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Novartis announces positive result of phase III study with radioligand therapy 177Lu-PSMA-617 in patients with advanced prostate cancer

On March 23, 2021 Novartis reported the first interpretable results of the Phase III VISION study evaluating the efficacy and safety of 177Lu-PSMA-617, a targeted radioligand therapy in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) compared to best standard of care alone (Press release, Novartis, MAR 23, 2021, View Source [SID1234576992]). The trial met both primary endpoints of overall survival and radiographic progression-free survival1, helping to move closer the ambition of becoming the targeted treatment for >80% of patients with advanced prostate cancer. The safety profile was consistent with data reported in previous clinical studies1. Results from the VISION trial will be presented at an upcoming medical meeting and included in US and EU regulatory submissions.

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"Patients with metastatic castration-resistant prostate cancer have a less than 1 in 6 chance of surviving 5 years2 and need new treatment options. These groundbreaking data confirm our belief in the potential of 177Lu-PSMA-617 to reimagine outcomes for these patients through phenotypic precision medicine. We intend to submit these data to regulatory authorities as soon as possible," said John Tsai, Head of Global Drug Development and Chief Medical Officer for Novartis. "We would like to thank the patients who volunteered to participate in this study as well as the clinical teams at each of the trial sites. We would not be able to realize our commitment to reimagining medicine without the partnership of patients and their families."

Radioligand therapy combines a targeting compound that binds to markers expressed by tumors and a radioactive isotope, causing DNA damage that inhibits tumor growth and replication. This therapeutic approach enables targeted delivery of radiation to the tumor, while limiting damage to the surrounding normal tissue. Novartis has established global expertise and specialized supply chain and manufacturing capabilities across its network of four radioligand therapy production sites, and is further increasing capacity to ensure delivery of radioligand therapies like 177Lu-PSMA-617 to patients in need.

Novartis is the only pharmaceutical company which is pursuing four different cancer treatment platforms. These include radioligand therapy, cell and gene therapy, and targeted therapy and immunotherapy, with an opportunity to combine these platforms for the best outcomes for each cancer patient.

About Advanced Prostate Cancer
Prostate cancer is a form of cancer that develops in the prostate gland, a small walnut shaped gland in the pelvis of men. In castration resistant prostate cancer (CRPC), the tumor shows signs of growth, such as rising Prostate Specific Antigen (PSA) levels, despite the use of hormone treatments that lower testosterone3. In metastatic CRPC (mCRPC), the tumor spreads to other parts of the body, such as neighboring organs or bones and remains unresponsive to hormone treatment4. The five-year survival rate for patients with mCRPC is approximately 15%2.

About Phenotypic Precision Medicine in Advanced Prostate Cancer
Despite advances in prostate cancer care, there is a high unmet need for new targeted treatment options to improve outcomes for patients with metastatic castration resistant prostate cancer. More than 80% of prostate cancer tumors highly express a phenotypic biomarker5 called Prostate Specific Membrane Antigen (PSMA)4,6-9, making it a promising diagnostic (through positron emission tomography (PET) scan imaging) and therapeutic target for radioligand therapy6.

About 177Lu-PSMA-617
177Lu-PSMA-617 is an investigational PSMA-targeted radioligand therapy for metastatic castration-resistant prostate cancer. It is a type of precision cancer treatment combining a targeting compound (ligand) with a therapeutic radioisotope (a radioactive particle)10-12. After administration into the bloodstream, 177Lu-PSMA-617 binds to prostate cancer cells that express PSMA13, a transmembrane protein, with high tumor-to-normal tissue uptake10,14,15. Once bound, emissions from the radioisotope damage tumor cells, disrupting their ability to replicate and/or triggering cell death. The radiation from the radioisotope works over very short distances to limit damage to surrounding cells14,16.

