Celyad Announces First Quarter 2018

Business Update

On May 15, 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 on key clinical and operational developments for the first quarter ended March 31, 2018 (Press release, Celyad, MAY 15, 2018, View Source [SID1234526684]).

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FIRST QUARTER 2018 AND RECENT HIGHLIGHTS

Robust clinical development plan is foundation for new trials focusing on AML and CRC

Steady progress related to ongoing THINK, SHRINK and LINK trials

Good safety profile of CYAD-01 confirmed

Lead publication Haematologica publishes THINK Study Case Report
Dr. Christian Homsy, CEO of Celyad commented: "We had a productive first quarter, further defining our strategy that will guide CYAD-01 in becoming the foundation for cancer therapies. Not only have we progressed in the THINK trial, we have also treated our first patients in the SHRINK and LINK trial. The absence of any observed critical on target/off tumor toxicity in our trials is an important signal validating our technology. The next months will be exciting for our company and we look forward communicating results on our clinical trials in scientific congresses."

FIRST QUARTER 2017 OPERATIONAL AND FINANCIAL REVIEW

In February 2018, Celyad provided a detailed clinical update summarizing the promising results achieved in 2017: the THINK trial resulted in signs of clinical activity ranging from Stable Disease (SD) to Complete Response (CR), despite the absence of preconditioning therapy and the lower doses used at that stage of the trial. The company also announced that it will further evaluate CYAD-01 in a series of additional Phase 1 clinical trials in patients with AML and CRC.

Also in February 2018, Celyad organized a Key Opinion Leader meeting on CAR-T therapy in New York, USA. The meeting featured a presentation by Marco Davila, MD, PhD (Moffitt Cancer Center). The numerous attendants received information on the unmet medical need in blood cancers as well as details on Celyad’s clinical strategy.

1 THerapeutic Immunotherapy with CAR-T NKG2D
2 Standard CHemotherapy Regimen and Immunotherapy with NKG2D
3 Loco-regional Immunotherapy with NKG2D

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15 May 2018

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On the operation side, during this first quarter, Celyad progressed well in the THINK trial as well as the in LINK trial:

– The company dosed the three CRC patients in the third dose cohort in the solid arm of the THINK trial, and the two last AML patients in the second dose. Celyad plans to initiate the third dose in the AML arm in May 2018, and complete the recruitement of three additional CRC patients at the higher dose by mid-2018. In 2018, all patients were dosed with the our new production process adopted in December 2017.

– The company also treated its first CRC patient in the LINK trial. This patient received three planned injections at the first Dose level (3×108). LINK adopts a loco-regional approach in treating CRC by administering CYAD-01 through multiple hepatic transarterial injections.

The company ended the quarter with €25.1 million in cash, cash equivalents and short-term investrments. Use of cash over the first quarter of 2018 amounted to €8.8 million, in line with expectations. The company confirms that existing cash and cash equivalents and short term investments are sufficient to fund operating expenses and capital expenditure requirements, based on the current scope of activities, until the end of Q1 2019.

EVENTS SUBSEQUENT TO QUARTER-END:

In April 2018, Celyad’s world’s first reported complete response by a CAR-T cell therapy in a patient with refractory and relapsed AML was published as a case report in the leading scientific publication Haematologica. The publication detailed the first objective response related to this patient that is still in remission, more than 9 months after study enrollment.

In May 2018, Celyad achieved an important milestone in its CYAD-01 clinical strategy by dosing the first metastatic CRC patients in the LINK and SHRINK trials. No drug related toxicity was observed in the first patients of both SHRINK and LINK trials.

Generally, Celyad’s progress is the result of the company’s clinical development plan aiming at defining the best of three approaches for CYAD-01 in patients with AML and CRC:

1) CYAD-01 as a stand-alone investigational therapy, currently being evaluated in the THINK trial with relapsed refractory AML and CRC patients. Promising results have already been reported including a complete response and stable diseases.

Based on data as of April 5, 2018, the date of Celyad’s most recent interim safety report for the THINK trial, Celyad had collected safety data from 20 patients treated with CYAD-01 in the THINK trial. Of the 20 patients included in the interim safety report of the THINK trial (11 solid and nine hematologic cancer patients), Celyad reported the following serious adverse events:

– Grade 4 serious adverse events occurred in two patients: one patient in the hematologic cohort experienced respiratory failure and other Grade 4

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adverse events after administration of dose level one of CYAD-01. The other patient, who was in the solid tumor cohort, experienced cytokine release syndrome and other Grade 4 adverse events after administration of dose level three of CYAD-01, which was adjudicated as a dose limiting toxicity (DLT).

– Those two patients each experienced a Grade 5 event that was deemed unrelated to administration of CYAD-01.

2) CYAD-01 administered concurrently with standard of care treatments. The SHRINK trial was recently initiated with the dosage of one CRC patient in April 2018. No Grade 4 or higher adverse event has been reported so far. This trial evaluates the concurrent administration of CYAD-01 with the standard chemotherapy FOLFOX. We expect that another similar trial aimed at AML patients, EPITHINK trial, will be initiated soon.

3) CYAD-01 administered after preconditioning of the patients using lymphodepletion. We expect trials in AML (DEPLETHINK AML) and CRC (DEPLETHINK CRC) patients to be initiated in the coming weeks.

Astellas Appoints Bernhardt Zeiher, M.D., as New Chief Medical Officer

On May 15, 2018 Astellas reported that Bernhardt G. Zeiher, M.D., F.C.C.P., F.A.C.P., ("Bernie"), was promoted to Chief Medical Officer (CMO), effective April 1, 2018 (Press release, Astellas Pharma US, MAY 15, 2018, View Source [SID1234526669]). Zeiher will continue serving as president of Development, while now also overseeing all other functions of Astellas’ Medical and Development (M&D) organization, including Clinical and Research Quality Assurance, Medical Affairs, Pharmacovigilance, Planning & Administration, and Regulatory Affairs.

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Reporting directly to Astellas president and CEO, Kenji Yasukawa, Ph.D., Zeiher will join the company’s top executive leadership team. He will continue to lead the organization from Northbrook, Ill., Astellas’ headquarters for M&D and its Americas operations.

In his new role, Zeiher will focus on further integrating and enhancing Astellas’ delivery of its global innovative pipeline and driving support for the appropriate use of its products.

"I am honored to lead the Astellas M&D organization during this critical time," said Zeiher. "As we embark upon a new Corporate Strategic Plan, it is my goal to continue and further implement the corporate vision of turning innovative science into value for patients."

Zeiher started his career at Astellas in 2010 as vice president and Therapeutic Area leader for Inflammation, Immunology and Infectious Diseases. He was promoted to senior vice president and Therapeutic Area Head for Immunology, Infectious Diseases and Transplant in 2012. Zeiher was later named executive vice president and Therapeutic Area Head when his organization was expanded to include the company’s CNS and Pain programs. Most recently, Zeiher was promoted to president of Development in 2015. Prior to joining Astellas, Zeiher served as the vice president of the Inflammation/Immunology therapeutic area at Pfizer.

He earned his Doctor of Medicine at the Case Western Reserve University School of Medicine, and completed an internal medicine residency at University Hospitals of Cleveland as well as a fellowship in Pulmonary and Critical Care Medicine at University of Iowa Hospitals and Clinics. Zeiher is a Fellow in the American College of Physicians and the American College of Chest Physicians. He has worked in the pharmaceutical industry since 1998

Savara to Present at Bank of America Merrill Lynch 2018 Healthcare Conference on May 15th

On May 15, 2018 Savara Inc. (NASDAQ:SVRA), an orphan lung disease company, reported that the Company’s Chief Executive Officer, Rob Neville, will present at the Bank of America Merrill Lynch 2018 Healthcare Conference on Tuesday May 15th, 2018 at 1:55 p.m. Pacific Time at the Encore Hotel in Las Vegas (Press release, Savara, MAY 15, 2018, View Source [SID1234526660]).

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Interested parties can access a live audio webcast on the Savara website at www.savarapharma.com. An archived presentation will be available on the website for 30 days.

Akebia Therapeutics to Present at Upcoming Investor Conference

On May 15, 2018 Akebia Therapeutics, Inc. (NASDAQ:AKBA), a biopharmaceutical company focused on delivering innovative therapies to patients with kidney disease through the biology of hypoxia-inducible factor (HIF), reported that its President and Chief Executive Officer, John P. Butler, will present at the UBS Global Healthcare Conference on Tuesday, May 22, 2018, at 4:00 p.m. Eastern Time, at the Grand Hyatt New York in New York City (Press release, Akebia, MAY 15, 2018, View Source [SID1234526659]).

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A live webcast of the presentation will be available on the company’s website at www.akebia.com. To access the webcast, please log onto the Akebia website at least 15 minutes prior to the webcast to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Akebia’s website following the conference.

CEL-SCI Corporation Reports Second Quarter Fiscal 2018 Financial Results

On May 15, 2018 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended March 31, 2018 (Press release, Cel-Sci, MAY 15, 2018, View Source [SID1234526658]).The Company also reported key clinical and corporate developments achieved during the quarter.

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Clinical and Corporate Developments included:

CEL-SCI’s Phase 3 head and neck cancer study has been fully enrolled since September 2016. All 928 patients are being followed per the protocol. The primary endpoint of the study, a 10% improvement in overall survival of the Multikine treatment regimen plus Standard of Care (SOC) vs. Standard of Care alone, will be determined after a total of 298 deaths have occurred in the two main comparator arms of the study and have been recorded in the study database. All that remains to be done in this pivotal Phase 3 study is to continue to track patient survival until the 298 deaths have occurred and it can be determined if the primary endpoint has been met.
Closing arguments in the arbitration against the former clinical research organization (CRO) for the Phase 3 trial concluded on April 25, 2018. The parties are now awaiting a decision from the arbitrator.
The U.S. Patent and Trademark Office allowed a patent for CEL-SCI’s LEAPS technology for methods of inducing immune responses.
"We expect two significant and material outcomes in the near term. In the very near term we are awaiting a decision from the arbitrator in our case against our former CRO. The read out of the Phase 3 study is expected to take longer than the decision by the arbitrator, however, based on overall survival data available in the scientific literature for the study’s patient population and the fact that the last patient was enrolled in the study over 1.5 years ago, we believe that the end of the Phase 3 trial is approaching as well," said CEL-SCI’s Chief Executive Officer, Geert Kersten. "Separately, we continue to advance our LEAPS technology through preclinical studies in preparation for human clinical studies. The current work with the Rheumatoid Arthritis vaccine is funded by a $1.5 million grant from the National Institutes of Health."

During the six months ended March 31, 2018, CEL-SCI’s cash increased by approximately $0.7 million. Significant components of this increase include net proceeds from the sale of CEL-SCI’s common stock of approximately $7.1 million offset by net cash used to fund the Company’s regular operations, including its Phase 3 clinical trial, of approximately $6.4 million.

CEL-SCI reported an operating loss of ($4,205,745) for the quarter ended March 31, 2018 versus an operating loss of ($7,906,557) for the quarter ended March 31, 2017. The operating loss was ($9,117,175) for the six months ended March 31, 2018 versus an operating loss of ($12,844,565) for the six months ended March 31, 2017.

The research and development expenses decreased by approximately $4.8 million compared to the six months ended March 31, 2017. The majority of CEL-SCI’s research and development expense relates to its on-going Phase 3 clinical trial. Clinical trial costs tend to be higher during the enrollment phase of the study and because the study is fully enrolled, the expenses incurred over the last six months have decreased. The general and administrative expenses increased by approximately $1.3 million compared to the six months ended March 31, 2017. This increase is primarily due to an increase of approximately $1.0 million in equity based compensation related to the Company’s shareholder approved 2014 Incentive Stock Bonus Plan, and a net increase of approximately $0.3 million in other general and administrative expenses primarily for accounting fees and public relations services.