On November 1, 2018 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of specialized CAR-T cell-based therapies, reported that two abstracts detailing updated clinical results from the Phase 1 THINK dose-escalation trial and anticipated clinical trials for the CYAD-01 program will be presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, December 1-4, 2018 (Press release, Celyad, NOV 1, 2018, View Source [SID1234530542]). Company management will also review the results of the THINK trial and provide an update on Celyad’s clinical development program for CYAD-01 at an Analyst/Investor event, which will also be available via webcast on December 3, 2018.

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"We are encouraged by the preliminary THINK study data evaluating CYAD-01, without preconditioning chemotherapy in patients with relapsed or refractory acute myeloid leukemia," said Dr. Christian Homsy, CEO of Celyad. "The data supplement a growing body of evidence that CYAD-01 shows encouraging clinical activity and is well-tolerated and suggests its potential for the treatment of acute myeloid leukemia, a challenging disease with limited therapeutic options. In addition to this important milestone, we continue to investigate CYAD-01 in alternative protocols to further optimize its clinical benefit."

Updated data from the THINK trial of CYAD-01 in patients with r/r AML will be presented by Principal Investigator David A. Sallman, M.D., of the Moffitt Cancer Center, on December 3, 2018. The presentation will include new information on safety, activity and correlative science data of the complete dose-escalation segment of the trial.

Top-line data from the abstract as of a data cut-off date of July 31, 2018, included:

Of the seven response-evaluable r/r AML patients enrolled in the trial who received the per-protocol dose of CYAD-01, the best overall response rate was 42% (three patients). Two additional patients experienced important clinical benefit with hematologic improvement and bone marrow blasts decrease, leading to clinical activity of 71% (five patients).
One patient experienced a complete remission with partial hematologic recovery (CRh) and two patients experienced a complete remission with incomplete marrow recovery (CRi). One CRh and one CRi occurred at dose level 1 (DL1) with an additional CRi at dose level 3. All three responders achieved a response by day 29 (i.e., prior to the third administration of CYAD-01).
The patient with CRh from DL1 was bridged to allogeneic hematopoietic stem cell transplantation (allo-HSCT) on day +97 post treatment with CYAD-01. This patient remains in durable complete molecular remission (CRMRD-) for more than one year (ongoing). A detailed case report of this patient was published in Haematologica in April 2018.
Of the two additional r/r AML patients who experienced a clinical benefit, one patient had a decrease in blast counts from 24% to 10%, while a second patient had a decrease from 9.8% to 5.5%. Disease stabilization in these patients were observed for three months and over four months (ongoing), respectively. Both patients were treated in dose level 2 of the study.
Overall, 12 patients with hematological malignancies (AML, myelodysplastic syndrome and multiple myeloma) treated with CYAD-01 in the cohort had reached the safety follow-up. The most common treatment-related adverse events (AEs) included pyrexia, cytokine release syndrome (CRS), hypoxia, lymphopenia, fatigue and nausea. CRS occurred in five patients (three grade 1/2 AEs and two grade 3 AEs), with rapid resolution following the appropriate treatment, including tocilizumab. Overall, five patients experienced grade 3/4 treatment-related AEs. No neurotoxicity AEs were observed in patients treated with CYAD-01.

CYAD-01 and THINK Trial Design

CYAD-01 is an investigational CAR-T therapy in which a patient’s T cells are engineered to express the chimeric antigen receptor NKG2D, a receptor expressed on natural killer (NK) cells that binds to eight stress-induced ligands expressed on tumor cells.

The THINK trial (NCT03018405) is an open-label, dose-escalation Phase 1 trial assessing the safety and clinical activity of multiple CYAD-01 administrations without prior preconditioning in two parallel cohorts: i) patients with hematological malignancies, including r/r AML, and ii) patients with metastatic solid tumors. The dose escalation segment of the study evaluates three dose levels (300 million, 1 billion and 3 billion cells per injection) of one cycle of three CYAD-01 administrations with two-week intervals.

ASH Analyst/Investor Event and Webcast Information

Celyad will host an Analyst/Investor event on Monday, December 3, 2018, beginning at 8:30 p.m. PT to review data presented at ASH (Free ASH Whitepaper). The event will be webcast live and can be accessed under Events & Webcasts in the Investors section of the Company’s website.

