On December 7, 2017 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported the commercial availability of new Vantage 3D Hematology assays as well as the highlights of more than 45 nCounter-based research abstracts that will be presented at the 59th Annual Meeting of the America Society of Hematology being held December 9-12, in Atlanta (Press release, NanoString Technologies, DEC 7, 2017, View Source [SID1234522444]).

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The company announced the launch of new Vantage 3D hematology assays. Three of the studies presented at ASH (Free ASH Whitepaper) (Abstracts 1758, 2481 and 3945) include data from early access versions of NanoString’s Vantage 3D DNA:RNA:Protein Heme Assays, which are launching commercially at ASH (Free ASH Whitepaper). These assays enable simultaneous, multi-analyte analysis of single nucleotide variants (SNVs), RNA, protein, and phospho-protein. The assay includes curated content that covers clinically actionable SNVs and Indels, and provide comprehensive expression profiling of RNA, protein, and phospho-protein in key pathways including PI3K, JAK-STAT, BCR, and TCR signaling.

One of the presentations will be made by Dr. Sergio Rutella, M.D. Ph.D. FRCPath., Professor of Cancer Immunotherapy at the John van Geest Cancer Research Centre at Nottingham Trent University. Dr. Rutella stated, "AML is a rare disease and we need to maximize the amount of information we collect. We are using NanoString’s Vantage 3D DNA:RNA:Protein Heme Assay to capture information about single nucleotide variants, mutations, and new antigens that could be compiled into a ‘super signature’ to better characterize the disease and stratify the treatment of patients."

"Our customers and collaborators are presenting a record body of nCounter-based research at the 59th annual ASH (Free ASH Whitepaper) conference," said Brad Gray, president and chief executive officer of NanoString Technologies. "This research demonstrates the advances that are being enabled with the nCounter platform in subtyping lymphomas and optimizing regimens to achieve better clinical outcomes."

The ASH (Free ASH Whitepaper) Annual Meeting will include at least four oral presentations and forty-one posters in various leukemia, lymphoma, and myeloma malignancies that demonstrate the utility of the nCounter platform across the heme-oncology spectrum. In particular, NanoString’s collaborators in Diffuse Large B-Cell Lymphoma (DLBCL) are presenting 11 abstracts highlighting the potential clinical relevance of NanoString’s Lymphoma Subtyping Test (LST) and its potential for directing treatment decisions, including:

Results from the frontline prospective Phase III BIO-DLBCL04 study conducted by the Fondazione Italiana Linfomi (FIL). The results show that ABC subtype determined by the NanoString LST assay is associated with a worse outcome in untreated, poor-risk, young DLBCL (Abstract #4010).

Researchers from the European Institute of Oncology found that the NanoString LST robustly identifies DLBCL subgroups according to the Cell-of-Origin (COO). The molecular definition of the COO can be used to identify patients at high risk of poor outcome when treated with R-CHOP and who may benefit by intensified high dose chemotherapy or experimental new treatments (Abstract #3998).

Results from an exploratory biomarker analysis of the Phase 3 GOYA Trial comparing the efficacy and safety of obinutuzumab plus CHOP (G-CHOP) with R-CHOP in patients with previously untreated DLBCL. Using the NanoString LST assay and a new cutoff on the Linear Predictor Score (LPS), a new distinct molecular subgroup of GCB DLBCL, referred to as "strong-GCB", was identified. Results from the exploratory analysis show that treatment with G-CHOP confers substantial clinical benefit over R-CHOP in this new subgroup of DLBCL (Abstract #1543).
The table below includes a selection of 2017 ASH (Free ASH Whitepaper) abstracts that best illustrate the potential clinical utility of nCounter across multiple tumor types. You can learn more about the capabilities of the nCounter platform by visiting NanoString at booth #2465 at ASH (Free ASH Whitepaper).

On December 7, 2017 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the company will be webcasting its corporate presentation at the BMO Capital Markets Healthcare Conference in New York, NY (Press release, Jazz Pharmaceuticals, DEC 7, 2017, View Source;p=RssLanding&cat=news&id=2321712 [SID1234522443]).

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Matt Young, executive vice president and chief financial officer, will provide an overview of the company and a business and financial update at the conference on Thursday, December 14, 2017 at 10:30 a.m. EST / 3:30 p.m. GMT.

