On July 25, 2019 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported that it will release its second quarter 2019 financial results on Thursday, August 1, 2019 (Press release, Insmed, JUL 25, 2019, View Source [SID1234537774]).

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Insmed management will host a conference call for investors beginning at 8:30 a.m. ET on Thursday, August 1, 2019 to discuss the financial results and provide a business update.

Shareholders and other interested parties may participate in the conference call by dialing (888) 317-6003 (domestic) or (412) 317-6061 (international) and referencing conference ID number 5579948. The call will also be webcast live on the company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately one hour after its completion through August 8, 2019 by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and referencing replay access code 10133256. A webcast of the call will also be archived for 90 days under the Investor Relations section of the company’s website at www.insmed.com.

On July 25, 2019 Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO – FR0010095596), ("Onxeo" or "the Company"), a clinical-stage biotechnology company specializing in the development of innovative drugs targeting tumor DNA Damage response (DDR) in oncology, in particular against rare or resistant cancers, reported its consolidated half-year financials, as of June 30, 2019, and provided a business update (Press release, Onxeo, JUL 25, 2019, View Source [SID1234537742]).

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Judith Greciet, Chief Executive Officer of Onxeo, said: "During the first half of 2019, we have achieved major progress in our developments that continue to enhance the value of our first-in-class lead drug candidate AsiDNA and our other R&D assets.

With regards to AsiDNA, the well-executed DRIIV-1 phase I study of AsiDNA in solid tumors provided positive results by meeting each of its core objectives and notably confirming both the activity and the tolerance of AsiDNA. Based on these sound data, we have launched the first phase 1b study of AsiDNA in combination with a reference chemotherapy (carboplatin and paclitaxel) in patients suffering from eligible solid tumors. In parallel, we plan to initiate a second combination clinical study with a PARP inhibitor by year-end to assess the ability of AsiDNA to abrogate the acquired resistance to PARP inhibitors, a major limitation for their clinical use.

We also recently expanded our pipeline with our new optimized lead OX401 that entered a proof-of-concept preclinical phase. OX401 is based on the same decoy agonist mechanism as AsiDNA and was designed to be a next-generation PARP inhibitor that does not induce resistance but triggers a strong immune response through the activation of the STING pathway. This new candidate is at the crossroads of DNA Damage Response and immuno oncology, the two most attractive domains in cancer treatment.

By renewing our equity financing line with Nice & Green last June, we have secured the needed financial resources over at least the next 12 months to confidently move forward the developments of these two high potential candidates."

On July 25, 2019 Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported that the U.S. Food and Drug Administration (FDA) has accepted for filing the company’s New Drug Application (NDA) for accelerated approval of tazemetostat, its lead investigational agent (Press release, Epizyme, JUL 25, 2019, View Source [SID1234537758]). Epizyme has proposed an indication of metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery. The FDA granted Priority Review for the NDA and has set a Prescription Drug User Fee Act (PDUFA) target action date of January 23, 2020. Priority Review is granted to investigational therapies that, if approved, may offer significant improvements in the treatment, prevention or diagnosis of a serious condition.

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"We are thrilled with FDA’s acceptance of this first tazemetostat NDA submission for priority review, and to be an important step closer to achieving our mission of rewriting treatment for patients with cancer and other serious diseases," said Robert Bazemore, president and chief executive officer of Epizyme. "This is a significant achievement in the development of this potentially first-in-class EZH2 inhibitor, and we look forward to working with FDA during the review. If approved, we believe tazemetostat could become an important new option in the treating physicians’ arsenal. We would like to extend our sincerest gratitude to those patients, families and medical teams who have participated in our clinical studies and helped bring tazemetostat to this stage."

Epizyme’s NDA submission is based primarily on data from the 62 patient epithelioid sarcoma cohort of its ongoing Phase 2 study of tazemetostat. These data, recently reported at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, showed that tazemetostat treatment resulted in clinically meaningful and durable responses, and was generally well-tolerated.

