ATHERSYS ANNOUNCES FINANCIAL RESULTS FOR FOURTH QUARTER AND FULL YEAR 2018

On March 14, 2019 Athersys, Inc. (NASDAQ: ATHX) reported its fourth quarter 2018 and annual 2018 financial results and recent highlights (Press release, Athersys, MAR 14, 2019, View Source [SID1234534341]).

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"As we have announced previously, we had a number of important accomplishments in 2018, including the initiation of our Phase 3 MASTERS-2 study and the expansion of our partnership with Healios. Additionally, we completed enrollment of, and recently announced positive results for, our exploratory clinical study of MultiStem treatment of acute respiratory distress syndrome patients," commented Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys. "We also finished the year in a meaningfully stronger financial position, which was an important objective."

Fourth Quarter 2018 and Recent Highlights:

Announced positive results from our exploratory clinical study of MultiStem cell therapy for treatment of acute respiratory distress syndrome (ARDS), further confirming the tolerability and safety profile of MultiStem treatment and demonstrating trends of lower mortality and greater ventilator-free and ICU-free days; the study has been selected for presentation at the American Thoracic Society International Conference in May 2019;
Our partner, HEALIOS K.K. (Healios), announced plans to initiate an ARDS trial using MultiStem therapy for patients in Japan, which, if successful could lead to registration under Japan’s regenerative medicine regulatory framework;
Advanced our ischemic stroke program through continued support of Healios’ Japan TREASURE trial and enrollment of our MASTERS-2 Phase 3 registration study for ischemic stroke;
Received a $2.0 million payment from Healios for a right of first negotiation through June 2019 for an option to develop and commercialize MultiStem therapy for certain indications in China; Healios may extend the negotiation period through December 2019 with an additional payment of $3.0 million;
Recognized revenues of $1.5 million and net loss of $11.3 million, or $0.08 net loss per share, for the quarter ended December 31, 2018; and
Ended 2018 with $51.1 million in cash and cash equivalents and February 28, 2019 with $51.5 million in cash and cash equivalents, reflecting a solid financial foundation.
Other 2018 Highlights:

Expanded our collaboration with Healios in June 2018 to include additional areas – such as development for the treatment of ARDS in Japan, iPSC and MultiStem cells in combination to treat dysfunction in certain organs in Japan, and potential use of MultiStem cells alone or with RPE cells for certain ophthalmological indications globally – for $20 million in license fees, plus potential milestone payments and royalties; this followed a $21.1 million investment by Healios through the purchase of our common stock in March 2018;
Commenced the MASTERS-2 Phase 3 registration study for ischemic stroke and started enrolling patients;
Completed the enrollment of our exploratory clinical study of MultiStem cell therapy treatment for ARDS, and announced positive results soon thereafter as noted above;
Announced grant funding and began preparations to conduct a Phase 2 clinical trial evaluating MultiStem cell therapy for early treatment and prevention of complications after severe traumatic injury, in collaboration with The University of Texas Health Science Center at Houston and Hermann Memorial Trauma Center;
Expanded our process development and manufacturing efforts, including strategic leadership hires and diversification in our manufacturing networks; and
Entered into a new equity facility during the first quarter of 2018 as a follow-on to the existing facility, giving us the right to sell up to $100 million of common stock over a three-year period, providing access to capital, as needed, to support operations.
"We believe we are well-positioned to capitalize on our innovative MultiStem product platform and to develop and deliver highly effective new treatments to patients in areas of substantial unmet medical need, particularly in the critical care area. The Healios’ TREASURE trial and our MASTERS-2 trial are making continued progress, and the results from our exploratory ARDS trial illustrate the potential of MultiStem therapy in other acute care settings. We continue to work toward the scale-up of our manufacturing capabilities and to focus on the further development of other core capabilities and programs, while we continue to explore additional partnering opportunities," concluded Dr. Van Bokkelen.

Fourth Quarter 2018 Financial Results

Revenues increased to $1.5 million for the three months ended December 31, 2018 compared to $1.2 million for the three months ended December 31, 2017. Our revenues are generally derived from license fees, manufacturing-related services for Healios, royalty and related contract revenue from our collaborations, and grant revenue.