A live audio webcast of the presentation may be accessed from the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the presentation to ensure adequate time for any software downloads that may be necessary to listen to the webcast.

An archive of the webcast will be available for at least one week following the presentation on the Investors section of the company’s website at www.jazzpharmaceuticals.com.

On December 7, 2017 Enzo Biochem Inc. (NYSE:ENZ) reported improved operating results for the first fiscal quarter ended October 31, 2017, including year over year double digit revenue growth at Enzo Clinical Labs (Press release, Enzo Biochem, DEC 7, 2017, View Source [SID1234522442])

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.

First Quarter’s Highlights (Year Over Year):

· Total revenue in the first fiscal quarter increased to $27.7 million, or 5%, from $26.3 million in the prior year period.
· Clinical Labs revenue totaled $20.3 million, or an increase of 10% from $18.6 million a year ago.
· Gross profit increased 1%, with operating income improving $0.7 million, or 53%. Gross margin was 44%.
· The GAAP and non-GAAP net loss was $0.6 million or ($0.01) per fully diluted share, an improvement of $0.8 million, or 56% over the prior year’s net loss of $1.5 million or $(0.03) per fully diluted share.
· Total cash and cash equivalents at October 31, 2017 was $66.8 million, an increase of $2.7 million from July 31, 2017. Tight cost controls and efficiencies resulted in positive $2.6 million cash flow from operations in the quarter.
· During the quarter, the New York State Health Department issued conditional approval of the final three women’s health related molecular diagnostic tests which completes a 13-analyte panel, placing Enzo in a leading position to benefit from higher-margin proprietary women’s health diagnostics.
· Plant facilities in Farmingdale, NY, undergoing expansion to accommodate anticipated growth in manufacturing of growing roster of new molecular diagnostic assays and to increase laboratory capacity and to support GMP certification.

Barry Weiner, President, Comments:

"The first quarter of fiscal 2018 saw additional progress in moving aggressively towards our model as an integrated, growth-oriented molecular diagnostics company, and a low-cost medically related assay provider and reference service organization. Revenues continued to grow, spurred by double digit growth in the quarter at Clinical Labs, the result of expanded product and service capabilities and geographical expansion, along with increasing volume from our association with leading healthcare insurance providers. Sales, general and administrative costs (SG&A) are being held in check, even as product development and research efforts continue. We are also completing preparations to launch our marketing and sales program of our comprehensive products and reference services menu, while also adding to our Lab’s capability, particularly in the women’s health diagnostic field.

"We are preparing to offer one of the industry’s most advanced and well-rounded 13-analyte women’s health diagnostics panel, which last month was presented at the annual meeting of the prestigious Association for Molecular Pathology. The study our group reported on demonstrated that Enzo’s AMPIPROBE technology provides a new proprietary approach to creating clinically sensitive and specific multiplex real-time PCR assays, which, when run in tandem, can serve as a comprehensive panel for the accurate detection and identification of vaginitis-associated microorganisms. Enhanced by our proprietary AMPIPROBE technology, it offers faster, more specific and more sensitive molecular vaginitis testing, which we expect to improve diagnoses of vaginitis, a leading medical concern among women. Increasingly, when measured against the performance of similar assays, Enzo products continue to outperform, as reported recently in a leading diagnostic pathology publication, which cited our Polyview technology for viewing the clinical morphology of HPV specimens as having no false positives. That compared favorably to other leading competitive products that did have false positives in their tests.

"Our focus is on gaining sustained profitability and advancing healthcare in today’s challenging environment by providing affordable and reliable diagnostic testing. With Medicare reimbursement rates expected to decline materially over the next three years based on a new formula being adopted in place of one used over the past 30 years, the challenges are clear. Enzo’s transformative blueprint may help to alleviate this new challenge to the nation’s independent labs. This opportunity is reflected in our expanding line of medically related, versatile, highly efficient platforms and assays that are increasingly professionally recognized for their sensitivity and economics."

First Quarter Results
Total revenues increased to $27.7 million or 5% higher than the prior year period driven by increased Clinical labs services revenue from MDx tests. Gross profit was $12.2 million or 44% of total revenue. Total operating expenses were $12.9 million, a decline of $0.5 million or 4% over a year ago.

