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Rubius Therapeutics Provides Update on Clinical Trial Progress for its Lead Oncology Product Candidate RTX-240 and Announces Planned Leadership Transition

On June 30, 2020 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines, reported that it has completed dosing of the first dose-escalation cohort with no observed adverse events to date in the Phase 1/2 clinical trial of RTX-240 (Press release, Rubius Therapeutics, JUN 30, 2020, View Source [SID1234561568]). RTX-240 is an allogeneic cellular therapy for the treatment of patients with relapsed/refractory or locally advanced solid tumors. Rubius also announced a planned change to its management team. Andrew Oh, Chief Financial Officer, will depart the Company following completion of a transition period, which will conclude on December 31, 2020. The Company has initiated a search for its next Chief Financial Officer.

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"After the successful completion of the first monotherapy dose escalation cohort in our Phase 1/2 clinical trial of RTX-240, with no observed adverse events to date, we have initiated the portion of the trial where we expect to see the biological effects of RTX-240 on innate and adaptive immunity, including potential activation and increased numbers of NK cells and T cells," said Pablo J. Cagnoni, M.D., president and chief executive officer of Rubius Therapeutics. "Based on our preclinical data, we believe these effects will translate into anti-tumor killing and clinical responses in patients. Our fully owned manufacturing facility in Smithfield, RI, continues to successfully manufacture clinical supply of RTX-240."

The Phase 1/2 clinical trial of RTX-240 is evaluating the safety, tolerability, pharmacokinetics, maximum tolerated dose and recommended Phase 2 dose and dosing regimen of RTX-240 in adult patients with relapsed/refractory or locally advanced solid tumors. The trial is also assessing the pharmacodynamic effects of RTX-240 as measured by increased proliferation and effector function of the NK and T cell populations relative to baseline. In addition to these proliferation biomarkers, the study will also evaluate production of granzyme B, which is an indicator of activated NK and T cells capable of tumor killing. The trial is evaluating anti-tumor activity as measured by overall response rate, progression-free survival and overall survival. The ongoing dose escalation phase will be followed by expansion cohorts in specified tumor types during Phase 2 of the trial. The extent to which the COVID-19 pandemic may impact Rubius’ ability to enroll patients in the trial is uncertain and will depend on future developments.

With this clinical update, Rubius also announced the planned transition of Andrew Oh as Chief Financial Officer.

"Andy joined Rubius in 2017, when the company was privately held, and, since that time, he has helped us raise more than $450 million in capital, including our initial public offering, to support the development of our proprietary pipeline of Red Cell Therapeutics," said Dr. Cagnoni. "He has built a world-class finance team and was instrumental in negotiating and securing our fully owned manufacturing facility that today is providing clinical supply for RTX-240. As we progress towards becoming an integrated clinical development organization with one product currently in the clinic and a second oncology Investigational New Drug Application planned for RTX-321 in HPV-positive cancers by year-end, now is the appropriate time for this important transition. We are thankful for Andy’s many contributions and wish him well on his next endeavor."

About RTX-240

RTX-240 is an allogeneic cellular therapy product candidate that simultaneously presents hundreds of thousands of copies of the costimulatory 4-1BB ligand (4-1BBL) and the trans-presented cytokine interleukin-15 (IL-15TP) in their native forms to activate and expand NK and T cells. 4-1BBL is a costimulatory molecule that can drive T and NK cell proliferation and activation and interferon gamma (IFNγ) production. IL-15 is a cytokine that bridges innate and adaptive immunity by promoting NK and T cell proliferation and NK cell cytotoxicity. IL-15TP is a fusion of IL-15 and IL-15 receptor alpha.

Magenta Therapeutics Announces Closing of Public Offering, Including Full Exercise of Underwriters’ Option to Purchase Additional Shares

On June 30, 2020 Magenta Therapeutics, Inc. (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported the closing of its previously announced underwritten public offering of 8,625,000 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,125,000 shares, at a price to the public of $8.00 per share (Press release, Magenta Therapeutics, JUN 30, 2020, View Source [SID1234561566]). The total gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be $69.0 million.

Goldman Sachs & Co. LLC and Cowen acted as joint bookrunning managers for the offering. Wedbush PacGrow acted as lead manager for the offering.

