvement over placebo throughout the entire duration of the trial as well as a favorable tolerability profile consistent with Phase 1 data.
Launched Phase 1 trial with subcutaneous formulation of ARGX-113 evaluating pharmacokinetics (PK), pharmacodynamics (PD), safety and tolerability.
Presented updates on Phase 1/2 clinical trials of ARGX-110 in acute myeloid leukemia (AML) and cutaneous T-cell lymphoma (CTCL) during American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
Raised approximately $266 million in gross proceeds in an upsized U.S. public offering.

FINANCIAL HIGHLIGHTS (as of December 31, 2017) (compared to financial highlights as of December 31, 2016)

Operating income of €41.3 million (December 31, 2016: €17.2 million).
Net loss of €28.1 million (December 31, 2016: €21.4 million).
Cash position of €359.8 million (cash, cash equivalents and current financial assets) allowing us to pursue development of our pipeline as planned.

DETAILS OF OPERATIONAL RESULTS

Products in Clinical Development:

ARGX-113 (efgartigimod)

Reported positive topline results from Phase 2 proof-of-concept trial of ARGX-113 in generalized MG. ARGX-113 treatment resulted in a strong clinical improvement over placebo as measured by all four predefined clinical efficacy scales throughout the entire duration of the study. Among ARGX-113 treated patients, 75% had a clinically meaningful and statistically significant improvement for a period of at least six consecutive study weeks versus 25% of patients on placebo. Primary endpoint analysis revealed ARGX-113 to be well-tolerated in all patients and consistent with Phase 1 data.
Nearing complete enrollment in the Phase 2 clinical trial of ARGX-113 in immune thrombocytopenia (ITP). Amended ITP trial protocol to extend the follow-up period from eight weeks to 21 weeks. In addition, a one-year open label extension study was added as a second amendment to allow (re)treatment of the ITP patients from the first study (dosed at 10 mg/kg).
Launched Phase 1 trial to evaluate the PK, PD, safety and tolerability of a subcutaneously administered formulation of ARGX-113 in healthy volunteers.

ARGX-110

Provided updates on Phase 1/2 clinical trials of ARGX-110 in AML and CTCL during ASH (Free ASH Whitepaper) Annual Meeting.
Data from the Phase 1/2 clinical trial of ARGX-110 in combination with azacitidine in six newly diagnosed AML patients unfit for intensive chemotherapy showed signs of clinical activity, including complete remission (3/6 patients), complete remission with incomplete blood count recovery (1/6 patients) and partial response (2/6 patients). One of the patients that achieved a complete remission bridged to allogeneic stem cell transplant after five cycles. The preliminary data from the first set of patients suggest ARGX-110 is active both at the circulating and bone marrow blast levels and at the leukemic stem cell level. The data showed a favorable tolerability profile with no exacerbation of azacitidine toxicity.
Amended Phase 1/2 AML trial protocol to add a 20mg/kg dose cohort to the dose escalation to best inform the recommended dose for a Phase 2 study.
Data from the Phase 1/2 clinical trial of ARGX-110 in relapsed/refractory CTCL patients. Of the 22 CTCL patients under analysis, we observed one complete response, two partial responses and 10 patients with stable disease. ARGX-110 was observed to have a favorable tolerability profile in these CTCL patients.

Collaborations

Bird Rock Bio, Inc. (BRB) and argenx have mutually agreed to terminate BRB’s license agreement to develop and commercialize ARGX-109 (gerilimzumab). Genor, a sublicensee of Bird Rock Bio, will continue to develop ARGX-109 for the Chinese market.

Corporate

Intellectual property portfolio now includes 80 granted and 109 pending patents as of December 31, 2017.
Company expanded to 95 employees and consultants in support of the growth of the business.
Established argenx US, Inc., a subsidiary located in Boston, MA.
Nominated James Michael Daly as a new member of the Board of Directors, subject to approval by the shareholders at the annual general meeting.

