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.

On September 30, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported that its tender offer to purchase any and all issued and outstanding shares of common stock of Pfenex Inc. (NYSE American: PFNX) at an offer price of $12.00 per share in cash, plus one non-transferable contractual contingent value right per share representing the right to receive a contingent payment of $2.00 in cash, if a certain specified milestone is achieved, expired at midnight (New York City time), at the end of the day on Tuesday, September 29, 2020 (Press release, Ligand, SEP 30, 2020, View Source [SID1234567818]).

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The depositary for the tender offer has advised that, as of the expiration of the tender offer, a total of approximately 27,591,554 shares were validly tendered and not withdrawn in the tender offer (including shares delivered through notices of guaranteed delivery), representing approximately 80.3% of Pfenex’s outstanding shares. Ligand’s wholly-owned subsidiary will accept for payment all shares that were validly tendered and not withdrawn prior to expiration of the tender offer, and payment for such shares will be made promptly, in accordance with the terms of the tender offer.

Ligand expects the merger to close on October 1, 2020, with Pfenex becoming a wholly owned subsidiary of Ligand. As a consequence of the merger, each outstanding Pfenex share not tendered and purchased in the offer (other than those as to which holders properly exercise dissenters’ rights and those owned at the commencement of the tender offer by Ligand or its direct and indirect subsidiaries) will be converted into the right to receive the same $12.00 per share in cash, plus one non-transferable contractual contingent value right per share representing the right to receive a contingent payment of $2.00 in cash, if a certain specified milestone is achieved, without interest and less any required withholding taxes, that was offered in the tender offer. Following completion of the merger, Pfenex’s common stock will cease to be traded on the New York Stock Exchange American.

On September 30, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that management will participate in a fireside chat at the Jefferies Cell Therapy Virtual Summit on Tuesday, October 6, 2020 and 2:30 p.m. E.T (Press release, Fate Therapeutics, SEP 30, 2020, View Source [SID1234567817]).

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A live webcast of the presentation will be available through the investor relations section of the Company’s website at www.fatetherapeutics.com.

On September 30, 2020 GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to improving the lives of patients with metabolic and liver diseases, reported its first half-year 2020 financial report, including the advances of its R&D portfolio and the new GENFIT corporate strategy (Press release, Genfit, SEP 30, 2020, https://ir.genfit.com/news-releases/news-release-details/genfit-first-half-year-2020-financial-report-and-new-corporate [SID1234567816]). The Half Year Business and Financial Report, including the new corporate strategy is available to the public and was filed with the French Autorité des marchés financiers (French Financial Markets Authority) today. The condensed consolidated financial statements are included in this press release and the complete financial statements are available on the "Investors" page of the GENFIT website.

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Conference Call in English on September 30, 2020 at 4:30pm EDT / 22:30 CEST, and in French on October 1, 2020 at 1:30am EDT / 7:30am CEST

Both the English and French conference calls will be accessible on the investor page of our website, under the events section at https://ir.genfit.com/ or by calling 877-407-9167 (toll-free U.S. and Canada), 201-493-6754 (international) or 0 800 912 848 (France) five minutes prior to the start time (no passcode needed). A replay will be available shortly after the call.

New corporate strategy and prospects

The company’s corporate strategy now focuses on two priority areas:

Phase 3 clinical trial ELATIVE evaluating elafibranor in PBC:
Patient enrolment now started, and results expected early 2023, given the current constraints due to the COVID-19 pandemic;
Following the positive Phase 2 data of elafibranor in PBC, the U.S. Food and Drug Administration (FDA) granted elafibranor Breakthrough Therapy designation. The ELATIVE study aims to confirm elafibranor’s previously successful results of efficacy, potential improvement in pruritus and safety in PBC patients;
Current market size for second line therapies in PBC is estimated at ~ $300MM in 2020 and is anticipated to experience double digit growth and estimates for 2025 are up to $1B. Elafibranor is a promising alternative therapy to the existing treatment in PBC, based on the significant unmet needs in this indication.

