On October 7, 2019 SHINE Medical Technologies LLC reported the closing of a $50-million financing with funds managed by Oaktree Capital Management L.P. ("Oaktree"), a leading global investment firm with more than $120 billion under management as of June 30, 2019 (Press release, Shine Medical Technologies, OCT 7, 2019, View Source;pk_kwd=shine-closes-50-million-financing [SID1234540975]). The financing supports the ongoing construction of SHINE’s medical isotope production facility and its commercialization of diagnostic and therapeutic isotopes, including molybdenum-99 (Mo-99) and lutetium-177 (Lu-177).

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"We are excited to welcome Oaktree to SHINE’s growing list of top-tier institutional investors," said Greg Piefer, founder and CEO of SHINE. "Oaktree has broad expertise in complex infrastructure projects like our medical isotope facility. It joins Deerfield Management, a leading health care investment firm, as one of our key partners.

"Oaktree and Deerfield will help us deliver on SHINE’s value proposition, including the completion of our isotope production facility and the development of our therapeutics business. Their participation validates the strength of our business case, team and vision for bringing our lifesaving products to market."

Construction of SHINE’s production facility is expected to be completed in 2021 and commercial-scale isotope production should begin in 2022. The facility will be the first of its kind, utilizing the company’s patented technology to produce Mo-99, which is used in more than 40 million procedures every year.

Chronic global shortages of medical isotopes routinely and significantly affect the diagnosis and treatment of patients around the world. SHINE’s production facility will be capable of supplying more than one-third of the global demand for Mo-99.

"SHINE is an outstanding company with a strong management team, exceptional technology and compelling story," said Milwood Hobbs Jr., managing director of Oaktree. "The need for SHINE’s medical isotope production facility is profound, as millions of patients are affected each year by the ongoing shortage of Mo-99. Oaktree is confident SHINE will play a major role in ending that shortage and we are pleased to support SHINE’s production facility construction."

SHINE also will commercialize Lu-177, a therapeutic isotope currently used to treat neuroendocrine cancers and showing promise for the treatment of metastatic prostate and other cancers. In May, the company entered into an agreement with the Institute of Organic Chemistry and Biochemistry of the CAS (IOCB Prague) that provides SHINE with a global, exclusive license to a novel separation technology that it will use to produce Lu-177. The technology enables SHINE to produce non-carrier-added Lu-177, which should provide the highest therapeutic efficacy.

"Oaktree is pleased to be a partner with SHINE as it executes a growth strategy that includes commercializing therapeutic isotopes and constructing a production facility in Europe," said Aman Kumar, senior vice president of Oaktree. "The global market for therapeutic isotopes, including Lu-177, continues to grow rapidly and SHINE is well positioned to capture significant value in that market. We are equally as excited by SHINE’s commitment to Europe, where the current isotope producers are expected to stop production during the next several years."

About Medical Isotopes
Medical isotopes are radioisotopes that are used in the diagnosis and treatment of disease. Molybdenum-99 (Mo-99) is a radioisotope that decays into the diagnostic imaging agent technetium 99m (Tc-99m). The workhorse of nuclear medicine, Tc-99m is used in more than 40 million medical imaging procedures each year, primarily in stress tests to diagnose heart disease and to stage cases of cancer. SHINE was founded to deploy a safe, cost-effective and environmentally friendly technology to produce a variety of medical isotopes, including Mo‑99. Roughly one percent of all Mo-99 in the world decays every hour, meaning it must be produced continuously. Current production is limited to only a handful of government-owned nuclear research reactors, the majority of which are overseas.

On October 7, 2019 Gilead Sciences, Inc. (NASDAQ: GILD) reported that Merdad Parsey, MD, PhD, will join the company as Chief Medical Officer, effective November 1 (Press release, Gilead Sciences, OCT 7, 2019, View Source [SID1234540090]). Dr. Parsey will be responsible for the company’s global clinical development and medical affairs organizations. He will join the company’s senior leadership team and will report directly to Daniel O’Day, Gilead’s Chairman and Chief Executive Officer. Under a new structure, William Lee, PhD, Gilead’s Executive Vice President, Research will separately report to Mr. O’Day.