The GAAP and Non-GAAP net loss amounted to $(0.6) million, or $(0.01) per fully diluted share, compared to the year ago net loss of $(1.5) million or $(0.03) per fully diluted share, an improvement of $0.8 million. EBITDA and Adjusted EBITDA were essentially break even compared with a loss of $(0.6) million a year ago, a $0.5 million improvement year over year.

As of October 31, 2017 working capital amounted to $71.4 million, cash and cash equivalents totaled $66.8 million, up $2.7 million from three months earlier. Apart from capital leases, there was no debt.

Quarterly Segment Results

Enzo Clinical Labs revenues increased to $20.3 million, or 10%, from $18.6 million a year ago. As noted above, expansion of its diagnostics services to additional nearby geographical locations, the increased product mix of diagnostic tests, particularly in the women’s health field, and greater volume, all are contributing to its steady test and service growth. Gross profit increased to $8.3 million, from $7.7 million, with gross margin as a percentage of revenues at 41% for both the current and year ago quarters. Total operating expenses increased by 3.6%, to $6.9 million. Operating income amounted to $1.4 million, a nearly 40% increase.

Enzo Life Sciences revenues were $7.1 million compared to $7.4 million a year ago, a decline of $0.3 million or 5%. Gross profit approximated $4 million and gross margin as a percentage of revenue was 54%, compared to $4.4 million and 57%, respectively, a year ago. With the continued focus on reducing costs and improving efficiency, SG&A declined by 11%, to $2.6 million, with total operating expenses declining 12%, to $3.2 million.

Conference Call

The Company will conduct a conference call Friday, December 8, 2017 at 8:30 AM ET. The call can be accessed by dialing 1-888-459-5609. International callers can dial 1-973-321-1024. Please reference PIN number 3678737.

Interested parties may also listen over the Internet at: View Source

To listen to the live call on the Internet, please go to the web site at least fifteen minutes early to register, download and install any necessary audio software. For those who cannot listen to the live broadcast, a replay will be available approximately two hours after the end of the live call, through midnight (ET) on December 22, 2017. The replay of the conference call can be accessed by dialing 1-855-859-2056, and when prompted, use PIN number 3678737. International callers can dial 1-404-537-3406, using the same PIN number.

NON-GAAP Financial Measures

To comply with Regulation G promulgated pursuant to the Sarbanes-Oxley Act, Enzo Biochem attached to this news release and will post to the Company’s investor relations web site (www.enzo.com) any reconciliation of differences between non-GAAP financial information that may be required in connection with issuing the Company’s quarterly financial results.

The Company uses EBITDA as a measure of performance to demonstrate earnings exclusive of interest, taxes, depreciation and amortization. Adjustments to EBITDA are for items of a non-recurring nature and are reconciled on the table provided. The Company manages its business based on its operating cash flows. The Company, in its daily management of its business affairs and analysis of its monthly, quarterly and annual performance, makes its decisions based on cash flows, not on the amortization of

assets obtained through historical activities. The Company, in managing its current and future affairs, cannot affect the amortization of the intangible assets to any material degree, and therefore uses EBITDA as its primary management guide. Since an outside investor may base its evaluation of the Company’s performance based on the Company’s net loss not its cash flows, there is a limitation to the EBITDA measurement. EBITDA is not, and should not be considered, an alternative to net loss, loss from operations, or any other measure for determining operating performance of liquidity, as determined under accounting principles generally accepted in the United States (GAAP). The most directly comparable GAAP reference in the Company’s case is the removal of interest, taxes, depreciation and amortization.

We refer you to the tables attached to this press release which includes reconciliation tables of GAAP to Non-GAAP net income (loss) and EBITDA to Adjusted EBITDA.

On December 7, 2017 Champions Oncology, Inc. (Nasdaq: CSBR), engaged in the development of advanced technology solutions and services to personalize the development and use of oncology drugs, reported its financial results for the second fiscal quarter and six months ended October 31, 2017 (Press release, Champions Oncology, DEC 7, 2017, View Source [SID1234522440]).

