On September 30, 2020 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company developing a new class of potent and selective precision oncology medicines, which it refers to as Pentarin miniature drug conjugates, reported the appointment of Brian Roberts as President and Chief Executive Officer effective September 30, 2020 (Press release, Tarveda Therapeutics, SEP 30, 2020, View Source [SID1234567826]). Mr. Roberts will succeed Drew Fromkin, who has resigned from his roles as Chief Executive Officer and Chairman of the Board to pursue other interests and will transition to an advisory role.

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"Brian has been instrumental in the growth and corporate development of Tarveda in his roles as President and Chief Financial Officer over the last several years, and brings the experience and skill needed to propel Tarveda forward," said Guido Magni, M.D., Ph.D., Partner at Versant Ventures. "On behalf of the Tarveda Board of Directors, I would like to express our deep thanks to Drew for his vision, leadership and years of service as President, Chief Executive Officer and Chairman. We appreciate his many contributions to Tarveda and wish him well in his new endeavors."

"I am grateful to the Tarveda Board and our investors for their ongoing support during this stage in the company’s evolution," said Mr. Roberts. "We have an exceptional team at Tarveda, and I am thrilled to continue to work with them in this new capacity, as we execute on our strategy to advance the company, our clinical programs and platform capabilities. This is an exciting time as both of our clinical programs, PEN-221 and PEN-866, rapidly progress towards data points over the coming quarters and as we continue to advance additional miniature drug conjugates from our HSP90 platform towards the clinic."

Mr. Roberts served as Chief Financial Officer of Tarveda since joining the company in January 2018, and in March 2020 was appointed to President and Chief Financial Officer. Prior to joining Tarveda, Mr. Roberts served as Chief Financial and Operating Officer at Avedro, Inc. During his tenure, he acted in the capacity of interim Chief Executive Officer, leading the company through FDA approval, manufacturing readiness and commercial launch of its lead combination drug and device system. Previously, he served as Chief Financial Officer at Insulet Corporation. During his tenure, Insulet grew rapidly from approximately $30 million to nearly $300 million in revenue, increased its market capitalization to over $2 billion and achieved operating profitability. Prior to Insulet, Mr. Roberts served as Chief Financial Officer for Jingle Networks, which was acquired by Marchex, Inc. Mr. Roberts also served as Chief Financial Officer at Digitas, where he was instrumental in building the organization to nearly $400 million in revenue, resulting in the successful sale of the company for $1.3 billion to Publicis Groupe. Mr. Roberts also held finance positions at Idiom Technologies, Inc., the Monitor Group and served as an auditor with Ernst & Young LLP. Mr. Roberts holds a Bachelor of Science in accounting and finance from Boston College and is a certified public accountant.

On September 30, 2020 Ultimovacs ASA ("Ultimovacs", ticker ULTIMO), reported positive topline results from the first cohort of 20 patients in its ongoing US-based Phase I clinical trial evaluating the Company’s lead candidate, UV1, in combination with PD-1 checkpoint inhibitor, pembrolizumab, as a first line treatment in patients with metastatic malignant melanoma (Press release, Ultimovacs, SEP 30, 2020, View Source [SID1234567825]). The results confirm achievement of the primary endpoints of safety and tolerability and indicate initial signs of clinical response.

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As per the cut-off date of September 30, 2020, every patient in the first cohort reached at least 12-months of follow-up post treatment with UV1 and pembrolizumab. At the one-year landmark, the overall survival (OS) rate was 85%. Median Progression-Free Survival (mPFS) was not reached at 12 months, indicating that more than half of the participating patients did not demonstrate disease progression. None of the patients experienced unexpected safety issues related to UV1 and the vaccine was well-tolerated. The safety events observed are in line with the established data on UV1 and pembrolizumab.

