On March 23, 2018 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell therapies, reported consolidated financial results for the twelve-month period ended 31 December 2017, prepared in accordance with IFRS (Press release, Celyad, MAR 23, 2018, View Source [SID1234524957]).

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Christian Homsy, CEO of Celyad: "2017 has been another milestone year for Celyad. The responses obtained to date in AML, and in solid tumors, we believe validate NKG2D as a target and allow us to move rapidly forward with the development of this product candidate in 2018. Our intellectual property related to the allogeneic technology, another of our core strengths, has been further strengthened: it was upheld by the US Patent and Trademark Office as part of multiple ex-parte re-examination requests and its importance has been recognized through our licensing agreement with Novartis. As far as 2018 is concerned: we believe it will be an exciting year as we expect to – among other things– complete the dose escalation segment of THINK and explore the various conditions that could lead to a potential registrational Phase 2 trial."

1 THINK: THerapeutic Immunotherapy with CAR-T NKG2D

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Press Release

22 March 2018

10:01 pm CET

Regulated Information

Key 2017 Highlights

First complete response by a CAR-T cell therapy reported in a patient with relapsed and refractory AML in the Phase 1 THINK trial. Complete response obtained without preconditioning therapy

CYAD-01 (CAR-T NKG2D) well tolerated and clinical activity seen in AML patients treated in THINK trial to date

First signs of clinical activity in Colorectal and Ovarian cance

Phase 1 SHRINK2 study initiated to evaluate the synergetic effect of the concurrent administration of CYAD-01 with standard chemotherapy in metastatic colorectal cancer patients

Non-exclusive license agreement signed with Novartis for its allogeneic TCR-deficient CAR-T cell patents

New agreements with Celdara Medical LLC and Dartmouth College following encouraging results from the THINK trial, giving right to an increased share of future revenues generated by our CAR-T platform

Strong central IP position in the allogeneic CAR-T cell field confirmed by USPTO
Expected Milestones for 2018 and Beyond

Dose escalation segment of THINK trial; Enrolment according to plans

Interim read-outs of the LINK3 and the SHRINK clinical trials

Initiation of DEPLETHINK4 study (non-myeloablative preconditioning chemotherapy in relapse/refractory AML or myelodysplastic syndrome (MDS) patients)

Initiation of EPITHINK5 study (CYAD-01 treatment administered concurrently with 5-azacytidine in treatment-naïve AML or MDS patients not candidates for intensive therapy).

Investigational New Drug (IND) filing for our allogeneic CYAD-101 product candidate and enrolment of the first patient of the trial

Finalization of preclinical development of CYAD-02, a CYAD-01 iteration aimed at enhanced in-vivo expansion and persistence

Proof of concept of the CARGO platform, a next generation CAR-T engineered with tumor micro-environment targeting tools and enhanced tumor infiltration capabilities (a cargo CAR). CYAD-03 is the NKG2D CAR of the CARGO platform

2 SHRINK: Standard CHemotherapy Regimen and Immunotherapy with CAR-T NKG2D
3 LINK: Locoregional Immunotheraoy with NKG2D
4 DEPLETHINK: LymphoDEPLEtion and THerapeutic Immunotherapy with NKG2D
5 EPITHINK: EPIgenetic drug treatment and THerapeutic Immunotherapy with NKG2D

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Press Release

22 March 2018

10:01 pm CET

Regulated Information

Commenting on the 2017 results, Dr. Debasish Roychowdhury, Member of the Board, said: "2017 was a watershed year for Celyad. We saw promising signs of tolerability and clinical activity in the THINK trial, validating NKG2D as a target, thus allowing us to be well positioned to broaden the scope of NKG2D platform and to initiate potential pivotal studies, respectively planned for 2018 and 2019."

Conference Call Details

A conference call will be held on Friday, 23 March 2018, at 2:00 p.m. CET / 9:00am EDT to review the financial results. Christian Homsy, Chief Executive Officer, and Patrick Jeanmart, Chief Financial Officer will deliver a brief presentation followed by a Q&A session.

