On December 15, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported that updated clinical data from an ongoing Phase 1A/1B trial of tislelizumab, an investigational anti-PD-1 antibody, were presented in an oral session and a poster at the European Society for Medical Oncology Immuno-Oncology (ESMO-IO) Congress, being held December 13-16 in Geneva, Switzerland (Press release, BeiGene, DEC 15, 2018, View Source;p=RssLanding&cat=news&id=2380786 [SID1234532073]).

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"We continue to be pleased with the results of tislelizumab in solid tumors," commented Amy Peterson, M.D., Chief Medical Officer, Immuno-Oncology, at BeiGene. "We believe that these updated results provide support for the continued development of tislelizumab in patients with bladder, esophageal, stomach, liver and non-small cell lung cancers, and we have registration-enabling studies ongoing or planned to start soon in each of these indications."

Summary of ESMO (Free ESMO Whitepaper)-IO Presentations from the Ongoing Phase 1A/1B Trial
The multi-center, open-label Phase 1A/1B trial (NCT02407990) of tislelizumab as monotherapy in advanced solid tumors is being conducted in Australia, New Zealand, the United States, Taiwan and South Korea and consists of dose-escalation and dose-expansion phases in disease-specific cohorts.

Updated Results in Patients with Urothelial Carcinoma (UC)
Data presented at ESMO (Free ESMO Whitepaper)-IO included updated results from an analysis of tislelizumab in 17 patients with UC. At the time of the data cutoff on August 31, 2018, median treatment duration was 4.1 months (0.7-30.4 months), with two patients still on treatment.

Treatment-related adverse events (TRAEs) as assessed by the investigator occurred in 15 patients (88.2%). Of those, fatigue (n=5), infusion-related reactions (n=3), rash (n=3), nausea (n=2), pain in extremity (n=2), peripheral adema (n=2), and proteinuria (n=2) occurred in two or more patients. Three treatment-related Grade 3 or 4 AEs occurred in two patients, fatigue (n=1), and hyperglycemia and latent autoimmune diabetes (n=1). One patient discontinued treatment due to recurrent infusion-related reactions considered related to tislelizumab.

At the time of the data cutoff, all 17 patients were evaluable for response, defined as having a baseline tumor assessment with at least one post-baseline tumor response assessment, or progression or death. The confirmed response rate was 29.4 percent, with one complete response (CR) and four partial responses (PR). Three additional patients achieved stable disease (SD) as their best response. There was one CR, one PR and one SD among the eight patients with PD-L1 high tumors and two PRs and two SDs among the eight patients with PD-L1 low or negative tumors (one tumor was not-evaluable for PD-L1 expression). The median duration of response was 18.7 months (6.2-18.7 months).

Updated Results in Patients with Esophogeal, Gastric, Hepatocellular and Non-Small Cell Lung Cancers
In an oral presentation at ESMO (Free ESMO Whitepaper)-IO, data on patients with esophageal (EC, n=54), gastric (GC, n=54), hepatocellular (HC, n=50) and non-small cell lung cancers (NSCLC, n=49) were reported.

TRAEs occurring in at least five percent of patients across all cohorts included fatigue (8.7%), pruritis (7.7%), hypothyroidism (7.2%), decreased appetite (6.8%), rash (6.8%) and nausea (6.3%). Ten patients experienced one or more serious adverse events considered related to tislelizumab, including pneumonitis (n=3) and one case each of acute hepatitis, dermatitis, diarrhea, increased ALT, increased AST, infusion-related reaction, pyrexia and vomiting. Grade 3 or 4 TRAEs occurring in more than one patient included increased AST (n=4), increased ALT (n=3) and pneumonitis (n=2). There were two fatal TRAEs reported, including acute hepatitis in a patient with HCC confounded by rapidly progressive disease, and pneumonitis in a patient with NSCLC with compromised pulmonary capacity at baseline.

Confirmed response rates and disease control rates in patients with EC were 11.1 percent and 37.0 percent, respectively; 13.0 percent and 29.6 percent in patients with GC, respectively; 12.2 percent and 51.0 percent in patients with HCC, respectively, and 13.0 percent and 63.0 percent in patients with NSCLC, respectively. For patients with EC and NSCLC, the median duration of response (mDOR) had not been reached. The mDOR in patients with GC was 8.5 months and for patients with HCC it was 15.7 months.

About Tislelizumab
Tislelizumab (BGB-A317) is an investigational humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells.

Discovered by BeiGene scientists, tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers. The new drug application (NDA) for tislelizumab in China for patients with relapsed/refractory (R/R) classical Hodgkin’s lymphoma (cHL) has been accepted by the China National Medical Products Administration (NMPA, formerly known as CFDA) and granted priority review status. BeiGene and Celgene Corporation have a global strategic collaboration for the development of tislelizumab in solid tumors in the United States, Europe, Japan and the rest of world outside Asia.

