Sierra Oncology to Attend Two Investor Conferences in May

On May 6, 2019 Sierra Oncology, Inc. (SRRA), a clinical stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported that members of its senior management team will be attending the SunTrust Robinson Humphrey 5th Annual Life Sciences Conference in New York on May 8th, and the Oppenheimer Oncology Insight Summit in New York on May 16th (Press release, Sierra Oncology, MAY 6, 2019, View Source [SID1234535737]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Sun Trust Robinson Humphrey 5th Annual Life Sciences Summit
Date: May 8
Format: One-on-one sessions

Oppenheimer Oncology Insight Summit
Date: May 16
Format: One-on-one sessions

Epizyme Provides Business Update and Reports First Quarter 2019 Financial Results

On May 6, 2019 Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported business and pipeline updates and reported first quarter 2019 financial results (Press release, Epizyme, MAY 6, 2019, View Source [SID1234535736]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This is an incredibly exciting time for our company, and 2019 is slated to be one of the most important years in Epizyme’s evolution," said Robert Bazemore, president and chief executive officer of Epizyme. "Our team is on track to submit two NDAs for tazemetostat this year, first for epithelioid sarcoma in the second quarter, which, if successful, would make tazemetostat the first commercially available EZH2 inhibitor, and then a second submission for follicular lymphoma in the fourth quarter. We are preparing for multiple clinical trials starting mid-year designed to expand the utility of tazemetostat, and to initiate clinical development of our novel G9a inhibitor in the second half of 2019. In addition, we have achieved the first milestone in our partnership with Boehringer Ingelheim, which further validates our research expertise and our collaboration strategy in epigenetic target discovery and drug development. With each milestone accomplished, we are one step closer to fulfilling our mission of bringing new treatments to patients with cancer and other serious diseases."

Tazemetostat Program Updates and Progress

NDA Submission for Epithelioid Sarcoma (ES) on Track for Second Quarter: Epizyme is well underway with its preparations to submit its first New Drug Application (NDA) for accelerated approval to the U.S. Food and Drug Administration (FDA) in the second quarter of 2019 for tazemetostat in patients with ES. This is an ultra-rare and difficult-to-treat sarcoma, and if approved, tazemetostat would be the first treatment specifically indicated for patients with ES. Updated data from the company’s ongoing Phase 2 trial in ES will be presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago; details will be disclosed at a future date.

NDA Submission for All-Comer Follicular Lymphoma (FL) Population Planned for Fourth Quarter: Epizyme plans to submit an NDA for accelerated approval of tazemetostat for patients with FL, regardless of their EZH2 mutational status, who have been previously treated with two or more systemic therapies. The ongoing Phase 2 study has completed enrollment, and the company is working toward the


submission of an NDA for accelerated approval in the fourth quarter of 2019 for this patient population. Updated data from the Phase 2 trial will be presented at a medical meeting in the second quarter of 2019. Details will be disclosed at a future date.

Planned Tazemetostat Clinical Studies in 2019

Epizyme is planning for multiple clinical trials designed to expand the benefit of tazemetostat into earlier treatment lines in follicular lymphoma, and to explore new combinations and potential indications in both FL and solid tumors. Planned clinical trials include:

a combination study of tazemetostat with the chemo-free treatment regimen "R2" (Revlimid plus Rituxan) for patients with relapsed/refractory FL who have received at least one prior therapy;

a combination study of tazemetostat with Rituxan for patients with relapsed/refractory FL;

a combination study of tazemetostat with R-CHOP for front-line patients with FL in collaboration with the Lymphoma Study Association (LYSA);

a combination study of tazemetostat with the standards-of-care for patients with castration-resistant prostate cancer; and

a combination study of tazemetostat with a PARP inhibitor for patients with platinum-resistant solid tumors, such as small-cell lung cancer, triple-negative breast cancer and ovarian cancer.

Initiation of Clinical Development of EZM8266 for Sickle Cell Disease

Upon approval of an Investigational New Application (IND) for EZM8266 for the treatment of patients with sickle cell disease, Epizyme anticipates beginning clinical development in the second half of 2019 with a Phase 1 trial of EZM8266, a novel, first-in-class G9a inhibitor.