A complete list of Celyad and collaborator presentations to be made at ASH (Free ASH Whitepaper) appears below:

Oral Presentation

Remissions in Relapse/Refractory Acute Myeloid Leukemia Patients Following Treatment with NKG2D CAR-T Therapy Without a Prior Preconditioning Chemotherapy (Abstract #111326 – Publication Number 902)

Presenter: David A. Sallman, M.D., Moffitt Cancer Center

Date: Monday, December 3, 2018, 4:45 p.m. Pacific Time

Location: Manchester Grand Hyatt San Diego, Seaport Ballroom F

Poster Presentation

Phase 1 Studies Assessing the Safety and Clinical Activity of Multiple Doses of a NKG2D-based CAR-T Therapy, CYAD-01, in Acute Myeloid Leukemia (Abstract #114747 – Publication Number 1398)

Presenter: Jason B Brayer, MD, Moffitt Cancer Center

Date: Saturday, December 1, 2018, 6:15 PM – 8:15 PM

Location: San Diego Convention Center, Hall GH

On November 1, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported it will report its third quarter 2018 financial results and business progress on Thursday, November 8, 2018, after the close of the U.S. financial markets (Press release, Tocagen, NOV 1, 2018, View Source;p=RssLanding&cat=news&id=2374897 [SID1234530570]).

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On November 1, 2018 4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 antagonist drugs to improve immune cell trafficking to treat rare disease and cancer, reported that an abstract highlighting X4P-001-RD, the company’s CXCR4 antagonist, has been selected for poster presentation at the 60th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting, taking place Dec. 1-4 in San Diego (Press release, X4 Pharmaceuticals, NOV 1, 2018, View Source [SID1234530651]). The presentation will describe Phase 2 results used to determine the Phase 3 dose in the ongoing Phase 2/3 study of X4P-001-RD in patients with WHIM syndrome, a rare genetic, primary immunodeficiency disease.

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At ASH (Free ASH Whitepaper): X4 Pharmaceuticals to present clinical data used to determine Phase 3 Dose from Phase 2/3 Study of X4P-001-RD in WHIM Syndrome.

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Details of the presentation on X4P-001-RD in WHIM syndrome are as follows:

Title: Determination of Phase 3 Dose for X4P-001 in Patients with WHIM Syndrome

Date & Time: Saturday, December 1, 6:15 PM – 8:15 PM

Location: San Diego Convention Center, Hall GH

Publication #: 1102

Poster Session: 201. Granulocytes, Monocytes, and Macrophages: Poster I

About WHIM Syndrome
WHIM syndrome is a primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene resulting in susceptibility to certain types of infections. WHIM is an abbreviation for the characteristic clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Within the overall category of primary immunodeficiencies, there are between 15,000 and 100,000 patients in the U.S. that are classified with primary immunodeficiency disease of unknown origin – of which WHIM is one.1,2,3 WHIM syndrome is a rare disorder and the precise prevalence or incidence of patients that have the genetic mutation responsible for WHIM syndrome is unknown. Individuals with WHIM syndrome are more susceptible to potentially life-threatening bacterial infections4. Additionally, WHIM syndrome is associated with significant morbidity beginning in early childhood and continuing throughout life. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin or G-CSF. There is no approved therapy for the treatment of WHIM syndrome.

On October 31, 2018 OncBioMune Pharmaceuticals, Inc. (OTCQB:OBMP) ("OncBioMune" or the "Company"), a clinical-stage biopharmaceutical company engaged in the development of a proprietary therapeutic cancer vaccine immunotherapy and targeted cancer therapies, is pleased to inform shareholders that the Company’s CEO, Dr. Jonathan Head, has been asked to present alongside other well renowned faculty speakers at Oncology Meeting Innovations’ 2018 Global Summit on Genitourinary Malignancies (Press release, Oncbiomune, OCT 31, 2018, View Source [SID1234530470]). This year’s conference is being held November 1-4, 2018 at Fairmont Banff Springs hotel in Banff, Alberta, Canada.

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Dr. Head is scheduled to speak at 11:30 AM CT on Friday, November 2, 2018 during the session titled, "Immunotherapy in GU Malignancies." Dr. Head will be discussing the qualities and therapeutic benefits of ProscaVax, the Company’s novel immunotherapeutic vaccine for prostate cancer, documented through a successfully completed Phase 1a clinical trial in late-stage prostate cancer patients. Dr. Head’s presentation will further include information on the upcoming Phase 2 clinical trials, one to be hosted at a teaching hospital of Harvard University enrolling early-stage prostate cancer patients and a second at Urology Clinics of North Texas planned to enroll recurrent prostate cancer patients.