On December 15, 2018 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported its results indicating that tab-cel (tabelecleucel) was generally well tolerated with responses for patients with Epstein-Barr virus-associated leiomyosarcoma (EBV+ LMS) (Press release, Atara Biotherapeutics, DEC 15, 2018, View Source [SID1234532076]). EBV+ LMS is a rare soft tissue sarcoma that occurs in transplant and immunosuppressed patients and is typically an aggressive radiation- and chemotherapy-resistant disease with poor patient outcomes. The results were presented in an oral session at the European Society for Medical Oncology Immuno-Oncology (ESMO I‑O) Congress 2018 taking place in Geneva, Switzerland.

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"The EBV+ LMS results presented at ESMO (Free ESMO Whitepaper) I-O are the second example, along with nasopharyngeal carcinoma (NPC), of a difficult-to-treat, EBV-associated solid tumor with encouraging responses to tab‑cel," said Dietmar Berger, M.D., Ph.D., Global Head of Research and Development of Atara Biotherapeutics. "Observations of responses based on standard-CT and metabolic PET-CT imaging, in the context of prolonged survival, further highlight the opportunity for tab‑cel and off-the-shelf, allogeneic T-cell immunotherapy in EBV-associated cancers beyond our ongoing studies for patients with post‑transplant lymphoproliferative disease (PTLD) and NPC."

The oral presentation summarized the evaluation of tab-cel in an analysis of EBV+ LMS patients from three clinical studies, 2 single-center, open-label studies (NCT00002663, NCT01498484) and the multi‑center expanded access protocol (EAP) study (NCT02822495). Twelve patients with EBV+ LMS received one or more doses of tab-cel, of whom 10 were assessed for responses with two patients not evaluable. Two of the 10 patients achieved a partial response via CT-based RECIST 1.1 criteria and eight patients achieved stable disease. In the two single-center studies with longer follow-up, six of eight patients survived more than 27 months and the estimated median survival was 77.4 months. At the time of this analysis, responses assessed by PET-CT imaging were available from the multi-center EAP study where 3 of the 4 patients achieved a metabolic response. Tab-cel was generally well tolerated and the safety appeared consistent with a favorable risk profile and previous clinical studies.

About tab-cel (tabelecleucel)
Atara’s most advanced T-cell immunotherapy in development, tab-cel, is a potential treatment for patients with Epstein-Barr virus (EBV)-associated post-transplant lymphoproliferative disorder (EBV+ PTLD) who have failed rituximab, as well as other EBV-associated hematologic and solid tumors, including nasopharyngeal carcinoma (NPC). In February 2015, the FDA granted tab-cel Breakthrough Therapy Designation for EBV+ PTLD following allogeneic hematopoietic cell transplant (HCT), and in October 2016, tab-cel was accepted into the EMA Priority Medicines (PRIME) regulatory pathway for the same indication, providing enhanced regulatory support. In addition, tab-cel has orphan status in the U.S. and EU. Tab-cel is in Phase 3 clinical development for the treatment of EBV+ PTLD following an allogeneic hematopoietic cell transplant (MATCH study) or solid organ transplant (ALLELE study), and Atara recently initiated a Phase 1/2 study in NPC.

On December 14, 2018 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that it exercised, and closed on its exclusive option to acquire Potenza Therapeutics, Inc. ("Potenza") on December 13, 2018, U.S. Eastern Time (Press release, Astellas Pharma, DEC 14, 2018, View Source [SID1234532074]). This acquisition marks the successful outcome of a collaboration agreement entered into in 2015 to build a portfolio of novel immuno-oncology (IO) therapies. The clinical IO therapies developed through this collaboration may also provide a platform for IO combinations with Astellas’ existing non-IO programs for life cycle management and future novel IO combinations.

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"This transaction underscores Astellas’ commitment to innovation and scientific partnerships to advance our creation of value for patients," said Kenji Yasukawa, Ph.D., President and CEO, Astellas. "In oncology, Astellas has focused on developing treatments for unmet medical needs with novel mechanisms of action and modalities. We believe the novel assets we have developed with Potenza have the potential to make an even more pronounced difference for patients in need."

Through the research and development collaboration over the past three and a half years, Astellas and Potenza have discovered and developed three novel investigational new drugs (INDs) with the potential to treat various cancers that are non-responsive or resistant to the current generation of IO therapies. This portfolio of programs targeting immune stimulation, immune checkpoint inhibition, and regulatory T cell function includes:

ASP8374/PTZ-201, an anti-TIGIT antibody (immune checkpoint inhibitor) and ASP1948/PTZ-329, an anti-NRP1 antibody (Treg function inhibitor), both of which are currently in Phase 1 clinical studies;
ASP1951/PTZ-522, a novel format GITR agonistic antibody (T cell priming & costimulation), which has recently achieved IND clearance.
Upon the closing of this transaction, Potenza became a wholly-owned subsidiary of Astellas, establishing a competitive and fully owned clinical IO pipeline.