About VISION
VISION is an international, prospective, randomized, open-label, multicenter, phase III study to assess the efficacy and safety of 177Lu-PSMA-617 (7.4 GBq administered by i.v. infusion every 6 weeks for a maximum of 6 cycles) plus investigator-chosen best standard of care in the investigational arm, versus best standard of care in the control arm17. Patients with PSMA PET-scan positive mCRPC, and progression after prior taxane and androgen receptor-directed therapy (ARDT), were randomized in a 2:1 ratio in favor of the investigational arm. The alternate primary endpoints were rPFS and OS. The study enrolled 831 patients1.

Nuformix plc ("Nuformix", the "Company" or the "Group") NXP001 (Oncology) – Exercise of Option by Oxilio

On March 22, 2021 Nuformix plc (LSE:NFX), a pharmaceutical development company targeting unmet medical needs in fibrosis and oncology via drug repurposing, reported that Oxilio Ltd ("Oxilio") has exercised its option to acquire a licence for NXP001 (a proprietary new form of aprepitant) for oncology indications (Press release, Nuformix, MAR 22, 2021, View Source [SID1234621610]).

· On 23 September 2020, Nuformix granted an exclusive option to Oxilio, a private pharmaceutical development company, to license NXP001 globally for oncology indications. This triggered the first upfront payment and the option period ran until 23 March 2021
· Nuformix and Oxilio will now work together to finalise a global licensing agreement for NXP001. Once this has been agreed, this will trigger a second undisclosed upfront payment, payable to Nuformix. Under this agreement, Nuformix will license its patent estate and know-how on NXP001 in return for development milestone payments and a royalty on any future net sales, capped at £2m per annum

Dr Anne Brindley, CEO of Nuformix, said: "We are very pleased that Oxilio has exercised its option for NXP001, allowing them to continue the development of NXP001 in oncology. We will now look to finalise the licensing agreement with Oxilio that will provide Nuformix with near term revenue and an opportunity to benefit from the upside of the significant global oncology market. This successful outcome endorses the Nuformix strategy of identifying new improved forms of existing drugs for repurposing into new indications with early-stage licensing during the pre-clinical or Phase 1 stages of development."

Dr Simon Yaxley, Co-Founder and Director of Oxilio said: "Exercising our option gives us the opportunity to continue to develop NXP001 in line with our approach to optimise the delivery of our drug for the effective treatment of certain cancers. We look forward to continuing our collaboration with Nuformix to realise the huge potential of this approach."

About NXP001
NXP001 is a proprietary new form of the drug aprepitant that is currently marketed as a product in the oncology supportive care setting (chemotherapy induced nausea and vomiting). A disadvantage of aprepitant is that its suboptimal properties necessitate a complex formulation. Nuformix discovered new forms of aprepitant (NXP001) with improved properties and it has granted patents on its new forms.

About NXP001 and Oxilio
Oxilio will develop and seek to exploit NXP001 globally for the treatment of cancer. Cancer is one of the world’s primary causes of death. The total worldwide annual economic cost of cancer is substantially in excess of US$1 trillion. Traditional cancer treatments such as surgery, chemotherapy and radiotherapy are expensive and often have debilitating consequences for the patient. Costs of cancer drug treatments are also increasing, in large part due to the expense of taking new drugs through clinical trials where historically there is a very low rate of success. Therefore, there is an urgent need to develop safe, effective, and readily available anticancer agents.

Oxilio is focused on alleviating the current dilemma of a shortage of effective drug candidates that have potential as new cancer therapies, by adopting a drug repurposing strategy (identifying new uses for approved or investigational drugs that are outside the scope of the original medical indication). The major advantage of this approach is that the pharmacokinetics, pharmacodynamics and toxicity profiles of these drugs are already reasonably well established. Thus, drug repurposing is a less risky development route with substantially lower associated development costs. The agreement with Nuformix allows Oxilio to focus on developing rapidly a unique formulation and dosage form with NXP001 and progressing into the clinic.

Oxilio is a privately held pharmaceutical development company focused on repurposing known drugs for the treatment of cancer through a programme of corporate alliances coupled with rapid proof of concept clinical development.