"It’s an honor to be selected to speak at OMI’s Global Summit. I look forward to discussing ProscaVax with the scientific and academic community as a new treatment candidate for prostate cancer, a disease greatly in need of new options for men," commented Dr. Head. "Now that we are closing in on commencing both Phase 2 trials, we intend to dedicate more time towards spreading the word about ProscaVax and the potential we believe it holds to fill a large gap in oncology."

Oncology Meeting Innovations’ Global Summit on Genitourinary Malignancies is a four-day meeting fostering discussion and debate on the evolution of treatment in prostate cancer, renal cell carcinoma, and bladder cancer. Topics will include current standards of care, and the evolving clinical landscape in the treatment of GU cancers. The summit features a focused approach towards individual patient populations through case-based discussions. Panel discussions will highlight clinical application of approved and emerging agents as well as the challenges within the field of GU Oncology. The Summit will assemble translational scientists, clinical researchers, and key community oncologists to further progress this rapidly evolving area of oncology.

About Prostate Cancer

According to the American Cancer Society (ACS), prostate cancer is the most common type of cancer in men other than skin cancer, with about 1 in 9 men diagnosed during their lifetime. ACS estimates that about 164,690 new cases of prostate cancer will be diagnosed during 2018 and approximately 29,430 men will die from the disease this year. Prostate cancer is the second leading cause of cancer death in men, trailing only lung cancer. Approximately 2.9 million men are living with prostate cancer today. The average age of diagnosis is 66, with the disease considered rare in men under the age of 40

On October 31, 2018 PRA Health Sciences, Inc. ("PRA," "we," "us" or the "Company") (NASDAQ: PRAH) reported financial results for the quarter ended September 30, 2018 (Press release, PRA Health Sciences, OCT 31, 2018, View Source;p=RssLanding&cat=news&id=2374565 [SID1234530527]).

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For the three months ended September 30, 2018, revenue was $717.6 million, which represents growth of 23.3%, or $135.6 million, compared to the third quarter of 2017 at actual foreign exchange rates. On a constant currency basis, revenue grew $139.7 million, an increase of 24.0% compared to the third quarter of 2017. On January 1, 2018, the Company adopted Accounting Standards Codification Topic 606, "Revenue from Contracts with Customers," or ASC 606, using the modified retrospective method for all contracts that were not completed as of January 1, 2018. Prior periods have not been restated under this guidance and remain as previously reported. The primary impact of applying this new guidance on our statement of operations is that (i) we now recognize reimbursements from our customers for payments to investigators as revenue, whereas these payments and costs were previously recorded on a net basis, and (ii) we include all reimbursed costs in the total project costs when measuring our progress under our research contracts instead of recording these amounts on a separate basis.

Excluding the impact of the adoption of ASC 606 and reimbursement revenue, revenue increased $78.4 million, which represents growth of 15.8% at actual foreign exchange rates and 16.3% on a constant currency basis. Organic revenue growth, excluding the adoption of ASC 606, reimbursement revenue and revenue attributable to our Data Solutions segment, was 7.7% at actual foreign exchange rates and 8.2% on a constant currency basis.

Net new business for our Clinical Research segment for the quarter ended September 30, 2018 was $657.2 million, representing a net book-to-bill ratio of 1.28 for the period. Our calculation of the net book-to-bill ratio excludes the revenue impact of adopting ASC 606, excludes reimbursement revenue and excludes $60.6 million of revenue from our Data Solutions segment. Net new business during the quarter contributed to an ending backlog of $4.1 billion at September 30, 2018.

"We are delighted to have delivered another quarter with strong financial results on many fronts" said Colin Shannon, PRA’s Chief Executive Officer. "Our key financial metrics continue to improve, as highlighted by our new business wins, our strong revenue growth and our expanding margins. Our financial performance reflects our commitment to client delivery and the efforts of our employees."

Direct costs were $371.4 million during the three months ended September 30, 2018 compared to $326.9 million for the third quarter of 2017. The increase in direct costs was primarily due to an increase in labor-related costs of $11.1 million in our Clinical Research segment as we continue to hire billable staff to ensure appropriate staffing levels. In addition, our Data Solutions segment resulted in $29.4 million of incremental direct costs when compared to the third quarter of 2017. We also had a favorable impact of $6.1 million from fluctuation in foreign currency exchange rates during the three months ended September 30, 2018. Excluding the impact of the adoption of ASC 606 and reimbursement revenue, direct costs were 64.8% of revenue during the third quarter of 2018 compared to 66.1% of revenue during the third quarter of 2017.

