On February 13, 2019 Cerus Corporation (Nasdaq:CERS) reported that its fourth quarter and full-year 2018 results will be released on Tuesday, February 26, 2019, after the close of the stock market (Press release, Cerus, FEB 13, 2019, View Source [SID1234533312]). The company will host a conference call and webcast at 4:30 P.M. EDT that afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook.

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To access the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on the company’s website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 2392137. The replay will be available approximately three hours after the call through March 12, 2019.

On February 13, 2019 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the fourth quarter and full year ended December 31, 2018 (Press release, Vanda Pharmaceuticals, FEB 13, 2019, View Source [SID1234533291]).

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"The combination of strong commercial performance and positive clinical results has set 2018 apart as a transformative year for Vanda," said Mihael H. Polymeropoulos, M.D., Vanda’s President and CEO. "The tradipitant clinical programs in gastroparesis and atopic dermatitis have emerged as potentially significant drivers of future growth with the possibility of bringing new treatment options to millions of patients."

Key Financial Highlights:

Total net product sales from HETLIOZ and Fanapt were $53.0 million in the fourth quarter of 2018, an 8% increase compared to $49.1 million in the third quarter of 2018 and a 20% increase compared to $44.3 million in the fourth quarter of 2017.
HETLIOZ net product sales were $32.4 million in the fourth quarter of 2018, an 8% increase compared to $29.9 million in the third quarter of 2018 and a 30% increase compared to $25.0 million in the fourth quarter of 2017.
Fanapt net product sales were $20.6 million in the fourth quarter of 2018, a 7% increase compared to $19.2 million in the third quarter of 2018 and a 7% increase compared to $19.3 million in the fourth quarter of 2017.
Cash, cash equivalents and marketable securities (Cash) were $257.4 million as of December 31, 2018, representing an increase to Cash of $113.9 million during 2018.
Research and Development Highlights:

Tradipitant – Clinical Development

In December 2018, Vanda announced positive results from a Phase II clinical study (2301) of tradipitant in gastroparesis. Gastroparesis patients treated with tradipitant demonstrated significant improvement in nausea and most of the core gastroparesis symptoms.
Vanda expects to meet with the U.S. Food and Drug Administration (the FDA) to further define and confirm the path towards approval of tradipitant in the treatment of patients with gastroparesis, including the planned initiation of a Phase III clinical study in the second quarter of 2019.
Enrollment in the Phase III clinical study (EPIONE) of tradipitant in atopic dermatitis is ongoing. Results are expected in the first half of 2020. A second Phase III clinical study is expected to begin in the first quarter of 2020.
In January 2019, Vanda initiated a Phase II clinical study of tradipitant in motion sickness. Study results are expected in the second quarter of 2019.
HETLIOZ (tasimelteon)

In December 2018, Vanda announced positive results from a clinical study of HETLIOZ in Smith-Magenis Syndrome (SMS). SMS patients treated with HETLIOZ demonstrated significant improvement in overall sleep quality and overall total nighttime sleep duration.
Vanda expects to meet with the FDA in the second quarter of 2019 to confirm the regulatory path forward for HETLIOZ in the treatment of patients with SMS and expects to file a supplemental New Drug Application (sNDA) in the third quarter of 2019.
In December 2018, Vanda announced that the FDA had accepted the HETLIOZ sNDA for the treatment of jet lag disorder with a Prescription Drug User Fee Act target action date of August 16, 2019.
Vanda plans in the third quarter of 2019 to initiate a Phase II clinical study of HETLIOZ in delayed sleep phase disorder (DSPD) in patients who have a mutation in the CRY1 gene which is believed to be causative in a subset of patients with the disorder.
Fanapt (iloperidone)

Enrollment is ongoing in a pharmacokinetic study for the once-a-month long acting injectable (LAI) formulation of Fanapt. A randomized clinical study of the LAI formulation in schizophrenia is planned to begin in 2019.
A randomized study of Fanapt in bipolar disorder is planned to begin in 2019.
VTR-297 (histone deactetylase (HDAC) inhibitor)

