On September 24, 2018 Epizyme, Inc. (NASDAQ: EPZM), a clinical-stage company developing novel epigenetic therapies, reported the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold that had paused U.S.-based enrollment of new patients in its tazemetostat clinical trials (Press release, Epizyme, SEP 24, 2018, View Source [SID1234529538]). Epizyme is now in the process of reopening enrollment in all of its company-sponsored trials in the U.S., including the follicular lymphoma (FL) EZH2 activating mutation cohort of its Phase 2 non-Hodgkin lymphoma trial.

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Epizyme’s formal response to the FDA included a comprehensive assessment of the risk of secondary malignancies, including T-cell lymphoblastic lymphoma (T-LBL) potentially associated with tazemetostat, which took into account both published literature and the company’s clinical experience to date. This followed a report of a single case of T-LBL in its tazemetostat pediatric study. Epizyme provided a thorough assessment of efficacy and safety data across all of its trials in hematological malignancies and solid tumors, in both adults and children, and convened a panel of external scientific and medical experts who reviewed and validated the findings.

"The Epizyme team has worked diligently to provide a comprehensive response back to the FDA, and through constructive dialogue, we successfully resolved the partial clinical hold. This allows us to turn our full attention to our key priorities: preparing for our first NDA submission for tazemetostat in epithelioid sarcoma and defining our registration path in FL," said Robert Bazemore, president and chief executive officer of Epizyme. "We, along with our investigators and the global experts we consulted to support our complete response, continue to believe in the positive benefit/risk of tazemetostat as we move forward in our clinical development program. We remain steadfast in our commitment to bringing this potential therapeutic option to cancer patients in need of safe and effective new treatments."

Epizyme will now engage with regulators in France and Germany to resolve the partial clinical holds and resume enrollment in those countries. The company is also working closely with its study partners to reach a similar resolution for their respective trials in which tazemetostat is being studied in combination with other therapies.

Investor Conference Call Notice
Company management plans to host a conference call and webcast at 8:30 a.m. EDT today to discuss the resolution of the partial clinical hold. To participate, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 3499753. A live webcast will be available in the investor section of the company’s website at www.epizyme.com. The webcast also will be archived on the website for 60 days.

About the Tazemetostat Clinical Trial Program
Tazemetostat, a potent, selective, orally available, first-in-class EZH2 inhibitor, is currently being studied as a monotherapy in ongoing Phase 2 programs in certain molecularly defined solid tumors, including epithelioid sarcoma and other INI1-negative tumors; follicular lymphoma (FL); and combination studies in diffuse large B-cell lymphoma (DLBCL) and non–small cell lung cancer (NSCLC).

On September 24, 2018 Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, reported that Douglas M. Fambrough, Ph.D., president and chief executive officer, will participate in two investor conferences in October (Press release, Dicerna, SEP 24, 2018, View Source [SID1234529539]).

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2018 Cantor Fitzgerald Global Healthcare Conference, Monday, October 1, 2018 at 7:45 a.m. ET at the InterContinental New York Barclay in New York. Dr. Fambrough will present a corporate update and overview of the Company’s most advanced GalXC pipeline programs, including DCR-PHXC, an investigational RNAi therapeutic in development for the treatment of all forms of primary hyperoxaluria. A live webcast of the presentation can be accessed on the Investors & Media section on the Dicerna website at www.dicerna.com. An archived replay of the webcast will be available on the Company’s website after the conference.

Leerink Partners Roundtable Series: Rare Disease and Oncology, Tuesday, October 2, 2018 at 2:30 p.m. ET at the Lotte New York Palace in New York. Dr. Fambrough will participate in an analyst-moderated fireside chat. A live webcast of the fireside chat can be accessed on the Investors & Media section on the Dicerna website at www.dicerna.com. An archived replay of the webcast will be available on the Company’s website after the conference.

On September 24, 2018 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections, reported that President and Chief Executive Officer Guy Macdonald will present a corporate overview at the Cantor Global Healthcare Conference on Monday, October 1, 2018 at 5:15 p.m. Eastern Time at InterContinental New York Barclay Hotel in New York City (Press release, Tetraphase, SEP 24, 2018, View Source [SID1234529560]).

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A live webcast of the presentation will be available on the Company’s website at View Source The archived presentation will be available for 30 days

On September 24, 2018 Affibody AB ("Affibody"), a clinical stage biopharmaceutical company developing a portfolio of innovative drug projects, reported that a major Nordic study ("Affibody-3") will begin using Affibody’s PET imaging agent ABY-025 (Press release, Affibody, SEP 24, 2018, View Source [SID1234575703]). ABY-025 is a novel Affibody molecule imaging tracer that with high precision can identify HER2 status in breast cancer patients . The researchers will be investigating a new ABY-025 based method developed at Uppsala University and the PET center at Uppsala University Hospital that has the potential to improve personalized breast cancer treatment.

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"Since 2015, we have performed two smaller studies at Uppsala University Hospital with more than 20 patients showing very promising results. If we can demonstrate that the method works in larger scale, we believe it can quickly become a new "gold" standard worldwide. This would mean that difficult and stressful biopsies can be avoided for many breast cancer patients," says Dr. Henrik Lindman, Physician at the Oncology Clinic, Uppsala University Hospital, who is running the study together with Dr. Jens Sörensen, Physician at the PET center in the hospital.

In patients with so-called HER2-positive breast cancer, a large amount of the HER2 protein is present on the surface of the cancer cells. The more concentrated the protein, the more aggressive the disease. The new PET method involves a small protein-tracer, ABY-025, labeled with radioactive gallium. This procedure is done at the PET center at Uppsala University Hospital. ABY-025 is administered to the patient, finding and attaching to the HER2 protein. The PET camera then records the tracer signals and calculates the patient’s exact HER2 status, enabling the physician to prescribe medication more accurately.