Joining the Conference Call:

1. In the 10 minutes prior to the call start time, call the appropriate participant dial-in number.

• Standard International Dial-In Number: +44 (0) 2071 928338
Local Call Dial-In Numbers:

• Belgium 027933847

• France 0170700781

• UK 08444819752

• Netherlands 0207956614

• US 18778709135

2. Provide the operator with the conference ID: 4391809
Helpful keypad commands: *0—Operator assistance.

2017 Financial and Operating Results

Celyad reported steady progress in 2017 with the advancement of the clinical development of CYAD-01. Data collected thus far from the THINK trial, which started in early 2017, show that CYAD-01 has been well-tolerated, offers an excellent safety profile and validates the activity of the NKG2D receptor.

At year-end 2017, there were no critical toxicity events related to the CYAD-01 product candidate reported by the THINK trial investigators. More importantly, the first signs of clinical activity were reported in both arms of the trial.

In the hematological arm of the THINK trial, Celyad announced in October 2017 a world’s first with the complete response in a patient with refractory and relapsed AML, obtained without preconditioning chemotherapy or other anti-tumor treatments combined with CYAD-01. Furthermore, preliminary signs of clinical activity were observed in all AML patients dosed in 2017. In the solid arm, cases of stable disease (SD) were reported in patients suffering from ovarian cancer and colorectal cancer.

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Press Release

22 March 2018

10:01 pm CET

Regulated Information

Celyad also made important progress on its IP position with the announcement of a non-exclusive license agreement with Novartis and three new patents covering the allogeneic CAR-T cell approach.

In terms of financing, Celyad reported €34 million at year end 2017. This, together with anticipated milestone payments expected to be received by Celyad in 2018 from its strategic partners, should enable the company to finance all its clinical programs and other needs through the first half of 2019.

Here are the operational and financial highlights of 2017 identified by the company’s board of directors:

Clinical Developments

• Data collected in 2017 from the THINK trial showed that CYAD-01 has been well-tolerated to date and validated activity of the NKG2D CAR.

• In October, Celyad achieved an important milestone in oncology with the first ever complete response in a patient with refractory and relapsed AML, obtained without preconditioning chemotherapy or other anti-tumor treatments combined with CYAD-01. Importantly, clinical activity has been observed in AML patients dosed in 2017, with all patients seeing a reduction in their blast counts in the bone marrow and/or improvements in their hematological parameters.

• Data for the THINK trial showed preliminary signs of activity of CYAD-01 in solid tumors. Stabilization of the disease was observed in an ovarian patient and in colorectal cancer patients.

• In late-2017, Celyad initiated the SHRINK trial, an open-label Phase 1 trial evaluating the safety and clinical activity of multiple doses of CYAD-01, administered concurrently with the neoadjuvant standard FOLFOX chemotherapy 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 of CYAD-01: 1×108, 3×108 and 1×109 CYAD-01 per administration. At each dose-level, patients will receive three successive administrations, two weeks apart, at the specified dose administered at a specific timing within the FOLFOX cycle. The dose escalation portion of the trial is designed to enrol three patients per dose level and the extension phase is expected to enrol twenty-one additional patients. SHRINK is being conducted in key oncology centers in Belgium.

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Press Release

22 March 2018

10:01 pm CET

Regulated Information

Intellectual property

• In January, the U.S. Patent and Trade Office (USPTO) upheld, for a third time, Celyad’s U.S. Patent No. 9,181,527 relating to allogeneic human primary T-cells that are engineered to be TCR-deficient and express a CAR. In March, the USPTO rejected another request for a re-examination of the same patent.

• In May, Celyad obtained a new patent related to its method of treating cancer by administering allogeneic primary human T cells that are engineered to be TCR-deficient and to express a CAR. U.S. Patent no. 9,663,763 is the third patent in Celyad’s allogeneic intellectual property portfolio awarded by the USPTO. This new patent claims specific methods of treating cancer patients with allogeneic TCR-deficient CAR-T immunotherapies.
The combination of this patent with earlier granted U.S. patents, consolidates Celyad’s strong intellectual property position in the allogeneic CAR-T field and strengthens the Celyad’s IP portfolio covering key elements in the allogeneic TCR-deficient CAR-T cells production value chain.

