On January 7, 2019 Rainier Therapeutics, Inc., a privately-held clinical stage drug development company, reported that its targeted antibody vofatamab has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for treatment of patients with advanced or metastatic urothelial cell carcinoma (bladder cancer) that is positive for FGFR3 mutation and/or fusion (Press release, Rainier Therapeutics, JAN 7, 2019, View Source [SID1234532539]).

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"This Fast Track designation underscores the great unmet medical need that exists for the treatment of bladder cancer," said Scott Myers, Chairman and CEO of Rainier Therapeutics. "As the only antibody specifically targeted to FGFR3 we know to be in clinical development, we believe vofatamab offers a promising therapeutic option. We look forward to further data from our ongoing trials and working to advance our development efforts."

The FDA’s Fast Track designation is a process to facilitate development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Through the Fast Track program, more frequent meetings may be scheduled with the FDA to discuss the drug’s development plan and to ensure the collection of appropriate data needed to support approval. Additionally, the drug may qualify for accelerated approval and priority review and, at the time of a biologics license application (BLA) filing, the drug candidate’s sponsor may be eligible to submit completed sections of the BLA on a rolling basis before the complete application is submitted.

"Fast Track designation offers the potential to reduce development time and cost associated with bringing a drug to patients," said Valerie Fauvelle, Vice President, Regulatory Affairs at Rainier Therapeutics. "We look forward to working with the FDA to rapidly advance vofatamab through the clinical development and regulatory processes."

Vofatamab (B-701) is an antibody specifically targeted against the fibroblast growth factor receptor 3 (FGFR3), a known driver of bladder and other cancers. Vofatamab is the most advanced targeted biologic specific for FGFR3 known by Rainier to be in clinical development.

Rainier Therapeutics has ongoing Phase 1b and Phase 2 clinical studies of vofatamab in metastatic bladder cancer – the Fierce 21 and Fierce 22 studies. In addition, Rainier Therapeutics plans to study vofatamab in early stage bladder cancer – the Fierce 23 trial.

The Fierce 21 trial is evaluating vofatamab alone and in combination with docetaxel versus docetaxel alone to determine safety and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer with FGFR3 mutant/fusion who have relapsed after, or are refractory to, at least one prior line of chemotherapy. For more on this trial, visit www.clinicaltrials.gov (NCT0240542).

The Fierce 22 trial is evaluating vofatamab in combination with pembrolizumab, an immune checkpoint inhibitor, to determine safety, tolerability and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer, who have progressed following platinum-based chemotherapy and who have not received prior immune checkpoint inhibitor therapy. For more on this trial, visit www.clinicaltrials.gov (NCT03123055).

The Fierce 23 trial will evaluate vofatamab monotherapy in non-muscle invasive bladder cancer (NMIBC). This trial is planned to start in 2019.

On January 7, 2019 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing novel cancer immunotherapies, reported it has established a collaboration with the GOG Foundation, Inc., (GOG Foundation), to conduct a registration-enabled study of TAVO (tavokinogene telseplasmid) in women with recurrent/persistent cervical cancer (OMS-150) (Press release, OncoSec Medical, JAN 7, 2019, View Source [SID1234532538]).

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In June, 2018, KEYTRUDA (pembrolizumab) received accelerated approval from the FDA for the treatment of advanced cervical cancer with disease progression during or after chemotherapy based on data from a single-arm 98 patient study that showed a 14% overall response rate (ORR). Previous data in other advanced solid tumors demonstrate that TAVO combined with KEYTRUDA can induce objective responses in patients who do not respond to anti-PD-1 antibody monotherapy.

In this registration-directed clinical trial, OncoSec and GOG will evaluate the combination of TAVO and commercially available KEYTRUDA with the goal of achieving a clinically meaningful response rate greater than what has already been demonstrated with KEYTRUDA alone (14%). OncoSec and the GOG Foundation plan to enroll approximately 80 to 100 patients, who qualify for standard of care treatment with KEYTRUDA, in this single-arm study with TAVO. The trial will be open to patients with surface or subcutaneous lesions that are accessible via TAVO’s current delivery system. Patient enrollment is expected to begin in the first half of 2019. Importantly, should a clinically meaningful increase be observed in patients receiving the TAVO beyond that which they receive from KEYTRUDA alone, OncoSec plans to seek accelerated approval of TAVO in this patient population.

"KEYTRUDA is only the second drug in 30 years to be approved for the treatment of cervical cancer and, though it represents significant progress, the number of patients who can benefit is limited. Our goal is to improve upon the 14% KEYTRUDA response rate with the addition of TAVO," said Daniel J. O’Connor, President and Chief Executive Officer of OncoSec. "We believe that TAVO, our proprietary intratumoral plasmid-based IL-12, is an excellent complement for expanding the clinical benefit of anti-PD-1 therapies, especially for those patients that are resistant to anti-PD-1 therapies. Given that KEYTRUDA is already approved and reimbursed for this indication, this study fits perfectly with our strategy of identifying opportunities to conduct small, relatively low-cost single-arm clinical studies that have the potential to offer a rapid path to drug approval and commercialization."