"The safety profile observed to date in this first cohort of the PD-L1 combination study is consistent with the promising safety profiles seen in our earlier Phase I trials. For the further development of our vaccine, it is important to demonstrate that UV1 can be combined with different classes of immunotherapies without compromising on safety," commented Jens Bjørheim, Chief Medical Officer at Ultimovacs. "From our perspective, an appropriate historical comparison for our trial is the cohort in the KEYNOTE006 trial in which patients with advanced melanoma without prior treatment history were treated with pembrolizumab only. Results from that study demonstrated a 68% OS and a mPFS of 11.6 months. Comparing this to our data presented today, we are encouraged by the results and look forward to additional data readouts over the next years that will demonstrate if the initial signs of efficacy in this cohort persist and mature into long-term added clinical benefit for the patients."

The Phase I trial in malignant melanoma is evaluating the safety, tolerability and initial signs of clinical response in patients treated with UV1 in combination with pembrolizumab. Pembrolizumab improves the ability of immune cells to kill tumor cells and is a current standard-of-care therapy for malignant melanoma. The 20 patients in the first cohort had no prior treatment history and received a 37.5 µg GM-CSF adjuvant dose per UV1 vaccination, combined to strengthen the ability of UV1 to stimulate the immune system. The study has completed full enrollment of 30 patients, as announced on August 18, 2020. The 10 patients in the second cohort have received the standard 75 µg GM-CSF adjuvant dose per UV1 vaccination. One-year of follow-up data on these patients will be available in the second half of 2021.

"In all four Phase I studies, UV1 has demonstrated a consistent and positive safety and tolerability profile. The topline results announced today complement our extensive and growing clinical data package, confirming that this strong safety profile holds when combining UV1 with a PD-1 checkpoint inhibitor," stated Carlos de Sousa, Chief Executive Officer at Ultimovacs. "As an update on other clinical development progress, we had announced in May this year a collaboration with a leading Big Pharma company and a European oncology clinical trial group to evaluate UV1 in an additional Phase II study, which we had planned to confirm with all details in the third quarter. Ultimovacs, the sponsor and the Big Pharma partner have been making improvements to the trial protocol and due to the challenges of operating in the current pandemic environment, the agreement is still in the process of finalization. We look forward to announcing the collaboration and trial timelines during the fourth quarter."

More complete data on the patients in the first cohort will be presented at an upcoming oncology conference in the first half of 2021.

About UV1 and the UV1 Phase II Clinical Trials

UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen, telomerase. UV1 is being developed as a therapeutic cancer vaccine which may serve as a platform for use in combination with other immuno-oncology drugs which require an ongoing T cell response for their mode of action. To date, UV1 has been tested in four Phase I clinical trials in a total of 82 patients and maintained a positive safety and tolerability profile as well as encouraging signals of efficacy.

UV1 is being tested in different randomized Phase II trials:

The INITIUM trial is an Ultimovacs-sponsored, global, randomized Phase II trial for patients with metastatic malignant melanoma. Patients will be treated with UV1 in combination with ipilimumab (CTLA-4 checkpoint inhibitor) and nivolumab (PD-1 checkpoint inhibitor). The trial will be conducted in the U.S. and Europe, including Norway. The first patient was dosed on June 15, 2020. A total of 154 patients will be enrolled in the study, 77 patients will receive nivolumab and ipilimumab and the second half of the patients will receive nivolumab, ipilimumab and UV1. The primary endpoint for the study is progression-free survival and the planned readout of the primary endpoint is in the second half of 2022.
The NIPU trial is a randomized, multi-center Phase II trial in which the universal cancer vaccine, UV1, is investigated in combination with the checkpoint inhibitors, ipilimumab and nivolumab, as second-line treatment in mesothelioma. Oslo University Hospital is the sponsor of the NIPU study. Bristol-Myers Squibb and Ultimovacs have entered into agreements with OUS to support the preparations and execution of the trial. The first patient was dosed on June 15, 2020. A total of 118 patients will be included in the NIPU study. Half of the patients will be treated with the combination of UV1, ipilimumab (CTLA-4 checkpoint inhibitor) and nivolumab (PD-1 checkpoint inhibitor), whereas the other half will receive nivolumab and ipilimumab only. The study is planned to be conducted at six national hospital centers specialized in treating mesothelioma in four countries (Norway, Sweden, Denmark and Australia). Planned readout of the primary endpoint progression-free survival is the second half of 2022.
A third Phase II clinical trial will evaluate UV1 in a new cancer indication in combination with indication-specific standard of care cancer therapies different from those to be tested in INITIUM (malignant melanoma, 154 patients) and NIPU (mesothelioma, 118 patients). In the collaboration, Ultimovacs will supply UV1 and a big pharma company will supply its proprietary cancer treatment to the clinical trial group which will sponsor the trial.