Corporate and financial highlights

• In May, Celyad announced a non-exclusive license agreement with Novartis regarding U.S. patents related to allogeneic CAR-T cells. The agreement includes Celyad’s intellectual property rights under U.S. Patent No. 9,181,527. This agreement is related to two undisclosed targets currently under development by Novartis.
Under the terms of the agreement, Celyad received an upfront payment and is eligible to receive payments in aggregate amounts of up to $96 million. In addition, Celyad is eligible to receive single digit percentage royalties based on net sales of the licensed target. Celyad retains all rights to grant further licenses to third parties for the use of allogeneic CAR-T cells.

• In August, Celyad amended its agreements with Celdara Medical LLC and Dartmouth College related to the CAR-T NK cell drug product candidates and related technology licensed in January 2015 following the acquisition of OnCyte LLC. Under the amended agreements Celyad is entitled to receive an increased share of future revenues generated by these assets, including revenues from its sub-licensees. In return, Celyad paid Celdara Medical LLC and Dartmouth College an upfront payment of $12.5 million (€10.6 million) and issued to Celdara Medical LLC $12.5 million worth of Celyad’s ordinary shares at a share price of €32.35.

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Press Release

22 March 2018

10:01 pm CET

Regulated Information

Financial highlights

For the full year ended December 31, 2017, our revenues generated by our strategic collaborations amounted to €3.5 million and corresponded to the non-refundable upfront payment received from Novartis, as a result of the non-exclusive license agreement signed in May 2017. The revenues of 2016 corresponded to the payment received from ONO.

In 2017, our research and development expenses decreased by €4.7 million at €23.0 million compared to 2016. The general and administrative expenses are almost stable over the 2 periods.

As a result, the operating loss of our recurring operations (REBIT) amounted to €26.6 million compared to €25.6 million in 2016.

In 2017, we recognized non-recurring expenses related to the amendment of the agreements with Celdara Medical, LLC and Dartmouth College and the write-off of the C-Cure and Corquest assets together with the derecognition of related liabilities (respectively for €24.3 million, €0.7 million and €1.2 million). There were no non-recurring items in the income statement of 2016.

At year end 2017, the loss from operations before financial results and taxes (EBIT) amounted to €52.9 million versus €25.6 million in 2016.

The 2017 financial income & charges covered mainly interest received on cash deposits and currency exchange rates differences and bank charges. Due the depreciation of the USD compared to EUR, the Group recognized an unrealized loss on foreign exchange differences of €4.4 million in 2017. In 2016, the unrealized gain on foreign exchange differences amounted to €0.8 million.

Taking into account the net financial loss, the net loss of 2017 amounted to €56.4 million versus a net loss of €23.6 million for same period in 2016.

For 2018, we expect to receive additional sublicensing income from our strategic partners and a reasonable increase of our operating expenses, mainly research and development expenses as we will be running multiple clinical trials in parallel.

Cash and short-term deposits were €34 million as of 31 December 2017.

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22 March 2018

10:01 pm CET

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Annual Report 2017

Celyad plans to publish its audited Annual Report for the year ended 31 December 2017 on 6 April 2018. The statutory auditor, BDO Réviseurs d’Entreprises SCCRL, represented by Bert Kegels, has confirmed that the audit, which is substantially complete, has not to date revealed any material misstatement in the draft consolidated financial statements, and that the accounting data reported in the press release are consistent, in all material respects, with the draft consolidated financial statements from which it has been derived.

***END***

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On March 23, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small-molecule drugs that address cancer, liver disease and inflammatory diseases, reported it has filed its 2017 Annual Report on Form 20-F with the U.S. Securities and Exchange Commission (Press release, Can-Fite BioPharma, MAR 23, 2018, View Source [SID1234524956]).

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Clinical Development Program and Corporate Highlights Include:

Piclidenoson (CF101)

A multi-million dollar agreement has been signed with Gebro Holdings for the distribution of piclidenoson in 3 European Countries

Under the terms of the distribution agreement, Gebro made a total upfront and milestone payment of approximately $2,200,000 to Can-Fite. In addition, the agreement provides that additional payments of up to approximately $7,000,000 will be received by Can-Fite upon the achievement of certain regulatory, launch and sales milestones plus double-digit percentage royalty payments on net sales.