OUTLOOK 2018
argenx continues to implement its business plan through advancing its deep pipeline of differentiated antibody-based therapies, including ARGX-113, ARGX-110, ARGX-115 and ARGX-112, the forging of collaborations with pharmaceutical companies and leading academic labs under its Innovative Access Program and the strengthening of its shareholder base.

In 2018, argenx aims to execute its ambitious business plan as follows:

Report full data from the Phase 2 proof-of-concept trial for ARGX-113 in generalized MG at the American Academy of Neurology conference (April 21-27, 2018,Los Angeles, CA)
Report topline data of the Phase 2 proof-of-concept trial for ARGX-113 in ITP and interim data of the Phase 2 proof-of-concept trial in pemphigus vulgaris in the second half of 2018. Report full data of the Phase 2 proof-of-concept trial for ARGX-113 in ITP before the end of the year.
Progress ARGX-113 into Phase 3 clinical development in generalized MG before the end of the year.
Report the full data of the Phase 1 healthy volunteer trial with the subcutaneous formulation of ARGX-113 during the second quarter of the year.
Provide an update on the Phase 1/2 clinical trial in AML in the second half of the year.
Report the full data of the AML Phase 1/2 and CTCL Phase 2 clinical trials of ARGX-110 by the end of the year.
Launch the Phase 2 proof-of-concept trial for ARGX-110 in AML before the end of the year.

KEY FIGURES (CONSOLIDATED)
Year Ended
December 31,
in thousands of € 2017 2016 Variance
Revenue € 36,415 € 14,713 € 21,702
Other operating income 4,841 2,439 2,402
Total operating income 41,256 17,152 24,104
Research and development expenses (51,740) (31,557) (20,183)
Selling, general and administrative expenses (12,448) (7,011) (5,437)
Operating loss € (22,932) € (21,416) € (1,516)
Financial income 1,250 73 1,177
Financial expenses – – –
Exchange losses (5,797) (31) (5,766)
Loss before taxes € (27,479) € (21,374) € (6,105)
Income tax income expense € (597) € – € (597)
TOTAL COMPREHENSIVE LOSS OF THE PERIOD € (28,076) € (21,374) € (6,702)

Net increase in cash, cash equivalents and current financial assets compared to year-end 2016 and 2015 263,047 54,402
Cash, cash equivalents and current financial assets at the end of the period 359,775 96,728

DETAILS OF THE FINANCIAL RESULTS

Cash, cash equivalents and current financial assets totaled €359.8 million for the year ended on December 31, 2017, compared to €96.7 million on December 31, 2016. The increase in the year-end cash balance on December 31, 2017 resulted primarily from €304.7 million of net proceeds received from the initial and follow-on U.S. public offerings of American Depositary Shares on the Nasdaq Global Select Market completed respectively in May and December 2017.

Operating income increased by €24.1 million for the year ended December 31, 2017 to reach €41.3 million, compared to €17.2 million for the year ended December 31, 2016. The increase primarily related to a €11.0 million increase in the recognition of upfront payments and a €9.2 million increase in milestone payments from our collaboration partners.

Research and development expenses totaled €51.7 million and €31.6 million for the years ended December 31, 2017 and 2016, respectively. The increase is mainly the result of higher external research and development expenses, reflecting higher clinical trial costs and manufacturing expenses related to the development of our product candidate portfolio.

Selling, general and administrative expenses totaled €12.4 million and €7.0 million for the years ended December 31, 2017 and 2016, respectively. The increase of €5.4 million in selling, general and administrative expenses for the year ended December 31, 2017 primarily resulted from higher personnel expenses, office costs and consulting fees incurred to support our growth and prepare the company to become and operate as a Nasdaq-listed company.

For the year ended December 31, 2017, financial income amounted to €1.3 million compared to €0.1 million for the year ended December 31, 2016. The increase of €1.2 million relates to (i) a €0.9 million realized gain on the sale of a participation in FairJourney Biologics LDA in December 2017 and (ii) an increase in the interest received on our cash, cash equivalents and current financial assets.

