Bristol-Myers Squibb Receives Positive CHMP Opinion Recommending Approval of Empliciti (elotuzumab) Plus Pomalidomide and Low-Dose Dexamethasone for Treatment of Patients with Relapsed and Refractory Multiple Myeloma

On July 26, 2019 Bristol-Myers Squibb Company (NYSE:BMY) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion on a Type-II variation application for Empliciti (elotuzumab) plus pomalidomide and low-dose dexamethasone (EPd) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI), and have demonstrated disease progression on the last therapy (Press release, Bristol-Myers Squibb, JUL 26, 2019, View Source [SID1234537784]). The CHMP recommendation will now be reviewed by the European Commission, which has the authority to approve medicines for the European Union.

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"The positive opinion by the CHMP further validates the potential of this Empliciti-based combination to help patients with multiple myeloma who have relapsed or are refractory to prior therapies, a setting particularly in need of additional treatment options given the majority of patients with multiple myeloma will relapse," said Fouad Namouni, M.D., head, Oncology Development, Bristol-Myers Squibb.

The application is based on data from ELOQUENT-3, a randomized Phase 2 study evaluating pomalidomide and dexamethasone with or without Empliciti in patients with refractory or relapsed and refractory multiple myeloma. The U.S. Food and Drug Administration approved EPd for the treatment of adult patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a PI, in November 2018.

Bristol-Myers Squibb and AbbVie are co-developing Empliciti, with Bristol-Myers Squibb solely responsible for commercial activities.

About ELOQUENT-3

ELOQUENT-3 is a randomized Phase 2 study evaluating the addition of Empliciti to pomalidomide and dexamethasone (EPd) versus pomalidomide and dexamethasone (Pd) in 117 patients with multiple myeloma who received two or more prior therapies and were either refractory or relapsed and refractory to lenalidomide and a PI. Patients were randomized 1:1 to receive either EPd (n=60) or Pd alone (n=57) in 28-day cycles until disease progression or unacceptable toxicity. Patients in both the EPd and Pd arms received 4 mg of pomalidomide for days 1-21 of each cycle, and the weekly equivalent of 40 mg or 20 mg dexamethasone for patients ≤75 years or >75 years, respectively. In the EPd arm, Empliciti was administered at the dose of 10 mg/kg IV weekly for the first two cycles and 20 mg/kg monthly starting from cycle 3. The primary endpoint of the study was investigator-assessed progression-free survival.

Bristol-Myers Squibb: Advancing Oncology Research

At Bristol-Myers Squibb, patients are at the center of everything we do. The focus of our research is to increase quality, long-term survival for patients and make cure a possibility. Through a unique multidisciplinary approach powered by translational science, we harness our deep scientific experience in oncology and Immuno-Oncology (I-O) research to identify novel treatments tailored to individual patient needs. Our researchers are developing a diverse, purposefully built pipeline designed to target different immune system pathways and address the complex and specific interactions between the tumor, its microenvironment and the immune system. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines, like I-O, a reality for patients.

About Empliciti

Empliciti is an immunostimulatory antibody that specifically targets Signaling Lymphocyte Activation Molecule Family member 7 (SLAMF7), a cell-surface glycoprotein. SLAMF7 is expressed on myeloma cells independent of cytogenetic abnormalities. SLAMF7 also is expressed on Natural Killer cells, plasma cells and at lower levels on specific immune cell subsets of differentiated cells within the hematopoietic lineage.

Empliciti has a dual mechanism of action. It directly activates the immune system through Natural Killer cells via the SLAMF7 pathway. Empliciti also targets SLAMF7 on myeloma cells, tagging these malignant cells for Natural Killer cell-mediated destruction via antibody-dependent cellular toxicity.

Empliciti was initially approved by the FDA in 2015 in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received one to three prior therapies.

U.S. FDA-APPROVED INDICATIONS FOR EMPLICITI

EMPLICITI (elotuzumab) is indicated in combination with lenalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received one to three prior therapies.

EMPLICITI (elotuzumab) is indicated in combination with pomalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor.