Patient enrolment for the ACRobat Phase III Trial in Rheumatoid Arthritis is ongoing

During the fourth quarter of 2017, Can-Fite commenced enrollment in the pivotal Phase III ACRobat trial of approximately 500 patients through clinical sites in Europe, Israel and Canada. The study aims to evaluate Piclidenoson (CF101) as a first line treatment and replacement for the current standard of care, Methotrexate (MTX), the most widely used drug for rheumatoid arthritis. The primary endpoint of ACRobat is low disease activity after 12 weeks of treatment in patients dosed with Piclidenoson compared to those dosed with MTX. Piclidenoson at 1 mg and 2 mg, or placebo, will be administered twice daily, and MTX or placebo will be administered once weekly. The total study duration will be 24 weeks.

The rheumatoid arthritis market is forecast to reach $34.6 billion by 2020.

A patent for Psoriasis has been approved in Korea

The Korean Intellectual Property Office issued patent No. 10-1741281 titled, "Pharmaceutical Composition Comprising A3 Adenosine Receptor Agonist (IB-MECA/CF-101) For Treatment of Psoriasis" for the Company’s lead drug candidate Piclidenoson in its psoriasis indication. Can-Fite has two distribution agreements in Korea, including one with Kwang Dong Pharmaceutical for Piclidenoson in the treatment of rheumatoid arthritis and another with Chong Kun Dang for Namodenoson in the treatment of liver cancer.

Namodenoson (CF102)

Progress of the Phase II Liver Cancer Namodenoson (CF102) Study in the treatment of advanced HCC – Data to be released H2/2018

Current data indicate potentially favorable drug safety profile. The global Phase II study is being conducted in the U.S., Europe and Israel. Patients with advanced HCC, Child Pugh B, who failed Nexavar (sorafenib) as a first line treatment are being treated twice daily with 25 mg of oral Namodenoson or placebo using a 2:1 randomization. The primary endpoint of the Phase II study is Overall Survival (OS). Secondary endpoints include Progression Free Survival (PFS), safety, and the relationship between outcomes and A3AR expression. A total of approximately 78 patients have been enrolled in the Phase II study. As of December 2017, 15 subjects have completed at least 12 cycles of treatment (each cycle is 28 days of treatment), of which two completed 24 cycles. The Company anticipates data release to occur in 2H 2018.

The market for hepatocellular carcinoma drugs is expected to generate $1.4 billion in sales in 2019.

Phase II NAFLD/NASH Study is enrolling patients; Data Release Expected in H1 2019

Can-Fite is currently enrolling patients in a Phase II study in the treatment of non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH) with Namodenoson which plans to enroll 60 patients. Can-Fite’s 12-week study is being led by key opinion leaders in the area of NASH and liver diseases. Clinical trial sites are some of the most prestigious medical institutions in Israel, including Hadassah Medical Center and Rabin Medical Center. Patients are being enrolled in three arms, including two different dosages of oral Namodenoson twice daily versus placebo. The study’s primary endpoints are the mean percent change from baseline in serum alanine aminotransferase (ALT) levels and safety. The secondary endpoint is percent change from baseline in hepatic steatosis measured by magnetic resonance imaging-determined by proton-density fat-fraction (MRI-PDFF) and additional metabolic parameters.

The Phase II trial design was based on preclinical studies showing Namodenoson’s efficacy in reducing liver steatosis, inflammation and fibrosis in experimental NASH models.

There is currently no U.S. FDA approved drug for the treatment of NASH, which is an addressable pharmaceutical market estimated to reach $35-40 billion by 2025.

"We’ve made several notable accomplishments during this past year which validate the strength of our drug portfolio and demonstrate our commitment in execution. We believe these events position us well for the year ahead in which we anticipate a marked progress with our Piclidenoson and Namodenoson drug candidates. We are pleased with the progress thus far and recently announced the submission of safety reports for Piclidenoson and Namodenoson to the FDA and other regulatory authorities so that we may continue with the various clinical studies. Our drug candidates are highly unique due to our favorable safety profile and the specific anti-inflammatory and anti-cancer effects. In the coming year 2018, we remain focused on execution with the goal of providing our patients a better alternative option and treatment for autoimmune inflammatory, oncology and NASH/NAFLD," stated Can-Fite CEO Dr. Pnina Fishman.