Selling, general and administrative expenses were $92.6 million during the three months ended September 30, 2018 compared to $79.3 million for the third quarter of 2017. Excluding the impact of the adoption of ASC 606 and reimbursement revenue, selling, general and administrative costs were 16.2% of revenue during the third quarter of 2018 compared to 16.0% of revenue during the third quarter of 2017.

GAAP net income was $1.5 million for the three months ended September 30, 2018, or $0.02 per share on a diluted basis, compared to GAAP net income of $48.2 million for the three months ended September 30, 2017, or $0.73 per share on a diluted basis.

EBITDA was $64.4 million for the three months ended September 30, 2018, representing an increase of 6.7% compared to the third quarter of 2017. Adjusted EBITDA was $120.9 million for the three months ended September 30, 2018, representing growth of 29.6% compared to the third quarter of 2017.

Adjusted net income was $74.8 million for the three months ended September 30, 2018, representing growth of 29.3% compared to the third quarter of 2017. Adjusted net income per diluted share was $1.13 for the three months ended September 30, 2018, representing growth of 28.4% compared to the third quarter of 2017.

A reconciliation of our non-GAAP measures, including EBITDA, adjusted EBITDA, adjusted net income, adjusted net income per diluted share and our 2018 guidance, to the corresponding GAAP measures is included in this press release.

Nine Months Ended September 30, 2018 Financial Highlights

For the nine months ended September 30, 2018, revenue was $2,142.3 million, which represents growth of 33.6%, or $538.8 million, compared to the nine months ended September 30, 2017 at actual foreign exchange rates. On a constant currency basis, revenue grew $520.3 million, representing growth of 32.4% compared to the nine months ended September 30, 2017.

Excluding the impact of the adoption of ASC 606 and reimbursement revenue, revenue increased $328.3 million, which represents growth of 23.8% at actual foreign exchange rates and 22.8% on a constant currency basis. Organic revenue growth, excluding the adoption of ASC 606, reimbursement revenue and revenue attributable to our Data Solutions segment, was 12.6% at actual foreign exchange rates and 11.6% on a constant currency basis.

Reported GAAP income from operations was $184.6 million, reported GAAP net income was $82.5 million and reported GAAP net income per diluted share was $1.24 for the nine months ended September 30, 2018.

Adjusted Net Income was $197.2 million for the nine months ended September 30, 2018, an improvement of 31.4% compared to the same period in 2017. Adjusted Net Income per diluted share was $2.98 for the nine months ended September 30, 2018, up 30.7% compared to the same period in 2017.

Guidance

The Company is maintaining its 2018 revenue guidance of between $2.87 billion and $2.92 billion, representing as reported growth of 47% to 50%, constant currency growth of 18% to 20% excluding the impact of adopting ASC 606 and reimbursement revenue, and constant currency organic growth of 10% to 12% excluding the impact of adopting ASC 606 and reimbursement revenue. We are updating our GAAP net income per diluted share to between $2.21 and $2.26 and Adjusted Net Income per diluted share to between $4.22 and $4.27. We continue to estimate our annual effective income tax rate at approximately 24%, which includes the expected impact of the U.S. Tax Cuts and Jobs Act. Our effective tax rate may differ from this estimate, due to, among other things, changes to estimates of the geographic allocation of our pre-tax income as well as changes in guidance from regulatory agencies related to interpretation, analysis and guidance of the U.S. Tax Cuts and Jobs Act.

Our guidance assumes a EURO rate of 1.17 and a GBP rate of 1.33. All other foreign currency exchange rates are as of September 30, 2018.

Conference Call Details

PRA will host a conference call at 9:00 a.m. ET on November 1, 2018, to discuss the contents of this release and other relevant topics. To participate, please dial (877) 930-8062 within the United States or (253) 336-7647 outside the United States approximately 10 minutes before the scheduled start of the call. The conference ID for the call is 9668676. The conference call will also be accessible, live via audio broadcast, on the Investor Relations section of the PRA website at investors.prahs.com. A replay of the conference call will be available online at investors.prahs.com. In addition, an audio replay of the call will be available for one week following the call and can be accessed by dialing (855) 859-2056 within the United States or (404) 537-3406 outside the United States. The replay ID is 9668676.

Additional Information

A financial supplement with third quarter 2018 results, which should be read in conjunction with this press release, may be found in the Investor Relations section of our website at investors.prahs.com in a document titled "Q3 2018 Earnings Presentation."