Business Updates

Epizyme recently earned a $5.5 million milestone payment from Boehringer Ingelheim, following the selection of a lead optimization candidate for the shared program targeting an enzyme within the helicase family. The companies are jointly researching and developing this program, with both parties sharing U.S. commercialization responsibilities and Boehringer Ingelheim assuming responsibility for commercialization outside the U.S. The companies will also share research responsibilities for a histone acetyltransferase (HAT) program that is under development.

In March of this year, Epizyme raised $172.50 million in aggregate gross proceeds, before deducting underwriting discounts and offering expenses, from two concurrent underwritten public offerings.

First Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $371.1 million as of March 31, 2019, as compared to $247.9 million as of March 31, 2018.

Revenue: Collaboration revenue for the first quarter of 2019 was $7.9 million. There was no collaboration revenue recognized for the first quarter of 2018. The increase in collaboration revenue is due to the company’s collaboration with Boehringer Ingelheim, which was initiated in November 2018.

R&D Expenses: Research and development (R&D) expenses were $26.9 million for the first quarter of 2019, compared to $25.6 million for the first quarter of 2018. The increase is

primarily due to greater tazemetostat manufacturing costs and costs incurred in preparation for two NDA submissions offset by decreases in clinical trial expenses.

G&A Expenses: General and administrative (G&A) expenses were $12.0 million for the first quarter of 2019, compared to $9.4 million for the first quarter of 2018. The increase is primarily due to a rise in medical affairs and commercial costs as a result of organizational development in preparation for tazemetostat commercialization.

Net Loss Attributed to Common Stockholders: Net loss attributable to common stockholders was $32.3 million, or $0.39 per share, for the first quarter of 2019, compared to $34.1 million, or $0.49 per share, for the first quarter of 2018.

Financial Guidance

Following its March financing, and based on its current operating plan, Epizyme expects its cash runway to extend into at least the first quarter of 2021.

The company will not hold a conference call in conjunction with these results.

About the Epizyme-Boehringer Ingelheim Collaboration

Epizyme and Boehringer Ingelheim established a worldwide collaboration agreement in November 2018 to develop novel epigenetic oncology therapies. Under the terms of the agreement, Boehringer Ingelheim and Epizyme will jointly research and develop a helicase program, with both parties sharing U.S. commercialization responsibilities and Boehringer Ingelheim assuming responsibility for commercialization outside the U.S. Epizyme and Boehringer Ingelheim will also share research responsibilities for a histone acetyltransferase (HAT) program, with Boehringer Ingelheim assuming responsibility for worldwide development and commercialization. Epizyme received an upfront payment of $15 million and will receive an additional $5 million in research funding in 2019, and is eligible to receive up to $280.5 million in research, development and commercialization milestones. For the helicase program, Epizyme will fund a portion of the global development costs, retain a share of U.S. profits and receive tiered royalties on ex-U.S. sales. For the HAT program, Epizyme is eligible to receive tiered royalties on worldwide sales.

Adaptimmune Reports First Quarter 2019 Financial Results

On may 6, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results for the first quarter ended March 31, 2019 (Press release, Adaptimmune, MAY 6, 2019, View Source [SID1234535735]). Adaptimmune shared a clinical update in a separate release (https://bit.ly/2IJpltR).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Financial Results for the three-month period ended March 31, 2019

·Cash / liquidity position: As of March 31, 2019, Adaptimmune had cash and cash equivalents of $49.9 million and Total Liquidity(1) of $168.2 million.

·Revenue: Revenue for the three-month period ended March 31, 2019 was nil, compared to $8.2 million for the same period in 2018. No revenue has been recognised for the three months ended March 31, 2019 as the NY-ESO SPEAR T-cell transition program and the PRAME pre-clinical development program were completed in 2018, and work has not commenced on the third target nominated by GSK under the Collaboration and License Agreement.

·Research and development ("R&D") expenses: R&D expenses for the three-month period ended March 31, 2019 were $22.0 million, compared to $25.7 million for the same period of 2018; this decrease being primarily due to a reduction in expenditure associated with NY-ESO, which was transferred to GSK on July 23, 2018.

· General and administrative ("G&A") expenses: G&A expenses for the three-month period ended March 31, 2019 were $11.8 million, compared to $11.2 million for the same period of 2018.