IMPORTANT SAFETY INFORMATION

Infusion Reactions

Infusion reactions were reported in 10% of patients treated with EMPLICITI in the ELOQUENT-2 trial [EMPLICITI + lenalidomide + dexamethasone (ERd) vs lenalidomide + dexamethasone (Rd)] and 3.3% in the ELOQUENT-3 trial [EMPLICITI + pomalidomide + dexamethasone (EPd) vs pomalidomide + dexamethasone (Pd)].

In the ELOQUENT-2 trial, all infusion reactions were Grade 3 or lower, with Grade 3 infusion reactions occurring in 1% of patients. The most common symptoms included fever, chills, and hypertension. Bradycardia and hypotension also developed during infusions. In the trial, 5% of patients required interruption of the administration of EMPLICITI for a median of 25 minutes due to infusion reactions, and 1% of patients discontinued due to infusion reactions. Of the patients who experienced an infusion reaction, 70% (23/33) had them during the first dose.

In the ELOQUENT-3 trial, the only infusion reaction symptom was chest discomfort (2%), which was Grade 1. All the patients who experienced an infusion reaction had them during the first treatment cycle.

If a Grade 2 or higher infusion reaction occurs, interrupt the EMPLICITI infusion and institute appropriate medical and supportive measures. If the infusion reaction recurs, stop the EMPLICITI infusion and do not restart it on that day. Severe infusion reactions may require permanent discontinuation of EMPLICITI therapy and emergency treatment.

Premedicate with dexamethasone, H1 blocker, H2 blocker, and acetaminophen prior to EMPLICITI infusion.

Infections

In the ELOQUENT-2 trial (N=635), infections were reported in 81% of patients in the ERd arm and 74% in the Rd arm. Grade 3-4 infections were 28% (ERd) and 24% (Rd). Discontinuations due to infections were 3.5% (ERd) and 4.1% (Rd). Fatal infections were 2.5% (ERd) and 2.2% (Rd). Opportunistic infections were reported in 22% (ERd) and 13% (Rd). Fungal infections were 10% (ERd) and 5% (Rd). Herpes zoster was 14% (ERd) and 7% (Rd).

In the ELOQUENT-3 trial (N=115), infections were reported in 65% of patients in both the EPd arm and the Pd arm. Grade 3-4 infections were reported in 13% (EPd) and 22% (Pd). Discontinuations due to infections were 7% (EPd) and 5% (Pd). Fatal infections were 5% (EPd) and 3.6% (Pd). Opportunistic infections were reported in 10% (EPd) and 9% (Pd). Herpes zoster was reported in 5% (EPd) and 1.8% (Pd).

Monitor patients for development of infections and treat promptly.

Second Primary Malignancies

In the ELOQUENT-2 trial (N=635), invasive second primary malignancies (SPM) were 9% (ERd) and 6% (Rd). The rate of hematologic malignancies was the same between ERd and Rd treatment arms (1.6%). Solid tumors were reported in 3.5% (ERd) and 2.2% (Rd). Skin cancer was reported in 4.4% (ERd) and 2.8% (Rd).

In the ELOQUENT-3 trial (N=115), invasive SPMs were 0% (EPd) and 1.8% (Pd).

Monitor patients for the development of SPMs.

Hepatotoxicity

In the ELOQUENT-2 trial (N=635), AST/ALT >3X the upper limit, total bilirubin >2X the upper limit, and alkaline phosphatase <2X the upper limit were 2.5% (ERd) vs 0.6% (Rd). Of 8 patients experiencing hepatotoxicity, 2 patients discontinued treatment while 6 patients had resolution and continued. Monitor liver enzymes periodically. Stop EMPLICITI upon ≥Grade 3 elevation of liver enzymes. Continuation of treatment may be considered after return to baseline values.

Interference with Determination of Complete Response

EMPLICITI is a humanized IgG kappa monoclonal antibody that can be detected on both the serum protein electrophoresis and immunofixation assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and possibly relapse from complete response in patients with IgG kappa myeloma protein.

Pregnancy/Females and Males of Reproductive Potential

There are no available data on EMPLICITI use in pregnant women to inform a drug-associated risk of major birth defects and miscarriage.

