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

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·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 Stelexis Therapeutics, LLC reported that it closed a $43 million Series A financing to expand its proprietary platform to discover and selectively target pre-cancerous stem cells (Press release, Stelexis Therapeutics, JAN 7, 2019, View Source [SID1234550413]). Deerfield established Stelexis in 2017 together with scientific founders, Ulrich Steidl, Evripidis Gavathiotis, Amit Verma, and Roman Perez-Soler of Albert Einstein College of Medicine, Montefiore Health, New York and Derrick Rossi of Boston Children’s Hospital, Harvard Medical School. Patrick Doyle serves as the founding CEO, and Keren Paz is the CSO of Stelexis.

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Stelexis’ proprietary drug discovery platform identifies the earliest definable pre-cancerous stem and progenitor cells that lead to the formation of human primary and recurrent tumors for therapeutic intervention and relapse prevention. Stelexis’ mission is to develop novel cancer drugs that selectively target these critical pre-cancerous events related to both hematopoietic and solid malignancies.

"The ability to identify, isolate, study and screen rare pre-cancerous stem cells, from within bulk tumors, is an enormous breakthrough that has the potential to change how cancer patients are treated," stated Dr. Steidl. "Our thesis is that targeting cancer at its very origin should not only be effective as first line therapy, but should also lead to long-lasting remission for patients," said Dr. Rossi, who, prior to co-founding Stelexis, has also co-founded numerous other successful biotechnology companies.

Utilizing Deerfield seed funding and operational support since 2017, Stelexis has established its labs in Albert Einstein College of Medicine facilities, hired key management and is poised to deliver clinical trial data that validate its platform using the proceeds of this Series A round.

"We are thrilled to announce the formation and funding of Stelexis, which has the platform technology to explore the role pre-cancer conditions play in cancer development and recurrence. The team has an outstanding track record and we look forward to a stream of transformative cancer medicines," stated Dr. Robert Jackson, director at Stelexis and partner at Deerfield Management.

"Deerfield’s holistic approach to forming, funding and providing operational support to Stelexis has been instrumental in creating a leadership position in a novel targeted approach to treating cancer," said Patrick Doyle, CEO of Stelexis. "With these funds we are now positioned to execute on our potential to transform patients’ lives."

On January 6, 2019 GE Healthcare and Vanderbilt University Medical Center (VUMC) reported a five-year partnership to enable safer and more precise cancer immunotherapies (Press release, GE Healthcare, JAN 6, 2019, View Source [SID1234532487]). Multiple diagnostic tools will be developed to help predict both the efficacy of an immunotherapy treatment and its adverse effects for a specific patient before the therapy is administered. This would allow physicians to better target immunotherapies to the right patients and avoid potentially damaging, ineffective and costly courses of treatments.

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Immunotherapies use the immune system to recognize and attack cancer cells and can be more effective than traditional treatments, but response rates are often low and side effects can be severe1. GE Healthcare and VUMC will retrospectively analyze and correlate the immunotherapy treatment response of thousands of VUMC cancer patients, with their anonymized demographic, genomic, tumor, cellular, proteomic and imaging data. They will then develop AI-powered apps that draw on this data to help physicians identify the most suitable treatment for each individual patient.

Simultaneously, GE Healthcare and VUMC will develop new positron-emission tomography (PET) imaging tracers, which together with the apps, will help physicians to stratify cancer patients for clinical trials. It currently takes an average of 12 years2 and costs almost $2bn3 to bring a drug to market. In many cases, inappropriate patients are recruited to participate in immunotherapy trials, incurring unnecessary expense and slowing down approvals of new therapies. It is hoped that the PET tracers will ultimately also be used to monitor the efficacy of immunotherapies in everyday practice.

"Immunotherapy offers tremendous promise but given the current unpredictability of some patients’ reactions to treatments, it is also associated with increased morbidity and cost. This partnership provides the opportunity to leverage strengths of both of our organizations to further personalize cancer care by creating new tools that allow clinicians to more accurately predict how patients will respond to a specific therapy," said Jeff Balser, MD, PhD, President and Chief Executive Officer, Vanderbilt University Medical Center and Dean of the Vanderbilt University School of Medicine.

"GE Healthcare and Vanderbilt will combine their data science, genomic, imaging and cellular analysis capabilities to help improve clinical decision making. This partnership is a great example of the increasing convergence of the tools, technologies and data used by therapy innovators and healthcare providers," said Kieran Murphy, President and Chief Executive Officer, GE Healthcare.

GE Healthcare and the Vanderbilt-Ingram Cancer Center, a world-renowned stem cell transplant facility, will also collaborate on methods to improve productivity, efficiency and cost of stem cell transplant processing operations by automating processes, digitizing workflows, improving throughput and industrializing operations.

The first analytics application prototype will be available by the end of 2019 and the PET tracer proof-of-concept by the end of 2020.