On January 7, 2019 Celgene Corporation (NASDAQ: CELG) reported a business update, including confirmation that the company achieved its guidance for full-year 2018; its financial guidance for 2019; and reaffirmation of its expected 2020 financial targets (Press release, Celgene, JAN 7, 2019, View Source [SID1234532535]).

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Based on a preliminary review of its results for the fourth-quarter ended December 31, 2018, Celgene achieved guidance for the full-year 2018 that it provided on October 25, 2018 and which is summarized in the table below. The company will report its results for the fourth-quarter and full-year 2018 on January 31, 2019.

Celgene today also provides guidance for the full-year 2019, including total revenue expected to be $17.0 billion to $17.2 billion, a 12 percent increase year-over-year, based on the mid-point of the range. Based on Generally Accepted Accounting Principles (GAAP) diluted earnings per share (EPS) for the full-year 2019 is expected to be in the range of $8.48 to $9.17. Adjusted diluted EPS for the full-year 2019 is expected to be in the range of $10.60 to $10.80.

"Our 2019 financial guidance reflects continued strong operating performance and momentum," said Mark J. Alles, Chairman and Chief Executive Officer of Celgene Corporation. "Multiple clinical and regulatory milestones are expected in 2019 to advance our late-stage portfolio and accelerate our early-stage pipeline."

*Year-over-year percentage change based on the mid-point of the range.

**Not meaningful as the 2019 measures exclude the impact of any strategic transactions, impairments, loss contingencies, changes in the fair value of equity investments, costs associated with the Bristol-Myers Squibb Company (Bristol-Myers Squibb) and Celgene transaction and non-operating tax adjustments that have not yet occurred.

Reaffirming Expected 2020 Long-Term Financial Targets*

2020 total revenue range of $19.0 billion to $20.0 billion

Adjusted diluted EPS to exceed $12.50

*At constant currency using an average of December 2018 spot rates

Overview of Key Milestones Expected Over the Next 12 Months

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REVLIMID

Approval expected by the U.S. Food and Drug Administration (FDA) on the supplemental New Drug Application (sNDA) for REVLIMID in combination with rituximab in relapsed/refractory indolent lymphoma (AUGMENT)

Approval expected by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for REVLIMID in combination with bortezomib and dexamethasone (RVd) in newly diagnosed multiple myeloma (NDMM)

Data expected from the phase III ROBUST trial with REVLIMID in combination with rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate and prednisone (R-CHOP) in patients with first-line ABC-subtype diffuse large B-cell lymphoma (DLBCL) (event-driven)

POMALYST/ IMNOVID

Approval expected by the EMA CHMP and Japan Pharmaceuticals and Medical Devices Agency (PMDA) for POMALYST/IMNOVID in combination with bortezomib and dexamethasone (PVd) in relapsed/refractory multiple myeloma (RRMM)

OTEZLA

Approval expected by the U.S. FDA for the sNDA in Behҫet’s disease with a Prescription Drug User Fee Act (PDUFA) action date of July 21, 2019. Approval by the PMDA in Japan is expected in H2:2019

Submission of the sNDA with label update for moderate to severe scalp psoriasis to the U.S. FDA expected in Q2:2019

ABRAXANE

Data from the phase III apact trial with ABRAXANE as adjuvant therapy in patients with surgically resected pancreatic cancer (event-driven)

PDUFA action date of March 12, 2019 for the supplemental Biologics License Application (sBLA) submission filed by Roche of Tecentriq (atezolizumab) in combination with ABRAXANE for the initial treatment of patients with PD-L1-positive, metastatic triple-negative breast cancer

Milestones Expected for Key Pivotal Assets

Ozanimod

U.S. NDA and EU Marketing Authorization Application (MAA) submissions in relapsing multiple sclerosis (RMS) on-track for Q1:2019

Phase III TRUE NORTH trial in ulcerative colitis (UC) expected to complete enrollment in H1:2019

Fedratinib

U.S. FDA approval expected by year-end 2019

EU MAA submission planned in H1:2019

Phase I/II combination trial with luspatercept planned

Luspatercept

U.S. and EU regulatory applications for transfusion-dependent, lower-risk myelodysplastic syndromes (MDS) with ring sideroblasts (RS+) and transfusion-dependent beta-thalassemia planned for H1:2019

Data expected from the phase II myelofibrosis trial in H2:2019

Liso-cel

Data from the pivotal TRANSCEND trial in relapsed/refractory DLBCL expected in 2019

U.S. BLA submission expected in H2:2019

Pivotal phase II trial in relapsed/refractory chronic lymphocytic leukemia (CLL) to be initiated in H1:2019

bb2121

Data from the KarMMa pivotal trial in RRMM expected in H2:2019

Phase II trial in NDMM to be initiated in H2:2019

Key Milestones Expected for Research & Early Development Pipeline

File at least 5 Investigational New Drug (IND) or Clinical Trial Applications (CTA) for novel assets

·Clinical data expected in 2019 from the following assets:

Asset

Indication

Status

CC-92480 (CELMoD)

RRMM

Phase I trial

CC-93269 (BCMA TCE)

RRMM

Phase I trial

CC-220 (CELMoD)

RRMM

Phase I/II trial

bb21217 (BCMA CAR T)

RRMM

Phase I trial

JCARH125 (BCMA CAR T)

RRMM

Phase I trial

CC-90009 (CELMoD)

Relapsed/refractory acute myeloid leukemia (AML)

Phase I trial

CC-90002 (anti-CD47 mAb)

Non-Hodgkin lymphoma (NHL)

Phase I trial

On January 7, 2019 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported 2018 highlights, 2019 guidance and commercialization plans for INBRIJA at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Acorda Therapeutics, JAN 7, 2019, View Source [SID1234532551]).

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"The approval of INBRIJA is a major milestone for Acorda. We are eager to bring this much-needed therapy to the Parkinson’s community," said Ron Cohen, M.D., Acorda’s President and CEO. "Acorda has one of the pre-eminent specialty neurology sales forces in the industry. Our team will immediately begin visiting key movement disorder centers to begin demonstrations and training on the appropriate use of INBRIJA. We expect INBRIJA to be available in the first quarter of 2019."

Burkhard Blank, M.D., Acorda’s Chief Medical Officer, added, "INBRIJA represents the first FDA approval of a treatment using the ARCUS technology, a platform that allows delivery of relatively large doses of medication through inhalation. ARCUS has the potential to be used in the development of a variety of inhaled medicines. In 2019, we will continue our development of an ARCUS-based treatment for migraine."

2018 Financials

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg unaudited net sales for 2018 are expected to be greater than $430 million, subject to change based on discounts and allowances recorded in the fourth quarter of 2018.
The Company is reiterating its 2018 non-GAAP operating expense guidance of R&D $100-$110 million and SG&A $170-$180 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."
2018 year-end cash and cash equivalents were approximately $445 million (unaudited).
Final results are subject to completion of the Company’s year-end audit.
2019 Guidance

During INBRIJA’s 2019 launch year, the Company expects to assess key metrics such as total and new prescriptions, unique prescribers, and managed care access, and does not expect to provide INBRIJA revenue projections.
The Company will no longer provide revenue guidance for AMPYRA, due to the unpredictable trajectory of revenue decline given the entrance of generics.
R&D expenses for the full year 2019 are expected to be $70-$80 million and SG&A expenses for the full year 2019 are expected to be $200-$210 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."
Presentation/Webcast Details

Dr. Cohen will provide a corporate overview at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9 at 8:00 a.m. Pacific/11:00 a.m. Eastern. The presentation is available via webcast at View Source or at www.acorda.com.

Non-GAAP Financial Measures

This press release includes financial measures that were not prepared in accordance with accounting principles generally accepted in the United States (GAAP). In particular, Acorda has provided 2018 and 2019 expense guidance for R&D and SG&A on a non-GAAP basis. Reconciliations of these measures to the most directly comparable GAAP financial measures are not available at this time because our analysis of 2018 financial performance (including share-based compensation expense) is ongoing, and because the 2019 financial measures are forward looking in nature and the amount of compensation charges and benefits needed to reconcile these measures to the most directly comparable GAAP financial measures is dependent on future changes in the market price of our common stock. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes the presentation of these non-GAAP financial measures, when viewed in conjunction with actual GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because they exclude non-cash charges that are substantially dependent on changes in the market price of our common stock. The Company believes these non-GAAP financial measures help indicate underlying trends in the Company’s business and are important in comparing current results with prior period results and understanding expected operating performance. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance

On January 7, 2019 Moleculin Biotech, Inc is presented the corporate presentation (Presentation, Moleculin, JAN 7, 2019, View Source [SID1234532567]).

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On January 7, 2019 Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) reported that the Company will report its financial results for the fourth quarter and full year ended December 31, 2018 before the US financial markets open on February 4, 2019 (Press release, Alexion, JAN 7, 2019, View Source [SID1234532504]). Following the release of the financial results, Alexion management will conduct a conference call and audio webcast at 8:00 a.m. Eastern Time (ET).

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To participate in this conference call, dial 866-762-3111 (USA) or 210-874-7712 (International), conference ID 5972194 shortly before 8:00 a.m. ET. The audio webcast can be accessed on the Investor page of View Source and an archived version will be available for a limited time following the presentation.

On January 7, 2019 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, reported that it is accelerating its clinical development strategy for AML and MDS and provided updates on clinical candidates CYAD-01 and CYAD-101 with key upcoming milestones for 2019 (Press release, Celyad, JAN 7, 2019, View Source [SID1234532520]).

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"We made progress in 2018 with our CAR-T clinical programs and we believe the Company is poised to achieve a number of important milestones in 2019," noted Dr. Christian Homsy, CEO of Celyad. "We continue to be encouraged by the clinical data observed with CYAD-01 for the treatment of hematological indications. As a result, we are prioritizing the clinical development program of CYAD-01 for the treatment of relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome towards Phase 2 trials."

