On May 9, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to CLR 131, the company’s lead Phospholipid Drug Conjugate (PDC) product candidate, for the treatment of rhabdomyosarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, MAY 9, 2018, View Source [SID1234526363]).

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"Rhabdomyosarcoma is the most common type of tissue sarcoma in children. While initial response to treatment is generally favorable, there is an important need for new treatments, especially in children who experience relapse." said John Friend, M.D., chief medical officer of Cellectar. "Cellectar is committed to working closely with the FDA to fully evaluate the potential for targeted delivery of CLR 131 to address this currently unmet medical need."

Orphan drug designation provides seven-year market exclusivity, increased engagement and assistance from the FDA, tax credits for certain research, research grants and a waiver of the New Drug Application user fee. Rhabdomyosarcoma is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people nationwide.

About Rhabdomyosarcoma

Rhabdomyosarcoma (RMS), a malignant tumor of mesenchymal origin, is the most common soft tissue sarcoma in children, accounting for approximately 40% of childhood soft tissue sarcomas in the United States. The incidence is about 4.5 cases per 1 million per year in children younger than 15 years and more than 50% are younger than 10 years at diagnosis. Approximately 340 new cases are diagnosed each year in North America and the prognosis is favorable with a 64% 5-year survival in children aged birth to 19 years [Ward 2014]. At least one-third of all patients will experience disease progression or relapse, and 95% of all failures occur within 3 years. The median progression free survival following the first recurrence or progression is approximately nine months.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. In 2018 the company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma, and a second Phase 1 study in combination with external beam radiation for head and neck cancer.

On May 9, 2018 NantHealth, Inc. (NASDAQ-GS: NH), a next-generation, evidence-based, personalized healthcare company, reported financial results for its first quarter ended March 31, 2018 (Press release, NantHealth, MAY 9, 2018, View Source;p=RssLanding&cat=news&id=2348335 [SID1234526379]).

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Molecular Analysis – Highlights

Expanded Molecular Analysis Portfolio: Molecular Analysis portfolio expanded to include proprietary blood-based tumor profiling services, with beta launch of 26-analyte profiling test.
In-Vitro Diagnostic (IVD) Filing with FDA for circulating free DNA (cfDNA) Liquid Biopsy Platform: In Q1 2018, the company submitted a medical device application with the FDA for its proprietary cfDNA liquid biopsy platform.
Commenced Beta Launch of GPS Ordering and Results Portal enabling ordering physicians to electronically receive GPS results and request molecular tumor board and Medical Affairs consultations.
Test Growth: The company reported 677 GPS Cancer commercial tests were ordered in Q1 2018, up from 606 in Q4 2017.
Key Publication: In Q2 2018, results of a company sponsored study were published in Oncotarget, a peer-reviewed bio-medical journal. The study results demonstrate the significant gains in accuracy by performing tumor/normal DNA and RNA sequencing and the risks associated with high error rates of tumor only sequencing. View Source Publication
New National GPS Cancer Payer: In Q1 2018, as previously announced, the company signed a new GPS Cancer reimbursement contract with a large, national healthcare IT company.
New Lab Services Arrangement: In Q1 2018, as previously announced, the company signed a laboratory services agreement with a 20+ facility hospital system for the availability of GPS Cancer testing to its patient community.
Expanded International Adoption: In Q1 2018, as previously announced, the company signed a strategic reseller agreement with a partner in the United Kingdom for the provision of molecular analysis services for clinical studies and other research initiatives.
FDA Submission: In Q1 2018, as previously announced, the company submitted a 510(k) premarket notification application to the FDA for tumor/normal DNA sequencing.
"We are excited about the opportunity to feature GPS Cancer and our new liquid biopsy platform in 11 presentations at next month’s American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, a significant and auspicious milestone for NantHealth," said Sandeep (Bobby) Reddy, M.D., Chief Medical Officer of NantHealth. "In conjunction with these presentations, we plan to unveil to the oncology community at ASCO (Free ASCO Whitepaper) and commence the commercial launch of our liquid biopsy test, a 26 analyte test for circulating-free DNA (cfDNA) and RNA (cfRNA) extracted from patient blood permits non-invasive detection of expressed biomarkers and monitoring of response to immunotherapies such as Keytruda or Opdivo or resistance to anti-androgens such as Xtandi."