Financial Results

Revenues for the year ended December 31, 2017 were NIS 2.9 million (U.S. $0.85 million), an increase of NIS 2.3 million (U.S. $0.66 million), or 350%, compared to NIS 0.65 million (U.S. $0.2 million) for the year ended December 31, 2016. The revenues during 2017 were mainly due to a portion of the NIS 0.76 million (U.S. $0.22 million or CAD 0.2 million) advance payment received in March 2015 under the distribution agreement with Cipher and from the recognition of the milestone payment of NIS 1.8 million (U.S. $0.5 million) and the recognition of a portion of the NIS 0.4 million (U.S. $0.1 million) advance payment received in December 2016 under the distribution agreement with CKD.

Research and development expenses for the year ended December 31, 2017 were NIS 18.3 million (U.S. $5.28 million), a decrease of NIS 5 million (U.S. 1.44 million), or 22%, compared to NIS 23.4 million (U.S. $6.74 million) for the year ended December 31, 2016. Research and developments expenses for the year ended 2017 comprised primarily of expenses associated with the Phase II studies for Namodenoson as well as expenses for ongoing studies of Piclidenoson. The decrease is primarily due to costs associated with CF602 expenses that decreased since the postponement of a planned IND submission for this indication and decreased costs associated with the ongoing clinical trial of Namodenoson for treatment in liver cancer. The Company expects that the research and development expenses will increase through 2018 and beyond.

General and administrative expenses were NIS 10.2 million (U.S. $2.94 million) for the year ended December 31, 2017 a decrease of NIS 0.2 million (U.S. $0.06 million), or 2%, compared to NIS 10.5 million (U.S. $3.02 million) for the year ended December 31, 2016. The minor decrease is primarily due to a decrease in investor and public relations expenses. The Company expects that general and administrative expenses will remain at the same level through 2018.

Financial income, net for the year ended December 31, 2017 aggregated NIS 6.6 million (U.S. $1.89 million) compared to financial income, net of NIS 6.31 million (U.S. $1.82 million) for the same period in 2016. The slight increase in financial income, net in the year ended December 31, 2017 was mainly due to issuance expenses recorded in 2017 which were not recorded in 2016, a decrease in net change in fair value of warrants exercisable into shares and a decrease in exchange rate difference expenses.

Can-Fite’s net loss for the year ended December 31, 2017 was NIS 17.3 million (U.S. $4.99 million) compared with a net loss of NIS 27 million (U.S. $7.78 million) for the year ended December 31, 2016. The decrease in net loss for the year ended December 31, 2017 was primarily attributable to a decrease in research and development expenses.

As of December 31, 2017, Can-Fite had cash and cash equivalents of NIS 12.1 million (U.S. $3.51 million) as compared to NIS 31.2 million (U.S. $8.99 million) at December 31, 2016. The decrease in cash during the year ended December 31, 2017 is due to use of cash to fund operating expenses.

For the convenience of the reader, the reported NIS amounts have been translated into U.S. dollars, at the representative rate of exchange on December 31, 2017 (U.S. $1 = NIS 3.467).

The Company’s consolidated financial results for the year ended December 31, 2017 are presented in accordance with International Financial Reporting Standards.

The 2017 Annual Report can be found on the Company’s website at www.canfite.com as well as on the SEC website at www.sec.gov. In addition, security holders may request a hard copy of the Annual Report, which includes the Company’s complete audited financial statements, free of charge. Requests can be made by contacting Can-Fite Investor Relations at 10 Bareket Street, Kiryat Matalon, Petah-Tikva 4951778, Israel or by phone at +972-3-9241114.

On March 22, 2018 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported that it filed a Registration Statement on Form F-1 with the U.S. Securities and Exchange Commission (SEC) for a proposed offering of ordinary shares in the form of American Depositary Shares ("ADSs") in the United States (Press release, MorphoSys, MAR 22, 2018, View Source [SID1234556339]). The final number of ADSs to be offered and the price for the offering have not yet been determined.