• 28th Healthcare Investment Forum, 24th March 2021

On March 22, 2021 Nanoligent reported that it has been selected to showcase at the 28th Healthcare Investment Forum the next 24th of March (Press release, Nanoligent, MAR 22, 2021, http://www.nanoligent.com/index.php/2021/03/22/28th-healthcare-investment-forum/ [SID1234578144]). The Healthcare Barcelona Investment Forum is a meeting point for entrepreneurs and investors in the healthcare sector. All kinds of companies can be presented with projects related to Biotechnology, Medical Devices, Health Services and Information Technologies related to health. The Healthcare Barcelona Investment Forum has positioned itself as a benchmark in the Catalan biomedical ecosystem, promoting innovation and entrepreneurship among the group with the aim of bringing together entrepreneurs and investors in the healthcare sector.

Coordinated by the Col·legi de Metges de Barcelona, Barcelona Activa – Ajuntament de Barcelona, ESADE-BAN and Biocat, it is the meeting point of reference between entrepreneurs and investors in the healthcare sector. Given the current situation, science, innovation and entrepreneurship in health must not be stopped, with funding being key.

NICE recommends Kyprolis triple combination treatment for multiple myeloma

On March 22, 2021 Amgen reported that The National Institute for Health and Care Excellence (NICE) has recommended a triple combination therapy comprised of Kyprolis, lenalidomide and dexamethasone for the treatment of previously-treated multiple myeloma patients.

In final guidance, NICE backed the use of Kyprolis (carfilzomib) plus lenalidomide and dexamethasone for people with multiple myeloma, who have had at least one previous therapy – including targeted therapy bortezomib (Janssen’s Velcade).

In clinical trials of the triple combination, Kyprolis with lenalidomide and dexamethasone was found to extend periods of remission and help people to live longer, compared to the current second-line treatment for multiple myeloma patients – lenalidomide and dexamethasone alone.

In a statement, NICE said that its independent appraisal committee saw evidence that shows the benefit of the triple therapy appears to continue for up to six years.

Following its review, the committee said the most-likely cost-effectiveness estimates for Kyprolis plus lenalidomide and dexamethasone are within what NICE considers a cost-effective use of NHS resources.

"The recommendation of our committee will be welcomed by people with multiple myeloma who have told us of the need of a new second line treatment option that gives longer periods of remission and improves survival," said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Assessment at NICE.

"The clinical data shows that the benefits of this triple therapy continue after treatment has stopped. A positive decision has been made possible after the company and NHS England came to a commercial arrangement which allows carfilzomib to be used on the NHS with a confidential discount," he added.

Verrica Announces Proposed Public Offering of Common Stock

On March 22, 2021 Verrica Pharmaceuticals Inc. (Nasdaq: VRCA), a dermatology therapeutics company developing medications for skin diseases requiring medical interventions, reported that it intends to offer and sell, subject to market conditions, shares of its common stock in an underwritten public offering (Press release, Verrica Pharmaceuticals, MAR 22, 2021, View Source [SID1234577030]). All of the shares of common stock to be sold in the offering will be offered by Verrica. Verrica also intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of its common stock offered in the public offering on the same terms and conditions. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.

Jefferies LLC, Cowen and Company, LLC and RBC Capital Markets, LLC are acting as joint book-running managers for the offering.

A shelf registration statement relating to the shares of common stock offered in the public offering described above was filed with the Securities and Exchange Commission (SEC) on March 13, 2020 and declared effective by the SEC on March 25, 2020. The offering will be made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus, when available, may also be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at 877-547-6340 or by email at [email protected]; or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at [email protected]; or RBC Capital Markets, LLC, Attention: Equity Syndicate, 200 Vesey Street, 8th Floor, New York, NY 10281, or by telephone at (877) 822-4089 or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy the securities being offered, nor shall there be any sale of the securities being offered in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.