Magenta intends to use the net proceeds from this offering to advance its clinical and earlier stage programs and for research and development, working capital and general corporate purposes.

The securities described were offered pursuant to a shelf registration statement on Form S-3 (File No. 333-233127), including a base prospectus. A preliminary prospectus supplement and accompanying prospectus relating to and describing the terms of the offering was filed with the U.S. Securities and Exchange Commission (the "SEC") on June 24, 2020. The final prospectus supplement relating to the offering was filed with the SEC on June 25, 2020. Copies of the final prospectus supplement and the accompanying prospectus relating to these shares can be obtained from: Goldman Sachs & Co. LLC, Attn: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 866-471-2526, facsimile: 212-902-9316, e-mail: [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].

Important Information

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any offer or sale of these securities 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.

Boston Scientific Announces Conference Call Discussing Second Quarter 2020 Financial Results

On June 30, 2020 Boston Scientific Corporation (NYSE: BSX) reported that it will webcast its conference call discussing financial results and business highlights for the second quarter ended June 30, 2020 on Wednesday, July 29, 2020 at 8:00 a.m. EDT (Press release, Boston Scientific, JUN 30, 2020, View Source [SID1234561565]). The call will be hosted by Mike Mahoney, chairman and chief executive officer, and Dan Brennan, executive vice president and chief financial officer. The company will issue a news release announcing financial results for the second quarter on Wednesday, July 29, 2020, prior to the conference call.

A live webcast of the conference call will be available on the Investor Relations section of the website at investors.bostonscientific.com.

A replay of the webcast will be archived and available at investors.bostonscientific.com beginning approximately one hour following the completion of the meeting.

Cambridge Spin-out PharmEnable Raises £1.8 million for Drug Discovery Expansion

On June 30, 2020 PharmEnable, a Cambridge-based drug discovery company using advanced medicinal chemistry and AI-enabled approaches to design the next generation of highly complex and specific drug candidate molecules, reported it has closed a £1.8 million seed financing to support its transition into a drug development company (Press release, PharmEnable, JUN 30, 2020, View Source [SID1234561564]). It aims to develop new treatments for conditions with significant unmet clinical need, by designing highly complex molecules for addressing challenging biological targets.

The round, which was significantly over-subscribed, was led by Cambridge Enterprise, the commercialisation arm of the University of Cambridge, as well as the University of Cambridge Enterprise Fund VI, managed by Parkwalk Advisors. It also attracted support from a wealth of angel investors and notable life science funds, including Jonathan Milner, serial entrepreneur and founder of Abcam; Andy Richards, Cambridge-based entrepreneur and investor; David Ford, Oxford-based life sciences angel investor; the family office of Paul Forster, co-founder of Indeed.com; Ian Tomlinson, chairman of several bio-incubators, entrepreneur and co- founder of Domantis; KQ Labs at the Francis Crick Institute; Martlet Capital, a Cambridge-based investor with a growing portfolio of innovative life science companies; the fast-growing o2h ventures Human Health EIS fund; and Wren Capital, the established London-based angel investor in science, engineering and software businesses.

A spin-out from the University of Cambridge in 2016, and financed to date by its founders and service-based revenues, PharmEnable will use the funding to evolve its business model and invest in a pipeline of drug discovery programmes across a number of disease areas including cancer and neurodegenerative disease. Additionally, PharmEnable will continue to engage in strategic partnerships with pharma, innovative biotechs and academia.

PharmEnable is led by co-founder and CEO Dr Hannah Sore. It has attracted an experienced Board and management team including Dr Jane Dancer, previously of F-star, who joined recently as the new Board Chair. The financing has enabled PharmEnable to expand its scientific team including Dr David Vidal as Director of Technology, with further expansion, including the addition of a Director of Drug Discovery planned for Q3 2020.

The PharmEnable platform technology can predict improved small molecule hits to targets across a range of disease areas. Its approach focuses on exploring and mapping the possible chemical universe and designing novel small molecules that are highly complex with shapes similar to those found in nature. This approach can identify hits with improved specificity compared with traditional screening methods, and allows PharmEnable to take on particularly challenging biological targets, such as protein-protein interactions and epitranscriptomic modifications that have been undruggable by existing approaches. Its solution consists of two elements: ChemUniverse a diversity-focused virtual database of chemically diverse molecules; and ChemSeek, a suite of gold standard AI-enabled tools for finding drug target matches from structure and ligand data.