The study will be conducted within GOG Foundation’s network under OncoSec’s investigational new drug (IND) application for TAVO. The GOG Foundation is a world-renowned non-profit organization with the purpose of conducting clinical research for the prevention and treatment of all gynecologic cancers, such as ovarian cancer, cervical cancer, endometrial cancer, vulvar cancer, and vaginal cancer. Its members make up a multi-disciplinary group, consisting of gynecologic oncologists, medical oncologists, pathologists, radiation oncologists, nurses, statisticians, and basic scientists.

"Conducting research that can lead to promising new therapies for women facing cervical cancer and other gynecological malignancies is central to our mission, and this collaboration is an exciting opportunity to bring our esteemed network and expertise in quality scientific research to the table," said Larry J. Copeland, MD, GOG Foundation President. "We’re grateful to play a role in this trial and look forward to advancing this therapy through the clinic."

On January 7, 2019 Stemline Therapeutics, Inc. (NASDAQ:STML), a biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that it has submitted the marketing authorization application (MAA) for ELZONRIS (tagraxofusp) to the European Medicines Agency (EMA) (Press release, Stemline Therapeutics, JAN 7, 2019, View Source [SID1234532537]). The MAA seeks approval for treating patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In November 2018, the EMA granted the ELZONRIS MAA accelerated assessment.

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On December 21, 2018, ELZONRIS was approved by the U.S. Food and Drug Administration (FDA) for the treatment of BPDCN in adult and pediatric patients, two years and older, in both treatment-naïve and previously-treated populations. ELZONRIS is the first treatment approved for BPDCN and the first approved CD123-targeted therapy.

"The submission of the ELZONRIS MAA is another major step forward for providing this important targeted treatment to patients, globally," said Ivan Bergstein, M.D., CEO of Stemline Therapeutics. "We look forward to working closely with the EMA to ensure this treatment is available to patients as quickly as possible. In parallel, our commercial team is continuing its ongoing effort to raise awareness of both CD123 testing and BPDCN worldwide. Potential European approval offers us an opportunity to significantly increase the number of patients who may benefit from ELZONRIS."

About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, was approved by the Food and Drug Administration (FDA) on December 21, 2018 for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment to the marketing authorization application (MAA), which was submitted to the EMA in January 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and other CD123 positive diseases.

About BPDCN
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

On January 7, 2019 Molecular Partners AG (SIX: MOLN), a clinical-stage biotech company pioneering the use of DARPin therapeutics* to treat serious diseases, reported that it will present at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019 at 8:00 AM Pacific Standard Time (11:00 AM Eastern Time; 5:00 PM CET) (Press release, Molecular Partners, JAN 7, 2019, View Source [SID1234532536]). The presentation, followed by a Q&A session, will be hosted by Dr. Patrick Amstutz, CEO of Molecular Partners.

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Beyond highlighting the progress of the clinical DARPin compounds and the evolution of the company’s therapeutic design space in oncology research, the presentation will outline the collaboration and license agreement for the clinical development and commercialization of MP0310 (FAP x 4-1BB) which the company and Amgen (NASDAQ:AMGN) announced on December 19, 2018. MP0310 is a preclinical molecule designed to locally activate immune cells in the tumor by binding to FAP on tumor stromal cells (localizer) and co-stimulating T cells via 4-1BB (immune modulator).

Audio webcast
The presentation will be webcast live. A copy of the presentation handout as well as a replay of the webcast will be made available on the company’s website www.molecularpartners.com under the Investors section. The replay will be available for 30 days following the presentation.

Financial Calendar
February 7, 2019 – Publication of Full-year Results 2018 (unaudited)
March 15, 2019 – Expected Publication of Annual Report 2018
April 16, 2019 – Annual General Meeting
May 9, 2019 – Interim Management Statement Q1 2019
View Source

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage more than 5 targets at once, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics.
The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their good safety profile, low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapeutics have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology, and is advancing a proprietary pipeline of DARPin drug candidates in oncology and immuno-oncology. The most advanced global product candidate is abicipar, a molecule currently in phase 3, in partnership with Allergan. Several DARPin molecules for various ophthalmic indications are also in development. The most advanced DARPin therapeutic candidate wholly owned by Molecular Partners, MP0250, is in phase 2 clinical development for the treatment of solid tumors and hematological tumors. MP0274, the second-most advanced DARPin drug candidate owned by Molecular Partners, has broad anti-HER activity; it inhibits HER1, HER2 and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. MP0274 is currently in phase 1. Molecular Partners is also advancing a growing preclinical pipeline that features several immuno-oncological development programs. DARPin is a registered trademark owned by Molecular Partners AG.

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

Maximize Commercial Assets

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