On June 18, 2018 TG Therapeutics, Inc. (NASDAQ: TGTX), reported an oral presentation of clinical data from its ongoing Phase 2 study evaluating umbralisib (TGR-1202), the Company’s PI3K delta inhibitor, in patients with relapsed or refractory Chronic Lymphocytic Leukemia (CLL) who are intolerant to prior BTK or PI3K delta inhibitor therapy (Press release, TG Therapeutics, JUN 18, 2018, View Source [SID1234527378]). Data from this trial were presented over the weekend during an oral session at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "We are pleased to present data evaluating umbralisib in patients intolerant to currently approved BTK or PI3K therapies during the EHA (Free EHA Whitepaper) annual congress. While there have been great advancements in recent years in the treatment of CLL, this study confirms that there are many patients still in need of an alternative treatment option and that umbralisib can be used safely and effectively in those patients who were not able to tolerate a prior BTK or PI3K therapy. The rate of patients withdrawing from kinase treatment for CLL in real world settings has been estimated to reach upwards of 40%, representing a significant unmet medical need." Mr. Weiss continued, "We are extremely pleased with the data presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) this month and we look forward to presenting the topline response rate data from the UNITY- CLL Phase 3 trial by the end of summer 2018."

Highlights from the oral presentation include the following:

Oral Presentation: A Phase 2 Study to Assess the Safety and Efficacy of Umbralisib (TGR-1202) In Patients with Chronic Lymphocytic Leukemia (CLL) Who Are Intolerant to Prior BTK or PI3K-delta Inhibitor Therapy (Abstract Number S808)

This presentation includes data from patients with CLL who are intolerant to prior BTK or PI3K delta inhibitor therapy who were then treated with single agent umbralisib (TGR-1202). To be eligible for the study patients had to have received prior treatment with a BTK inhibitor (ibrutinib, acalabrutinib) or a PI3K delta inhibitor (idelalisib, duvelisib) and discontinued therapy due to intolerance within 12 months of starting treatment on this study. Forty-seven patients were evaluable for safety of which 46 were evaluable for Progression Free Survival (PFS), (1 patient had a confirmed Richter’s Transformation (RT) at enrollment which did not meet eligibility criteria).

Highlights from this presentation include:

●Umbralisib demonstrated a favorable safety profile in patients intolerant to prior BTK or PI3K therapy

●Only 13% discontinued due to an adverse event, of which only one patient discontinued due to a recurrent adverse event (AE) also experienced with prior kinase inhibitor therapy

●Median progression free survival (PFS) and overall survival has not been reached with a median follow-up of 9.5 months

●In this relapsed/refractory CLL population, of which 77% required treatment within 6 months of prior KI discontinuation, 64% had a high-risk molecular / genetic marker and 6% had an ibrutinib resistance mutation, significant clinical activity has been observed

PRESENTATION DETAILS
The above referenced presentation is now available on the Publications page, located within the Pipeline section, of the Company’s website at www.tgtherapeutics.com/publications.cfm.

On June 18, 2018 Crinetics Pharmaceuticals, Inc., a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported the appointment of Alan S. Krasner, M.D. as Chief Medical Officer (Press release, Crinetics Pharmaceuticals, JUN 18, 2018, View Source [SID1234527377]).

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"We are delighted to welcome Alan to the Crinetics management team," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Alan has extensive experience in clinical development of endocrine drugs at both big pharma and smaller biotech companies. We are fortunate to have his leadership to oversee our development programs as we advance our product candidates into and through the clinic."

Dr. Krasner joins Crinetics from Shire Pharmaceuticals where he was a Senior Medical Director and served as Global Development Lead for Natpara, the first recombinant human intact parathyroid hormone treatment for hypoparathyroidism. Prior to Shire, he worked at Biodel and Pfizer conducting clinical research at various stages of development in diabetes and obesity. He obtained his undergraduate and M.D. degrees from Northwestern University and received his clinical training in Internal Medicine and Endocrinology at Johns Hopkins University.

On June 18, 2018 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its lead program, mirvetuximab soravtansine (Press release, ImmunoGen, JUN 18, 2018, View Source [SID1234527375]). The designation is for the treatment of patients with medium to high folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer who received at least one, but no more than three prior systemic treatment regimens, and for whom single-agent chemotherapy is appropriate as the next line of therapy.

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"We are pleased the FDA has granted Fast Track designation for mirvetuximab soravtansine," said Mark Enyedy, ImmunoGen’s President and Chief Executive Officer. "Patients with platinum-resistant ovarian cancer have a poor prognosis and we are encouraged by the FDA’s recognition of the significant need for new therapeutic options that may be addressed by mirvetuximab as monotherapy. This important designation is based on the promising safety and activity findings observed to-date and we look forward to working closely with the FDA as we advance the development of mirvetuximab."

