On November 12, 2019 PharmaCyte Biotech (OTCQB: PMCB) reported that has reduced a long list of FDA-required items to just one last important component—release testing—before it can submit an Investigational New Drug application (IND) to the U.S. FDA (Press release, PharmaCyte Biotech, NOV 12, 2019, View Source [SID1234550944]). PharmaCyte’s IND application will request permission from the FDA to conduct a Phase 2b clinical trial in locally advanced, inoperable pancreatic cancer (LAPC). With the FDA’s approval to begin a clinical trial, PharmaCyte’s opportunities for funding improve dramatically.

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PharmaCyte has already conducted and completed a long list of tests and entered the data from those tests into the IND application. PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of completing the IND for submission to the FDA, "Most of the work needed for PharmaCyte to submit an IND to the FDA has been completed."

PharmaCyte recently announced that it has encapsulated the live cells from its Master Cell Bank (MCB) into its live-cell encapsulation technology, Cell-in-a-Box, in what is the second of two staggered and back-to-back manufacturing runs. In that announcement the company’s CEO stated that after reviewing pictures of the growing cells, he is confident that those cells are growing as well as those in the first run, which ended successfully after a host of changes were implemented to improve the manufacturing process.

And, with the second manufacturing run well underway and growing as expected, this final run should end in success over the next week or so, and then the release testing for this second run combined with the release testing from the first run will complete what has been a long and arduous path to submitting the much-anticipated IND.

Now, let’s walk through each of the tests (release testing) that are required by cGMP Validation, the company that is taking responsibility for "releasing" the clinical trial product into the U.S. for use in PharmaCyte’s upcoming clinical trial. Many of these tests are also FDA-required tests that must be completed in order to obtain the data necessary to complete and submit the IND and for PharmaCyte to receive a Certificate of Analysis (CoA) from Austrianova. A CoA is a document issued by Austrianova’s Quality Assurance and Quality Control Department (QA/QC) that confirms that PharmaCyte’s product meets its product specifications. The CoA will contain the actual results obtained from the testing performed as part of the QA/QC of a representative sample of PharmaCyte’s clinical trial product.

After a manufacturing run is completed successfully, 300 Cell-in-a-Box capsules are placed into 400 of PharmaCyte’s clinical trial syringes and then frozen. A representative sample of those syringes will be thawed and undergo release testing.

All release testing related to "safety" of the encapsulated cells is being outsourced by PharmaCyte to independent third-party laboratories in Europe. They include tests for sterility, endotoxin, pH, mycoplasma and cell identity.

A sterility test confirms that all syringes are free from the presence of viable microorganisms or microbial contamination.

An endotoxin test of a biologic product is necessary to ensure the product is endotoxin free. This means that the cells do not contain bacterial toxins.

A pH test will confirm how much hydrogen is in the product and that the product meets the specified pH balance for a human being.

A mycoplasma test confirms that the syringes are free from mycoplasma. Mycoplasma are small, simple bacteria which lack a cell wall and represent one of the most prevalent and serious sources of cell line contamination.

A cell identity test confirms that the cell line used in production is positively identified as the 22P1G cell line. This is the human cell line that PharmaCyte cloned from the original cell line used in the first two clinical trials using the Cell-in-a-Box technology.

All release testing related to "functionality" of the encapsulated cells is being conducted by Austrianova at its GMP facility in Thailand. Those tests include enzymatic activity, cell viability, capsule count in each syringe, and capsule diameter.

An enzymatic activity test shows that the requisite enzymes are being produced by the cells inside the capsules to create the desired chemical reaction in the body. In PharmaCyte’s case, this means that the correct enzymes are being produced to convert the cancer-killing prodrug, ifosfamide, from its inactive form to its active form. Ifosfamide is the chemotherapy drug that PharmaCyte uses with the encapsulated cells that are implanted in a patient as close to the tumor as possible.

A cell viability test measures the number of cells that remain alive and viable after thawing a syringe of encapsulated cells.

A capsule count test confirms that each syringe contains 300 capsules. One syringe full of the encapsulated cells is implanted in a pancreatic cancer patient before ifosfamide is given intravenously to treat LAPC.

A capsule diameter test confirms that the diameter of each capsule inside a syringe of encapsulated cells meets a specified range.