On September 30, 2020 TargetCancer Foundation, with support from Bayer and in partnership with Foundation Medicine, Inc. and investigators from leading academic institutions, reported the first patients enrolled in the TCF-001 TRACK (Target Rare Cancer Knowledge) Study (Press release, TargetCancer Foundation, SEP 30, 2020, View Source [SID1234567824]). TRACK aims to provide individualized treatment recommendations to patients with rare cancers, defined as cancers with an incidence of six per 100,000 people per year in the U.S., informed by genomic analysis and in consultation with field-leading rare cancer clinicians and researchers. An expert virtual molecular tumor board (VMTB) will share these recommendations with patients and their healthcare providers (HCPs), ensuring the widest range of options are considered. To learn more about TRACK and how to enroll, visit www.targetcancerfoundation.org/track.

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For people diagnosed with cancer, it is important that they discuss genomic testing with their HCPs. Genomic testing can identify DNA alterations, or changes, within cancer cells that determine how a tumor behaves or why it grows.1 The results of genomic testing may help HCPs match patients to approved or investigational therapies based on the specific alteration identified.

"It wasn’t until years after my initial diagnosis that I learned a genomic tumor test might help identify what was driving my cancer and which therapy would be appropriate for me," said Susan, thyroid cancer patient and advisor on the TRACK Patient and Caregiver Advisory Council. "Ultimately, the testing identified an NTRK gene fusion, a rare genomic driver of multiple tumor types. I’m hopeful TRACK will enable patients like me with rare cancers to gain access to testing and make better informed decisions throughout their treatment journey."

TRACK participants’ subsequent treatments and responses will be prospectively tracked for at least one year, observing participant outcomes based on molecularly-informed treatment decisions. Through a remote consenting process, participants can fully enroll in the study from home without travelling to a clinical trial site. Beyond potentially informing treatment options, the data produced through TRACK will also contribute to the field’s understanding of genomics in rare cancers, addressing a critical unmet need where such knowledge is otherwise lacking. Studies show rare cancers generally receive less scientific consideration and financial support than trials of more common tumors.2

As a company dedicated to new and innovative approaches to treat cancer and broader access to genomic testing, Bayer’s financial support of the TRACK Study is another key initiative it has undertaken along with various laboratory and patient advocacy groups to advance patient care.

"People with rare cancers deserve better informed, individualized and targeted treatment choices, and we look forward to having these patients enrolled in TRACK," said Jim Palma, Executive Director, TargetCancer Foundation. "We are making significant strides in shifting clinical practice norms by challenging traditional methods of clinical trial enrollment and introducing virtual care into the treatment continuum, and we are grateful for Bayer’s longstanding support of these efforts."

"Those with rare cancers are especially vulnerable to reductions in research and limited access to treatments, especially during these uncertain times," said Joseph Germino, M.D., Vice President of Medical Affairs, Oncology at Bayer. "Bayer’s recent initiatives, including the support of TRACK, encourage genomic testing and subsequent tailored treatment approaches for patients to ensure optimal care."

TRACK is sponsored and managed by TargetCancer Foundation, in partnership with Foundation Medicine. In addition, the research team for TRACK includes Principal Investigator Razelle Kurzrock, M.D. (University of California San Diego), as well as co-Principal Investigators Vivek Subbiah, M.D. (University of Texas MD Anderson Cancer Center), James Cleary, M.D., Ph.D. (Dana-Farber Cancer Institute), and Roman Groisberg, M.D. (Rutgers Cancer Institute of New Jersey).