The FDA’s Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.

Mirvetuximab soravtansine is being evaluated in the FORWARD I Phase 3 trial. The trial is designed to randomize 333 patients 2:1 to receive either mirvetuximab soravtansine or the physician’s choice of single-agent chemotherapy. Eligibility criteria include patients with platinum-resistant ovarian cancer that express medium or high levels of FRα who have been treated with up to three prior regimens. The primary endpoint of this study is Progression Free Survival, which is being assessed in the entire study population and in the subset of patients with high FRα expression. Enrollment of FORWARD I was completed ahead of schedule in April 2018 and ImmunoGen expects to report top-line results from the FORWARD I trial in the first half of 2019.

ImmunoGen is partnering with the Gynecologic Oncology Group Foundation Inc., a leader in clinical research in gynecologic malignancies, on FORWARD I, which is being conducted in North America and Europe. This trial is intended to support full marketing approval of mirvetuximab for patients with platinum-resistant ovarian cancer.

Mirvetuximab soravtansine is also being assessed in multiple combinations in the FORWARD II trial. FORWARD II is a Phase 1b/2 study of mirvetuximab in combination with Avastin (bevacizumab), or Keytruda (pembrolizumab) in patients with FRα-positive platinum-resistant ovarian cancer, primary peritoneal, or fallopian tube tumors, as well as a triplet combination of mirvetuximab plus carboplatin and Avastin in patients with platinum-sensitive ovarian cancer.

About Mirvetuximab Soravtansine

Mirvetuximab soravtansine (IMGN853) is the first folate receptor alpha (FRα)-targeting ADC. It uses a humanized FRα-binding antibody to target the ADC specifically to FRα-expressing cancer cells and a potent anti-tumor agent, DM4, to kill the targeted cancer cells.

About Ovarian Cancer and FRα

It is estimated that 22,000 women are diagnosed annually with ovarian cancer in the US. With more than 14,000 deaths each year, ovarian cancer accounts for more deaths than any other cancer of the female reproductive system.1

Standard first-line therapy for ovarian cancer is a platinum-based combination regimen. Once the cancer becomes platinum-resistant, treatment options include single-agent cytotoxic therapies such as pegylated liposomal doxorubicin, paclitaxel, or topotecan, and combination therapies that include Avastin.

There is a significant need for more effective, better-tolerated therapies for recurrent ovarian cancer. It is estimated that approximately 19,000 women in the US and approximately 24,000 women in the EU have platinum-resistant ovarian cancer requiring second-line or later treatment.2 ImmunoGen estimates that 60% of ovarian cancer cases have medium or high FRα expression.

On June 18, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that positive clinical data from ELZONRISTM (tagraxofusp; SL-401) trials were presented at the 23rdCongress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Sweden (Press release, Stemline Therapeutics, JUN 18, 2018, View Source [SID1234527374]).

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Results from the completed pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN) were presented for the first time to a European audience. This oral presentation also included positive survival and safety updates. Updated results from ongoing clinical trials in patients with relapsed/refractory chronic myelomonocytic leukemia (CMML) or relapsed/refractory myelofibrosis (MF) were also presented and continue to indicate the potential of ELZONRIS in indications beyond BPDCN.

Copies of all three presentations are available on Stemline’s website (www.stemline.com) under the Scientific Presentations tab.

Highlights from the Pivotal Trial – BPDCN (ELZONRIS 5-day regimen, multicycle)

ELZONRIS demonstrated high response rates and high rate of stem cell transplant (SCT) in blastic plasmacytoid dendritic cell neoplasm (BPDCN)
° 90% overall response rate (ORR) in first-line (12 mg/kg; n=29); 69% ORR in relapsed/refractory (n=13)
• Majority of responses are complete responses
° 45% of patients treated with ELZONRIS in first-line (12 mg/kg) were bridged to stem cell transplant in remission (n=13)
No apparent cumulative adverse events, including in the bone marrow, over multiple cycles
Updated data: Median overall survival (OS) in first-line (12 mg/kg; n=29) not reached
Rolling BLA submission in BPDCN underway; on track for completion this quarter
Highlights from the CMML Phase 1/2 Trial (ELZONRIS 3-day regimen, multi-cycle)

ELZONRIS monotherapy demonstrated efficacy, including bone marrow complete responses and improvements in splenomegaly, with a manageable safety profile in patients with relapsed/refractory CMML, an area of high unmet medical need
° Patient enrollment and follow up continues
Manageable safety profile
° Most common treatment-related adverse events (TRAEs) included hypoalbuminemia (38%), thrombocytopenia (25%), and fatigue (25%). Most common TRAEs, grade 3+, include thrombocytopenia (25%) and nausea (6%)
2 bone marrow complete responses (BMCRs)
100% of evaluable patients had reduction in baseline splenomegaly (spleen response)
° 75% had reduction by ≥50%
° 60% with baseline spleen size ≥5cm had reduction by ≥50%
55% (6/11) evaluable patients with treatment duration 6+ months, including 8+ and 14+ months
Based on these encouraging results, registrational trial designs in patients with relapsed/refractory CMML are under evaluation
Highlights from the MF Phase 1/2 Trial (ELZONRIS 3-day regimen, multi-cycle)

