On November 17, 2017 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 an update on key clinical and operational developments for the third quarter ended Sept. 30, 2017 (Press release, Celyad, NOV 17, 2017, View Source [SID1234522222]).

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THIRD QUARTER 2017 AND RECENT HIGHLIGHTS

• Reported first complete response by a CAR-T therapy in a patient with relapsed refractory AML (Acute Myeloid Leukemia) in the Phase 1b THINK1 trial

• Reported promising safety and clinical activity of CYAD-01 (CAR-T NKG2D) in all AML patients treated so far in THINK trial

• Announced amended agreements with Celdara Medical and Dartmouth College following encouraging results from the THINK trial

• Initiated the Phase 1 SHRINK2 study in Belgium to evaluate the synergetic effect of the concurrent administration of CYAD-01 with standard chemotherapy in patients suffering from metastatic colorectal cancer
Christian Homsy, CEO of Celyad commented: "We are pleased with our progress during the third quarter, particularly with the complete response in a relapse refractory AML patient to the CYAD-01 therapy in our THINK trial. This, together with the clinical activity detected in all AML patients treated so far encourages us to rapidly progress in our development. Having seen this activity, we want to build upon it and explore how to strengthen it and make it more robust. The research steps are behind us now, and the development strategy we are engaged in will investigate approaches that will both strengthen the responses and make them more durable, when needed."

The clinical responses obtained by Celyad are perceived as a key milestone not only for the company, but also for the CAR-T field as a whole, as this is the first time that a patient has shown a complete response to a CAR-T therapy without pre-conditioning. Christian Homsy added: "The results validate CYAD-01 and NKG2D as a target in AML, a severe disease that affects approximately 20,000 people in the US and almost as many people in Europe. Celyad will further investigate CYAD-01 in this indication as well as in colorectal cancers, an indication for which CYAD-01 has also demonstrated interesting results."

1 THINK: THerapeutic Immunotherapy with CAR-T NKG2D
2 SHRINK: Standard CHemotherapy Regimen and Immunotherapy with CAR-T NKG2D

www.celyad.com | 1
LOGO Press Release
17 November 2017
07:00 am CET

Regulated Information

THIRD QUARTER 2017 OPERATIONAL AND FINANCIAL REVIEW
In July 2017, Celyad initiated the SHRINK trial, an open-label Phase 1 study evaluating the safety and clinical activity of multiple doses of CYAD-01, administered concurrently with the neoadjuvant FOLFOX treatment in patients with potentially resectable liver metastases from colorectal cancer. The trial includes a dose escalation and an extension stage. The dose escalation design will include three dose levels adjusted to body weight: up to 1×108, 3×108 and 1×109 CYAD-01 cells. At each dose, patients will receive three successive administrations, two weeks apart, at the specified dose. The dose escalation portion of the study will enroll up to 18 patients and the extension phase will enroll 21 additional patients. SHRINK is being conducted in oncology centers in Belgium.

In August 2017, Celyad amended its existing agreements with Celdara Medical LLC and Dartmouth College. Under the amended agreements, Celyad will receive an increased share of future revenues generated by these assets, including revenues from its sublicensees. In return, Celyad paid Celdara Medical and Dartmouth College an upfront payment of $12.5 million (€10.6 million) and $12.5 million worth of Celyad shares at a share price of €32.35 corresponding to a 14% premium versus the prior trading day.
Patrick Jeanmart, CFO of Celyad, added, "Our revised agreements with Celdara Medical and Dartmouth College reflect our strong belief in the value-creating potential of our allogeneic CAR-T cell patent portfolio and our ongoing confidence in CYAD-01. By shifting some of the value of the original deal upfront, we have increased our share of potential future revenues from sublicenses."
The Company ended the quarter with €40 million in cash. Use of cash over the third quarter of 2017 amounted to €29 million, of which €18 million paid to Celdara Medical and Dartmouth College as a result of our new license agreements. The cash burned by our operations was €11 million over the third quarter and €27 million over 2017, in line with our expectations. The company confirms its previous guidance, that existing cash, cash equivalents and short-term investments are sufficient to fund operating expenses and capital expenditure requirements, based on the current scope of activities, through to the first half 2019.

www.celyad.com | 2
LOGO Press Release
17 November 2017
07:00 am CET

Regulated Information

EVENTS SUBSEQUENT TO QUARTER-EN

In October 2017, Celyad announced a first ever morphologic complete response (MLFS3) with gene-engineered T-cells without prior pre-conditioning chemotherapy for a patient with relapsed refractory AML. At the first dose-level, 3×108, CYAD-01 T-cells were administered without any prior conditioning chemotherapy to a cohort of three patients with hematologic cancer (two with AML and one with multiple myeloma): One AML patient achieved a MLFS administered at the H. Lee Moffitt Cancer Center and Research Institute in Tampa, Florida. A second AML patient treated also reached a Complete Response (CR) and more precisely a Complete Response with incomplete hematologic recovery. Unlike the patient that reached MLFS, this second patient progressed after a while and has since moved to another treatment.
The first AML patient treated at the second dose-level (1×109) was reported as Stable Disease with improvement of his hematological parameters at 2-month follow-up post treatment.
To date, all AML patients have shown varying clinical responses that are attributed to the CYAD-01 treatment.