Research and development expenses decreased to $10.2 million for the three months ended December 31, 2018 from $12.1 million for the comparable period in 2017. In 2017, approximately $4.7 million of license fees were expensed (of which $3.2 million was non-cash) related to a settlement and license agreement. After factoring in this one-time charge, the net $2.8 million increase is associated with increased clinical development costs of $1.6 million, personnel costs of $0.6 million, internal research supplies of $0.2 million and other expenses of $0.4 million. The $1.6 million increase in our clinical costs during the period is primarily related to clinical product manufacturing, covered in part by Healios, technology transfer services associated with planned Japan manufacturing for Healios, process development activities to support large-scale manufacturing, and our MASTERS-2 clinical trial that began enrolling patients in the third quarter of 2018.

General and administrative expenses increased to $2.8 million for the three months ended December 31, 2018 from $2.1 million in the comparable period in 2017. The $0.7 million increase was due primarily to increases in personnel costs, professional fees, stock compensation costs and other administrative costs compared to the same period last year.

Net loss for the fourth quarter was $11.3 million in 2018 compared to a net loss of $13.1 million in the fourth quarter of 2017. The difference of $1.8 million reflects the above variances, as well as an increase of $0.3 million in other income items.

Full Year 2018 Financial Results

Revenues increased to $24.3 million for the year ended December 31, 2018 from $3.7 million in 2017. Our contract revenues from our collaboration with Healios increased $21.4 million year over year, reflecting the expansion of our collaboration in June 2018 to include additional licensed indications, among other things. Included in our 2018 revenues were royalties and other contract revenues of $1.5 million ($1.9 million in 2017) primarily related to our collaboration with RTI Surgical, Inc., which recently announced that it will cease distribution of its bone graft product that utilizes our technology.

Research and development expenses increased to $38.7 million for the year ended December 31, 2018 from $27.8 million for the year ended December 31, 2017. The increase in research and development expenses year-over-year of $10.9 million related to increases in clinical trial and manufacturing process development costs of $11.4 million, personnel costs of $1.6 million, and internal supply and other costs of $1.6 million. These increases were partially offset by a decrease in license fees of $3.7 million related to the settlement and license agreement in 2017 with one-time payments of cash and stock that concluded in 2018.

General and administrative expenses increased to $10.4 million in 2018 from $8.5 million in 2017. The $1.9 million increase was due primarily to increases in personnel costs, legal and professional services and stock compensation expense.

Net loss was $24.3 million in 2018 compared to a net loss of $32.2 million in 2017. The difference of $7.9 million reflects the above variances, as well as a decrease of $0.1 million in other net expenses.

In the twelve months ended December 31, 2018, net cash used in operating activities was $13.4 million compared to $24.0 million in the twelve months ended December 31, 2017. The difference reflects in part license fees paid by Healios in connection with the collaboration expansion being partially offset by an increase in clinical development activity in 2018.

At December 31, 2018, we had $51.1 million in cash and cash equivalents, compared to $29.3 million at December 31, 2017.

Conference Call

Gil Van Bokkelen, Chairman and Chief Executive Officer, William (B.J.) Lehmann, President and Chief Operating Officer, and Laura Campbell, Senior Vice President of Finance, will host a conference call today to review the results as follows:

Date March 14, 2019
Time 4:30 p.m. (Eastern Time)
Telephone access: U.S. and Canada (877) 396-3286
Telephone access: International (647) 689-5528
Encore Password (needed for the replay only) 7677927
Live webcast www.athersys.com, under the Investors section
We encourage shareholders to listen using the webcast link, and to use the phone line if you intend to ask a question. A replay will be available on the webcast at www.athersys.com under the investors section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening shortly after the completion of the call until 11:59 PM Eastern Time on March 21, 2019 by dialing (800) 585-8367 or (416) 621-4642 and entering Encore passcode 7677927. The archived webcast will be available for one year at the aforementioned URL.

Moleculin Announces First Patients Treated in European Annamycin Clinical Trial

On March 14, 2019 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported the first patients have been treated in the Company’s second clinical trial to study Annamycin for the treatment of relapsed and refractory adults with acute myeloid leukemia (Press release, Moleculin, MAR 14, 2019, View Source [SID1234534336]). The Company further reported that the initial treatment of the first patient appeared to be well tolerated.

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”We are encouraged to see such ready access to qualified patients in Poland,” commented Walter Klemp, Moleculin’s Chairman and CEO.” We consider it a positive indication to have completed the treatment of the first European patient so soon after beginning recruitment. In addition, we have already begun treatment of the second patient. We also believe that the higher starting dosage in the European trial as compared to the US trial may be contributing to a faster rate of recruitment.”