About Oncology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. The oncology franchise at Bayer now expands to six marketed products and several other assets in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways with the potential to impact the way that cancer is treated.

On September 30, 2020 Lantheus Holdings, Inc. (the "Company") (NASDAQ: LNTH), the parent company of Lantheus Medical Imaging, Inc. and Progenics Pharmaceuticals, Inc., and a global leader in the development, manufacture and commercialization of innovative diagnostic and therapeutic agents and products, reported the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for PyL (18F-DCFPyL), a prostate specific membrane antigen (PSMA)-targeted positron emission tomography (PET) imaging agent for prostate cancer (Press release, Lantheus Medical Imaging, SEP 30, 2020, View Source [SID1234567823]). The NDA includes a request for Priority Review, which if granted, could shorten the FDA’s review of the NDA to six months from the time of acceptance, versus the standard review timeline of 10 months from acceptance. The Company expects to receive notification from the FDA confirming acceptance of the filing for substantive review in early December 2020.

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"The completion of our NDA submission marks a significant milestone for Lantheus and our PyL clinical development program," said Mary Anne Heino, President and Chief Executive Officer of Lantheus. "Prostate cancer is the second leading cause of cancer death in men. Fortunately, men can live for a long time with their disease if managed appropriately. There are approximately 3.2 million men in the United States annually living with this disease.1 We believe that PyL, if approved, will play an ongoing role in the diagnosis and management of prostate cancer."

The NDA is supported by data from two pivotal studies (OSPREY and CONDOR), designed to establish the safety and diagnostic performance of PyL imaging across the disease continuum of prostate cancer. Results from OSPREY Cohort A demonstrated improvement in specificity and positive predictive value (PPV) of PyL PET imaging over conventional imaging in men with high risk prostate cancer. OSPREY Cohort B and CONDOR studied men with prostate cancer in various disease states, including biochemical recurrent prostate cancer, hormone sensitive prostate cancer, non-metastatic castrate resistant prostate cancer, and metastatic castrate resistant prostate cancer. OSPREY Cohort B demonstrated a sensitivity in detecting metastatic lesions, while CONDOR, in patients with biochemical recurrent prostate cancer and non-informative baseline findings, demonstrated a high correct localization rate and high detection rate, including patients with low PSA values. In the CONDOR study, 63.9% of patients had a change in intended disease management plans due to the PyL imaging results. We believe the results from these two studies, taken as a whole, demonstrate the ability of PyL to reliably detect and localize disease and could enable more appropriate patient management.

PyL has been administered in approximately 3,500 subjects globally, including the two Company sponsored studies, multiple investigator sponsored studies, as well as clinical use reported in the literature. Across all of these studies PyL has shown an attractive safety profile.

"We are extremely grateful to the prostate cancer patients and investigators who participated in PyL’s clinical development program," said Istvan Molnar, MD, Chief Medical Officer of Lantheus. "We believe that the demonstrated strong diagnostic performance of PyL, will assist in treatment decisions and, ultimately, may improve patient outcomes. We look forward to working with the FDA during the regulatory process in pursuit of our goal of bringing PyL to patients."

About PyL for PET Imaging of Prostate Cancer

PyL (also known as 18F-DCFPyL) is a fluorinated PSMA-targeted PET imaging agent that enables visualization of localized prostate cancer as well as bone and soft tissue metastases to determine the presence or absence of recurrent and/or metastatic prostate cancer.