· Other income, net: Other income for the three-month period ended March 31, 2019 was $5.4 million, compared to $7.1 million for the same period of 2018. Other income primarily comprises unrealized foreign exchange gains, which fluctuate depending on exchange rate movements and the amount of foreign currency assets and liabilities.

· Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three-month period ended March 31, 2019 was a loss of $27.4 million, ($(0.04) per ordinary share) compared to a loss of $21.1 million ($(0.04) per ordinary share) in the same period of 2018.

Financial guidance

The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations into the third quarter of 2020.

(1) Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

Unum Therapeutics to Host First Quarter 2019 Financial Results Conference Call and Webcast on May 13, 2019 at 8:00 A.M. ET

On May 6, 2019 Unum Therapeutics Inc. (Nasdaq: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies to treat cancer using its novel T cell technology platforms, reported that the company will host a conference call and live audio webcast on Monday, May 13, 2019 at 8:00 a.m. ET to discuss financial results for the first quarter of 2019 (Press release, Unum Therapeutics, MAY 6, 2019, View Sourcenews-releases/news-release-details/unum-therapeutics-host-first-quarter-2019-financial-results" target="_blank" title="View Sourcenews-releases/news-release-details/unum-therapeutics-host-first-quarter-2019-financial-results" rel="nofollow">View Source [SID1234535734]). Unum management will also provide an update on the Company’s recent progress and upcoming milestones.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Participants may access the conference call by dialing 866-300-3411 (domestic) or 636-812-6658 (international) and refer to conference ID number 1443149. To join the live webcast, please visit the investor relations section of the Unum Therapeutics website at View Source at least 10 minutes before the event begins.

A webcast replay will be available at the same location on the Unum Therapeutics website beginning approximately two hours after the event and will be archived for 90 days.

Significant Clinical Progress as Adaptimmune Announces Responses with ADP-A2M4 in Synovial Sarcoma and Antitumor Activity in Other Solid Tumors

On May 6, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported significant clinical progress with partial responses in 4 out of 5 synovial sarcoma patients treated with ~10 billion cells in the ADP-A2M4 pilot study, and tumor shrinkage seen in nearly all assessed synovial sarcoma patients (Press release, Adaptimmune, MAY 6, 2019, View Source;p=RssLanding&cat=news&id=2397170 [SID1234535732]). Based on these data, the Company will initiate the SPEARHEAD-1 trial in patients with synovial sarcoma and myxoid/round cell liposarcoma (MRCLS) later this year.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Beyond sarcoma, there is evidence of antitumor activity with ADP-A2M4 and ADP-A2M10 in other solid tumors. Based on these data and translational findings, the Company is expanding its clinical trial program by initiating a radiation sub-study, as well as opening a next-generation ADP-A2M4CD8 study (SURPASS trial), for which the IND has been filed. Finally, the first patient with HCC was treated in Cohort 2 (1 billion SPEAR T-cells) of the ADP‑A2AFP study and showed good tolerability to treatment and a transient decrease in serum AFP as well as tumor shrinkage at first scan.

"Today is a watershed moment for the Company. We now have confirmed responses in an unmet medical indication, synovial sarcoma, with our wholly owned ADP-A2M4 SPEAR T-cells. As we prepare to start the SPEARHEAD-1 study, we are one step closer to realizing our ambition to be the first T-cell company with an approved therapy in solid tumors in 2022," said James Noble, Adaptimmune’s Chief Executive Officer. "There is also early evidence of activity in other solid tumors with all three products and I am delighted to announce that we have filed an IND for a more potent, next-generation program with MAGE-A4 as the target. Working with world-class clinical trial centers and having a robust manufacturing and supply capability, we look forward to making further progress across the entire portfolio in the coming months."