Additionally, a pyrogenicity test must be completed to ensure that the capsules themselves do not produce a fever. This FDA-required test is not in the list of release tests. The pyrogenicity test is being conducted in the United States.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: View Source

On November 12, 2019 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for people living with kidney disease, reported financial results for the third quarter ended September 30, 2019 (Press release, Akebia, NOV 12, 2019, View Source [SID1234550943]). The Company will host a conference call today, Tuesday, November 12, 2019, at 9:00 a.m. Eastern Time to discuss its third quarter 2019 financial results and recent business highlights.

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Akebia also announced that it has entered into a $100 million non-dilutive, definitive term loan agreement with funds managed by Pharmakon Advisors LP, the investment manager of the BioPharma Credit funds. The loans provide Akebia with up to $100 million of borrowing capacity available in two tranches. Subject to the satisfaction of customary conditions, Akebia expects to draw $80 million at an initial closing later this month, and an additional tranche of $20 million is available for draw at Akebia’s option until December 31, 2020. Additional information on the loan agreement will be included in the Company’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2019 that is expected to be filed with the U.S. Securities and Exchange Commission today, November 12, 2019.

"Akebia continues to make great progress advancing our strategy. We achieved a primary objective of the Company by strengthening our balance sheet with $80 to $100 million non-dilutive, tranched term loans, on very competitive terms, to further support our clinical development program for vadadustat, our investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) for the treatment of anemia due to chronic kidney disease (CKD), and other strategic goals. Importantly, we believe these loans, the first tranche of which is expected to close later this month, in combination with our other cash resources, are expected to extend our cash runway into 2021, well past our expected top-line data readouts of our global Phase 3 clinical studies of vadadustat. Auryxia product revenue allows us to service the debt," stated John P. Butler, Chief Executive Officer of Akebia. "As we believe we are now within two quarters of our first readout of our global Phase 3 studies of vadadustat with top-line data of INNO2VATE on track for Q2’FY20 and PRO2TECT for mid-2020, subject to the accrual of major adverse cardiovascular events (MACE), we’re working to sharpen the timelines for completion and keying in on NDA and MAA related activities and commercial plans for vadadustat, upon approval."

Butler continued, "We have a tremendous amount of confidence in the program that we’ve designed for vadadustat and believe we are positioned well for clinical, regulatory and commercial success. We expect vadadustat to be the first drug of the HIF class to deliver clear data that directly compares its outcomes to the current standard of care in both dialysis and non-dialysis patients for the treatment of anemia due to CKD. We believe these data will be highly informative for physicians, patients and payers as they make important decisions about patient care, and a key consideration when differentiating between HIFs in the class."

Recent Highlights

Auryxia (ferric citrate) net product revenue increased 13 percent year-over-year to $30.0 million for the third quarter of 2019. Total Auryxia prescriptions increased 15.5 percent year-over-year to 51,700 in the third quarter of 2019.

In November, Mitsubishi Tanabe Pharma Corporation (MTPC), Akebia’s development and commercialization collaboration partner in Japan for vadadustat, presented positive 24-week and 52-week data from two Phase 3 active-controlled pivotal studies evaluating the efficacy and safety of vadadustat in Japanese patients with anemia due to CKD, at the American Society of Nephrology (ASN) Kidney Week 2019. Each study met its primary endpoint based on mean hemoglobin level at week 20 and 24, and showed vadadustat’s effect on hemoglobin was sustained through to 52 weeks in each study. (See recent related press release here.) In July, MTPC submitted a Japanese New Drug Application (JNDA) to the Ministry of Health, Labor and Welfare in Japan for marketing approval of vadadustat as a treatment for anemia due to CKD. The JNDA is the first regulatory submission for marketing approval of vadadustat and, if approved, is expected to lead to the first launch of vadadustat worldwide, next year.

Nine abstracts, including several associated with vadadustat and Auryxia, Akebia’s FDA-approved drug, were presented at ASN in November.

In October, the Independent Data Monitoring Committee reviewed unblinded safety and efficacy data from Akebia’s global Phase 3 studies of vadadustat, as planned, and recommended continuation of the studies without modifications.