Caladrius Biosciences Reports 2018 Fourth Quarter and Year End Financial Results

On March 14, 2019 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a late-stage therapeutics development biopharmaceutical company committed to the development of innovative products that have the potential to restore the health of people with chronic illnesses and with a focus on select cardiovascular indications, reported financial results for the three and twelve months ended December 31, 2018 (Press release, Caladrius Biosciences, MAR 14, 2019, View Source [SID1234534335]).

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"2018 was an exciting and productive year for Caladrius, featuring a number of significant developments. Specifically, we made great progress advancing and expanding our clinical CD34+ cell technology platform while maintaining strong fiscal prudence," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "Notably, we initiated the ESCaPE-CMD phase 2 study in the U.S. for CLBS14-CMD and subsequently acquired a data license for our no-option refractory disabling angina program, CLBS14-NORDA, for which we plan to initiate a phase 3 trial in the fall of this year pending finalization of the protocol with the U.S Food and Drug Administration. We continue to expect to report top-line data in the ESCaPE-CMD trial by the end of 2019 or early 2020, and we continue to enroll in our CLI study in Japan for CLBS12 for which we expect to report topline data in the first half of 2020.

"We are excited about what lies ahead in 2019 and expect the momentum to continue as we advance our clinical development pipeline and achieve a number of important development milestones throughout the balance of the year," concluded Dr. Mazzo.

Fourth Quarter Financial Highlights

Research and development expenses for the fourth quarter of 2018 were $1.5 million, a 68% decrease compared with $4.7 million for the fourth quarter of 2017. Expenses in the fourth quarter of 2018 principally comprised costs in our ischemic repair programs for CLBS12 and CLBS14-CMD and preparation for our CLBS14-NORDA program. Conversely, the prior year quarter expenses were focused primarily on our T-Rex study for CLBS03, which completed enrollment in December 2017.

General and administrative expenses for the fourth quarter of 2018 were $2.3 million, a 14% decrease compared with $2.7 million for the fourth quarter of 2017, due to lower corporate-related activities compared with the prior year period.

The net loss from continuing operations for the fourth quarter of 2018 was $3.6 million, or $0.36 per share, compared with $4.0 million, or $0.40 per share, for the fourth quarter of 2017.

2018 Financial Highlights

Research and development expenses for 2018 were $7.6 million, a 52% decrease compared with $15.8 million for 2017. The current year expenses were principally comprised of costs related to our ischemic repair programs for CLBS12 and CLBS14-CMD as well as initial preparation for our CLBS14-NORDA program. Conversely, the prior year expenses were focused primarily on our T-Rex study for CLBS03, which trial completed enrollment in December 2017.

General and administrative expenses for 2018 were $9.4 million, a 20% decrease compared with $11.8 million for 2017. The decrease was due to lower corporate-related activities compared with the prior year period, along with the sale of our counter-flow centrifugation system to Hitachi in the second quarter of 2018, which resulted in a one-time $1.4 million gain included in general and administrative expenses.

Net loss from continuing operations for the twelve months ended December 31, 2018 was $16.2 million, or $1.67 per share, compared with $16.2 million, or $1.78 per share, for the same period of 2017.

Balance Sheet Highlights

As of December 31, 2018, Caladrius had cash, cash equivalents and marketable securities of $43.1 million. Based on existing programs and projections, the Company remains confident that its cash balances will allow it to fund its current business plan through mid-2020.

Conference Call

Caladrius’ management will host a conference call for the investment community beginning at 4:30 p.m. ET on Thursday, March 14, 2019 to discuss the financial results, provide a company update and answer questions.

Shareholders and other interested parties may participate in the conference call by dialing (866) 595-8403 (domestic) or (706) 758-9979 (international), using the conference ID number: 2168777. The conference call will also be webcast live and can be accessed from the Company’s website at www.caladrius.com/investors/news-events.

For those unable to participate in the live conference call or webcast, an audio recording of the call will be available for replay approximately two hours after the conclusion of the call until 11:59 p.m. ET on March 21, 2019. To access the audio replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and provide conference ID number: 2168777.

A webcast replay of the conference call will remain available on the Company’s website for 90 days.

Deciphera Pharmaceuticals, Inc. Announces Fourth Quarter and Year-end 2018 Financial Results

On March 14, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported financial results for the fourth quarter and year ended December 31, 2018 and provided an update on clinical and corporate developments (Press release, Deciphera Pharmaceuticals, MAR 14, 2019, View Source [SID1234534334]).