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MorphoSys’s ordinary shares are listed on the Prime Standard Segment in Frankfurt, Germany. Application has been made to list the ADSs to be offered in the proposed offering on the Nasdaq Global Market in the United States under the ticker symbol "MOR".

Goldman Sachs & Co. LLC, J.P. Morgan Securities LLC and Leerink Partners LLC, are acting as lead book-running managers, and Berenberg Capital Markets, LLC and JMP Securities LLC are acting as co-managers for the proposed ADS offering.

A Registration Statement relating to these securities has been filed with the U.S. Securities and Exchange Commission but has not yet become effective. The securities may not be sold, nor may offers to buy be accepted, prior to the time the Registration Statement becomes effective.

The securities referred to in this release are to be offered only by means of a prospectus. A copy of the preliminary prospectus, when available, can be obtained from Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 1-212-902-9316 or by e-mailing [email protected]; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, telephone: 1-866-803-9204; Leerink Partners LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at 1-800-808-7525, ext. 6132, or by emailing [email protected].

This announcement does not constitute an offer to sell nor a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On March 22, 2018 Astellas Pharma Inc. (President and CEO: Yoshihiko Hatanaka, "Astellas" ) reported that the Japanese Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation to gilteritinib for the treatment of FLT3mut+ Acute Myeloid Leukemia (AML), the drug’s prospective indication (Press release, Astellas, MAR 22, 2018, View Source [SID1234536693]).

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Orphan drug designation system in Japan aims to support the development of drugs for diseases that, despite there being a significant medical need for treatments, affect only a small number of patients, and for which research and development is virtually nonexistent. As stipulated in Article 77 of the Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act of Japan, the designation is granted by the minister of Health, Labour and Welfare for drugs that meet the designation criteria which include the following: the number of patients who may use the drug is less than 50,000 in Japan; there is no alternative appropriate drug or treatment in Japan; high efficacy or safety is expected compared to existing products. Specific measures to support the development of orphan drugs include subsidies for research and development expenditures, prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period1

AML is a cancer that impacts the blood and bone marrow, and its incidence increases with age. In Japan, approximately 5,500 patients are diagnosed with AML each year2. Gilteritinib is an investigational compound that has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) as well as FLT3 tyrosine kinase domain (TKD), two common types of FLT3 mutations that are seen in approximately one-third of patients with AML. Further, gilteritinib has also demonstrated inhibition of the AXL receptor in AML cell lines.

Astellas will continue to develop gilteritinib to provide it to FLT3mut+ AML patients as early as possible.

(1): View Source
(2): KantarHealth. TREATMENT ARCHITECTURE: JAPAN LEUKEMIA, ACUTE MYELOID. CancerMPact Japan, February 2017.

On March 22, 2018 Rheos Medicines, Inc. was launched with $60 million in Series A financing backed by Third Rock Ventures, LLC. Rheos is pioneering immunometabolism as a novel approach to tune metabolic pathways in immune cells to treat disease (Press release, Rheos Medicines, MAR 22, 2018, View Source [SID1234532790]). The company will translate recent breakthroughs in the understanding of immune cell metabolism to develop precision medicines for immune-mediated diseases.

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The number of patients with immune-mediated diseases is large and growing in Western society, with an estimated 7-10 percent of the population with autoimmune diseases1 and 5-7 percent of the population with immune-mediated inflammatory diseases.2 Despite recent advances in treatment, there remains a significant unmet need. Understanding how variations in immune cell metabolism underlie patient heterogeneity provides an opportunity to bring precision treatments to patients.

New, in-depth understanding of immune cell physiology based upon research by leading academic scientists, including Rheos’s founders, points to cellular metabolism as a key driver of immune cell response. Rheos’s product engine takes a fundamentally novel approach to immune-mediated diseases by encompassing the full range of immune cells and their functions, and how they are regulated by cellular metabolism. In addition, by identifying new therapeutic targets and related biomarkers, the Rheos engine will address patient heterogeneity inherent to many immune-mediated diseases.

"The emerging field of immunometabolism offers a tremendous opportunity to set a higher standard for how immune-mediated diseases are treated. This opens an opportunity for Rheos to direct our medicines to a new aspect of disease pathogenesis by targeting the underlying cellular metabolism of immune cells," said Abbie Celniker, PhD, Chief Executive Officer of Rheos. "By building on the discoveries of our founders, Rheos is developing a biomarker and drug discovery engine that will allow us to address disease biology and patient variability by ‘tuning’ immune cells in select patient populations with precision medicines."