Exchange losses totaled €5.8 million for the year ended December 31, 2017 compared to €0.03 million for the year ended December 31, 2016. The increase is mainly attributable to unrealized exchange rate losses on our cash and current financial assets position in U.S. dollars due to the unfavourable fluctuation of the EUR/USD exchange rate.

The total comprehensive loss for the year ended December 31, 2017 was €28.1 million compared to €21.4 million for the year ended December 31, 2016.

U.S. SEC and statutory financial reporting
argenx’s primary accounting standard for quarterly earnings releases and annual reports is International Financial Reporting Standars (IFRS) as issued by the International Accounting Standards Board (IASB). Quarterly summarized statements of profit and loss based on IFRS as issued by the IASB are available on www.argenx.com.

In addition to reporting financial figures in accordance with IFRS as issued by the IASB, argenx also reports financial figures in accordance with IFRS as adopted by the European Union (EU) for statutory purposes. The consolidated statement of financial position, the consolidated statements of profit and loss, the consolidated statements of cashflow, and the consolidated statement of changes in equity are not affected by any differences between IFRS as issued by the IASB and IFRS as adopted by the EU.

The consolidated statement of profit and loss data of argenx SE as of December 31, 2017 presented in this press release are unaudited.

Annual Report 2017
argenx expects to publish its 2017 Annual Report based on IFRS as issued by the IASB and its 2017 Annual Report for statutory purposes based on IFRS as adopted by the EU on March 26, 2018. These Annual Reports will be available on www.argenx.com.

FINANCIAL CALENDAR:

May 8, 2018: Annual General Meeting
May 9, 2018: Q1 2017 Business Update and financial results
August 2, 2018: Half-year 2017 Business Update and financial results
October 25, 2018: Q3 2017 Business Update and financial results

Dial-in numbers:
Please dial in 5-10 minutes prior to 3 pm CET/ 9 am EST using the number and conference ID below.

Confirmation Code: 6683242
Belgium 0800 58228
Belgium +32 (0)2 404 0659
France 0805 101 219
France +33 (0)1 76 77 22 74
Netherlands 0800 023 1436
Netherlands +31 (0) 20 721 9251
United Kingdom 0800 358 6377
United Kingdom +44 (0)330 336 9105
United States 888-394-8218
United States +1 323-794-2149

A question and answer session will follow the presentation of the results. Go to www.argenx.com to access the live audio webcast. The archived webcast will also be available (90 days) for replay shortly after the close of the call from the "Downloads" section of the argenx website.

On february 28, 2018 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and wet age‐related macular degeneration, reported financial results for the fourth quarter and year ended December 31, 2017 (Press release, Tracon Pharmaceuticals, FEB 28, 2018, View Source [SID1234524975]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Fourth Quarter 2017 and Recent Corporate Highlights

In February, TRACON’s partner, Santen Pharmaceutical Co. Ltd. (Santen), reported Phase I/II data for DE‑122 (carotuximab) in patients with refractory wet age-related macular degeneration (AMD) at the 15th Annual Angiogenesis, Exudation, and Degeneration meeting organized by Bascom Palmer Eye Institute. The study assessed the safety, tolerability and bioactivity of a single intravitreal injection of DE-122 at four dose levels in 12 subjects with wet AMD refractory to vascular endothelial growth factor (VEGF) inhibitors. Improvement was observed in mean change in central retinal subfield thickness (CST) based on the spectral domain optical coherence tomography (SD-OCT) suggesting bioactivity of DE-122 in these refractory wet AMD patients. No serious adverse events were reported. Based on these results, Santen previously advanced DE-122 into a Phase 2a randomized controlled trial assessing the efficacy and safety of intravitreal injections in combination with Lucentis (ranibizumab) compared to Lucentis monotherapy in patients with wet AMD that continues to enroll subjects, and which resulted in TRACON receiving a $7.0 million milestone payment.