In October, Akebia filed a complaint in federal district court against the Centers for Medicare & Medicaid Services (CMS) and the U.S. Department of Health and Human Services (HHS). The lawsuit challenges a September 2018 decision by CMS that rescinded Medicare Part D coverage of Auryxia, when used for the treatment of iron deficiency anemia (IDA) in adult patients with CKD not on dialysis. The legal action also seeks to reverse a related decision by CMS that imposed a prior authorization requirement for Auryxia when used for the control of serum phosphorus levels in adult patients with CKD on dialysis.

In April and August, Akebia completed enrollment in its global Phase 3 program, INNO2VATE and PRO2TECT, respectively, evaluating the safety and efficacy of vadadustat in dialysis-dependent and non-dialysis dependent CKD subjects with anemia due to CKD. The Company continues to expect to report top-line data from the INNO2VATE and PRO2TECT studies in Q2’FY20 and mid-2020, respectively, subject to the accrual of MACE.

Financial Results

Total revenue for the third quarter of 2019 was $92.0 million, compared to $53.2 million in the pre-merger third quarter of 2018.

Auryxia net product revenue for the third quarter of 2019 was $30.0 million, compared to $26.6 million, as reported by Keryx Biopharmaceuticals, Inc. (Keryx) prior to its merger with the Company, during the same period in 2018. This represents a 13 percent increase in net product revenue from the third quarter of 2018.

Collaboration and license revenue for the third quarter of 2019 was $62.0 million, compared with $53.2 million in the third quarter of 2018. The increase was primarily due to increased collaboration revenue of $6.8 million from Otsuka Pharmaceutical Co. Ltd (Otsuka). In accordance with the Company’s collaboration agreements, Otsuka began funding 80 percent of the development costs for vadadustat in the second quarter of 2019.

Cost of goods sold was $38.3 million for the third quarter of 2019, consisting of $11.2 million of costs associated with the manufacture of Auryxia and non-cash charges of $27.1 million related to the application of purchase accounting as a result of the merger with Keryx. These non-cash, merger-related charges include a $18.0 million inventory step-up charge and $9.1 million of amortization of intangibles.

Research and development expenses were $74.5 million for the third quarter of 2019 compared to $70.6 million for the third quarter of 2018. The increase was primarily attributable to an increase in headcount and other costs to support its research and development programs and clinical and preclinical activities. These increases were partially offset by a decrease in external costs related to PRO2TECT and INNO2VATE Phase 3 studies as they advance toward readout.

Selling, general and administrative expenses were $34.2 million for the third quarter of 2019 compared to $10.4 million for the third quarter of 2018. The increase was primarily attributable to commercialization costs associated with Auryxia, as there were no comparable commercialization costs in the third quarter of 2018.

The Company reported a net loss for the third quarter of 2019 of $54.6 million, or ($0.46) per share, as compared to a net loss of $26.0 million, or ($0.46) per share, for the third quarter of 2018. The Company’s net loss for the third quarter of 2019 includes the impact of non-cash charges of $27.1 million related to the application of purchase accounting as a result of the merger with Keryx, offset by an income tax benefit of $1.3 million.

The Company ended the quarter with cash, cash equivalents and available-for-sale securities of $145.6 million. "We are pleased to have further strengthened our balance sheet and extended our operating cash runway with our very recent non-dilutive, tranched term loans for up to $100 million, with the first $80 million tranche expected to close later this month. We expect these loans, coupled with the committed research and development funding from our collaborators and the receipt of a regulatory milestone from MTPC, assuming approval of vadadustat in Japan, to provide us with the cash resources to fund our current operating plan into Q1 of 2021," stated Jason A. Amello, Chief Financial Officer of Akebia.

Conference Call

Akebia will host a conference call today, Tuesday, November 12, 2019, at 9:00 a.m. Eastern Time to discuss its third quarter 2019 financial results and recent business updates. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 9996464. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through November 18, 2019. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 9996464. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On November 12, 2019 Myovant Sciences (NYSE: MYOV), a healthcare company focused on developing innovative treatments for women’s health and prostate cancer, reported recent corporate updates and reported financial results for the second fiscal quarter ended September 30, 2019 (Press release, Myovant Sciences, NOV 12, 2019, View Source [SID1234550942]).