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Dr. Parsey joins Gilead from Genentech, Inc., a member of the Roche Group, where he currently holds the position of Senior Vice President, Early Clinical Development in the Genentech Research and Early Development (gRED) group. In this role, he leads clinical development, quality, compliance, informatics and clinical operations functions. He brings to Gilead significant clinical expertise across a broad array of therapeutic areas, including inflammation, oncology and infectious diseases.

"I am delighted to welcome Merdad, a seasoned and highly respected scientist, clinician and leader, to Gilead," said Mr. O’Day. "Throughout his career, he has built a reputation as an outstanding leader among academic, industry and medical communities alike. I know Merdad’s exceptional skills and expertise will be of great benefit to Gilead as we focus on rapidly expanding our pipeline and clinical development portfolio through internal efforts and external partnerships."

Prior to his most recent tenure with Genentech, Dr. Parsey served as President and CEO of 3-V Biosciences. He has also held development roles at Sepracor, Regeneron and Merck, Inc. and has served as Assistant Professor of Medicine and Director of Critical Care Medicine at New York University School of Medicine. Dr. Parsey completed his MD and PhD at the University of Maryland, Baltimore, his residency in Internal Medicine at Stanford University and his fellowship in Pulmonary and Critical Care Medicine at the University of Colorado.

"I have had many opportunities over the course of my career to help shape clinical development strategies and programs, and I am profoundly excited to bring those experiences to Gilead, an organization I have long admired," said Dr. Parsey. "I am looking forward to working with the company’s talented teams to advance clinical development programs and build a robust pipeline that has the opportunity to change the trajectory of disease and transform the care of many more people in need around the world."

On October 7, 2019 ITM Isotopen Technologien München AG (ITM), a biotechnology and radiopharmaceutical group of companies, reported that ITM´s subsidiary, ITG Isotope Technologies Garching GmbH (ITG) and Nordic Nanovector ASA (OSE: NANO) have signed long-term global supply agreements for the medical radioisotope no-carrier-added Lutetium-177 (n.c.a. 177Lu) EndolucinBeta to support R&D, clinical and commercial supply of Betalutin (177Lu-Lilotomab-Satetraxetan) (Press release, ITM Isotopen Technologien Munchen, OCT 7, 2019, View Source [SID1234540091]).

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Under the terms of the agreement for the study phase, ITM has partnered with Nordic Nanovector to support its clinical development. At the same time, both parties signed a commercial agreement for the supply of EndolucinBeta post Marketing Approval of Betalutin. Additional terms of the agreement are not disclosed.

EndolucinBeta, radiolabeled to anti-CD37 murine antibody lilotomab, is an active component of Betalutin, a next generation radioimmuno conjugate (RIC) currently under clinical development in patients who suffer from Non-Hodgkin Lymphoma (NHL). Betalutin is a drug candidate with an excellent profile. As well as leveraging an alternative therapeutic target (anti-CD37 antigen) in recurrent lymphoma patients who have relapsed following anti-CD20-based therapy, it has shown durable responses in heavily pre-treated NHL patients after a one-time administration, combined with a predictable and manageable toxicity, an important feature for elderly NHL patients who may not be suited to chemotherapy.

EndolucinBeta, a radiopharmaceutical precursor, is used in Targeted Radionuclide Therapy in the field of Precision Oncology and has marketing authorization in the EU. Radiolabeled to disease-specific targeting molecules like antibodies or peptides, the tumor tissue is precisely destroyed by cytotoxic doses of ionizing radiation. ITM has developed a unique methodology to produce a particularly highly pure form of Lutetium-177. No-carrier-added Lutetium-177 contains no metastable Lutetium-177m, therefore there is no need for cost intensive clinical waste management. This is especially important in countries with a release limit of Lutetium-177m into public sewage systems.

Marco Renoldi, Chief Operating Officer at Nordic Nanovector, said: "We are pleased to extend our collaboration with a long-standing and reliable partner such as ITM. This global supply agreement is a key milestone in the implementation of our CMC (Chemistry, Manufacturing and Controls) strategy for gaining regulatory approval for Betalutin and its subsequent commercial rollout, as it provides certainty of continued supply of n.c.a. Lutetium-177 throughout clinical development as well as after launch. The agreement with ITM, alongside other manufacturing supply and development agreements in place with specialist manufacturers at all stages of the manufacturing and supply chain for Betalutin strengthens our confidence in the ability to deliver a reliable and sustainable supply chain in support of the launch of our lead asset."