In January, initial data from the ongoing open-label, non-randomized Phase 1b/2 study of TRC105 and Nexavar (sorafenib) in patients with advanced hepatocellular carcinoma (HCC) were presented by Dr. Kanwal Raghav from the University of Texas MD Anderson Cancer Center at the 2018 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium. Partial responses by RECIST 1.1 occurred in 25% (2 of 8) of evaluable patients and a reduction of 50% or greater in alpha fetoprotein (AFP) concentration occurred in 38% (3 of 8) of evaluable patients, including both partial responses. Reduction in AFP, a tumor marker expressed in patients with HCC, in early treatment may help identify a favorable response to treatment. Adverse events typical of each drug were observed but did not increase in frequency or severity when the drugs were administered concurrently. The 25% response rate seen in this trial is consistent with the 25% (5 of 20) response rate reported in a Phase 1/2 trial of TRC105 and Nexavar conducted by the National Cancer Institute and published in Clinical Cancer Research in 2017. The response rates observed in both trials compare favorably with the 2% and 3% response rates in HCC patients reported for single agent Nexavar in its pivotal studies. The trial is currently ongoing, and we expect to complete the enrollment of approximately 33 patients by the end of 2018.

In December, TRACON licensed development and commercialization rights outside of ophthalmology indications to TRC105 in China, Hong Kong, Macau, and Taiwan to Ambrx. Deal terms included a $3.0 million upfront payment to TRACON, up to $140.5 million in development and commercial milestones and potential high single digit to low teen royalties from net sales of TRC105 in the Ambrx territories. Ambrx is expected to file an IND later this year and to subsequently begin clinical development of TRC105 in China.

In December, Brian Daniels, M.D., was appointed to TRACON’s Scientific Advisory Board. Dr. Daniels was Senior Vice President at Bristol-Myers Squibb until 2014, where he chaired the development investment committee and oversaw the development of Sprycel (dasatinib), Ixempra (ixabepilone), Yervoy (ipilumumab), Empliciti (elotuzumab), and Opdivo (nivolumab). He is currently a venture partner at 5AM Ventures, and previously held senior level development-related positions at Merck and Genentech. In addition, Dr. Daniels previously served in multiple academic roles in the Department of Medicine at the University of California, San Francisco.

In November, the first patient was dosed in a Phase 1b clinical trial of TRC105 in combination with Opdivo in patients with non-small cell lung cancer. The Phase 1b clinical trial is an open-label, dose-escalation and expansion cohort study of TRC105 and Opdivo in patients with non-small cell lung cancer that have received prior chemotherapy. The primary objectives of the Phase 1b study are to assess the safety of TRC105 when dosed with Opdivo, determine its recommended Phase 2 dose and evaluate the response rate. The trial incorporates tumor biopsy testing to determine if there is a correlation of tumor myeloid cell infiltration with response, in order to allow for potential biomarker-directed therapy in lung cancer patients.

In November, TRACON presented updated data from the Phase 1b/2 study of TRC105 and Votrient (pazopanib) in patients with soft tissue sarcoma, including angiosarcoma, at the Connective Tissue Oncology Society (CTOS) annual meeting. Median progression-free survival (PFS) was 7.8 months in 13 VEGF inhibitor naïve angiosarcoma patients treated with the combination of TRC105 and Votrient using either 10 mg/kg weekly dosing or the hybrid dosing schedule of TRC105. The reported median PFS compares favorably to the median PFS of 3 months reported in a retrospective study of single agent Votrient in patients with angiosarcoma. In the 17 patients who received prior treatment for metastatic disease, treatment duration on TRC105 and Votrient exceeded the duration of the most recent prior therapy in 7 of 12 VEGF naïve angiosarcoma patients and 2 of 5 patients who received a VEGF inhibitor as part of their most recent prior therapy. Treatment with the combination of TRC105 and Votrient continued to be well-tolerated and allowed for dosing of the combination for more than two years in the two patients who experienced complete responses to treatment.