On November 17, 2017 PledPharma AB ("PledPharma") (STO: PLED) and Solasia Pharma K.K. ("Solasia") (TSE: 4597) reported that they have entered a license agreement pertaining to the clinical development and commercialization of PledOx in Japan, China, Hong Kong, Macau, South Korea and Taiwan (Press release, Solasia, NOV 17, 2017, View Source [SID1234532512]).

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Under the terms of this agreement, PledPharma grants exclusive development and commercialization rights to PledOx in the territories mentioned and Solasia will pay upfront, development, regulatory and sales milestones of up to ~USD 83 million (SEK 700 million)*. In addition, Solasia will pay industry standard royalty rates on sales applicable for a deal pertaining to an in-licensed asset in Phase III development. Solasia will also fully finance an expansion of the Phase III program to include Asian patients subject to regulatory consultations.

The license agreement is initially focused on the use of PledOx as prevention of chemotherapy induced peripheral neuropathy in colorectal cancer patients. The agreement with Solasia facilitates an expansion of the recently announced global Phase III-program for PledOx with Asian patients, subject to regulatory consultations, aiming to gain sufficient documentation for regulatory approvals in the major Asian markets. In addition, a Phase I study in Japanese and Caucasian Healthy Volunteers with focus on safety, tolerability and pharmacokinetics will be conducted. Following potential regulatory approvals, Solasia will be responsible for the commercialization of PledOx in Japan, China, Hong Kong, Macau, South Korea, and Taiwan.

"We are very excited to announce our partnership with Solasia – an ideal partner during the development, regulatory process and commercialization of PledOx in this very important region. The collaboration will ensure an optimized expansion of the Phase III program to include Asian patients, aiming at further realising the global commercial potential of our drug candidate," said Nicklas Westerholm, Chief Executive Officer and President, PledPharma.

"We are convinced that PledOx, as a novel first in class therapy, will play an important role in fulfilling the significant unmet medical need of preventing chemotherapy induced peripheral neuropathy. Solasia is ideally equipped to support PledPharma during the remaining clinical development and local regulatory processes in Japan, and to effectively launch the product in key Asian markets," said Yoshihiro Arai, President and Chief Executive Officer, Solasia.

As PledPharma announced earlier in November, following interactions with the regulatory authorities, EMA and FDA, the company has finalized the design of the global Phase III program for the drug candidate PledOx. The Phase III studies are anticipated to be initiated at the end of 2017 with top line results expected during 2020.

* The total value of upfront and milestone payments is up to JPY 9.3 billion. The amount given in USD and SEK is subject to exchange rate.

Invitation to corporate presentation
PledPharma will attend the Redeye Life Science Seminar on November 24 at 11:00 CET where PledPharma will provide a company update and an overview of the license agreement with Solasia. The event will be live streamed from Redeyes website www.redeye.se. After the event, the presentation will be available on PledPharma’s website.

On November 16, 2017 Innovation Pharmaceuticals Inc. (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, reported additional perspectives on the planned continued development of Brilacidin-OM for the prevention and treatment of Oral Mucositis (OM) in Head and Neck Cancer (HNC) patients (Press release, Innovation Pharmaceuticals, NOV 16, 2017, View Source [SID1234522105]).

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As previously announced, the Phase 2 randomized, placebo-controlled clinical trial of Brilacidin-OM (see NCT02324335), enrolling a total of 61 patients, recently was completed, with top-line analysis to be reported shortly.

After unblinding, should final study results compare favorably to those observed at interim, in which patients treated with Brilacidin-OM showed a markedly reduced rate of severe OM (WHO Grade ≥3 ) compared to those on placebo, the Company will initiate an aggressive development plan going forward, including: applying to the Food and Drug Administration (FDA) for Breakthrough Therapy Designation and possibly applying for a similar expedited path in Europe; enhancing oral rinse administration with patient-friendly formulation and packaging; and, dedicating internal resources to Phase 3 trial design planning and execution.

Formal collaboration with pharmaceutical companies that have expressed an interest in partnering Brilacidin-OM may well assist further with expediting the drug candidate’s development timetable. Some of these partnering conversations have matured to the point of potentially structuring mutually beneficial licensing agreements, pending the final Phase 2 study results.

"We very much look forward to taking what could be the final step in Brilacidin’s development for the prevention of OM—top-line results reporting followed by possible advancement into a pivotal Phase 3 trial," said Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "The OM market comprises a huge unmet medical need. To think we might one day help end the pain of so many patients suffering from OM, better enabling them to get the critical cancer care they need, would be an incredible accomplishment for Innovation Pharmaceuticals’ staff and a proud moment for our shareholders. Furthermore, securing partnerships with Pharma would represent a significant milestone for the Company, further anchoring the rapidly expanding Brilacidin Franchise."