About OSPREY

The Phase 2/3 OSPREY trial assessed the diagnostic performance of PyL to detect prostate cancer in pelvic lymph nodes in subjects with high risk locally advanced prostate cancer (Cohort A) and distant metastases in subjects with metastatic or recurrent prostate cancer (Cohort B). In the trial, the diagnostic performance of PyL in detecting disease in pelvic lymph nodes (Cohort A) showed specificity of 96-99%, sensitivity of 31-42%, and PPV of 78-91% although the trial did not meet one of its the primary endpoints. In the metastatic or recurrent prostate cancer setting (Cohort B), PyL exhibited sensitivity of 93-99% and PPV of 81-88% in detecting metastatic lesions. Overall, PyL demonstrated high diagnostic performance in reliably detecting nodal and distant metastatic prostate cancer.

About CONDOR

The Phase 3 CONDOR trial evaluated the diagnostic performance and clinical impact of PyL in men with biochemical recurrence of prostate cancer and uninformative baseline imaging based on conventional modalities. The CONDOR trial achieved its primary endpoint, with a correct localization rate (CLR) of 84.8% to 87.0% among the three blinded independent readers (the lower bound of the 95% confidence intervals ranging from 77.8% to 80.4%). CLR is based on positive predictive value, defined as the percentage of subjects with a one-to-one correspondence between localization of at least one lesion identified on PyL PET/CT and a composite truth standard comprised of histopathology, conventional imaging and/or changes in PSA levels following radiation therapy. 63.9% of subjects in the CONDOR trial had a change in intended disease management plans due to PyL imaging results, a key secondary endpoint of the trial. The changes to treatment management plans due to the PyL results included salvage local therapy to systemic therapy, observation to initiating therapy, noncurative systemic therapy to salvage local therapy, and planned treatment to observation.

About Prostate Cancer

Prostate cancer is the second most common form of cancer affecting men in the United States: an estimated one in nine men will be diagnosed with prostate cancer in his lifetime. The American Cancer Society estimates that each year approximately 192,000 new cases of prostate cancer will be diagnosed, and 33,000 men will die of the disease. Approximately 3.2 million men in the U.S. currently count themselves among prostate cancer survivors.1

On September 30, 2020 CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, reported that congratulated Orphazyme A/S (ORPHA.CO) (NASDAQ:ORPH) ("Orphazyme") on the September 29, 2020 pricing of its global offering, consisting of an initial public offering of American Depositary Shares in the U.S. and a concurrent private placement of ordinary shares in Europe. According to Orphazyme, the aggregate gross proceeds from its global offering will amount to approximately DKK 534,534,637 ($83,777,606 using a DKK/USD exchange rate of 6.3804) (assuming no exercise of the option to purchase additional shares) and DKK 614,714,770 ($96,344,237 using a DKK/USD exchange rate of 6.3804) (assuming full exercise of the option to purchase additional shares). CytRx has an agreement with Orphazyme that can yield milestone payments and royalties based on potential future sales of arimoclomol.

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In connection with Orphazyme’s global offering, it disclosed receipt of a filing communication from the U.S. Food and Drug Administration ("FDA") relating to the agency’s ordinary course review of its new drug application ("NDA") for arimoclomol in the treatment of Niemann-Pick disease Type C ("NPC"). The filing communication follows acceptance on a Priority Review basis by the FDA of Orphazyme’s NDA for arimoclomol in NPC and the agency’s establishment of the Prescription Drug User Fee Act ("PDUFA") target action date of March 17, 2021. Orphazyme stated that its receipt of the filing communication does not impact the FDA’s acceptance of its NDA, the target PDUFA action date or the Priority Review determination. Orphazyme’s disclosure notes that the filing communication constitutes preliminary notice from the FDA of potential review issues as part of its ordinary course review of the NDA and is not necessarily indicative of deficiencies that may be identified during the review. Orphazyme has stated that it intends to discuss the filing communication with the FDA.

In accordance with applicable securities laws, Orphazyme amended the registration statement on Form F-1 on file with the U.S. Securities and Exchange Commission. For further information concerning the FDA filing communication, we refer you to the "Recent Developments" section on Page 6 of Orphazyme’s amended Form F-1 filed on September 28, 2020.

CytRx will continue to provide updates that are relevant to its agreement with Orphazyme.