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"2018 was punctuated by significant progress in advancing our diverse pipeline of targeted drug candidates," said Michael D. Taylor, Ph.D. President and Chief Executive Officer. "We continued to add to the growing body of data that we believe supports ripretinib’s potential to provide improved, durable clinical benefit for GIST patients across multiple lines of therapy. Notably, we initiated two pivotal Phase 3 studies in 2018, INVICTUS and INTRIGUE, in fourth-line and fourth-line plus and second-line GIST, respectively."

Dr. Taylor continued, "As we near the reporting of top-line data from the INVICTUS study, expected in mid-2019, we are actively working to build out our commercial capabilities for ripretinib’s potential launch in the United States. In parallel, we continue to advance our earlier stage pipeline candidates, DCC-3014 and rebastinib, and expect to announce one new clinical candidate and commence IND-enabling studies during 2019."

Recent Clinical Updates

Ripretinib (DCC-2618)
Deciphera announced completion of enrollment in the INVICTUS pivotal Phase 3 clinical study evaluating the safety and efficacy of ripretinib (DCC-2618), the Company’s investigational broad-spectrum KIT and PDGFRα inhibitor, in fourth-line and fourth-line plus gastrointestinal stromal tumor (GIST) patients. The Company expects to report top-line data from this study in mid-2019 and is building commercial capabilities to support the planned launch of ripretinib in the United States, if approved.
Deciphera announced the initiation of its INTRIGUE pivotal Phase 3 clinical study evaluating the efficacy and tolerability of ripretinib compared to sunitinib in second-line GIST patients.
At the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress in October, Deciphera presented updated preliminary Phase 1 clinical study results of ripretinib in patients with GIST that the Company believes demonstrate the potential of ripretinib to provide improved, durable clinical benefit for GIST patients from second-line through fourth-line-plus. These data were also presented at the Annual Meeting of the Connective Tissue Oncology Society (CTOS) in November 2018.
Deciphera expanded the ongoing Phase 1 study of ripretinib to include additional cohorts for patients with various solid tumors, including melanoma, non-small cell lung cancer, germ cell cancer, penile cancer, soft tissue sarcoma, and GIST or other solid tumor patients with renal impairment.
During a poster session at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium in November 2018, Deciphera presented preclinical data on the effects of the combination of ripretinib and MAPK pathway inhibitors on cell death and apoptosis in cellular assays of GIST and mastocytosis.
Rebastinib
Deciphera recently announced the initiation of two open-label, multicenter, Phase 1b/2 combination studies of rebastinib, the Company’s investigational small molecule switch control inhibitor of TIE2 kinase:
Phase 1b/2 study in combination with carboplatin in patients with advanced or metastatic solid tumors.
Phase 1b/2 study in combination with paclitaxel in patients with advanced or metastatic solid tumors. The Company expects to report initial data from this study in 2019.
DCC-3014
Deciphera announced positive, preliminary, top-line data from the ongoing dose escalation portion of the Phase 1 clinical study of DCC-3014, the Company’s investigational small molecule switch control inhibitor of CSF1R, in patients with advanced malignancies. A review of further data from this Phase 1 study is planned to be presented at a medical meeting in 2019.
The Company also announced a plan to expand the Phase 1 study to evaluate DCC-3014 in patients diagnosed with Tenosynovial Giant Cell Tumors (TGCT).
Corporate Update

Earlier this month, Deciphera announced the appointment of Steve Hoerter as President & Chief Executive Officer, effective March 18, 2019. Mr. Hoerter, who currently serves as a member of the Deciphera Board of Directors, joins the Company from Agios, where he was Chief Commercial Officer. He will succeed Dr. Taylor, who will retire as President & Chief Executive Officer of the Company. Dr. Taylor will be available to support the transition and will remain as a member of the Company’s Board of Directors.
Fourth Quarter 2018 Financial Results