Enrollment is ongoing in a Phase I clinical study (1101) of VTR-297 in hematologic malignancies.
Tradipitant – Partial Clinical Hold and FDA Dispute

In April 2018, Vanda submitted a protocol amendment to the FDA, proposing a 52-week open-label extension (OLE) period for patients who had completed the tradipitant Phase II clinical study (2301) in gastroparesis. In May 2018, based on feedback from the FDA, Vanda amended the protocol limiting the duration of treatment in the 2301 study to a total of three months, while continuing to seek further dialogue with the FDA on extending the study duration to 52-weeks. As a part of this negotiation process, in September 2018, Vanda submitted a new follow-on 52-week OLE protocol to the FDA (2302) for patients who had completed the 2301 study. While waiting for further feedback, no patients were ever enrolled in any study beyond 12 weeks.
On December 19, 2018, the FDA imposed a partial clinical hold (PCH) on the two proposed studies, stating that Vanda is required first to conduct additional chronic toxicity studies in canines, monkeys or minipigs before allowing patients access in any clinical protocol beyond 12 weeks. The PCH was not based on any safety or efficacy data related to tradipitant. Rather, the FDA informed Vanda that these additional toxicity studies are required by a guidance document.
On February 5, 2019, Vanda filed a lawsuit against the FDA in the United States District Court for the District of Columbia, challenging the FDA’s legal authority to issue the PCH, and seeking an order to set it aside.
Vanda does not expect the PCH to have any material impact on its ongoing clinical studies in atopic dermatitis and motion sickness or the planned Phase III study in gastroparesis. At present, the PCH has not had any impact on the potential timing of an NDA filing or approval for these indications. Vanda will continually reassess this situation as events unfold.
Non-GAAP Financial Results

Non-GAAP net income was $13.7 million for the fourth quarter of 2018, or $0.26 per share, compared to a Non-GAAP net income of $1.4 million, or $0.03 per share, for the fourth quarter of 2017. Vanda Non-GAAP net income was $38.4 million for the full year 2018, compared to a Non-GAAP net loss of $3.4 million for the full year 2017.

Vanda provides Non-GAAP financial information, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP figures. Refer to the sections of this press release entitled "Non-GAAP Financial Information" and "Reconciliation of GAAP to Non-GAAP Financial Information" for more detailed information regarding Non-GAAP financial information.

2019 Financial Guidance

Conference Call

Vanda has scheduled a conference call for today, Wednesday, February 13, 2019, at 4:30 PM ET. During the call, Vanda’s management will discuss the fourth quarter and full year 2018 financial results and other corporate activities. Investors can call 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 1579398. A replay of the call will be available on Wednesday, February 13, 2019, beginning at 7:30 PM ET and will be accessible until Wednesday, February 20, 2019, at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 1579398.

The conference call will be broadcast simultaneously on Vanda’s website, www.vandapharma.com. Investors should click on the Investor Relations tab and are advised to go to the website at least 15 minutes early to register, download, and install any necessary software or presentations. The call will also be archived on Vanda’s website for a period of 30 days.

Non-GAAP Financial Information

Vanda believes that the Non-GAAP financial information provided in this press release can assist investors in understanding and assessing the ongoing economics of Vanda’s business and reflect how it manages the business internally and sets operational goals. Vanda’s "Non-GAAP Selling, general and administrative expenses" and "Non-GAAP Research and development expenses" exclude stock-based compensation. Vanda’s "Non-GAAP Net income (loss)," "Non-GAAP Net income (loss) per share" and "Non-GAAP Operating expenses excluding Cost of goods sold" exclude stock-based compensation and intangible asset amortization.

Vanda believes that excluding the impact of these items better reflects the recurring economic characteristics of its business, as well as Vanda’s use of financial resources and its long-term performance.

These Non-GAAP financial measures, as presented, may not be comparable to similarly titled measures reported by other companies since not all companies may calculate these measures in an identical manner and, therefore, they are not necessarily an accurate measure of comparison between companies.