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"Myovant continues to make great progress towards our milestones and is poised for a strong commercial launch of relugolix if approved, with two positive Phase 3 studies evaluating relugolix combination therapy in women with uterine fibroids and approximately $500 million in total cash and committed financing, including the Sumitomo Dainippon Pharma term loan facility," said Lynn Seely, M.D., President and CEO of Myovant Sciences. "Bringing a new treatment to the millions of women who suffer from uterine fibroids and endometriosis has been the key vision for the development of Myovant’s relugolix combination tablet. The use of a priority review voucher enables us to expedite the review of our NDA while strengthening the data package in the submission to support this objective."
Second Fiscal Quarter 2019 and Recent Business Highlights
Relugolix Phase 3 Clinical Programs

In July 2019, Myovant announced positive top-line data from LIBERTY 2, the second of two Phase 3 studies evaluating relugolix combination therapy (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg) in women with uterine fibroids and heavy menstrual bleeding. Results from a separate bioequivalence study support submission to the FDA of a potential one tablet, once-daily dosing regimen of relugolix combination therapy. In addition, the 12-month safety data from the LIBERTY open-label extension study are expected in the first quarter of calendar year 2020.

Roivant Sciences and Sumitomo Dainippon Pharma have committed to Myovant a priority review voucher (PRV) expected to become available in early December 2019. Myovant plans to use the PRV in conjunction with its NDA submission for a once-daily, relugolix combination tablet for the treatment of heavy menstrual bleeding and uterine fibroids, potentially decreasing the standard FDA review time. Myovant has decided to defer its NDA submission for a once-daily, relugolix combination tablet for the treatment of heavy menstrual bleeding and uterine fibroids until April 2020, which would allow inclusion of the complete 12-month safety data from the LIBERTY open-label extension study, key data that may positively impact the labeled duration of use of the combination tablet. The terms regarding how the PRV will be transferred to Myovant will be determined in connection with the closing of the Roivant-Sumitomo Dainippon Pharma transaction. The transfer is expected to be a related-party transaction and will not involve the issuance of Myovant shares. Myovant still plans to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency in the first quarter of calendar year 2020.

In July 2019, Myovant completed enrollment of an additional cohort of 139 men with metastatic prostate cancer in the Phase 3 HERO study in order to assess the secondary objective of demonstrating that relugolix can delay the time to progression of the lethal state of the disease, castration-resistant prostate cancer, as compared to leuprolide. Myovant expects the top-line data readout for the HERO study by the end of calendar year 2019, with results from this additional cohort, including the castration resistance-free survival endpoint, expected in the third quarter of calendar year 2020. Myovant also anticipates submitting its NDA for its once-daily, oral relugolix monotherapy tablet for men with advanced prostate cancer in the second quarter of calendar year 2020.

In August and October 2019, Myovant completed patient recruitment for the Phase 3 SPIRIT 2 and SPIRIT 1 studies, respectively, evaluating the safety and efficacy of relugolix combination therapy in women with pain associated with endometriosis. Myovant expects to report top-line results from SPIRIT 2 and SPIRIT 1 in the first and second quarters of calendar year 2020, respectively.
Corporate

In October 2019, Myovant entered into a landmark agreement with Sumitomo Dainippon Pharma to secure a $350 million low-interest, five-year term loan facility, with no payments due until the end of the term, and an Investors Rights Agreement, which is intended to provide safeguards to minority shareholders. The term loan facility and Investors Rights Agreement are expected to become effective in connection with the closing of Sumitomo Dainippon Pharma’s transaction with Roivant.
Second Fiscal Quarter 2019 Financial Summary
Research and development (R&D) expenses for the quarter ended September 30, 2019, were $50.8 million compared to $53.8 million for the comparable prior year period. R&D expenses in both periods primarily include expenses related to Myovant’s Phase 3 clinical studies, manufacturing expenses as well as personnel-related expenses for employees engaged in R&D activities. R&D expenses related to Myovant’s clinical studies have continued to decline driven primarily by the wind down of Myovant’s LIBERTY Phase 3 studies. The decrease in study costs were partially offset by increases in other R&D expenses related predominantly to Myovant’s manufacturing activities in connection with preparations for Myovant’s anticipated commercial launches and regulatory submissions for relugolix in multiple indications and jurisdictions, as well as increases in personnel expenses, share-based compensation expense, and other R&D expenses.
General and administrative (G&A) expenses for the quarter ended September 30, 2019, were $16.6 million compared to $10.3 million for the comparable prior year period. The increase primarily reflects increases in personnel-related expenses, share-based compensation, professional service fees, expenses related to commercial operations activities in advance of potential regulatory approvals of relugolix, and other general overhead and administrative expenses to support Myovant’s headcount growth and expanding operations which was in part driven by the assumption of activities previously provided by Roivant.
Interest expense for the quarter ended September 30, 2019, was $3.8 million compared to $1.6 million in the comparable prior year period. The increase for the quarter was primarily the result of higher outstanding debt balances under Myovant’s financing agreements as compared to the prior year period.