Cash Position: As of December 31, 2018, cash and cash equivalents were $293.8 million, compared to cash and cash equivalents of $196.8 million as of December 31, 2017. This increase was primarily related to proceeds obtained from the Company’s June 2018 underwritten public offering, offset by cash used in operating activities. We expect our current cash and cash equivalents will enable us to fund our operating and capital expenditures and debt service payments into the second half of 2020.
R&D Expenses: Research and development expenses for the fourth quarter of 2018 were $27.4 million, compared to $15.7 million for the same period in 2017. The increase was primarily due to an increase in spending on the ripretinib (DCC-2618) program of $5.8 million as a result of clinical trial start-up activities related to the Phase 3 INTRIGUE study in second-line GIST, which the Company initiated in December 2018. Expenses related to the rebastinib program increased $1.8 million, primarily due to the Phase 1b/2 study of rebastinib in combination with paclitaxel, which the Company initiated in October 2018, and start-up activities related to the second Phase 1b/2 clinical trial of rebastinib in combination with carboplatin, which the Company initiated in January 2019. Personnel-related costs increased $2.5 million due primarily to increased headcount in our research and development functions. Personnel-related costs for the fourth quarters of 2018 and 2017 included non-cash stock-based compensation expense of $1.0 million and $0.5 million, respectively. Facility-related and other costs included in unallocated expenses increased $2.0 million primarily due to increased costs incurred in connection with our early-stage drug discovery programs.
G&A Expenses: General and administrative expenses for the fourth quarter of 2018 were $6.5 million, compared to $4.7 million for the same period in 2017. The increase was primarily due to an increase in legal and professional fees as a result of various advisory fees related to ongoing operations as a public company. Facility-related and other costs increased due to insurance costs and higher rent expense related to the Company’s new lease. Non-cash stock-based compensation was $1.8 million and $2.3 million for the fourth quarters of 2018 and 2017, respectively.
Net Loss: For the fourth quarter of 2018, Deciphera reported a net loss of $32.3 million, or $0.86 per share, compared with a net loss of $19.9 million, or $0.62 per share, for the same period in 2017.

Agenus Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Corporate Update

On March 14, 2019 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology (I-O) company with a pipeline of immune checkpoint antibodies, cancer vaccines and adoptive cell therapies1, reported and update and reported financial results for the fourth quarter and full year of 2018 (Press release, Agenus, MAR 14, 2019, View Source [SID1234534331]).

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Agenus Logo

"We are rapidly advancing with the discovery and clinical development of our innovative I-O agents," said Garo H. Armen, Ph.D., Chairman and CEO of Agenus. "In the past year, we entered into an important partnership with Gilead, delivered 6 INDs, and confirmed benefit in the majority of patients treated with our lead CTLA-4 and PD-1 antibodies. Our next steps will target submission and commercial launch readiness for our first two antibodies."

Achievements
Strengthened balance sheet with the Gilead collaboration and payments from milestones achieved with Incyte and Merck
$150M payment from Gilead
$21.5M additional milestones for advancing LAG-3 (INCAGN02385), TIM-3 (INCAGN02390), ILT4 (MK-4830) and FDA acceptance of the IND for AGEN1423, licensed to Gilead
Advanced lead programs and reported clinical benefit in majority of patients across multiple solid tumors, including cervical cancer
Ongoing trials in cervical cancer are designed to support BLA via accelerated pathway
We plan to expand PD-1 development in additional indications
Advanced new discoveries, which will enter the clinic this year
Next-Gen CTLA-4, AGEN1181
First-in-class bispecific, AGEN1223 (a Gilead option program)
Revenues of GSK’s Shingrix, containing our QS-21 Stimulon, exceed $1Bn (USD)
Bill & Melinda Gates Foundation award Agenus ~$1M to develop novel technology for QS-21
AgenTus Cell Therapy Business:
2019 INDs are on track
Partnership and private financing discussions are underway
Fourth Quarter and Full Year 2018 Financial Results

We ended 2018 with a cash balance of $53 million followed by the $150 million received from Gilead in 2019.

For the fourth quarter ended December 31, 2018, we reported a net loss of $49 million or $0.40 per share compared to a net loss for same period in 2017 of $35 million, or $0.35 per share. In the fourth quarter, we recognized revenue of $6.5 million which includes non-cash royalties earned.

For the year ended December 31, 2018, we reported a net loss of $162 million or $1.44 per share compared to a net loss for the year ended 2017 of $121 million or $1.23 per share. The increased net loss reflects reduced revenue during 2018 due to an accelerated milestone received during 2017 from Incyte, the 2018 loss on early extinguishment of debt and increased non-cash interest on our liability related to the sale of future royalties.

Conference Call, Webcast and Prepared Statement Information

Date: Thursday, March 14, 2019

Time: 8:30 a.m. ET

Domestic Dial-in Number: 1-844-492-3727

International Dial-in Number: 1-412-317-5118

Conference ID: Agenus

Live Webcast: accessible from the Company’s website at View Source or with this link View Source

A replay will be available on the Company’s website approximately two hours after the call and will remain available for 90 days.