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Second Quarter and Recent Business Highlights:

•
Record quarterly revenue of $5.2 million, an increase of 16.7% year-over-year
•
Record Translational Oncology Services ("TOS") revenue of $4.8 million, an increase of 21.8% year-over-year
•
Awarded a two-year, $2 million, competitively-bid contract through the Small Business Innovation Research (SBIR) program to build and study an ethnically diverse cohort of metastatic prostate patient-derived xenograft (PDX) models
•
Clinical correlation between patient-derived xenograft (PDX) model responses and patient responses to oncology therapies highlighted in journal, Annals of Oncology; the report was published by the European Society for Medical oncology (ESMO) (Free ESMO Whitepaper), the leading European professional organization for medical oncology in Europe.
•
Reiterated expectations for fiscal year 2018 revenue growth of at least 20% over fiscal year 2017

Ronnie Morris, CEO of Champions, commented, "The continued execution of our strategic growth plan and disciplined management of expenses resulted in quarterly operating profitability and another quarter of record revenue, reinforcing our confidence in achieving at least 20% revenue growth for fiscal 2018 and the longer-term prospects for our business. As expected, revenues reached a level that exceeded our expenses, establishing a trajectory for long-term profitability. As we continue to scale, we expect to further minimize the impact of quarter-to-quarter fluctuations and generate consistent profitability."

"Engagement with existing and potential new clients remains solid, and we are encouraged by the level of interest being expressed for our services and the future growth opportunities we see before us," added Morris. "As we continue to expand our presence and collaborate with pharmaceutical companies on new projects, we are further increasing the value and importance of our models in the pre-clinical and clinical drug development process."

Financial Results

Exhibit 99.1

For the second quarter of fiscal 2018, revenue increased 16.7% to $5.2 million, as compared to $4.5 million for the second quarter of fiscal 2017. Total operating expenses for the second quarter fiscal 2018 and 2017 was $5.3 million and $5 million, respectively, an increase of $300,000 or 6.5%. Revenue was $10.2 million and $8.1 million for the six months ended October 31, 2017 and 2016, respectively, an increase of $2.1 million or 26%. Total operating expenses for the six months ended October 31, 2017 and 2016 was $10.9 million and $11.1 million, respectively, a decrease of $200,000 or (2%).

For the second quarter of fiscal 2018, Champions reported a loss from operations of $70,000, including $148,000 in stock-based compensation, an improvement of $423,000 or 85.8% compared to the loss from operations of $493,000, inclusive of $535,000 in stock-based compensation, in the second quarter of fiscal 2017. Excluding stock based compensation and depreciation, Champions recognized net income from operations of $175,000 for the second quarter 2018.

Net cash generated was $229,000 for the three months ended October 31, 2017. Net cash used for the same period last year was $134,000. The improvement in cash flow is the result of revenue growth and expense management.

The Company ended the quarter with $660,000 of cash and cash equivalents and reiterates its position that it does not currently intend to raise capital in the equity market.

TOS revenue was $4.8 million for the three months ended October 31, 2017 compared to $4 million for the three months ended October 31, 2016, an increase of $800,000 or 21.8%.

TOS cost of sales was $2.4 million for the three months ended October 31, 2017, an increase of $600,000, or 30.9%, compared to $1.8 million for the three months ended October 31, 2016. For the three months ended October 31, 2017 and 2016, gross margin for TOS was 50.4% and 53.8%, respectively. The increase in TOS cost of sales was due to an increase in the number and size of TOS studies. The gross margin often fluctuates quarter to quarter, resulting from timing differences between revenue and expense recognition.

Personalized Oncology Services ("POS") revenue was $378,000 and $497,000 for the three months ended October 31, 2017 and 2016, respectively, a decrease of $119,000 or (23.9%). The decrease is due mainly to a decrease in implant and drug study revenue.

POS cost of sales was $259,000 for the three months ended October 31, 2017, a decrease of $115,000, or (30.7%), compared to $374,000 for the three months ended October 31, 2016. For the three months ended October 31, 2017 and 2016, gross margin for POS was 31.5% and 24.8%, respectively. The improvement is attributed to the increase in higher margin sequencing revenue.

Research and development expense was $1.1 million for the three months ended October 31, 2017, an increase of $100,000, or 10.6%, compared to $1 million for the three months ended October 31, 2016. Sales and marketing expense for the three months ended October 31, 2017 was $551,000, a decrease of $166,000, or (23.2%), compared to $717,000 for the three months ended October 31, 2016. The decrease is mainly due to a reduction of marketing resources for the POS division. General and administrative expense was $1 million for both the three months ended October 31, 2017 and 2016.