To support full approval of tazemetostat for epithelioid sarcoma, Epizyme will initiate a global confirmatory trial. The company plans to conduct a 1:1 randomized, controlled clinical trial in the front-line treatment setting comparing tazemetostat in combination with doxorubicin versus placebo plus doxorubicin in approximately 150 patients. The primary efficacy endpoint will be progression-free survival, and secondary efficacy endpoints will include overall survival, disease control rate, overall response rate and duration of response. The confirmatory study will include a safety run-in that is expected to begin in the second half of 2019.

Investor Conference Call
Epizyme will host an investor conference call and webcast today, July 25, 2019, at 8:30 a.m. To participate in the call, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 5749851. A live webcast will be available in the investor section of the company’s website at www.epizyme.com. The webcast will be archived on the website for 60 days.

About the Tazemetostat Clinical Trial Program
Tazemetostat, an oral, potent, first-in-class EZH2 inhibitor, is currently being studied as a monotherapy in ongoing clinical programs in patients with certain molecularly defined solid tumors, including epithelioid sarcoma and other INI1-negative tumors, and in patients with follicular lymphoma, both with and without EZH2 activating mutations. Multiple clinical studies are underway through collaborations assessing tazemetostat as a combination treatment for patients with diffuse large B-cell lymphoma. Epizyme also plans to conduct multiple additional clinical trials designed to evaluate the potential benefit of tazemetostat in earlier lines of therapy for follicular lymphoma, as well as new combinations and cancer indications.

On July 25, 2019 4D pharma plc (AIM: DDDD), a pharmaceutical company leading the development of Live Biotherapeutics, reported an update on its oncology programmes (Press release, 4d Pharma, JUL 25, 2019, View Source [SID1234537775]). New preclinical data on the Company’s oncology candidates, MRx0518 and MRx1299, were presented at 1st Microbiome Movement Oncology Response Summit in Boston, USA by Research Director Imke Mulder. A clinical progress update on MRx0518 was also provided by 4D’s Chief Scientific Officer, Alex Stevenson.

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MRx1299

MRx1299 is a second-generation Live Biotherapeutic identified by 4D’s MicroRx discovery platform. One of the target host pathways identified for this Live Biotherapeutic is its potent histone deacetylase (HDAC) inhibition mediated by bacterial short-chain fatty acids (SCFAs). HDAC inhibition is thought to have multiple modes of anti-cancer activity, acting on both tumour and immune cells. Treatment with MRx1299 and its metabolites gives cytotoxic T lymphocytes the ability to reduce tumour growth in preclinical cancer models.

MRx0518

0MRx0518, the Company’s lead Live Biotherapeutic candidate in oncology, induces strong innate and adaptive immune responses in vitro and in vivo. New data presented demonstrates the ability of orally administered MRx0518 to modulate the frequency of immune cell populations with anti-tumour activity in the gastrointestinal tract, systemically and in the tumour microenvironment, acting through signalling pathways known to drive anti-cancer immunity.

The Company has previously published data on the mechanism of action of MRx0518 (Lauté-Caly et al. 2019), demonstrating that the bacterial flagellin protein, FliC, strongly activates immunostimulatory NF-κB signalling, via the TLR5 receptor. Research presented at the conference indicates a role for additional host receptors, including TLR9, in mediating the immuno-stimulatory effects of MRx0518.

Alex Stevenson, 4D’s Chief Scientific Officer, commented, "The identification of our new Live Biotherapeutic candidate, MRx1299, strengthens our leading oncology portfolio and expands the scope of oncology indications for our Live Biotherapeutics. MRx1299 has a different mechanism of action to MRx0518 and as such may be suitable for the treatment of different types of cancer. Studies of MRx1299’s efficacy in preclinical models of additional tumour types are ongoing, as is scale-up and process development."

He added, "The additional preclinical data on MRx0518 strengthens our knowledge of its mechanism of action and capacity to launch an immune response against cancer cells. In parallel, data generated from our ongoing clinical studies will assess the ability of MRx0518 to induce these anti-tumour immunological effects in patients. Our Phase I/II study of MRx0518 in combination with KEYTRUDA (pembrolizumab) in patients with solid tumours will provide us with specific biomarker data from both parts of the study. We anticipate the first of this data to be available by the end of 2019."

A copy of the presentations given can be found at View Source

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.