ELZONRIS monotherapy demonstrated efficacy, namely improvements in splenomegaly, with a manageable safety profile, in patients with relapsed/refractory MF, an area of high unmet medical need
° Patient enrollment and follow up continues
Manageable safety profile
° Most common TRAEs include hypoalbuminemia and thrombocytopenia (each 27%), and alanine aminotransferase increased, anemia, dizziness, fatigue, headache and nausea (each 20%). Most common TRAEs, grade 3+, include anemia (20%) and thrombocytopenia and fatigue (each 7%)
50% of evaluable patients, with baseline spleen size ≥5cm, had reduction in baseline splenomegaly
° 33% had reduction by ≥33%
° 25% had reduction by ≥35%
67% (6/11) of patients with spleen response had treatment duration 8+ months, including 8+, 12+ and 14+ months (all 3 ongoing)
° 4 patients with baseline thrombocytopenia had treatment durations 8+ months, 3 ongoing
Initial Quality of Life assessments appear promising, and a full TSS (Total Symptom Score) analysis is ongoing
Based on these encouraging results, next steps for the program are being evaluated including single agent, combination, and registration-directed trials in patients with relapsed/refractory MF
Ivan Bergstein, M.D., Stemline’s CEO, commented, "We had another strong showing at a major oncology conference. Our ELZRONRIS pivotal data in BPDCN, which included positive updates around survival and safety, were showcased and very well-received at EHA (Free EHA Whitepaper). The completion of our rolling BLA submission remains on-track for this quarter, and we continue to aggressively advance our pre-launch activities." Dr. Bergstein continued, "Additionally, given the encouraging data we continue to see with ELZONRIS in patients with previously-treated CMML and previously-treated MF, we are actively evaluating registrational opportunities in these areas of unmet medical need."

About ELZONRISTM (tagraxofusp; SL-401)
ELZONRISTM (tagraxofusp; SL-401) is a novel targeted therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. ELZONRIS has successfully completed a pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN), an indication for which it was granted Breakthrough Therapy Designation (BTD). A rolling Biologics License Application (BLA) submission is underway. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

On June 18, 2018 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported that the U.S. Food and Drug Administration (FDA) has accepted its supplementary premarket approval (sPMA) application for BRACAnalysis CDx to be used as a companion diagnostic with Pfizer’s PARP (poly ADP ribose polymerase) inhibitor, talazoparib (Press release, Myriad Genetics, JUN 18, 2018, View Source [SID1234527373]). The New Drug Application (NDA) for talazoparib has been granted priority review by the U.S. Food and Drug Administration and has a Prescription Drug User Fee Act (PDUFA) goal date of December 2018.

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Myriad’s sPMA and Pfizer’s NDA submissions are based on results from the Pfizer-sponsored EMBRACA trial, which evaluated talazoparib versus chemotherapy in patients with germline (inherited) BRCA-mutated (gBRCAm), HER2-negative locally advanced or metastatic breast cancer (MBC). The primary results of the study were presented at the San Antonio Breast Cancer Symposium in December 2017.

"Myriad was the pioneer in developing companion diagnostics for PARP inhibitors and this submission is another milestone in our collaborations to expand access to this class of drugs across multiple cancers," said Mark C. Capone, president and CEO, Myriad Genetics. "We are excited to pursue a simultaneous diagnostic approval along with talazoparib, as another outstanding opportunity to advance personalized medicine for oncology patients."

Myriad estimates there are approximately 125,000 patients with metastatic breast cancer who would immediately qualify for the BRACAnalysis CDx test, followed by 60,000 new patients per year on an ongoing basis.

About BRACAnalysis CDx
BRACAnalysis CDx is an in vitro diagnostic device intended for the qualitative detection and classification of variants in the protein coding regions and intron/exon boundaries of the BRCA1 and BRCA2 genes using genomic DNA obtained from whole blood specimens collected in EDTA. Single nucleotide variants and small insertions and deletions (indels) are identified by polymerase chain reaction (PCR) and Sanger sequencing. Large deletions and duplications in BRCA1 and BRCA2 are detected using multiplex PCR. Results of the test are used as an aid in identifying cancer patients with deleterious or suspected deleterious germline BRCA variants who may be candidates for a PARP inhibitor. This assay is for professional use only and is to be performed only at Myriad Genetic Laboratories, a single laboratory site located at 320 Wakara Way, Salt Lake City, UT 84108.