2019 Milestones

Building upon its 2018 accomplishments, the Company intends to achieve the following key milestones over the next 12 months:

Report additional data from the Phase 1 dose-escalation THINK trial for CYAD-01 in r/r AML or MDS patients, including initial data from the schedule optimization portion of the trial;

Complete enrollment for and report initial data from the Phase 1 dose-escalation DEPLETHINK trial evaluating CYAD-01 with preconditioning chemotherapy in r/r AML or MDS patients;

Accelerate the development strategy and refine the regulatory pathway plan for CYAD-01 for the treatment of r/r AML or MDS patients, including the initiation of a Phase 2 clinical trial;

Present in vivo preclinical data for our proprietary non-gene edited allogeneic shRNA platform and progress towards an Investigational New Drug (IND) application for the program; and

Further evaluate the potential for next-generation autologous and allogeneic NKG2D-based CAR-T therapies in the treatment of solid tumors.

Press Release

07 January 2019

7:00am CET

Clinical Update for CYAD-01 in Hematological Malignancies

Celyad’s lead clinical candidate, CYAD-01, is currently being evaluated in multiple clinical trials for the treatment of patients with hematological malignancies, including r/r AML and MDS.

THINK Phase 1 Trial

In December 2018 at the 60th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting, Celyad reported an encouraging objective response rate in r/r AML patients of 38% (three out of eight) from the THINK Phase 1 trial, evaluating CYAD-01 without preconditioning chemotherapy.

Preliminary data for the last two patients enrolled and treated at dose level 3 show one patient with relapsing MDS with refractory anemia with excess blasts achieved a marrow complete response after two injections of CYAD-01, while the second patient with r/r AML experienced disease stabilization after the first cycle of CYAD-01. Both patients plan to receive a second (consolidation) cycle of therapy
.
Additional results from the THINK Phase 1 trial are expected to be announced during the first half of 2019.

Dr. Frédéric Lehmann, Vice President of Clinical Development and Medical Affairs at Celyad, commented, "Current clinical data for the last two patients treated at dose level 3 of the THINK trial provide additional support for the further development of CYAD-01 as a potential treatment of r/r AML and MDS and our decision to rapidly identify the best clinical path forward for the investigational therapy."

DEPLETHINK Phase 1 Trial

In December 2018, Celyad reported initial data from Cohort 1 of the trial, in which the administration of CYAD-01 following the preconditioning regimen of cyclophosphamide and fludarabine was well-tolerated, with no dose-limiting toxicity or treatment-related grade 3 or above adverse events observed. Based on these preliminary safety data from Cohort 1, enrollment has been initiated in Cohort 2 of the trial. Preliminary data from the DEPLETHINK Phase 1 trial are expected in mid-2019.

EPITHINK Phase 1 Trial

Based on the data generated to date for CYAD-01 from the THINK trial and the recent update in the treatment landscape for newly diagnosed AML patients, the Company has put the EPITHINK trial on hold to focus its efforts on the development of CYAD-01 for the treatment of r/r AML patients. Celyad plans to reassess the opportunity for CYAD-01 in newly diagnosed AML patients after the optimal treatment design for the therapy is determined. The EPITHINK Phase 1 dose-escalation trial planned to assess the administration of CYAD-01 concurrently with 5-azacytidine in treatment-naïve and/or elderly AML patients ineligible for intensive treatment.

Press Release

07 January 2019

7:00am CET

Solid Tumor Clinical Program Update

In November 2018 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting, the Company reported an interim analysis from the Phase 1 dose-escalation SHRINK trial evaluating the safety and activity of CYAD-01 administered concurrently with FOLFOX chemotherapy (a combination of 5-fluorouracil, leucovorin and oxaliplatin) in patients with metastatic colorectal cancer (mCRC). Follow-up assessment of patients based on response evaluation criteria in solid tumors (RECIST) from dose level 1 of the trial confirmed one patient achieved a partial response and two patients experienced disease stabilization. Full data from the SHRINK Phase 1 trial are expected in 2019.

In December 2018, Celyad initiated the alloSHRINK trial evaluating the non-gene edited allogeneic CAR-T therapy, CYAD-101, administered concurrently with FOLFOX chemotherapy in the treatment of patients with unresectable mCRC. To date, no relevant treatment related toxicity has been observed in the first subject enrolled in the trial. Topline data from alloSHRINK trial are expected in the second half of 2019.

About CYAD-01 and CYAD-101

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. CYAD-101 is an investigational, non-gene edited, allogeneic (donor derived) CAR-T therapy that co-expresses the chimeric antigen receptor NKG2D of CYAD-01 and the novel inhibitory peptide TIM (T cell receptor [TCR] Inhibiting Molecule). The expression of TIM reduces signaling of the TCR complex, which is responsible for Graft versus Host Disease (GvHD) and could therefore reduce or eliminate GvHD in patients treated with CYAD-101.