Year-to-Date Financial Results

Exhibit 99.1

For the first six months of fiscal 2018, revenue increased 26.0% to $10.2 million, as compared to $8.1 million for the first six months of fiscal 2017. Total operating expenses for the first six months of fiscal 2018 were $10.9 million compared to $11.1 million for the first six months of fiscal 2017, a decrease of $200,000 or (1.9%).

For the first six months of fiscal 2018, Champions reported a loss from operations of $689,000, including $711,000 and $132,000 in stock-based compensation and depreciation, respectively, an improvement of $2.3 million, or 77.2%, compared to the loss from operations of $3 million, inclusive of $1.7 million and $87,000 in stock-based compensation and depreciation, respectively, in the first six months of fiscal 2017. Excluding stock-based compensation and depreciation, Champions recognized income from operations of $155,000 for the six months ended October 31, 2017 compared to net loss of $1.3 million for the six months ended October 31, 2016.

TOS revenue was $9.4 million for the six months ended October 31, 2017 compared to $7.1 million for the six months ended October 31, 2016, an increase of $2.3 million or 32.3%.

TOS cost of sales was $4.6 million for the six months ended October 31, 2017, an increase of $700,000, or 19.8%, compared to $3.9 million for the six months ended October 31, 2016. Gross margin for TOS was 50.7% for the first six months of fiscal 2018 compared to 45.5% for the first six months of fiscal 2017.

POS revenue was $818,000 for the six months ended October 31, 2017 compared to $1.0 million for the six months ended October 31, 2016, a decrease of $182,000 or (18.8%).

POS cost of sales was $646,000 for the six months ended October 31, 2017, a decrease of $201,000, or (23.7%), compared to $847,000 for the six months ended October 31, 2016. Gross margin for POS for the six months ended October 31, 2017 was 21% compared to 15.9% for the six months ended October 31, 2016.

Research and development expense was $2.2 million for both the six months ended October 31, 2017 and 2016. Sales and marketing expense for the six months ended October 31, 2017 was $1.2 million, a decrease of $400,000, or (24.8%), compared to $1.6 million for the six months ended October 31, 2016. General and administrative expense was $2.2 million for the six months ended October 31, 2017, a decrease of $400,000 or (15.3%), compared to $2.6 million for the six months ended October 31, 2016.

Net cash used in operations was $1.7 million for the six months ended October 31, 2017 compared to $2.6 million for the six months ended 2016, a decrease of $900,000 or 34.3%.

Conference Call Information:

The Company will host a conference call today at 4:30 p.m. EST (1:30:00 p.m. PST) to discuss its second quarter financial results. To participate in the call, please call 888-567-1602 (domestic) or 404-267-0373 (international) 10 minutes ahead of the call and give the verbal reference "Champions Oncology."

Full details of the Company’s financial results will be available Friday, December 15, 2017 in the Company’s Form 10-Q at www.championsoncology.com.

* Non-GAAP Financial Information

See the attached Reconciliation of GAAP net loss to non-GAAP net loss for an explanation of the amounts excluded to arrive at non-GAAP net loss and related non-GAAP net loss per share amounts for the three and six months ended October 31, 2017 and 2016. Non-GAAP financial measures provide investors and management with supplemental measures of operating performance and trends that facilitate comparisons between periods before and after certain items that would not otherwise be apparent on a GAAP basis. Certain unusual or non-recurring items that management does not believe affect the Company’s basic operations do not meet the GAAP definition of unusual or non-recurring items. Non-GAAP net loss and non-GAAP loss per share are not, and should not be viewed as a substitute for similar GAAP items. Champions’ defines non-GAAP dilutive loss per share amounts as non-GAAP net loss divided by the weighted average number of diluted shares outstanding. Champions’ definition of non-GAAP net loss and non-GAAP diluted loss per share may differ from similarly named measures used by others.

On December 7, 2017 Ablynx NV [Euronext Brussels and Nasdaq: ABLX] reported that the Ablynx management team will host a conference call and webcast to present additional data from the Phase III HERCULES study of caplacizumab in acquired thrombotic thrombocytopenic purpura (aTTP), following its presentation by Professor Marie Scully in the late-breaking abstracts session at the 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Atlanta, GA, USA (Press release, Ablynx, DEC 7, 2017, View Source [SID1234522437]).