"What’s important to understand about Brilacidin-OM, at this stage of its development, is that it truly represents a potential breakthrough treatment in OM," commented Arthur P. Bertolino, MD PhD, MBA, President and Chief Medical Officer at Innovation Pharmaceuticals. "A majority of OM treatments currently being evaluated in clinical trials still target reduction in duration of OM symptoms, rather than going after OM prevention—what we’ve established as our primary efficacy outcome in the completed Phase 2 trial. We’ve set for ourselves a high bar, to be sure, but feel confident that if we deliver strong top-line results, Brilacidin-OM will stand apart from any future competition—a potential first-to-market and best-in-class OM treatment."

About Oral Mucositis

Oral Mucositis (OM) is a frequent, painful and debilitating complication of chemoradiation. Head and Neck Cancer (HNC) patients—comprising an estimated 65,000 newly diagnosed cases in the U.S. alone in 2017, and an estimated 700,000 worldwide (source: GLOBOCAN)—are at the greatest risk of developing OM (a 90 to 100 percent rate of occurrence). By 2030, the global incidence of HNC cases is expected to exceed 1 million per year. Moreover, between 25 and 60 percent of cancer patients, regardless of cancer type, also will experience OM. Characterized by inflammation and ulceration, patients suffering from OM are often unable to speak and eat (requiring the insertion of a feeding tube) and are more susceptible to infections, with severe cases leading to hospitalization at increased treatment costs of up to $25,000. There currently are no approved medications for the prevention of OM in the HNC population, with only limited palliative care options available. Worldwide, the potential market for OM is expected to exceed $1 billion in the next few years.

On November 16, 2017 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 John P. Butler, President and Chief Executive Officer, will participate in the following investor conferences(Press release, Akebia, NOV 16, 2017, View Source [SID1234522110]):

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The 29th Annual Piper Jaffray Healthcare Conference on Wednesday, November 29, 2017, at 12:30 p.m. Eastern Time, to be held at the Lotte New York Palace in New York, NY.
The Evercore ISI Biopharma Catalyst/Deep Dive Conference on Thursday, November 30, 2017, at 9:30 a.m. Eastern Time, to be held at the Boston Harbor Hotel in Boston, MA.
The Global Mizuho Investor Conference on Tuesday, December 5, 2017, to be held at the Lotte New York Palace in New York, NY.
A live audio webcast from the presentation at Piper Jaffray and fireside chat at Evercore will be available on the Company’s website at View Source, with archives available for 90 days.

On November 16, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare diseases, reported the successful completion of a $27 million Series B financing (Press release, X4 Pharmaceuticals, NOV 16, 2017, View Source [SID1234522108]). Proceeds from the financing will be used to advance X4’s two lead drug candidates in clinical efficacy studies in immuno-oncology and a pivotal study in WHIM syndrome, a rare primary immunodeficiency disease. X4’s novel therapeutics are directed against the CXCR4 pathway to correct the trafficking of key immune cells that regulate healthy immune surveillance throughout the body.

"We are incredibly pleased to have the support of Cormorant Asset Management and additional new and existing investors to support our mission of bringing innovative treatments to patients with cancer and rare diseases," said Paula Ragan, PhD, President and CEO of X4. "We are now in a strong position to advance our lead candidate in WHIM syndrome into a pivotal study in 2018 and to advance our proof of concept studies in oncology. This financing recognizes the progress we have made as well as the important milestones that lie ahead in 2018."

"Having been an investor in the earliest stages of X4, we have witnessed the strong progress the company has made and the impact these treatments are having for patients," said Bihua Chen, CEO and Portfolio Manager, Cormorant Asset Management. "X4 has built a differentiated approach rooted in strong science and clear regulatory pathways. We are pleased to support X4’s continuing work in developing and commercializing innovative therapies for primary immunodeficiencies and cancer."

In the two years since X4 launched in 2015, the company has rapidly entered clinical studies with lead programs for two different diseases: as an immuno-oncology agent for solid tumors and as a treatment for the rare disease WHIM syndrome. The company’s oral, small molecule drug candidates inhibit the binding of chemokine CXCL12 to C-X-C receptor type 4 (CXCR4), a receptor-ligand pair that plays an essential role in normal immune surveillance. X4P-001-IO has demonstrated encouraging results in Phase 1/2 testing in refractory clear cell renal cell carcinoma, as well as in melanoma where results support activation of the immune system. In addition, X4P-001-RD has demonstrated initial proof-of-concept in the Phase 2 portion of an on-going Phase 2/3 study in patients with WHIM syndrome.

About Renal Cell Carcinoma

Kidney cancer is among the ten most common cancers in both men and women with more than 60,000 new diagnoses each year in the United States.1 Clear cell renal cell carcinoma (ccRCC) is the most common form of kidney cancer, and advanced ccRCC accounts for approximately 20% of the patient population. Therapies for advanced ccRCC include immunotherapies, mammalian target of rapamycin (mTOR) kinase inhibitors, and angiogenesis inhibitors, such as vascular endothelial growth factor (VEGF) inhibitors.2 There continue to be unmet medical needs with advanced ccRCC because durable responses remain a serious clinical challenge for patients with advanced disease.

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency disease ("PID") 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 US that are classified with PID of unknown origin — of which WHIM is one.3,4,5 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. Because patients are highly susceptible to infections, 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.

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