"We are extremely proud of what the experienced Potenza team has accomplished to discover and create innovative therapeutics for the treatment of cancers," said Dan Hicklin, Ph.D., President and CEO, Potenza. "Over the past three and a half years, we have enjoyed a successful and productive partnership with Astellas. I am pleased that these therapies will now have access to the resources of a large international company, with world-class R&D and the strategic and financial backing to support the development of these innovative potential new medicines for cancer patients in need."

By exercising its options under the Warrant Purchase Agreement, Astellas paid an upfront fee of $164.6 million to acquire Potenza and Potenza’s shareholders will be eligible for additional payments that total up to $240.1 million, depending on the progress of various programs in clinical development.

Astellas is reviewing the impact of the acquisition on its financial result for the fiscal year ending March 31, 2019.

Acquisition Summary

Acquiring company: Astellas Pharma Inc.
Major shareholders of Potenza Therapeutics:
MPM Capital, InterWest Partners, Astellas Pharma Inc., Founders and others (including stock options)
Payment: Cash on hand
Amount:
$164.6 million to make Potenza a wholly-owned subsidiary of Astellas
Up to $240.1 million in future contingent payments based on the advances in clinical programs
Overview of Acquired Company

Corporate Name: Potenza Therapeutics, Inc.
Location: Cambridge, MA
Representative: President and CEO Dan J. Hicklin, Ph.D.
Founded year: 2014
Number of employees: 19
Relationship with Astellas: Equity-method affiliate, research and development partner

On December 14, 2018 RadioMedix Inc reported that it has been selected by the National Cancer Institute (NCI) Small Business Innovation Research Development Center (SBIR) to participate in 2018 Bio Investor Forum (Press release, RadioMedix, DEC 14, 2018, View Source [SID1234532075]). The company received federal funds from the National Cancer Institute, National Institutes of Health, Department of Health and Human Services (HHSN261201600015C, HHS261201800048C/75N91018C00048) for the development of radiotherapeutic alpha-emitter labeled agents.

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Dr. Izabela Tworowska, Chief Science Officer of RadioMedix, presented the corporate overview of the company pipeline at the 2018 Bio Investor Forum held in San Francisco, California. The presentation was sponsored by NCI SBIR Development Center’s Investor Initiatives program. Dr. Tworowska provided an overview of the research and financial growth of the RadioMedix, discussed the company pipeline and the status of the clinical development of radiotheranostic drugs.

"We have been invited to attend the Bio Investor Forum in San Francisco for two consecutive years and we are grateful the NCI SBIR Investor Initiatives for this invitation and also for the feedback on our presentation", said Dr. Tworowska. "It is a great opportunity to attend One-on-One Partnering meetings and gain the interest of the pharmaceutical companies and investors", added Dr. Tworowska.

"RadioMedix’s rich clinical stage pipeline of innovative radiopharmaceuticals makes an attractive case for investors, and large companies, familiar with our space", said Dr. Delpassand, Chairman and CEO of RadioMedix. "Targeted radioligand therapy will be the strongest segment in the field of nuclear medicine and RadioMedix is well-positioned to take advantage of this opportunity", added Dr. Delpassand.

On December 14, 2018 RadioMedix Inc reported that it has been selected by the National Cancer Institute (NCI) Small Business Innovation Research Development Center (SBIR) to participate in 2018 BIO Investor Forum (Press release, RadioMedix, DEC 14, 2018, View Source [SID1234532131]). The company received federal funds from the National Cancer Institute, National Institutes of Health, Department of Health and Human Services (HHSN261201600015C, HHS261201800048C/75N91018C00048) for the development of radiotherapeutic alpha-emitter labeled agents.

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Dr. Izabela Tworowska, Chief Science Officer of RadioMedix, presented an overview of the company pipeline, the financial growth of RadioMedix, and status of the clinical development of radiotheranostic drugs. The presentation was sponsored by NCI SBIR Development Center’s Investor Initiatives program.

"We have been invited to attend the BIO Investor Forum in San Francisco for two consecutive years and we are grateful to the NCI SBIR Investor Initiatives for this invitation ", said Dr. Tworowska. "It is a great opportunity to attend BIO One-on-One Partnering meetings, discuss the future goals of our company and gain the interest of investors and business partners", added Dr. Tworowska.

"RadioMedix’s rich clinical stage pipeline of innovative radiopharmaceuticals makes an attractive case for investors, and large companies, familiar with our space", said Dr. Delpassand, Chairman and CEO of RadioMedix. "Targeted radioligand therapy will be the strongest segment in the field of nuclear medicine and RadioMedix is well-positioned to take advantage of this opportunity", added Dr. Delpassand.