There is a risk of fetal harm, including severe life-threatening human birth defects, associated with lenalidomide and pomalidomide, and they are contraindicated for use in pregnancy. Refer to the respective product full prescribing information for requirements regarding contraception and the prohibitions against blood and/or sperm donation due to presence and transmission in blood and/or semen and for additional information.

Adverse Reactions

ELOQUENT-2 trial:

Serious adverse reactions were 65% (ERd) and 57% (Rd). The most frequent serious adverse reactions in the ERd arm compared to the Rd arm were: pneumonia (15%, 11%), pyrexia (7%, 5%), respiratory tract infection (3.1%, 1.3%), anemia (2.8%, 1.9%), pulmonary embolism (3.1%, 2.5%), and acute renal failure (2.5%, 1.9%).
The most common adverse reactions in ERd and Rd, respectively (≥20%) were fatigue (62%, 52%), diarrhea (47%, 36%), pyrexia (37%, 25%), constipation (36%, 27%), cough (34%, 19%), peripheral neuropathy (27%, 21%), nasopharyngitis (25%, 19%), upper respiratory tract infection (23%, 17%), decreased appetite (21%, 13%), and pneumonia (20%, 14%).
ELOQUENT-3 trial:

Serious adverse reactions were 22% (EPd) and 15% (Pd). The most frequent serious adverse reactions in the EPd arm compared to the Pd arm were: pneumonia (13%, 11%) and respiratory tract infection (7%, 3.6%).
The most common adverse reactions in EPd arm (≥20% EPd) and Pd, respectively, were constipation (22%, 11%) and hyperglycemia (20%, 15%).

Pfenex to Announce Second Quarter 2019 Financial Results on August 8

On July 25, 2019 Pfenex Inc. (NYSE American: PFNX), a clinical-stage development and licensing biotechnology company focused on leveraging its Pfēnex Expression Technology to improve protein therapies for unmet patient needs, reported that it will report its financial results for the second quarter and six months ended June 30, 2019, after the market close on Thursday, August 8, 2019, and will host a conference call and webcast at 4:30 PM Eastern Time (Press release, Pfenex, JUL 25, 2019, View Source [SID1234538433]).

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Conference Call & Webcast
Thursday, August 8th @ 4:30pm Eastern Time/1:30pm Pacific Time
Domestic:866-376-8058
International:412-542-4131
Webcast: View Source

Replays, Available Through August 15th:
Domestic:877-344-7529
International:412-317-0088
Replay Access Code:10133889

About Pfenex Inc.
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Wayshine Biopharm holding limited Announces ORPHAN DRUG DESIGNATION GRANTED By The FDA For WSD0922 IN GLIOMA (including GLIOBLASTOMA AND ANAPLASTIC ASTROCYTOMA)

On July 25, 2019 Wayshine Biopharm, a clinical-stage pharmaceutical company, reported that its First-in-Class CNS penetrable EGFR/EGFRvIII inhibitor, namely WSD0922, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of glioma (including Glioblastoma and Anaplastic Astrocytoma) (Press release, Wayshine Biopharm, JUL 25, 2019, View Source [SID1234537798]). Orphan drug designation was created to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases.

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WSD0922 has previously received IND approval from FDA for the treatment of Glioblastoma, Anaplastic Astrocytoma and cancers with CNS metastasis patients. Phase I/IIA to evaluate safety, tolerability, pharmacokinetics and anti-tumor activity of WSD0922 is ongoing at Minnesota, Arizona and Florida, the three campuses of Mayo Clinic.

"Orphan Drug Designation by the FDA for glioma is another significant milestone in the WSD0922 development program," commented Dr. Wei Zhong, Ph.D., CEO and Founder of Wayshine Biopharm. " We are very pleased that the FDA has granted broader indication than the indication proposed (GBM and AA). Fast grant for orphan drug designation by the FDA based on encouraging preclinical data truly reflects our innovation and commitments and the clinical potential of WSD0922 has been recognized and endorsed by the FDA, for this substantial unmet medical need and expansion to lower grade brain cancer."

