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 Transgene (Paris:TNG) (Euronext Paris: TNG) a biotechnology company that designs and develops virus-based immunotherapies against cancers and infectious diseases, reported myvacTM, an individualized, viral vector-based immunotherapy against cancer that will enter clinical development in 2019 (Press release, Transgene, SEP 24, 2018, View Source [SID1234621824]).

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myvacTM, an individualized, MVA-based immunotherapy
myvacTM is designed to stimulate and educate the immune system of patients to recognize and destroy tumor cells. The personalized immunotherapy product is based on the mutations that are identified in the patient’s own tumor. These mutations are highly relevant targets since they lead to the expression of tumor neoantigens which are known to trigger a stronger immune response than "classic"2 tumor antigens.

Once administered to the patient, myvacTM triggers a cascade of immune responses against a variety of targets found in the cancer cells.

The neoantigens which are the basis for the myvacTM approach are identified by sequencing and selected using artificial intelligence algorithms, and then integrated into the genome of the viral vector (MVA).

myvacTM differs from autologous treatments since no biological material from the patient is used in the manufacturing process and as such is much easier to manufacture; it is a truly individualized approach that uses the information that is specific to the characteristics of each patient’s tumor.

Transgene has combined its expertise in viral vectors with highly innovative technologies to develop myvacTM.

myvacTM features several key advantages:

It is expected to deliver the benefits of an individualized treatment without the disadvantages of autologous approaches (Transgene does not modify the patient’s cells but integrates the neoantigen panel into the virus);
It is based on a viral strain (MVA) whose safety, tolerability, immunogenicity and efficacy have already been demonstrated in the clinic with TG4010 and TG4001;
The myvacTM viral vector (MVA) has repeatedly shown that it can induce a strong immune response from the patient against the tumor antigens incorporated in its viral genome as well as an enlargement of the antitumoral immune repertoire (epitope spreading);
An "all-in-one" formula, requiring neither adjuvant nor association of different peptides.
Éric Quéméneur, PhD, Executive VP, Chief Scientific Officer of Transgene, said: "With myvacTM, Transgene is at the forefront of innovation in cancer immunotherapy. Based on our know-how in virotherapy, we have successfully integrated sequences coding for neoantigens to create an individualized immunotherapy. By combining sequencing and artificial intelligence with the design of the virus, myvacTM marks the entry of viral vector-based approaches in the era of digital transformation. Importantly we have also set up an organization able to design and manufacture myvacTM for each patient in a timely and cost-competitive manner. The myvacTM innovation is a logical evolution of our expertise and a new therapeutic option that promises a major improvement over existing therapies. myvacTM is also the result of our policy of open innovation which is based on working with partners developing technologies that are complementary to our expertise allowing us to benefit from a multidisciplinary approach. We look forward to demonstrating the transformative potential of myvacTM in the clinical trials we plan to start in 2019."

myvacTM, an ambitious project expected to enter the clinic in 2019
myvacTM will be administered to patients with solid tumors. Two clinical trials are being set up in Europe and in the United States, including HPV-negative head and neck cancers and ovarian cancer. These trials are expected to start in 2019.
The first preclinical and translational results will be presented soon at immuno-oncology conferences.

Our innovative network combines bioengineering and digital transformation

To design myvacTM, Transgene and its collaborative network had to overcome many scientific and technical challenges. The network’s expertise covers all the required know-how:

Institut Curie (Cancer Immunotherapy Center, led by Professor Amigorena) is involved in the generation of translational data and the characterization of the mechanism of action;
HalioDx studies biomarkers to maximize the effectiveness of the therapy;
Traaser automates, secures and manages genomic data, including predictive algorithms provided by a recognized partner in artificial intelligence;
Transgene has developed a unique manufacturing pilot unit in France to vectorize neoantigens and provide myvacTM within a timeframe compatible with clinical treatment schemes.
This innovative project has obtained the labeling of the Biovalley France and Eurobiomed French competitiveness clusters.

Transgene holds the intellectual property of the myvacTM viral vector platform and is actively working on the translational development of this innovative technology.

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.