Steffen Schuster, CEO of ITM, added: "Nordic Nanovector is one of our longstanding partners, as ITM has been supplying n.c.a. Lutetium-177 to Nordic Nanovector since 2010. We are delighted that Nordic Nanovector has reaffirmed their confidence through this long term supply agreement for EndolucinBeta. In addition to the development of our own pipeline, we have once again been able to gain a strategic partner for the development of targeted radiopharmaceuticals in Precision Oncology, thereby making a significant contribution to advancing a promising treatment option for difficult-to-treat cancers. With our manufacturing facilities around the world and our unrivaled logistics network, we feel well equipped to reliably meet our partners’ needs and to enter into further strategic relationships."

On October 7, 2019 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported the publication of an expert panel consensus statement that includes appropriate use criteria for DecisionDx-Melanoma in patients with cutaneous melanoma (Press release, Castle Biosciences, OCT 7, 2019, View Source [SID1234540092]). The study was published in the peer-reviewed journal SKIN: The Journal of Cutaneous Medicine.

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A panel of nine expert dermatologists, dermatologic surgeons, and dermatopathologists performed a systematic review of published evidence and developed appropriate use criteria supporting the integration of DecisionDx-Melanoma and two other validated, widely used molecular tests in melanoma patient care. The systematic review of peer-reviewed medical literature identified studies relating to clinical validity, outcomes or utility of DecisionDx-Melanoma. Employing a modified Delphi consensus technique, the panel determined the level of evidence for publications as well as an overall strength of recommendation for selected indications using standard Strength of Recommendation Taxonomy (SORT) methodology.

Key Findings

Eleven publications were identified as achieving quality levels 1 or 2, with two publications achieving the highest level of evidence (Level 1).
Eight consensus-based appropriate use criteria recommendations achieved a rating of "A" or "B", with "A" rating indicating the highest strength of recommendation.
The expert consensus-based appropriate use criteria provide an evidence-based framework to integrate the DecisionDx-Melanoma test into the management of patients with cutaneous melanoma.
"This important publication is the first to provide expert consensus-based appropriate use criteria to help clinicians integrate technologies like the DecisionDx-Melanoma test into their melanoma practice," said study co-author Darrell S. Rigel, M.D., M.S., Clinical Professor at New York University (NYU) School of Medicine. "The DecisionDx-Melanoma test has been well validated as a predictor of risk of metastasis or recurrence. The appropriate use criteria recommendations align with the current clinical use of DecisionDx-Melanoma to guide the post-diagnostic decisions to perform a sentinel lymph node biopsy surgical procedure in certain patients, and determine appropriate management plans regarding follow-up regimens, inclusion of imaging and referral to oncology."

The full published study can be accessed at the journal’s website.

On November 7, 2019 BridgeBio Pharma, Inc. (NASDAQ: BBIO) reported financial results for the third quarter, which ended September 30, 2019, and provided recent highlights across the company’s research and development portfolio (Press release, BridgeBio, NOV 7, 2019, View Source [SID1234552609]).

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"We continue to make progress towards achieving our goals, which ultimately is the delivery of meaningful medicines to patients with genetic diseases," said Neil Kumar, Ph.D., founder and chief executive officer of BridgeBio. "We will present new data from our TTR stabilizer program this month at the American Heart Association (AHA). We are also on track to begin filing this year our rolling new drug application for BBP-870, a treatment for the often-fatal genetic disease molybdenum cofactor deficiency type A. We are growing our pipeline as well, adding BBP-418, a novel treatment for the neuromuscular disorder limb-girdle muscular dystrophy type 2i. We remain on track to put five or more programs into the clinic next year on a risk-adjusted basis."

Recent Highlights:

BBP-831 – FGFR1-3 inhibitor for achondroplasia: Initiated PROPEL, a prospective observational study in children with achondroplasia, the most common genetic form of short stature (NCT04035811). The study will establish annualized growth velocity (AGV) for each child over a minimum period of six months. PROPEL is designed to provide baseline measurements for children who enroll in PROPEL2, a Phase 2 study of low-dose infigratinib in achondroplasia which is on track to start in 2020. Additionally, in October 2019, new preclinical data supporting tolerability and efficacy of infigratinib in the mouse model of achondroplasia were reported at the American Society of Human Genetics conference (link to poster).

BBP-870 – cPMP replacement therapy for MoCD type A: Presented natural history study data at the Society of Inborn Errors of Metabolism Conference (link to poster) in September. These data suggest an urgent need for new therapies in molybdenum cofactor deficiency (MoCD) type A, an often-fatal rare genetic disease, and will be a critical component of the planned new drug application.

BBP-812 – Gene therapy candidate for Canavan disease: Opened a natural history study in Canavan disease (treatcanavan.com). Presented preclinical data (link to poster) demonstrating intravenous (IV) dosing of BridgeBio’s experimental therapy for Canavan disease (BBP-812) achieved broad central nervous system delivery; the IV approach is much less invasive compared to intrathecal or intracerebroventricular alternatives.

BBP-631 Gene therapy candidate for CAH: Presented preclinical update (link to poster) for gene therapy candidate BBP-631 in congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency wherein IV dosing of non-human primates with BBP-631 resulted in durable delivery and expression of the gene product to the adrenal tissue.

BBP-398 – SHP2 inhibitor for treatment-resistant cancer: Presented data (link to poster) highlighting the discovery and preclinical efficacy of BBP-398, a potent and selective SHP2 inhibitor, which is currently being prepared for the submission of an IND in 2020 for evaluation in RTK-driven cancer.

BBP-265 (AG10) – TTR stabilizer for ATTR: Granted Alexion Pharmaceuticals, Inc. an exclusive license to develop and commercialize AG10 in Japan for an upfront payment of $25 million and an equity investment of $25 million.

Pipeline growth: Announced addition of a new asset, BBP-418, to the pipeline. BBP-418 is a substrate supplementation therapy for the treatment of limb-girdle muscular dystrophy type 2i and is in IND-enabling studies. This condition affects an estimated 7,000 patients in the United States and European Union with no currently approved therapies.

Organizational growth: Added two new members to the senior leadership team. Brian Stolz joined as Chief Operating Officer of BridgeBio. Mr. Stolz most recently held the position of Chief People Officer at Activision Blizzard. Yi Ching Yau joined as Chief Accounting Officer of BridgeBio. Prior to joining BridgeBio, Ms. Yau served as the VP of Finance at Nektar Therapeutics.

Upcoming Milestones:

BBP-265 (AG10) – TTR stabilizer for ATTR: Plan to present interim analysis of the ongoing Phase 2 open label extension study (NCT03536767) of AG10 in patients with TTR amyloid cardiomyopathy, an inherited form of heart failure, at the American Heart Association 2019 Scientific Sessions in a Late-Breaking Featured Science Oral Presentation. A Phase 3 study of AG10 in ATTR-PN (ATTRibute-PN) is on track to begin in the first quarter of 2020.

BBP-870 – cPMP replacement therapy for MoCD type A: On track to initiate a rolling new drug application submission for our first-in-class therapy for molybdenum cofactor deficiency (MoCD) type A, BBP-870, by the end of 2019.

BBP-589 – COL7A protein replacement therapy for recessive dystrophic epidermolysis bullosa: Plan to share topline data from the ongoing Phase 1/2 study (NCT03752905) during 2020.

BBP-831 (infigratinib) – FGFR1-3 inhibitor for FGFR2+ cholangiocarcinoma: Plan to complete enrollment of the ongoing pivotal Phase 2 study (NCT02150967) in second line cholangiocarcinoma (bile duct cancer) and present updated results at a major oncology meeting in 2020. Remain on track to submit new drug application for FDA approval in 2020.

Consolidated cash, cash equivalents and marketable securities, excluding restricted cash, totaled $611.9 million as of September 30, 2019. Excluding Eidos, BridgeBio’s cash balance as of September 30, 2019 was $446.1 million compared to $162.4 million as of June 30, 2019. The net change in cash balance of $283.7 million reflects net proceeds received from BridgeBio’s initial public offering of $368.7 million, offset by the repurchase of a non-controlling interest for $26.4 million and approximately $58.6 million primarily for operating expenses.

Operating Expenses

Operating expenses for the three months that ended September 30, 2019 were $81.3 million, as compared to $41.5 million for the same period in the prior year. The increase in operating expenses of approximately $39.8 million was mainly attributable to increased research and development expenses related to the progression of our programs