Orphan drug designation by the FDA is granted to promote the development of drugs that target diseases affecting 200,000 or fewer U.S. patients annually and that are expected to provide significant therapeutic advantage over existing treatments. Orphan drug designation qualifies a company for benefits that apply across all stages of drug development, including an accelerated approval process, eligibility for orphan drug grants, seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, and a waiver of certain administrative fees.

Cytokinetics to Announce Second Quarter Results on August 8, 2019

On July 25, 2019 Cytokinetics, Incorporated (Nasdaq:CYTK) reported that it is scheduled to report second quarter results on August 8, 2019 at 4:00 PM Eastern Time (Press release, Cytokinetics, JUL 25, 2019, View Source [SID1234537787]). Following the announcement, Cytokinetics’ senior management will host a conference call at 4:30 PM Eastern Time to discuss operational and financial results and the company’s outlook for the future.

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The conference call will be simultaneously webcast and can be accessed from the homepage and in the Investors & Media section of Cytokinetics’ website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 1778276.

An archived replay of the webcast will be available via Cytokinetics’ website until August 15, 2019. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 1778276 from August 8, 2019 at 7:30 PM Eastern Time until August 15, 2019.

Snap Bio and i2020 Accelerator (Torrey Pines Investment) Reach Lead Compounds Milestone for Pediatric Oncology and Advanced Liver Cancer Programs

On July 25, 2019 Snap Bio, Inc. and Torrey Pines Investment (TPI) reported that their joint early drug discovery program within the framework of the i2020 Accelerator has resulted in achieving the projected milestone in developing several small molecule lead candidates for treatment of Hepatocellular carcinoma (HCC) and Hepatoblastoma (Press release, Snap Bio, JUL 25, 2019, View Source [SID1234537776]). The milestone is marked with an outstanding selectivity profile and promising overall animal data in rodents for the lead candidates.

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This timeline-driven achievement was enabled­­ through the synergistic efforts of TPI’s i2020 Accelerator network, which includes ChemDiv, Inc. with its pertinent technical life-sciences expertise and its unique integrated drug discovery platform.

"The i2020 Accelerator was created to help take early stage research programs with differentiated biology and established development paradigms to the level of advanced leads and clinical candidates," comments Ronald Demuth, President of Torrey Pines Investment. "We were excited to start working with Snap Bio, their unique platform that integrates structure-based drug design and advanced synthetic biology allows for scaffold modification to be taken to a new level with improved optimization for all the key properties of highly effective and selective treatments. Snap Bio has been a great fit for our i2020 Accelerator portfolio, and we are glad to be seeing the first fruits of our collaboration thanks to the collective efforts of our global net of partners, including ChemDiv."

"With the help of TPI and the i2020 Accelerator, Snap Bio has discovered novel bispecific inhibitors, opening the door to a platform that offers small molecules with unique combinations of well-defined activities," comments Mark Burk, President and CEO at Snap Bio. "Bispecific small molecules can simultaneously and selectively inhibit two mechanistically distinct targets, which can provide important polypharmacological benefits, such as higher efficacy, fewer side effects, and reduced drug resistance. In addition, relative to drug combination therapies, bispecific inhibitors can target multiple disease pathways without pharmacokinetic disparity, leading to easier, faster, cheaper, and lower risk clinical trials."

About Torrey Pines Investment and i2020

Torrey Pines Investment (San Diego, CA) is a specialty life-science investment firm that invests in development stage molecules, diagnostics, and therapeutics in areas of high unmet medical need. [View Source By rapidly de-risking novel science and enriching partner pipelines with best-in-class molecules, i2020 platform helps accelerate early stage drug discovery platforms towards advanced lead and clinical candidate level within a two-year framework. i2020 Accelerator’s vast scientific and global resource network tailored specifically to the needs of early drug development programs allows it to take on projects in a wide array of therapeutic areas, from immunology to infectious diseases and beyond. By successfully leveraging agile development principles and flexible partnering business models, i2020 Accelerator plans to create and invest in multiple small-molecule early drug discovery programs. [www.i2020accelerator.com]