The presentation of these Non-GAAP financial measures is not intended to be considered in isolation or as a substitute for guidance prepared in accordance with GAAP. The principal limitation of these Non-GAAP financial measures is that they exclude significant elements that are required by GAAP to be recorded in Vanda’s financial statements. In addition, they are subject to inherent limitations as they reflect the exercise of judgments by management in determining these Non-GAAP financial measures. In order to compensate for these limitations, Vanda presents its Non-GAAP financial guidance in connection with its GAAP guidance. Investors are encouraged to review the reconciliation of our Non-GAAP financial measures to their most directly comparable GAAP financial measure.

On February 13, 2019 Veru Inc. (NASDAQ: VERU), an oncology and urology biopharmaceutical company developing novel medicines for the prostate cancer continuum of care and urology specialty pharmaceuticals, reported financial results and business highlights for its fiscal 2019 first quarter ended December 31, 2018 (Press release, Veru, FEB 13, 2019, View Source [SID1234533313]).

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Fiscal 2019 First Quarter Financial Results

Company net revenues up 146% to $6.4 million from $2.6 million in the prior-year first quarter. Company reported FC2 Female Condom / FC2 Internal Condom (FC2) sales growth in both its prescription business and in its public sector business;

Company gross profit up 254% to $4.6 million from $1.3 million in the prior-year first quarter;

Company gross margin increased to 73% from 51% in the prior-year first quarter;

FC2 prescription business net revenues up 1,500% to $2.4 million from $0.15 million in the prior-year first quarter. Compound Annual Growth Rate (CAGR) of 100% over last five quarters for net revenues of FC2 prescription business;

FC2 public sector business net revenues up 60% to $3.9 million from $2.4 million in the prior-year first quarter;

CAGR of 25% over the last five quarters for net revenues of total FC2 business;

Commercial Segment (FC2, PREBOOST and drug commercialization costs) operating income was $3.7 million versus $0.1 million in the prior-year first quarter;

Operating loss significantly narrowed by 86% to $1.0 million from $7.4 million in the fiscal 2018 first quarter (the fiscal 2018 first quarter included a $3.8 million loss for the settlement of Brazilian receivables); and

Net loss of $2.1 million, or $0.03 per share, was significantly less than $4.3 million, or $0.08 per share, in the first quarter of fiscal 2018.

"We are pleased to report strong financial results in the fiscal 2019 first quarter, including excellent topline growth, substantially higher gross profit and a significantly lower operating loss," said Mitchell Steiner, M.D., Chairman, President and Chief Executive Officer of Veru Inc. "The improvement was driven by increased sales of FC2 in our prescription business as well as in our public sector business. We recently changed the sales strategy of our FC2 prescription business, which has a higher gross margin, making the growth of that part of our business particularly gratifying. Growing prescription sales of FC2 should help smooth out some of the fluctuations in total FC2 net revenues that we have historically seen due to ordering patterns related to our FC2 public sector business."

Recent Business and Operational Highlights

Announced the Company’s strategy to become known as "the prostate cancer company" and to provide a "continuum of care" for prostate cancer patients. Prostate cancer remains the second most frequent cause of cancer deaths in men and drugs to manage prostate cancer are large market opportunities. Our drug development and drug commercial activities will largely align with the clinical management of prostate cancer patients. Anticipated revenue from our urology specialty pharma business and existing commercial products will help to support these efforts; .

Initiated a Phase 1b/2 clinical trial and enrolled patients for VERU-111, a novel, proprietary, next generation, first-in-class oral selective antitubulin agent that targets and disrupts alpha and beta tubulin for advanced prostate cancer and potentially other cancers, with clinical data expected in 2019. Drugs for advanced prostate cancer currently have over $3 billion in U.S. annual sales;

Initiated a Phase 2 clinical trial and enrolled patients for zuclomiphene citrate, a novel, proprietary, oral, nonsteroidal, estrogen receptor agonist to treat hot flashes caused by androgen deprivation therapy, or hormone treatment for men with advanced prostate cancer; top line results expected Summer 2019. Based on an independent market analysis sponsored by the Company, the Company estimates the U.S. market potential for zuclomiphene citrate is over $600 million annually;

Completed a successful bioavailability and bioequivalence clinical trial for the Company’s proprietary tadalafil and finasteride combination tablet (TADFIN) for benign prostatic hyperplasia with a New Drug Application (NDA) to be submitted in 2019 and approval expected in 2020. BPH is an established multi-billion-dollar market;

Signed a multi-year exclusive supply and distribution agreement to supply the Company’s PREBOOST premature ejaculation wipes to Roman Health Ventures Inc., a premier and fast-growing men’s health and telemedicine company that discreetly sells men’s health products via the internet;

Four abstracts accepted for presentation for VERU-111 and zuclomiphene citrate at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium in February 2019;

Three abstracts accepted for presentation for VERU-111 at the European Association of Urology Congress in March 2019; and

One abstract accepted for presentation for VERU-111 at the American Urological Association Annual Meeting in May 2019.

"We are focused on advancing our pipeline of drug candidates for the continuum of care for prostate cancer, which have a combined addressable market of multiple billions of dollars, noted Dr. Steiner. We continue to run a lean operation, with our existing commercial businesses helping to fund the development of our prostate cancer and our urology specialty pharmaceuticals. Even after costs associated with all of our pharmaceutical clinical development programs in the most recently completed quarter, our operating loss significantly narrowed to approximately a $1 million loss for the quarter. Anticipated strong growth in revenues from our current commercial products combined with anticipated new revenue from our TADFIN for BPH, which we expect to launch in 2020, should allow us to continue to significantly invest in our prostate cancer proprietary pharmaceuticals, to seek strong commercialization partnerships, and to access large, well-established urology and prostate cancer markets around the globe."

Conference Call Event Details

Veru Inc. will host a conference call today at 8 a.m. ET to review the Company’s performance. Interested investors may access the call by dialing 800-341-1602 from the U.S. or 412-902-6706 from outside the U.S. and asking to be joined into the Veru Inc. call.

In addition, investors may access a replay of the conference call the same day beginning at approximately 12 p.m. (noon) ET by dialing 877-344-7529 for US callers, or 412-317-0088 from outside the U.S., passcode 10128045. The replay will be available for one week, after which, the recording will be available via the Company’s website at View Source

On February 13, 2019 ADC Therapeutics, an oncology drug discovery and development company that specializes in the development of proprietary antibody drug conjugates (ADCs), reported that the first patient has been dosed in a Phase I clinical trial evaluating the safety, tolerability, pharmacokinetics and anti-tumor activity of ADCT-402 (loncastuximab tesirine) plus AstraZeneca’s IMFINZI (durvalumab) in patients with advanced diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL) or follicular lymphoma (FL) (Press release, ADC Therapeutics, FEB 13, 2019, View Source [SID1234596065]).

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ADCT-402, an ADC designed to target and kill CD19-expressing malignant B-cells, is also being evaluated in an ongoing pivotal Phase II clinical trial in patients with relapsed or refractory (R/R) DLBCL. Durvalumab is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumor’s immune-evading tactics and releasing the inhibition of immune responses.

Jay Feingold, MD, PhD, Chief Medical Officer and Senior Vice President of Clinical Development at ADC Therapeutics, said, "Data from our 183-patient first-in-human clinical trial of ADCT-402, which were presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, demonstrated its acceptable safety profile and promising anti-tumor activity as a single agent in patients with relapsed or refractory B-cell non-Hodgkin lymphomas. We are now excited to explore the possible impact of ADCT-402 plus durvalumab in patient populations that would greatly benefit from new treatment options."

Craig Moskowitz, MD, Physician in Chief for the Cancer Service Line of the Sylvester Comprehensive Cancer Center, Professor of Medicine in the Miller School of Medicine at University of Miami Health System, and an investigator for the trial, said, "While the majority of patients with non-Hodgkin lymphoma typically respond to initial treatment, many patients relapse and face a poor prognosis. I look forward to evaluating this combination therapy of ADCT-402 and a PD-L1 blocker to determine its safety and potential anti-tumor activity in patients with relapsed or refractory diffuse large B-cell lymphoma, mantle cell lymphoma and follicular lymphoma who have failed or are intolerant to established therapies, or who don’t have other available treatment options."

The open-label, single-arm trial will include a dose-escalation part, followed by a dose-expansion part. The dose-expansion part will consist of up to three expansion cohorts – one for DLBCL, one for MCL and one for FL – to obtain additional safety and preliminary anti-tumor activity information at the maximum tolerated dose. Approximately 75 patients will be enrolled in the trial. For more information, please visit www.clinicaltrials.gov (identifier NCT03685344).

ADCT-402 Interim First-in-Human Data

Updated data from the ongoing 183-patient Phase I clinical trial of ADCT-402 were presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. In a subpopulation of 139 evaluable patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who had failed or were intolerant to established therapies, ADCT-402 demonstrated manageable toxicity. At doses >120 μg/kg, the overall response rate (ORR) was 43.3 percent (55/127 patients with DLBCL), comprising 23.6 percent complete responses and 19.7 percent partial responses. In a subgroup of 15 patients with R/R mantle cell lymphoma (MCL) and 14 patients with R/R follicular lymphoma (FL), ADCT-402 demonstrated manageable toxicity. In MCL patients, ORR was 46.7 percent (7/15) and median duration of response (DoR) was not reached after a median follow-up time of 8.7 months. In FL patients, ORR was 78.6 percent (11/14) and median DoR was not reached after a median follow-up time of 11.6 months.

About ADCT-402

ADCT-402 (loncastuximab tesirine) is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody that binds to human CD19, conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer toxin. Once bound to a CD19-expressing cell, ADCT-402 is internalized into the cell where enzymes release the PBD-based warhead. CD19 is a clinically validated target for the treatment of B-cell malignancies. The PBD-based warhead has the ability to form highly cytotoxic DNA interstrand cross-links, blocking cell division and resulting in cell death. ADCT-402 is being evaluated in a pivotal Phase II clinical trial in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) (NCT03589469) and a Phase I clinical trial in combination with IMFINZI (durvalumab) in patients with R/R DLBCL, mantle cell lymphoma or follicular lymphoma (NCT03685344). The U.S. Food and Drug Administration granted orphan drug designation to ADCT-402 for the treatment of DLBCL and MCL.

On February 13, 2019 Achilles Therapeutics ("Achilles"), a biopharmaceutical company developing personalised cancer immunotherapies, reported the approval by the UK Medicines and Healthcare products Regulatory Agency (MHRA) of its Clinical Trial Application (CTA) to conduct a Phase I/II study using clonal neoantigen targeting T cells (cNeT) in patients with metastatic or recurrent melanoma (Press release, Achilles Therapeutics, FEB 13, 2019, View Source [SID1234533294]). The study is expected to enrol the first patient later in 2019.

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"To have received our second CTA approval in as many weeks is highly encouraging," said Dr Iraj Ali, CEO of Achilles Therapeutics. "We look forward to bringing this potentially transformative treatment option into the clinic later this year."

Professor James Larkin, Consultant Medical Oncologist at The Royal Marsden and Reader at the Institute of Cancer Research, said: "The Achilles approach to leveraging the leading science in tumour evolution to tackling solid tumours using cNeT has the potential to change the immune-oncology space and bring life-changing treatment options to patients. We look forward to the start of clinical development."

Achilles is developing personalised T cell therapies for solid tumours targeting clonal neoantigens: protein markers unique to each patient that are present on the surface of all cancer cells. Using its PELEUS bioinformatics platform, Achilles can identify clonal neoantigens from each patient’s unique tumour profile which are present on every cancer cell. Achilles uses its proprietary process to manufacture T cells (cNeT) which exquisitely target a specific set of clonal neoantigens in each patient. Targeting multiple clonal neoantigens that are present on all cancer cells, but not on healthy cells, reduces the risk that new mutations can induce immune evasion and therapeutic resistance, and allows individualised treatments to target and destroy tumours without harming healthy tissue.