Software and Services Highlights:

Payer Engagement (NaviNet):
In Q1 2018, NantHealth’s industry leading Document Exchange solution was upgraded to include an enhanced document viewer and the ability for payers to tag and categorize documents.
In Q1 2018, as previously announced, the company signed a three-year renewal contract with a total contract value of approximately $17 million.
Clinical Decision Support (Eviti):
In Q1 2018, introduced new dual eligibility features, enabling payers to concurrently manage dual membership patients covered under Medicare and Medicaid, and drug shortage configuration features that provide improved management of high cost drugs.
Connected Care:
In Q1 2018, released DeviceConX5.14 (MDE), the first MDI solution to adopt the Fast Healthcare Interoperability Resources (FHIR) standard.
In Q1 2018, released VitalsConX2.1, with support for offline mode, enabling clinicians to continue nurse rounding when connectivity is lost and to submit data to the EMR once connectivity is restored.
In Q2 2018, completed first CE Mark submission for the DeviceConX software platform.
"Our 2018 first quarter performance reflects a 17% increase in consolidated revenue and a substantially improved gross margin over the prior year period," said Paul Holt, Chief Financial Officer of NantHealth. "We were delighted to see continued growth of our SaaS business, with revenue increasing 9% over the same quarter last year. Our year over year improvement in operating results was positively impacted by our revenue growth and the restructuring program, implemented late last year."

Business and Financial Highlights

In August 2017, NantHealth sold its provider/patient engagement assets to Allscripts to focus on core competencies and accelerate the plan to achieve profitability. As a result, the company has classified the current and prior period operating results of its provider/patient engagement business as discontinued operations. All results presented below represent the company’s continuing operations.

The company adopted a new revenue recognition standard on January 1, 2018. Please note that the financial results presented below include both amounts "as presented," which reflect implementation of the new revenue recognition standard, as well as amounts prior to the impact of the new revenue recognition standard to allow for comparability against historical results. Starting in fiscal year 2019, the company will no longer present its GAAP and Non-GAAP financial results under the previous revenue recognition standard. For additional information and reconciliations of our financial results between the new and previous revenue recognition standard, see the additional tables included in this press release and in the company’s Form 10-Q to be filed with the Securities and Exchange Commission.

For the 2018 first quarter, total net revenue as presented was $22.3 million. Total 2018 first quarter net revenue prior to the impact of the new revenue recognition standard increased 14% to $21.7 million from $19.1 million in 2017 first quarter. Gross profit as presented was $11.2 million, or 50% of total net revenue. Gross profit prior to the impact of the new revenue recognition standard was $10.7 million, or 49% of total net revenue, compared with $7.6 million, or 40% of total net revenue, for the prior-year first quarter. Selling, general and administrative (SG&A) expenses as presented were $20.7 million. SG&A prior to the impact of the new revenue recognition standard was $21.2 million compared with $17.4 million. Research and development (R&D) expenses as presented was $5.2 million decreased from $8.9 million; the new revenue recognition standard did not impact R&D expenses.

Net loss from continuing operations, net of tax, as presented was $22.0 million, or $0.20 per share. Net loss from continuing operations, net of tax, prior to the impact of the new revenue recognition standard narrowed to $22.8 million, or $0.21 per share, from $28.1 million, or $0.23 per share for the 2017 first quarter. Loss from discontinued operations, net of tax, as presented was $193,000, or breakeven on per share basis, compared with $13.0 million, or $0.11 per share; the new revenue recognition standard did not impact loss from discontinued operations. Net loss as presented was $22.2 million, or $0.20 per share. Net loss prior to the impact of the new revenue recognition standard was $23.0 million, or $0.21 per share, compared with $41.1 million, or $0.34 per share, for 2017 first quarter.