Oncopeptides submits a New Drug Application to the FDA for accelerated approval of melflufen in triple-class refractory multiple myeloma patients

On June 30, 2020 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO) reported that the Company submits a New Drug Application (NDA) to the U.S. Food and Drug Administration, FDA, for accelerated approval of melflufen (INN melphalan flufenamide) in combination with dexamethasone for the treatment of adult patients with multiple myeloma whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one anti-CD38 monoclonal antibody (i.e., triple-class refractory multiple myeloma patients) (Press release, Oncopeptides, JUN 30, 2020, View Source [SID1234561555]).

Melflufen is the lead candidate coming out of the Oncopeptides’ proprietary PDC-platform. The product is a first-in-class aminopeptidase-targeting peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. The submission is based on the results from the pivotal phase 2 study HORIZON, evaluating intravenous melflufen in combination with dexamethasone in patients with relapsed refractory multiple myeloma (RRMM).

The results from the HORIZON study demonstrates that melflufen in combination with dexamethasone, has a potential to provide a therapeutic option for patients with RRMM that are hard to treat and have a poor prognosis, including patients with triple class refractory myeloma and patients with extramedullary disease (EMD). The responses in the HORIZON study were durable and often deepened with prolonged treatment, suggesting that patients could benefit from staying on treatment for as long as possible.

"I am very proud and humbled by the organizations ability to timely submit the NDA for accelerated approval of melflufen. This is a major milestone for Oncopeptides and is a result of dedicated research and development activities throughout the last decade", says Jakob Lindberg CEO of Oncopeptides. "I would like to express my sincere gratitude to all patients, co-workers, investigators and shareholders who have provided relentless support to enable a novel treatment option for a fast-growing patient population with a significant unmet medical need".

Following the submission to the FDA Oncopeptides will initiate an Expanded Access Program (EAP) in the U.S. to enable melflufen treatment for patients with a significant unmet medical need.

About the HORIZON study
In total 157 multiple myeloma patients have been enrolled and evaluated in the pivotal phase 2 HORIZON study. The study was fully recruited in October 2019, the final data cut was made on January 14th and the final data was presented at the EHA (Free EHA Whitepaper) meeting in June. The patients in the study were refractory to pomalidomide and/or daratumumab after failing on immunomodulatory drugs (IMiDs) and proteasome inhibitors (PIs). The HORIZON study population includes subgroups of patients who were triple?class refractory and/or had extramedullary disease and/or had cytogenetic high?risk features.

Summary of results

End Points Intention to Treat (n=157) Triple Class Refractory (n=119) Extra Medullary Disease (n=55)
Overall Response Rate (ORR) 29% 26% 24%
Median Progression Free Survival (PFS)) 4.2 months 3.9 months 2.9 months
Median Overall Survival (OS) 11.6 months 11.2 months 6.5 months
Responding patients n=45 n=31 n=13
Median Duration of Response (DOR) 5.5 months 4.4 months 5.5 months
Median Progression Free Survival (PFS) 8.5 months 8.5 months 17.3 months
All data were confirmed by the Independent Review Committee (IRC), with only minimal discordance.

About melflufen
Melflufen (INN melphalan flufenamide) is a first-in-class aminopeptidase-targeting peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately hydrolyzed by peptidases to deliver an entrapped hydrophilic alkylator payload. Peptidases play a key role in protein homeostasis and feature in cellular processes such as cell-cycle progression and programmed cell death. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the increased intracellular alkylator concentration. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies.

Expanded access policy
The preparations for an Expanded Access Program (EAP) in the US are well underway and the program will open in Q3. Oncopeptides encourages awareness of and participation in its clinical trials and believes that participating in clinical trials is a good way for patients to access investigational drugs prior to regulatory approval. Individuals interested in participating in clinical trials for melflufen may visit View Source for information about ongoing clinical trials. Patients are encouraged to consult their physician regarding the possibility of participating in one of the ongoing clinical trials.