NIS4TM technology for (NASH) diagnosis:
NIS4TM technology data, recently published in The Lancet Gastroenterology & Hepatology, confirmed the technology’s diagnostic performance and garnered the support of leading NASH experts;
The recently announced exclusive licensing agreement with Labcorp for NIS4 TM technology will enable a large-scale commercial launch as of next year;
NIS4TM addresses patients’ and payers’ requirements as liver biopsy remains the only – although imperfect – approved diagnostic option in the clinical development field and cannot be replicated on a large scale due to its painful and invasive nature, and its cost for healthcare systems. It would be impossible to diagnose all patients with biopsies given the limited number of procedures that can be performed. The blood test commercialized by Lacorp will address these multiple challenges;
The NASH therapeutic market is potentially significant, however, the opportunity is dependent on quick, reliable and easy to execute diagnostic solutions to identify patients. NIS4TM technology represents the essential first step in managing patients with NASH and is the first step for patients to take control, even in the absence of treatments, of their disease.
GENFIT’s new strategy includes a plan, which aims to create two distinct operational subsidiaries by 2021 to enable more independent management and growth:

The first entity would be dedicated to the development of specialty indications, starting with the Phase 3 trial in PBC;

The second entity would house NASH solutions, including all programs related to the identification, evaluation and monitoring of patients with NASH. This independent structure would facilitate future partnerships for NIS4.
The two entities would remain a part of GENFIT as a listed company, who would ensure adequate decision making between both "business" entities, the goal being to best highlight each of the activities to benefit the Group’s valuation.

Concurrently, GENFIT is adopting a plan to reallocate and rationalize all capital with an objective to reduce the cash burn by more than 50% by 2022 compared to our cash burn prior to the RESOLVE-IT Phase 3 data. The program aims to reduce the current cash burn rate from €110M annually before our Phase 3 data, to approximately €45MM annually, beginning in 2022. Due to the residual expenses related to the termination of RESOLVE-IT, 2021 will be a transition year.

This plan incorporates the following key components:

The overall clinical development program for elafibranor in NASH and all activities associated with the commercial launch of elafibranor in NASH have been terminated given the low probability of success compared to required expenses. The termination includes the NASH combination therapy trials, the pediatric trials, and other trials such as the evaluation of the impact of elafibranor on liver fat composition;

A comprehensive cost-saving plan has been implemented, including the redirection of R&D activities and the termination of secondary programs (i.e. the RORgT program);

A workforce restructuring plan aims to reduce the overall workforce by 40%, encompassing both the U.S and France in order to align the company size to the new scope of activity.
Lastly, GENFIT plans to propose to the holders of its OCEANE bond (€180 million nominal amount with a maturity of October 16, 2022) and its shareholders, an adjustment of the terms of the OCEANE convertible bond. The Company’s objective is to begin this process towards the end of the year, in order to have a balance sheet which is structured in line with its new strategy.

Pascal Prigent, CEO of GENFIT, stated: "The decisions we have taken allow us to move GENFIT forward towards 2021 with a clear and precise roadmap. We are confident in the probability of success, and the potential of our two priority programs. The evolution of the company also ensures the structure adapts to our strategy, with an approach that is both organizationally and financially sound."

Key aspects of the half-year 2020 results

Key aspects of the half-year 2020 results are:

Cash and cash equivalents of €225.7 million at June 30, 2020 (€276.7 million at December 31, 2019);
Operating income of €5.9 million (€5.4 million at June 30, 2019), essentially from the Research Tax Credit, which amounted to €5.2 million for the first half 2020 (€5.3 million in the preceding half year);
Operating expenses of €55.0 million (€51.3 million at June 30, 2019) of which 67% represented R&D expenses.
The increase in operating expenses is due to increases in marketing and pre-commercialization expenses, which amounted to €9.5 million in the first half 2020 (€2.9 million in the first half 2019). Marketing and pre-commercialization expenses will significantly decrease as of the second half 2020 due to the discontinuation of the pre-commercialization work for elafibranor in NASH following the termination of this program in July 2020.

General and administrative expenses (€8,2 million in the first half 2020 compared to €9.5 million in the first half 2019) and research and development expenses (€36.9 million in the first half 2020 compared to €38.9 million in the first half 2019) have decreased slightly between 2019 and 2020. These expenses will progressively decrease as of the second half 2020 following the Company’s decision to begin the process of terminating the clinical trials for elafibranor in NASH, terminating secondary programs and to execute a comprehensive cost saving plan over 3 years. Significant expenses related to the termination of the RESOLVE-IT trial will be due in the second half 2020 and in 2021.

As a result of changes in revenues and expenses, the net loss amounted to €53.0 million at June 30, 2020 (€51.1 million at June 30, 2019). The net loss for 2019 was €65.1 million.