Enrollment continues in the Phase 1/2 trial of TRC253, TRACON’s product candidate for treating prostate cancer that was in-licensed from Janssen. The Phase 1/2 trial is designed to assess the safety, determine a recommended Phase 2 dose and assess response by prostate-specific antigen (PSA) levels. If Janssen opts to reacquire TRC253 prior to or following completion of the Phase 1/2 trial, TRACON is entitled to receive a $45.0 million opt-in payment, up to $137.5 million in potential milestone payments and a low single digit royalty.
"We achieved important progress across our pipeline and continued to position the Company well for long-term success during the fourth quarter and throughout 2017," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Most importantly, our pivotal Phase 3 TAPPAS trial of TRC105 in angiosarcoma continues to enroll well across sites in the U.S. and now Europe, with the key interim analysis projected for later this year. Moreover, our recent partnership with Ambrx expands the development and potential commercial opportunity of TRC105 into China."

Expected Additional 2018 Milestones

Completion of the dose escalation portion of the Phase 1/2 trial of TRC253 in patients with prostate cancer is expected in mid-2018.

Announcement of top-line data from the randomized Phase 2 TRAXAR trial of TRC105 in combination with Inlyta for patients with advanced or metastatic renal cell carcinoma is expected in mid-2018.

Announcement of the results of the interim analysis from the Phase 3 pivotal TAPPAS trial of TRC105 in angiosarcoma is expected in the second half of 2018.

Presentation of data from the Phase 1b trial of TRC105 in combination with Opdivo in patients with non-small cell lung cancer is expected in the second half of 2018.
Fourth Quarter 2017 Financial Results

Cash, cash equivalents and short-term investments were $34.5 million at December 31, 2017, compared to $44.4 million at December 31, 2016.

There was no collaboration revenue for the fourth quarter of 2017 compared to $0.6 million for the fourth quarter of 2016.

Research and development expenses for the fourth quarter of 2017 were $4.6 million compared to $4.8 million for the fourth quarter of 2016.

General and administrative expenses for the fourth quarter of 2017 were $1.7 million compared to $1.9 million for the fourth quarter of 2016.

Net loss for the fourth quarter of 2017 was $6.6 million compared to a net loss of $6.3 million for the fourth quarter of 2016.
Investor Conference Call

The Company will hold a conference call today at 4:30 p.m. EST / 1:30 p.m. PST to provide an update on corporate activities and to discuss the financial results of its fourth quarter of 2017. The dial-in numbers are (855) 779‑9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 1894785. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Carotuximab (TRC105)

TRC105 is a novel, clinical stage antibody to endoglin, a protein overexpressed on proliferating endothelial cells that is essential for angiogenesis, the process of new blood vessel formation. TRC105 is currently being studied in a pivotal Phase 3 trial in angiosarcoma and multiple Phase 2 clinical trials, in combination with VEGF inhibitors. TRC105 has received orphan designation for the treatment of soft tissue sarcoma in both the U.S. and EU. The ophthalmic formulation of TRC105, DE-122, is currently in a randomized Phase 2 trial for patients with wet AMD. For more information about the clinical trials, please visit TRACON’s website at www.traconpharma.com/clinical_trials.php.

About TRC253

TRC253 is a novel, orally bioavailable small molecule that is a potent, high affinity competitive inhibitor of the androgen receptor (AR) and AR mutations, including the F876L (also known as F877L) mutation. The AR F876L mutation results in an alteration in the AR ligand binding domain that confers resistance to therapies for prostate cancer. Activation of the AR is crucial for the growth of prostate cancer at all stages of the disease. Therapies targeting the AR have demonstrated clinical efficacy by extending time to disease progression, and in some cases, the survival of patients with metastatic castration-resistant prostate cancer. However, resistance to these agents is often observed and several molecular mechanisms of resistance have been identified, including gene amplification, overexpression, alternative splicing, and point mutation of the AR.