"Several drugs effective against HER2 positive breast cancer have been available many years now. However, when selecting treatment, it is extremely important to know if the woman has HER2-positive metastases and how much HER2 protein is present on the cell surface. Our new method has two major advantages – you do not have to take tissue samples to get answers, an unpleasant experience for the patient, and we can also simultaneously map all tumors in the body," says Dr. Lindman.

"We are very proud of the way by which the ABY-025 development is conducted. The unique partnership that Affibody has with the principle investigators of Affibody-3 reflects our corporate ambition to build an extensive network of renowned researchers and clinicians in order to be able to fully capture the value of our proprietary platforms. We look forward to continue to build more such relationships", said David Bejker, CEO of Affibody.

About Affibody-3

Affibody-3 is a pan-Nordic clinical study led from Uppsala University Hospital. In total, 120 women with breast cancer from 7-8 hospitals in Sweden, Denmark and Finland will be included in the study. Eligible patients should have HER2 positive or borderline HER2 positive, breast cancer with proliferation in the body or only in the breast. The purpose is to find out if the patients have HER2-positive tumors with the aid of a radioactively labeled tracer, ABY-025, and PET camera, something which is of great importance for the correct choice of treatment.

About ABY-025

The PET imaging agent ABY-025 is based on an Affibody molecule that binds strongly to HER2. The high affinity and rapid clearance of ABY-025 from blood and normal organs allows HER2 assessment within hours.

On September 24, 2018 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported results for its investigational medicine entrectinib, from an integrated analysis of the pivotal Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA trials, which showed that entrectinib shrank tumors (objective response rate; ORR) in 77.4 percent of people with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) (Press release, Genentech, SEP 24, 2018, View Source [SID1234529540]). In addition, entrectinib demonstrated a durable response of more than two years (duration of response [DoR] = 24.6 months). Importantly, entrectinib was shown to shrink tumors in more than half of people with cancer in the central nervous system (CNS) (intracranial ORR: 55 percent). The safety profile of entrectinib was consistent with that seen in previous analyses, and no new safety signals were identified. Based on the integrated analysis of these studies, Genentech plans to submit these data to global health authorities.

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"These results show the potential of precision medicines to deliver tailored and effective treatment options for people with non-small cell lung cancer, including those whose tumors have spread to the central nervous system," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "We are also investigating entrectinib in NTRK fusion-positive tumors across several different cancer types, and look forward to presenting those results in the near future."

ROS1 gene fusions have been identified in 1-2 percent of people with NSCLC. NSCLC is the most common type of lung cancer and accounts for 85 percent of all lung cancer diagnoses. Approximately 30-40 percent of people with ROS1-positive NSCLC have brain metastases at time of diagnosis.

These ROS1 results will be presented at the 2018 World Conference on Lung Cancer (WCLC) in Toronto, Canada on Monday, September 24 from 10:30 – 10:40 a.m. EDT (Abstract 13903), and featured in the WCLC press program on Monday, September 24 from 9:45 – 10:30 a.m. EDT. Follow Genentech on Twitter via @Genentech and keep up to date with WCLC 2018 congress news and updates by using the hashtag #WCLC2018.

About the integrated analysis

The integrated analysis included data from 53 people with ROS1-activating gene fusions from the Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA trials. The studies enrolled people across 15 countries and more than 150 clinical trial sites.

STARTRK-2 is a Phase II, global, multicenter open-label basket study in people with solid tumors that harbor an NTRK1/2/3 or ROS1 gene fusion. The primary endpoint is ORR. Secondary outcome measures include DoR, time to response, clinical benefit rate, intracranial tumor response, progression-free survival (PFS), CNS PFS and overall survival (OS).
STARTRK-1 is a Phase I, multicenter, open-label dose escalation study of a daily continuous dosing schedule in people with solid tumors with NTRK1/2/3 or ROS1 gene fusions in the U.S. and South Korea. The trial assessed the safety and tolerability of entrectinib via a standard dose escalation scheme and determined the recommended Phase II dose.
ALKA is a Phase I, multicenter, open-label dose escalation study of an intermittent and continuous entrectinib dosing schedule in people with advanced or metastatic solid tumors with ROS1 gene fusions in Italy.
Adverse events were consistent with previous data. The most commonly reported adverse events include those affecting the nervous system, as well as constipation, altered sense of taste (dysgeusia), and fatigue.

About entrectinib

Entrectinib (RXDX-101) is an investigational, oral medicine in development for the treatment of locally advanced or metastatic solid tumors that harbor NTRK1/2/3 or ROS1 gene fusions. It is a selective tyrosine kinase inhibitor designed to inhibit the kinase activity of the TRKA/B/C and ROS1 proteins, whose activating fusions drive proliferation in certain types of cancer. Entrectinib can block ROS1 and NTRK kinase activity and may result in the death of cancer cells with ROS1 or NTRK gene fusions. Entrectinib is being investigated across a range of solid tumor types, including non-small cell lung cancer, pancreatic cancer, sarcomas, thyroid cancer, salivary cancer, gastrointestinal stromal tumors (GIST) and cancers of unknown primary (CUP).

About lung cancer

According to the American Cancer Society, it is estimated that more than 234,000 Americans will be diagnosed with lung cancer in 2018, and NSCLC accounts for 85 percent of all lung cancers. It is estimated that approximately 60 percent of lung cancer diagnoses in the United States are made when the disease is in the advanced stages. While the ROS1 gene fusion can be found in any person with NSCLC, young never-smokers have the highest incidence of ROS1-positive NSCLC.

About Genentech in Lung Cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have four approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.