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The late-breaking data presentation will include positive topline results from the Phase III HERCULES study of caplacizumab announced on 2 October 2017 together with important data from additional analyses on the use of plasma exchange (PEX) and length of intensive care unit and hospital stay. The slides will be made available on the Ablynx website under Events & Presentations immediately after the presentation on 12 December 2017 at 7.30 am ET/1.30 pm CET.

Following the late-breaking data presentation, the Ablynx management team will host a conference call and webcast on 12 December 2017 at 4.00 pm CET/10.00 am ET. The live webcast and replay will be available via this link. If you wish to participate in the Q&A session, please dial +32(0)2 400 69 26 or +1 646 828 8193 and use confirmation code 9477994.

Late-breaking abstracts session at ASH (Free ASH Whitepaper), Atlanta, GA, USA
Date: Tuesday 12 December 2017
Abstract (LBA-1): Results of the Randomized, Double-Blind, Placebo-Controlled, Phase III HERCULES study of Caplacizumab in Patients with Acquired Thrombotic Thrombocytopenic Purpura
Presentation Time: 7.30 am ET (1.30 pm CET)
Presenter: Professor Marie Scully, M.D., Department of Haematology, University College London Hospitals NHS Trust, London, UK
Room: Building C, Level 1, Hall C2-C3 (Georgia World Congress Center)

About HERCULES
The HERCULES study recruited 145 patients and is the largest randomised, double-blind, placebo-controlled study conducted in patients with aTTP. Patients with an acute episode of aTTP were randomised 1:1 to receive either caplacizumab or placebo in addition to daily plasma exchange and immunosuppression. Patients received a single intravenous bolus of 10mg caplacizumab or placebo followed by a daily subcutaneous dose of 10mg caplacizumab or placebo for 30 days after the last daily PEX. If at the end of this treatment period there was evidence of persistent underlying disease activity (indicative of an imminent risk for recurrence), treatment could be extended for additional seven-day periods up to a maximum of 28 days and was to be accompanied by optimisation of immunosuppression. Patients were followed for a further 28 days after discontinuation of treatment.
A three-year follow-up study (NCT02878603) of patients who have completed the HERCULES study is in progress and will further evaluate the long-term safety and efficacy of caplacizumab and repeated use of caplacizumab, as well as characterising the long-term impact of aTTP.

About caplacizumab
Caplacizumab is a bivalent anti-vWF Nanobody that received Orphan Drug Designation in Europe and the United States in 2009. Caplacizumab blocks the interaction of ultra-large vWF multimers (ULvWF) with platelets and, therefore, has an immediate effect on platelet aggregation and the ensuing formation and accumulation of the micro-clots that cause the severe thrombocytopenia, tissue ischemia and organ dysfunction in aTTP. This immediate effect of caplacizumab has the potential to protect the patient from the manifestations of the disease while the underlying disease process resolves.

In February 2017, based on the Phase II study results, a Marketing Authorisation Application (MAA) was submitted to the European Medicines Agency (EMA) for approval of caplacizumab in aTTP. In July 2017, Ablynx received Fast Track designation from the Food and Drug Administration (FDA) for caplacizumab for the treatment of aTTP. In October 2017, positive results from the Phase III HERCULES study, meeting primary and two key secondary endpoints, were announced. These data are expected to further support the MAA, as well as a planned Biologics License Application (BLA) filing in the United States in 2018. If approved by regulatory authorities, caplacizumab would be the first therapeutic specifically indicated for the treatment of aTTP.
About aTTP

aTTP is a rare, acute, life-threatening, autoimmune blood clotting disorder. It is caused by impaired activity of the ADAMTS13 enzyme, leaving ULvWF molecules uncleaved (vWF is an important protein involved in the blood clotting process). These ULvWF molecules spontaneously bind to blood platelets, resulting in severe thrombocytopenia (very low platelet count) and clot formation in small blood vessels throughout the body1, leading to ischemia and widespread organ damage2.

Despite the current standard-of-care treatment consisting of PEX and immunosuppression, episodes of aTTP are still associated with a mortality rate of up to 20%, with most deaths occurring within 30 days of diagnosis3. Furthermore, patients are at risk of acute thromboembolic complications (e.g. stroke, myocardial infarction) and of recurrence of disease. Some patients are refractory to therapy1, which is associated with a poor prognosis for survival of an acute episode of aTTP. Long term, patients are at increased risk for hypertension, major depression, and premature death4.