Rheos Product Engine and Pipeline

Rheos has developed a product engine that offers unprecedented insight into the drivers of immune-mediated diseases by characterizing how different immune cell types impact disease progression in different patient subpopulations. By simultaneously identifying new drug targets and characterizing biomarkers of disease, the Rheos product engine enables a precision medicine approach to treatment of immune-mediated diseases. Using the latest technological advances– including DNA sequencing, transcriptional and metabolomic profiling – along with incorporating primary patient samples, the Rheos product engine is an integrated immune cell metabolism and physiology platform.

Central to this product engine is a proprietary Immune Cell Encyclopedia (ICE), which maps the metabolic pathways used by different types of immune cells to regulate their fate and function in disease and in health. The initial focus of Rheos’s product pipeline is on therapeutics that target CD4 and CD8 T cell subtypes, which are involved in diseases such as inflammatory bowel disease, psoriasis, vitiligo and in immuno-oncology applications. These immune cell subtypes are critical to disease pathogenesis, and Rheos’s product engine is providing insight into how these cells drive immune response in disease.

"I have firsthand experience treating patients with immune-mediated diseases based on my years in clinical practice, and it is exhilarating to apply the wealth of expertise and technology at Rheos to create new treatments and address these patient needs," said Larry Turka, MD, Chief Scientific Officer and co-founder of Rheos. "I am excited to work with the talented team at Rheos and use our product engine to translate the powerful science of immunometabolism into the reality of new treatments that can make a lasting difference for the millions of patients with immune-mediated diseases."

Expert team of immunometabolism scientists and immune disease clinicians

The Rheos leadership team includes recognized leaders in immunometabolism, target discovery, translational medicine and company building. Company leaders include Abbie Celniker, PhD, interim Chief Executive Officer; Cary Pfeffer, MD, interim Chief Business Officer; Laurence Turka, MD, Chief Scientific Officer; Edward Driggers, PhD, Chief Technology Officer; Ryan Cohlhepp, PharmD, Senior Vice President, R&D Strategy and Operations; Brian Albrecht, PhD, Vice President, Drug Discovery; and Hozefa Bandukwala, PhD, Senior Director, Head of Discovery Biology.

Rheos’s internal team is working alongside a founding team of leading scientists whose discoveries opened the field of immunometabolism and clinicians with deep understanding of immune-mediated diseases. The company’s scientific founders are:

Richard Flavell, PhD, Sterling Professor of Immunobiology, Yale University; Investigator, Howard Hughes Medical Institute. Flavell is a world-renowned immunologist who pioneered the use of transgenic mouse models to study autoimmune and inflammatory diseases. Richard is a member of the National Academy of Sciences, National Academy of Medicine (USA), and a Fellow of the Royal Society.
Edward Pearce, PhD, Senior Group Leader, Max Planck Institute of Immunobiology and Epigenetics; Faculty of Biology, University of Freiburg. Edward Pearce is a pioneer in understanding how macrophage and dendritic cell metabolism influences the function of those cells.
Erika Pearce, PhD, Director, Max Planck Institute of Immunobiology and Epigenetics. Erika Pearce is a world leader in T cell metabolism and understanding how different metabolic pathways tune T cell function and fitness.
Ken Smith, MD, PhD, Professor of Medicine and Head of the Department of Medicine, University of Cambridge. Smith is an international expert in identifying biomarkers which predict prognosis in patients with autoimmune disease.
William St. Clair, MD, Professor of Medicine and Immunology, Duke University Medical Center. Dr. St. Clair is a former President of the American College of Rheumatology, he has extensive experience in translational immunology and the design and implementation of trials in autoimmune diseases.
Laurence Turka, MD, CSO, Rheos Medicines. Turka is former President of the American Society of Transplantation, a leader in the fields of T cell costimulation and regulatory T cell biology. Prior to joining Rheos, Laurence was the Harold and Ellen Danser Professor of Surgery and Professor of Medicine at Harvard Medical School and Massachusetts General Hospital.