ADP-A2M4 responses and data in synovial sarcoma patients

10 patients treated
8 patients assessed, with 6 showing tumor shrinkage
3 confirmed partial responses, 1 unconfirmed partial response
3 stable diseases (SD) and 1 progressive disease (PD)
ADP-A2M4 SPEAR T-cells appear to show a favorable benefit:risk profile in patients with synovial sarcoma
Good tolerability overall. Most adverse events are consistent with those typically experienced by cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies
SPEARHEAD‑1 protocol summary
Single-arm, Phase 2 study in more than 20 centers (North America & Europe) to include 60 patients with:
Advanced (metastatic or inoperable) synovial sarcoma or MRCLS patients who have received prior chemotherapy
HLA-A*02 & MAGE-A4 antigen positive
MAGE-A4 expression 30% (2+, 3+)
Primary endpoint will be overall response rate by RECIST v1.1 by independent review
Interim futility: 3 or more responses in the first 15 patients for study continuation
Safety endpoints with Independent Data Safety Monitoring Board
Exploratory endpoints with translational data and patient-reported outcomes
Treatment
Lymphodepletion: Flu: (30 mg/m2/ day) x 4 days; Cy (1800 mg/ m2/ day) x 2 days
Dose: up to 10 billion transduced SPEAR T-cells
Antitumor activity in other indications with ADP-A2M4 and ADP-A2M10

Tumor shrinkage seen in lung patients (ADP-A2M10), and melanoma and ovarian patients (ADP-A2M4)
7 patients treated with ADP-A2M4 in indications other than sarcoma in Cohorts 3 and Expansion phase
3 SDs, 3 PDs, and 1 patient expired due to disease progression before the first scan
7 patients treated with ADP-A2M10 in Cohort 3 and Expansion phase in the NSCLC and triple tumor studies
4 SDs, with one patient receiving a second infusion at Week 16, and 3 PDs to date
Adaptimmune continues to examine patient data to gain a clearer understanding of the best path forward to enhance RECIST responses
Adaptimmune is planning to start two new studies to transform currently observed activity in epithelial tumors into durable responses
Radiation sub-study at MD Anderson Cancer Center (MDACC) to initiate 2H 2019
Sub-study of the ADP-A2M4 clinical trial with up to 10 patients to be treated
Primary endpoint is safety and secondary endpoint is RECIST v1.1 responses
Radiation is 7Gy (low dose) per lesion or isocenter to be administered before lymphodepletion
SURPASS trial (ADP-A2M4CD8) – the first next-generation approach in the clinic in multiple solid tumors to initiate later this year
IND filed April 2019
Preclinical proof-of-concept data for this next-generation SPEAR T-cell therapy were presented at AACR (Free AACR Whitepaper) 2019 (https://bit.ly/2FGlRHN)
These data indicate that addition of the CD8α homodimer to the ADP-A2M4 cells effectively enables CD4+ "helper" T-cells to adopt a CD8+ "killer" like phenotype in vitro
This is intended to speed up initial antitumor activity and broaden the antitumor response in patients
Protocol summary:
Up to 30 subjects (HLA-A*02 with MAGE-A4+)
Primary endpoint: safety and tolerability
Secondary endpoint: antitumor activity
Lymphodepletion: Flu (30 mg/m2/day) × 4 days; Cy (1800 mg/m2/day) × 2 days
Shorter stagger between patients – anticipate faster dose escalation
Starting doses of ~1 billion cells (Cohort 1) and escalation through:
Cohort 2 (1.2 to 3.0 billion cells)
Cohort 3 (3.0 to 6.0 billion cells)
Expansion phase with up to 10 billion cells
Data expected in 2020

ADP-A2M10 studies are continuing and research plans will be reassessed as more data is accumulated across the two studies
ADP-A2AFP

Data from first HCC patient treated in Cohort 2
Continuing favorable safety data as was seen in Cohort 1 presented at AACR (Free AACR Whitepaper) 2019 (https://bit.ly/2FR8Lse)
Shows transient serum AFP decrease and tumor shrinkage at first scan
Data update will be provided in 1H 2020
Off-the-shelf program (allogeneic platform)

Substantial progress with update provided in an oral presentation at the American Society of Gene & Cell Therapy Annual Meeting 2019 by Dr. Jo Brewer, VP Allogeneic Research (https://bit.ly/2UWmqma)
Conference Call and Webcast Link for Clinical and Business Update Slide Presentation today (May 6th)
The Company will host a live teleconference and slide presentation at 8:00 a.m. EDT (1:00 p.m. BST) today (Monday May 6, 2019). The live webcast of the conference call and slides will be available at View Source An archive will be available after the call at the same address. To participate in the live webinar, if preferred, please dial (833) 652-5917 (U.S. and Canada) or
+1 (430) 775-1624 (International).