Interest income for the quarter ended September 30, 2019, was $0.9 million. There was no interest income for the quarter ended September 30, 2018. During the quarter ended September 30, 2019, a portion of Myovant’s cash was invested in a combination of money market funds, commercial paper, and short-term corporate bonds. There were no such investments during the comparable prior year period.
Net loss for the quarter ended September 30, 2019, was $70.6 million, compared to $65.8 million for the comparable prior year period. The increase in the net loss for the quarter was driven primarily by the increase in costs outlined above. On a per common share basis, net loss was $0.79 and $0.99 for the quarters ended September 30, 2019, and 2018, respectively. The decrease in the net loss per common share for the quarter was due to an increase in the weighted-average common shares outstanding primarily as a result of Myovant’s underwritten public equity offering in June 2019.
Capital resources: Cash, cash equivalents, and marketable securities totaled $157.6 million as of September 30, 2019. Myovant’s term loan facility to be entered into with Sumitomo Dainippon Pharma, which is expected to become effective in connection with the close of the Sumitomo-Roivant transaction, is expected to provide an additional $350.0 million of capital to support Myovant’s operations.
About Relugolix
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces ovarian estradiol production, a hormone known to stimulate the growth of uterine fibroids and endometriosis, and testicular testosterone production, a hormone known to stimulate the growth of prostate cancer. Myovant Sciences is developing a relugolix combination tablet (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg) for women with heavy menstrual bleeding associated with uterine fibroids and for women with endometriosis-associated pain. Myovant is also evaluating relugolix monotherapy (120 mg once daily) in men with advanced prostate cancer.
About MVT-602
MVT-602 is an oligopeptide kisspeptin-1 receptor agonist. Kisspeptin, the ligand, is a naturally-occurring peptide that stimulates GnRH release and is required for puberty and maintenance of normal reproductive function, including production of sperm, follicular maturation and ovulation, and production of estrogen and progesterone in women and testosterone in men. A Phase 2a clinical study in healthy female volunteers to characterize the dose-response curve in a minimal controlled ovarian stimulation setting has been completed.

On November 12, 2019 Verastem, Inc. (NASDAQ: VSTM) (the "Company") reported that it has entered into privately negotiated agreements among the Company and a limited number of investors who are accredited investors (within the meaning of Rule 501 promulgated under the Securities Act of 1933, as amended (the "Securities Act")) and/or qualified institutional buyers (as defined in Rule 144A under the Securities Act) who are holders of the Company’s existing 5.00% convertible senior notes due 2048 (the "existing notes") to exchange approximately $114.3 million aggregate principal amount of existing notes for (i) approximately $62.9 million aggregate principal amount of newly issued 5.00% convertible senior second lien notes due 2048 (the "exchange notes") and (ii) an aggregate of approximately $11.6 million in cash (such transactions, collectively, the "exchange") (Press release, Verastem, NOV 12, 2019, View Source [SID1234550941]). For each $1,000 principal amount of existing notes being exchanged, the Company will deliver to the exchanging investor $550 principal amount of exchange notes and a cash payment of $100, together with accrued and unpaid interest on the existing notes (plus, if applicable, an adjustment in lieu of issuing fractional exchange notes). The exchange is expected to close on November 14, 2019, subject to customary closing conditions. The Company will not receive any cash proceeds from the exchange.

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The exchange notes will be issued pursuant to an indenture by and between the Company and Wilmington Trust, National Association (the "indenture"). After giving effect to the issuance of the exchange notes and the exchange of the existing notes pursuant to the exchange, approximately $62.9 million aggregate principal amount of the exchange notes is expected to be issued and outstanding and approximately $35.7 million aggregate principal amount of the existing notes is expected to remain issued and outstanding.