On February 28, 2018 Foamix Pharmaceuticals Ltd. (NASDAQ: FOMX) ("Foamix Pharmaceuticals" or the "Company"), a clinical stage specialty pharmaceutical company focused on developing and commercializing proprietary topical foams to address unmet needs in dermatology, reported financial results for the fiscal year ended December 31, 2017 (Press release, Foamix, FEB 28, 2018, View Source [SID1234524231]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Clinical and Corporate Update:

FMX101, the Company’s minocycline foam 4% is currently being evaluated in a third Phase 3 study (Study 22) in patients with moderate-to severe acne.
Top-line results are expected in the third quarter of 2018.
The expected timing of an NDA filing for FMX101 is planned for the end of 2018.
FMX103, 1.5% minocycline foam, is currently being evaluated in two Phase 3 studies (Studies 11 and 12) in patients with moderate-to severe papulopustular rosacea. Long term safety is concurrently being evaluated in the open label Study 13.
Top-line results are expected in late third quarter or early fourth quarter of 2018.
Enrollment in the open-label long term safety study (Study 13) continues to progress as scheduled. A significant number of patients have enrolled into this study and we continue to observe low discontinuation rates.
The expected timing of an NDA filing for FMX103 is planned for 2019.
In January and February 2018, Bayer and Foamix filed joint complaints against each of Teva and Perrigo, respectively, alleging patent infringement under U.S. patent laws arising out of the submissions by Teva and Perrigo of ANDAs seeking approval to manufacture and sell generic versions of Bayer’s Finacea Foam. We are committed to defending our own intellectual property rights globally, including patents we have licensed to other pharmaceutical companies as part of our collaboration efforts.
In January 2018, as part of the orderly transition in management, CEO David Domzalski was appointed to the Board of Directors to replace former CEO and co-founder Dr. Dov Tamarkin.
In January 2018, the Company changed its filing status with the SEC and Nasdaq from a "Foreign Private Issuer" to a U. S. Domestic filer.

FMX101 Open Label Safety Study Results

A total of 657 patients were enrolled in the open-label safety extension after an initial 12 weeks of double-blinded therapy in Phase 3 studies 04 and 05. Of these, 291 completed an additional 40-weeks for a total of 52 weeks on FMX101 therapy. The primary endpoint was safety.
As previously communicated, no serious drug-related adverse events were reported during the open-label safety extension, validating earlier data demonstrating that FMX101 appears to be well tolerated with an acceptable safety profile. Specific findings included:
Non-dermal adverse events were comparable in type and frequency with those reported during the double-blinded portion of Studies 04 and 05, with the most frequently reported treatment-emergent adverse event being nasopharyngitis (common cold).
Application-site adverse events occurred in less than 2% of patients during the additional 40 weeks of open-label treatment. Four patients discontinued the study for an application-site adverse event.
In the assessment of facial dermal tolerability at week 52, more than 95% of patients had "none" or "mild" scoring of erythema, dryness, hyperpigmentation, peeling, and itching. No severe local tolerability scores were recorded.

Efficacy Results at 52 weeks:

Efficacy was also measured as a secondary endpoint in the open-label study for FMX101. During the study, patients were allowed to discontinue therapy with FMX101 if they believed their acne had sufficiently improved. Patients could re-start therapy as needed and were also allowed to use other acne medications concomitantly.
The following summarizes efficacy results for subjects who had been assigned FMX101 therapy for 52 weeks:
At week 52, 37.7% of patients from Study 04 had an Investigator’s Global Assessment (IGA) score of 0 (clear) or 1 (almost clear); 50.3% of subjects from Study 05 had an IGA score of 0 or 1.
At week 52, patients from Study 04 had a 64.3% reduction in inflammatory lesions; patients from Study 05 had a 78% reduction in inflammatory lesions.
At week 52, patients from Study 04 had a 52.5% reduction in non-inflammatory lesions; patients from Study 05 had a 59.6% reduction in non-inflammatory lesions.