Financial results for the 2018 first quarter included approximately $3.3 million loss from related party equity method investment including impairment loss, $2.7 million of stock-based compensation expense, $2.2 million of intangible amortization, and $1.2 million of non-cash interest expense related to convertible notes, totaling $0.09 per share. On a non-GAAP basis, adjusted net loss from continuing operations as presented was $13.5 million, or $0.12 per share, for the 2018 first quarter. On a non-GAAP basis, adjusted net loss from continuing operations prior to the impact of the new revenue recognition standard was $14.3 million, or $0.13 per share, compared with $18.8 million, or $0.15 per share, for the 2017 first quarter.

Conference Call Information and Forward-Looking Statements

Later today, the company will host a conference call at 1:30 p.m. PT (4:30 p.m. ET) to review its results of operations for the first quarter ended March 31, 2018. The conference call will be available to interested parties by dialing 844-309-3709 from the U.S. or Canada, or 281-962-4864 from international locations, passcode 8963439. The call will be broadcast via the Internet at www.nanthealth.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary audio software. A playback of the call will be archived and accessible on the same website for at least three months.

Discussion during the conference call may include forward-looking statements regarding topics such as the company’s financial status and performance, regulatory and operational developments, and other comments the company may make about its future plans or prospects in response to questions from participants on the conference call.

Use of Non-GAAP Financial Measures

This news release contains references to Non-GAAP financial measures, including adjusted net loss and adjusted net loss per share, which are financial measures that are not prepared in conformity with United States generally accepted accounting principles (U.S. GAAP). The Company’s management believes that the presentation of Non-GAAP financial measures provides useful supplementary information regarding operational performance, because it enhances an investor’s overall understanding of the financial results for the Company’s core business. Additionally, it provides a basis for the comparison of the financial results for the Company’s core business between current, past and future periods. Other companies may define these measures in different ways. Non-GAAP financial measures should be considered only as a supplement to, and not as a substitute for or as a superior measure to, financial measures prepared in accordance with U.S. GAAP. Non-GAAP per share numbers are calculated based on one class of common stock and do not incorporate the effects, if any, of using the two-class method.

On May 9, 2018 Thermo Fisher Scientific Inc. (NYSE: TMO), the world leader in serving science, reported that Marc N. Casper, president and chief executive officer, will present at the Bank of America Merrill Lynch 2018 Health Care Conference on Wednesday, May 16, 2018, at 8:00 a.m. (PT) at the Encore at the Wynn Las Vegas, Las Vegas, Nev (Press release, Thermo Fisher Scientific, MAY 9, 2018, View Source [SID1234526399]).

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You can access the live webcast of the presentation via the Investors section of our website, www.thermofisher.com.

On May 9, 2018 Endocyte, Inc. (Nasdaq:ECYT), a biopharmaceutical company developing targeted therapeutics for personalized cancer treatment, reported financial results for the first quarter ending Mar. 31, 2018 and provided an operational update (Press release, Endocyte, MAY 9, 2018, View Source [SID1234526314]).

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"We made important progress during the first quarter in establishing the design of our phase 3 VISION trial of 177Lu-PSMA-617, securing clinical supply of no-carrier-added Lutetium, and raising sufficient capital to fund the company through expected completion of the trial," said Mike Sherman, president and CEO of Endocyte. "We continue to expect the first patient visit in the VISION trial in the second quarter and are working to advance EC17/CAR T-cell therapy, our folate-targeted CAM-based therapy, for which we expect to have an IND submitted in the fourth quarter of 2018."

Mr. Sherman continued, "In addition, we are encouraged by the updated 30 patient data from the ongoing phase 2 trial at Peter MacCallum Cancer Centre in Melbourne, Australia, published today in The Lancet Oncology. We anticipate an update at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June with early data on the additional 20 patients dosed in the expansion phase of that trial. Enrollment also continues in the phase 2 TheraP trial in Australia comparing 177Lu-PSMA-617 to cabazitaxel in 200 patients."

First Quarter and Recent Highlights

Finalized the design for the phase 3 VISION trial evaluating 177Lu-PSMA-617 in patients with metastatic castration-resistant prostate cancer (mCRPC) following a successful End-of-Phase 2 meeting with the U.S. Food and Drug Administration.

Announced an agreement with ITM Isotopen Technologien München AG to supply no-carrier-added Lutetium (177Lu) to support the phase 3 VISION trial.

Presented data on the chimeric antigen receptor T-cell (CAR T) adaptor molecule (CAM) platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018 confirming the anti-tumor activity of Endocyte’s folate-targeted EC17/CAR T-cell therapy.

·

Completed an underwritten registered public offering of 20,535,714 shares of its common stock, including full exercise of the underwriters’ option to purchase additional shares of common stock, at a public offering price of $4.20 per share. Endocyte received aggregate net proceeds from the offering of approximately $80.9 million.

·

Hired additional experienced clinical trial professionals to ensure strong execution and support the success of its clinical programs.

·

Elected Patrick Machado, J.D., co-founder and former chief business and financial officer of Medivation, and Dawn Svoronos, former president of Merck’s Europe/Canada region, to serve on the Board of Directors, bringing significant commercial leadership and understanding of the prostate cancer market to the Board.

Expected 2018 Milestones

·

First patient visit for phase 3 VISION trial of 177Lu-PSMA-617 in mCRPC (2Q 2018).

·

50-patient response rate data readout of investigator-initiated trial of 177Lu-PSMA-617 in mCRPC patients at Peter MacCallum Cancer Centre in Melbourne, Australia, to be presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (June 2018).

·

Publications on additional ongoing investigator-initiated clinical trials of 177Lu-PSMA-617 in prostate cancer patients (2018).

·

IND for phase 1 trial of EC17/CAR T-cell therapy in patients with osteosarcoma (4Q 2018).

First Quarter 2018 Financial Results

Endocyte reported a net loss of $8.6 million, or $0.16 per basic and diluted share, for the first quarter of 2018, compared to a net loss of $11.5 million, or $0.27 per basic and diluted share for the same period in 2017.

Research and development expenses were $5.3 million for the first quarter of 2018, compared to $8.0 million for the same period in 2017. The decrease was primarily attributable to a strategic portfolio review announced in June 2017 which led to a reduction in workforce and the discontinuation of certain research and development activities, including: a decrease of $1.4 million in expenses related to pre-clinical work and general research, including the development of EC2629; a decrease of $0.8 million in EC1169 trial expenses; a decrease of $0.6 million in EC1456 trial expenses; a decrease of $0.5 million in compensation expense as a result of employee terminations since March 31, 2017, and a decrease of $0.4 million in manufacturing expense for EC1169 and EC1456. These decreases were partially offset by: an increase of $0.8 million in expenses related to development of PSMA-617; and an increase of $0.2 million related to our CAR T-cell therapy program.

General and administrative expenses were $3.8 million for the first quarter of 2018, which were consistent with the $3.7 million of expenses for the same period in 2017.

Cash, cash equivalents and investments were $173.1 million at Mar. 31, 2018, compared to $127.6 million at Mar. 31, 2017, and $97.5 million at Dec. 31, 2017. Cash, cash equivalents and investments of $173.1 million at Mar. 31, 2018 included $80.9 million of net proceeds from our public offering of 20,535,714 shares of our common stock that closed in March 2018.

Financial Expectations

The company anticipates its cash, cash equivalents and investments balance at the end of 2018 to exceed $130 million. Based on current operational assumptions, Endocyte has sufficient cash to fund its activities through the expected end of the VISION trial and potential proof of concept of its EC17/CAR T-cell therapy.

Conference Call

Endocyte management will host a conference call today at 8:30 a.m. EDT.

U.S. and Canadian participants:(877) 845-0711

International:(760) 298-5081

A live, listen-only webcast of the conference call may be accessed by visiting the Investors & News section of the Endocyte website, www.endocyte.com.

The webcast will be recorded and available on the company’s website for 90 days following the call.

Website Information

Endocyte routinely posts important information for investors on its website, www.endocyte.com, in the "Investors & News" section. Endocyte uses this website as a means of disclosing material information in compliance with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the "Investors & News" section of

Endocyte’s website, in addition to following its press releases, SEC filings, public conference calls, presentations and webcasts. The information contained on, or that may be accessed through, Endocyte’s website is not incorporated by reference into, and is not a part of, this document.

On May 9, 2018 ChemoCentryx, Inc., (Nasdaq:CCXI), reported financial results for the first quarter ended March 31, 2018 and provided an overview of the Company’s recent corporate highlights (Press release, ChemoCentryx, MAY 9, 2018, View Source [SID1234526364]).

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"Positive momentum continues to build throughout our product pipeline," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "A major goal is on the near horizon: the completion of patient enrollment in our global Phase III ADVOCATE pivotal trial of avacopan for the treatment of ANCA-associated vasculitis, while a second trial of avacopan in the orphan kidney disease of C3G is well underway. Standing also on the threshold of a new value-creating era, we intend to expand avacopan’s scope into orphan dermatological disease with the launch of clinical trials in HS in 2018. Advances continue with our CCR2 inhibitor CCX140, with trials launched in primary FSGS, a devastating disease with no approved therapies. With such progress continuing, we are laying the foundation for commercialization of these novel therapies in the U.S. in order to bring the benefits of our precision medicines to those enduring these serious diseases."

Recent Highlights

Patient enrollment in ChemoCentryx’s Phase III ADVOCATE pivotal trial of avacopan for the treatment of ANCA-associated vasculitis is nearing completion with over 85% of the 300 patient target enrolled to date. The trial will evaluate the safety and efficacy of avacopan following 52 weeks of treatment. The ADVOCATE trial is designed to show the effect of avacopan on improving active vasculitis, as well as testing durable clinical benefit, which is one of the major limitations of the current standard of care. The Company’s CMA application for avacopan for the treatment of ANCA-associated vasculitis is currently under review by the EMA.

ChemoCentryx has reached approximately 30% of the patient enrollment target in its clinical trial evaluating avacopan for C3G. C3G is a rare disorder that often affects the young, requiring dialysis and often kidney transplant with relapsing disease common. There is no approved effective treatment.

In 2018, ChemoCentryx plans to launch clinical trials of avacopan in HS, an inflammatory and chronic skin disease characterized by recurrent, painful, boil-like nodules under the skin.

The Company’s CCR2 inhibitor CCX140 is currently being studied in two sub-populations of primary FSGS, an orphan kidney disease with no approved treatment option. One trial involves sub-nephrotic primary FSGS patients, whose disease cause is idiopathic; and the other trial is for primary FSGS patients with nephrotic syndrome, where reduction in proteinuria may constitute the registration endpoint. Further support of CCR2’s role in FSGS is highlighted in the peer reviewed findings published in March in the journal PLOS-ONE.
First Quarter 2018 Financial Results

Cash, cash equivalents and investments totaled $177.1 million at March 31, 2018.

Revenue was $9.5 million for the first quarter of 2018, compared to $8.2 million for the same period in 2017. Revenue recognized represents amortization of the upfront license fee commitments, milestone payments and collaboration funding from Vifor pursuant to the Avacopan Agreement, Avacopan Amendment and CCX140 Agreement. The increase from 2017 to 2018 was primarily due to the Company’s adoption of Accounting Standards Codification (ASC) Topic 606, Revenue from Contracts with Customers effective January 1, 2018.

Research and development expenses were $14.7 million for the first quarter of 2018, compared to $10.0 million for the same period in 2017. The increase from 2017 to 2018 was primarily due to continued patient enrollment of the avacopan Phase III ADVOCATE pivotal trial in patients with ANCA-associated vasculitis and start-up expenses related to the CCX140 and avacopan Phase II clinical trials in patients with FSGS and C3G, respectively.

General and administrative expenses were $4.7 million for the first quarter of 2018, compared to $4.6 million for the same period in 2017. The increase from 2017 to 2018 was primarily due to higher employee-related expenses partially offset by a decrease in professional legal fees.

Net loss for the first quarter of 2018 was $9.4 million, compared to $6.0 million for the same period in 2017.

Total shares outstanding at March 31, 2018 were approximately 49.1 million shares.

The Company expects to utilize cash and investments between $65 million and $75 million for the twelve months ending December 31, 2018.

Conference Call and Webcast

The Company will host a conference call and webcast today, May 9, 2018 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial 877-303-8028 (Domestic) or 760-536-5167 (International). The conference ID number is 2479786. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.