The exchange notes will be senior secured obligations of the Company and pay interest semiannually in arrears at a rate of 5.00% per annum on May 1 and November 1 of each year, beginning on May 1, 2020. The exchange notes will mature on November 1, 2048 (the "maturity date"), unless earlier converted, redeemed or repurchased by us, and will be secured by a second lien on the assets of the Company securing indebtedness issued under its existing senior secured credit facility. The exchange notes will be convertible at the option of the holder into shares of common stock of the Company, par value $0.0001 per share ("common stock"), at any time prior to the close of business on the scheduled trading day immediately preceding the maturity date. The conversion rate for the exchange notes is 606.0606 shares of the Company’s common stock per $1,000 principal amount of the exchange notes (subject to certain adjustments), which is equivalent to an initial conversion price of approximately $1.65 per share, representing an approximately 52.8% premium to the last reported sale price of $1.08 per share of common stock on November 11, 2019, as reported on the Nasdaq Global Market.

Upon conversion of the exchange notes, holders will receive a cash payment equal to the accrued and unpaid interest on the converted exchange notes. In addition, prior to November 1, 2020, holders who convert their exchange notes (other than in connection with the exercise of the Company’s issuer conversion option) will also be entitled to an interest make-whole equal to the sum of all regularly scheduled stated interest payments, if any, due on such exchange notes on each interest payment date occurring after the conversion date for such conversion and on or before November 1, 2020.

The Company will have the right, exercisable at its option, to cause all exchange notes then outstanding to be converted automatically if the volume-weighted average price per share of the Company’s common stock equals or exceeds 121% of the conversion price for a specified period of time and certain other conditions are satisfied.

Prior to November 1, 2022, the Company will not have the right to redeem the exchange notes. On or after November 1, 2022, the Company may elect to redeem the exchange notes, in whole or in part, at a cash redemption price equal to the principal amount of the exchange notes to be redeemed, plus accrued and unpaid interest, if any.

Holders of the exchange notes will have the right, at their option, to require the Company to purchase their exchange notes (i) on November 1, 2023, November 1, 2028, November 1, 2033, November 1, 2038 and November 1, 2043, or (ii) if a "fundamental change" (as defined in the indenture) occurs, in each case, at a repurchase price equal to 100% of the principal amount of the exchange notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the applicable repurchase date.

In connection with the exchange, the Company intends to enter into an amendment to its senior secured credit agreement.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy the notes or any other securities, and shall not constitute an offer, solicitation or sale in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful.

The offer and sale of the exchange notes or the shares of common stock issuable upon their conversion have not been registered under the Securities Act or any state securities laws. The notes and such shares may not be offered or sold except pursuant to an exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and any applicable state securities laws.

On November 12, 2019 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the third quarter ended September 30, 2019, and provided a corporate update (Press release, Cellectar Biosciences, NOV 12, 2019, View Source [SID1234550940]).

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Third Quarter and Recent Corporate Highlights

. Presented data from the DLBCL cohort in the company’s Phase 2 CLOVER-1 study of CLR 131 in relapsed or refractory select B-cell malignancies at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2019. The oral presentation featured data from 6 subjects, who received up to a single 30-minute intravenous (IV) dose of 25mCi/m2 of CLR 131. Data showed durable responses with a mean of 312 days (ongoing) and includes a 33% overall response rate (ORR), a 16.6% complete response rate (CR) and a 50% clinical benefit rate (CBR). CLR 131 had activity in both DLBCL subtypes (germinal center B-cell and activated B-cell) and patients with highly resistant/aggressive cytogenetics (known as "dual hit"). All patients enrolled in the study received an average of 3 prior lines of systemic therapy, 5 of 6 patients were refractory to at least one prior line of therapy.

·Presented a poster entitled: "Phospholipid ether delivery vehicle shows specificity for a broad range of tumor cells and provides a novel and improved approach for targeted therapy," at the Cancer Research UK-AACR Joint Conference on Engineering and Physical Sciences in Oncology. The poster featured data demonstrating that phospholipid ether drug conjugates (PDCs) were capable of delivering small molecule cytotoxins selectively to tumor cells and were well tolerated in animal models.