Details on the open-label study results for FMX101 are contained within the most recent Investor Presentation, available on the Company’s website at View Source

Financial Results for the Year Ended December 31, 2017

Revenues
Revenues for the year ended December 31, 2017 were $3.7 million, compared with $5.5 million for the same period in 2016. The decrease is mainly due to a decrease of $2.5 million in contingent payments from Bayer, that were payable for 2016 due to Bayer’s achievement of certain sale targets during that year, offset by an increase in royalty payments in the amount of $565,000 from Bayer for the sales of Finacea Foam.

Operating Expenses
Research and Development Expenses
Research and development expenses for the year were $57.8 million, compared to $25.9 million in 2016. The increase in research and development expenses resulted primarily from an increase of $28.0 million in costs relating predominantly to FMX101 and FMX103 clinical trials and an increase of $3.0 million in payroll and payroll related expenses primarily due to an increase in headcount.

Selling, General and Administrative Expenses
Selling, general and administrative expenses for the year were $11.5 million, compared to $9.2 million in 2016. The increase in selling, general and administrative expenses resulted primarily from an increase of $1.9 million in payroll and other payroll-related expenses mostly due to an increase in headcount and salary raises.

Net Loss
For the year ended December 31, 2017, the Company recorded a net loss of $65.7 million, or $1.76 per share, basic and diluted, compared with a loss of $29.3 million or $0.91 per share, basic and diluted, for the year ended December 31, 2016.

Cash & Cash Equivalents
At December 31, 2017, the Company had $76.4 million in cash and investments compared to $131 million at the end of December 2016. The Company believes, based on its current business plan, that its existing cash, cash equivalents and marketable securities will fund operating expenses and capital expenditure requirements throughout the completion of its third pivotal Phase 3 clinical trial for its lead product candidate FMX101 and its two pivotal Phase 3 clinical trials for FMX103.

Conference Call & Webcast
Wednesday, February 28 @ 8:30am Eastern Time
Toll Free: 800-289-0438
International: 323-794-2423
Conference ID: 2903389
Webcast: View Source

Replays, Available through March 14:
Toll-Free: 844-512-2921
International: 412-317-6671
Conference ID: 2903389

On February 28, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, today explains PharmaCyte’s plans to conduct a Phase 2b clinical trial (Press release, PharmaCyte Biotech, FEB 28, 2018, View Source [SID1234524256]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

When PharmaCyte met with the FDA in January of 2017, PharmaCyte’s pre-IND meeting submission was predicated on PharmaCyte conducting a Phase 2b trial. During discussions with the U.S. Food and Drug Administration (FDA), PharmaCyte asked whether the data from that planned trial could be considered "pivotal" and thus support registration for marketing purposes. The FDA indicated that this was a possibility, but that the trial would have to be much larger than PharmaCyte was planning and the data would have to be markedly superior to the data seen with the comparator treatment.

PharmaCyte’s decision to conduct a Phase 2b trial rather than a pivotal trial was made relatively recently based on advice from PharmaCyte’s consulting oncologists, Chief Medical Officer and information obtained from PharmaCyte’s Advisory Board. PharmaCyte must rely on data from two European trials from 27 patients that were conducted about 20 years ago. The data from these trials are incomplete when compared to what is now required by the FDA to support a pivotal trial.

The Phase 2b trial is designed to determine how effective and safe multiple courses of ifosfamide will be in patients with locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) and how PharmaCyte’s treatment compares to a commonly used treatment for LAPC after patients’ tumors no longer respond following 4 to 6 months of the combination therapy of gemcitabine and Abraxane. PharmaCyte is designing a Phase 2b clinical trial that, if successful, it believes will give the company a much more solid foundation for dealing with the FDA with the goal of bringing its pancreatic cancer therapy to market.

The planned Phase 2b trial will be significantly larger than the original Phase 2b trial PharmaCyte previously discussed with the FDA and will include multiple courses of low dose ifosfamide (the earlier trials used only two courses). This trial will also provide better statistical analyses of PharmaCyte’s therapy for LAPC and the comparator arm in terms of survival and safety. Also, the possibility exists that if the data from PharmaCyte’s therapy are significantly better than the data from the comparator arm, this may allow PharmaCyte to apply to the FDA for accelerated approval.