·Presented data from cohort 6 of its Phase 1 dose escalation study of CLR 131 in relapsed or refractory MM, in a late breaker poster at the 17th International Myeloma Workshop. Data highlighted 4 subjects in cohort 6 who received a fractionated dose of 37.5mCi/m2. Subjects in this cohort achieved a 50% ORR, with two subjects achieving partial responses (PR) and two subjects achieving minimal responses (39% and 48% reduction in M protein). CLR 131 was deemed safe and tolerated in all subjects with cytopenias being the only reported treatment emergent adverse events of grade 3 or higher. The majority (75%) of the subjects had high risk cytogenetics where median bone marrow plasma cell involvement was 25%. Patients’ median age was 72.5 and they averaged 5 prior systemic therapies, with one patient being dual class refractory, one being quad-refractory, and two penta-refractory.

·Received a second FDA Fast Track Designation for CLR 131 for relapsed/refractory DLBCL. CLR 131 is currently being evaluated in patients with select B-cell lymphomas, including MM and DLBCL in the Phase 2 CLOVER-1 clinical study.

·Successfully completed the first cohort of malignant brain tumor patients in the ongoing Phase 1 study of CLR 131 in children and adolescents with select solid tumors, lymphoma, and malignant brain tumors, including relapsed or refractory neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma, and osteosarcoma. The independent Data Monitoring Committee determined the 15mCi/m2 single dose to be safe and tolerated and recommended the company progress to a second cohort utilizing a 30mCi/m2 single dose of CLR 131.

· Received orphan drug designation from the European Commission for CLR 131 in the treatment of multiple myeloma. The designation provides ten years of market exclusivity for the treatment of multiple myeloma.

·Strengthened the management team with the appointment of Dov Elefant, Chief Financial Officer.

"We continue to effectively manage our financial resources to fund our budgeted operations into 2021 while advancing multiple clinical and preclinical programs. The third quarter proved to be highly productive for Cellectar with CLR 131 receiving FDA Fast Track Designation for DLBCL and Orphan Drug Designation for multiple myeloma from the European Commission. In addition, we presented incremental positive data at international scientific conferences," said Jim Caruso, CEO of Cellectar. "We continue to anticipate data from our ongoing clinical studies by the end of this year and in early 2020."

Third Quarter and Nine Months Summary of Financial Results

Cash and Cash Equivalents: As of September 30, 2019, cash and cash equivalents were approximately $13.3 million compared to $13.3 million as of December 31, 2018. The company believes the cash balance is adequate to fund its budgeted operations into the first quarter 2021. Cash used in operating activities was approximately $9.0 million during the nine months ended September 30, 2019 as compared to $8.7 million used during the nine months ended September 30, 2018.

Research and Development Expense: Research and development (R&D) expense for the three months ended September 30, 2019 was $2.7 million compared to $2.0 million in the three months ended September 30, 2018. The cumulative R&D spending for the first nine months of 2019 was $6.8 million as compared to $5.8 million for the first nine months of 2018. The majority of the company’s R&D spend for year-to-date 2019 was dedicated to the start-up costs and support of our pediatric study. Clinical project costs and manufacturing expenses were $2.1 million and $5.0 million for the three and nine months ending September 30, 2019, respectively.

General and Administrative Expense: General and administrative (G&A) expense for the three months ended September 30, 2019 was approximately $1.3 million compared to approximately $1.1 million in the three months ended September 30, 2018. The cumulative G&A spending for the first nine months of 2019 were of $4.0 million as compared to $3.6 million for the first nine months of 2018.

Net Loss: Net loss attributable to common stockholders for the three months ended September 30, 2019 was $(3.9) million, or a loss of $(0.42) per diluted share, compared to a net loss of $(5.3) million, or a loss of $(1.65) per diluted share, in the three months ended September 30, 2018. Net loss attributable to common stockholders for the nine months ended September 30, 2019 was $(10.7) million, or a loss of $(1.51) per diluted share, compared to a net loss of $(11.7) million, or a loss of $(5.29) per diluted share, in the nine months ended September 30, 2018.

About CLR 131

CLR 131 is a small-molecule, targeted Phospholipid Drug Conjugate (PDC) designed to deliver cytotoxic radiation directly to cancer cells, while limiting exposure to healthy cells. CLR 131 is the company’s lead product candidate and is currently being evaluated in a Phase 2 study in B-cell lymphomas, and two Phase 1 dose-escalating clinical studies, one in multiple myeloma and one in pediatric solid tumors and lymphoma. CLR 131 was granted Orphan Drug designation for the treatment of multiple myeloma, and was granted Orphan Drug and Rare Pediatric Disease designations for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma.