Insmed Announces Pricing of Public Offering of Common Stock

On May 5, 2020 Insmed Incorporated (Nasdaq: INSM) reported that it priced a registered underwritten public offering of 9,700,000 shares of its common stock at a price to the public of $23.25 per share (Press release, Insmed, MAY 5, 2020, View Source [SID1234557062]). The gross proceeds to Insmed from the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Insmed, are expected to be approximately $225.5 million. In addition, Insmed has granted the underwriters a 30-day option to purchase up to an additional 1,455,000 shares of common stock.

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Insmed intends to use its net proceeds from this offering to continue to commercialize ARIKAYCE (amikacin liposome inhalation suspension); conduct further trials of ARIKAYCE, including Insmed’s required confirmatory trial to assess and describe the clinical benefit of ARIKAYCE in patients with Mycobacterium avium complex (MAC) lung disease; conduct further trials of brensocatib (formerly known as INS1007), including its planned Phase 3 program in bronchiectasis; fund further clinical development of treprostinil palmitil (formerly known as INS1009); invest in third-party manufacturing capacity; fund business expansion activities in Europe and Japan; fund working capital, potential debt repayment, capital expenditures, and general research and development; and for other general corporate purposes, which may include the acquisition or in-license of additional compounds, product candidates, technology or businesses.

SVB Leerink is acting as sole bookrunning manager for the offering. Credit Suisse and Stifel are acting as co-lead managers for the offering. JMP Securities and H.C. Wainwright & Co. are acting as co-managers for the offering. The offering is expected to close on May 7, 2020, subject to the satisfaction of customary closing conditions.

A shelf registration statement on Form S-3 relating to the public offering of the shares of common stock described above has been filed with the Securities and Exchange Commission (SEC), as amended by Post-Effective Amendment No. 1 thereto, and became automatically effective upon filing on May 19, 2017. A preliminary prospectus supplement relating to and describing the terms of the offering was filed with the SEC and is available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus related to this offering may be obtained, when available, from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 02110, by telephone: 1-800-808-7525, ext. 6218 or by email at [email protected].

PTC Therapeutics to Participate at Upcoming Investor Conferences

On May 5, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that management will present in a virtual format at the following conferences (Press release, PTC Therapeutics, MAY 5, 2020, View Source [SID1234557061]):

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2020 BofA Securities Health Care Conference
Tuesday, May 12th at 3:40 p.m. ET

2020 RBC Capital Markets Global Healthcare Conference
Wednesday, May 20th at 8:35 a.m. ET

The presentations will be webcast live on the Events and Presentations page under the investor relations section of PTC Therapeutics’ website at www.ptcbio.com and will be archived for 30 days following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

VolitionRx Limited Schedules First Quarter 2020 Earnings Conference Call and Business Update

On May 5, 2020 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported it will host a conference call on Friday, May 8, at 8:30 a.m. Eastern time to discuss its financial and operating results for the first quarter 2020, in addition to providing a business update (Press release, VolitionRX, MAY 5, 2020, View Source [SID1234557060]).

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Event: VolitionRx Limited First Quarter 2020 Earnings and Business Update Conference Call
Date: Friday, May 8, 2020
Time: 8:30 a.m. Eastern time
U.S. & Canada Dial-in: 1-877-407-9716 (toll free)
U.K. Dial-in: 0 800 756 3429 (toll free)
Toll/International: 1-201-493-6779
Conference ID: 13703451

Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with David Vanston, Chief Financial Officer and Scott Powell, Executive Vice President, Investor Relations. The call will provide an update on recent developments and Volition’s activities, including details of new and ongoing clinical trials, important events which have taken place in the first quarter of 2020, and milestones for 2020 and beyond.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source In addition, a telephone replay of the call will be available until May 22, 2020. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13703451.

Antengene Announces Expansion of Partnership with Karyopharm in Asia Pacific Markets

On May 5, 2020 Antengene Corporation (Antengene) reported a broadened partnership and territory expansion agreement with Karyopharm Therapeutics Inc. (NASDAQ: KPTI) (Karyopharm) for development and commercialization of four oral novel drugs and drug candidates to support its mission of treating patients beyond borders (Press release, Antengene, MAY 5, 2020, View Source [SID1234557059]). This agreement broadens Antengene’s rights in the Asia Pacific region for XPOVIO (selinexor, aka ATG-010), the first-in-class selective inhibitor of nuclear export (SINE); eltanexor (ATG-016), a second-generation SINE compound; verdinexor (ATG-527), a lead compound in development for anti-viral and other non-oncology indications; and KPT-9274 (ATG-019), a dual inhibitor of PAK4 and NAMPT.

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In May 2018, Antengene and Karyopharm entered into a strategic collaboration for the development, manufacturing and commercialization of XPOVIO and eltanexor for all human oncology indications in mainland China and Macau; and KPT-9274 in all human oncology indications and verdinexor in human non-oncology indications in mainland China, Macau, Taiwan, Hong Kong, South Korea, and the ASEAN markets.

As part of the expanded territory, Antengene has received extended rights to develop, manufacture and commercialize XPOVIO and eltanexor in Australia, New Zealand, South Korea, Taiwan, Hong Kong, and the entire ASEAN markets; KPT-9274 and verdinexor rights have also been extended to Australia and New Zealand.

This expanded collaboration broadens Antengene’s portfolio and geographic footprint to additional Asian markets. In July 2019, the U.S. FDA approved XPOVIO in combination with low-dose dexamethasone for the treatment of adult patients with relapsed/refractory multiple myeloma (RRMM). The data from the recently released phase 3 BOSTON trial comparing SVd to Vd in 2nd and later lines in RRMM patients, has been selected as a late-breaking abstract for oral presentation at the upcoming virtual ASCO (Free ASCO Whitepaper) conference later this month.

Antengene is presently expanding its commercial operations to support the launch of XPOVIO in mainland China and other APAC markets; and conducting clinical trials with XPOVIO in RRMM, diffuse large B-cell lymphoma (DLBCL) and T-cell lymphomas in China. Antengene has also announced the ongoing recruitment of patients with advanced solid tumor and non-Hodgkin’s lymphoma in a clinical trial with ATG-019 (KPT-9274). In addition, clinical trials of ATG-016 (eltanexor) and ATG-527 (verdinexor) will be initiated in these markets in 2020.

"Upon the achievement of significant regulatory and clinical milestones in mainland China and other markets in Asia, Antengene is now expanding to additional Asia Pacific markets with clinical development and commercialization. We are pleased to have expanded our collaboration and partnership with Karyopharm in developing these novel drugs for patients in Asia Pacific," said Jay Mei, MD, PhD, Chairman and Chief Executive Officer of Antengene. "This critical geographic expansion is a step in our development and commercialization strategy, and we are building a world-class commercial team while continuing to add clinical and commercial-stage first-in-class novel drugs to address unmet medical needs and to further expand our strong pipeline. This is a further testament to our mission of treating patients beyond borders."

"We are delighted to have the opportunity to expand our very productive, efficient and results-driven collaboration with Antengene which began two years ago," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "The exemplary partnership between the two companies enables Karyopharm to fulfill its mission to develop and deliver novel drug candidates to patients around the world."

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. Karyopharm received accelerated U.S. Food and Drug Administration (FDA) approval of XPOVIO in July 2019 in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. Karyopharm has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval of selinexor. A supplemental New Drug Application was accepted by the FDA seeking accelerated approval for selinexor as a new treatment for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), and selinexor has received Fast Track and Orphan designation and Priority Review from the FDA with a scheduled PDUFA date of June 23, 2020 for this patient population. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), for which Karyopharm announced positive top-line results in March 2020. Additional, ongoing trials for selinexor include as a potential backbone therapy in combination with approved myeloma therapies (STOMP), in liposarcoma (SEAL) and in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

In China, Antengene is conducting registration clinical trials in relapsed refractory multiple myeloma (MARCH) and in diffuse large B-cell lymphoma (SEARCH), and has initiated the clinical trial for the treatment of peripheral T-cells lymphoma and NK / T-cell lymphoma (TOUCH).

About Eltanexor

Eltanexor (ATG-016) is a second generation oral SINE compound. Eltanexor functions by binding to and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. Eltanexor has demonstrated minimal brain penetration in animals, which has been associated with reduced toxicities in preclinical studies while maintaining potent anti-tumor effects. A Phase 1/2 clinical study is currently ongoing evaluating eltanexor in myelodysplastic syndrome, colorectal cancer and castrate-resistant prostate cancer.

About Verdinexor

Verdinexor (ATG-527) is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound being investigated across a variety of non-oncology indications in humans with an initial focus as a potential broad-spectrum treatment for viral diseases. Verdinexor functions by binding to and inhibiting the nuclear export protein XPO1 (also called CRM1), which is believed to be responsible for the movement of critical host cell and pathogen encoded cargoes across the nuclear membrane into the cytoplasm. Inhibition of this process with verdinexor results in accumulation of these cargoes in the nucleus, where they promote an anti-inflammatory state and prevent key steps in pathogen replication from occurring. Prior preclinical research showed efficacy of verdinexor in several viral models, including HIV and promising pre-clinical data has also been observed in multiple additional non-oncology indications. In a previously conducted randomized, double-blind, placebo-controlled, dose-escalating Phase 1 clinical trial in healthy human volunteers, verdinexor was found to be generally safe and well tolerated, with adverse events occurring in similar number and grade as placebo.

About KPT-9274

KPT-9274 (ATG-019) is a first-in-class, orally bioavailable, small molecule immunometabolic modulator that works through non-competitive dual inhibition of p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT). NAMPT and NAPRT (Nicotinate Phosphoribosyltransferas) are the two main pathways for production of the NAD (nicotinamide dinucleotide). About 15-30% of all solid tumors are deficient in NAPRT, making them reliant on NAMPT for NAD production. Co-inhibition of PAK4 and NAMPT is believed to lead to synergistic anti-tumor effects through suppression of ß-catenin by blocking PAK4, leading to both immune cell activation and inhibition of tumor growth, blockade of DNA repair, cell cycle arrest, and energy depletion through NAMPT inhibition, and ultimately apoptosis. KPT-9274 may therefore have both immune-activating and direct antitumor effects. Tumors deficient in NAPRT may be particularly susceptible to KPT-9274’s actions. In contrast, normal cells are less sensitive to inhibition by KPT-9274 due in part to their relative genomic stability and lower metabolic demands. KPT-9274 is currently being evaluated in a Phase 1 clinical study in advanced solid tumors and non-Hodgkin’s lymphoma.

KYPROLIS® (carfilzomib) Patents Upheld Against Generic Manufacturer

On May 5, 2020 Amgen (NASDAQ:AMGN) reported that the U.S. District Court in Delaware issued a decision upholding the validity of patent claims from three patents that protect Amgen’s multiple myeloma therapy KYPROLIS (carfilzomib) (Press release, Amgen, MAY 5, 2020, View Source [SID1234557057]). Today’s decision will prevent Cipla Limited, and Cipla USA, Inc. (collectively "Cipla") from making, using, selling, offering to sell, or importing its generic version of KYPROLIS until expiration of these three U.S. patents. The latest patent expiry is in December 2027.

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Onyx Therapeutics, Inc., an indirect, wholly-owned subsidiary of Amgen Inc., brought a patent infringement suit against Cipla in 2016. The decision comes after the Delaware court held a bench trial in May 2019. Prior to trial, Cipla acknowledged that its generic product would infringe all of the asserted claims, leaving only the issue of validity of the asserted patents to be addressed by the court.

KYPROLIS is approved for the treatment of patients with relapsed or refractory multiple myeloma who previously received one to three lines of other therapy. KYPROLIS is also indicated in combination with dexamethasone or with lenalidomide plus dexamethasone. Three patents that are the subject of the decision are: U.S. Patent No. 7,417,042, U.S. Patent No. 7,737,112 and U.S. Patent No. 8,207,125.

About KYPROLIS

INDICATIONS

KYPROLIS (carfilzomib) is indicated in combination with dexamethasone or with lenalidomide plus dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy.

KYPROLIS is indicated as a single agent for the treatment of patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy.

IMPORTANT SAFETY INFORMATION FOR KYPROLIS

Cardiac Toxicities

New onset or worsening of pre-existing cardiac failure (e.g., congestive heart failure, pulmonary edema, decreased ejection fraction), restrictive cardiomyopathy, myocardial ischemia, and myocardial infarction including fatalities have occurred following administration of KYPROLIS. Some events occurred in patients with normal baseline ventricular function. Death due to cardiac arrest has occurred within one day of administration.

Monitor patients for signs or symptoms of cardiac failure or ischemia. Evaluate promptly if cardiac toxicity is suspected. Withhold KYPROLIS for Grade 3 or 4 cardiac adverse events until recovery, and consider whether to restart at 1 dose level reduction based on a benefit/risk assessment.

While adequate hydration is required prior to each dose in Cycle 1, monitor all patients for evidence of volume overload, especially patients at risk for cardiac failure. Adjust total fluid intake as clinically appropriate.

For patients ≥ 75 years, the risk of cardiac failure is increased. Patients with New York Heart Association Class III and IV heart failure, recent myocardial infarction, conduction abnormalities, angina, or arrhythmias may be at greater risk for cardiac complications and should have a comprehensive medical assessment prior to starting treatment with KYPROLIS and remain under close follow-up with fluid management.

Acute Renal Failure

Cases of acute renal failure, including some fatal renal failure events, and renal insufficiency adverse events (including renal failure) have occurred. Acute renal failure was reported more frequently in patients with advanced relapsed and refractory multiple myeloma who received KYPROLIS monotherapy. Monitor renal function with regular measurement of the serum creatinine and/or estimated creatinine clearance. Reduce or withhold dose as appropriate.

Tumor Lysis Syndrome

Cases of Tumor Lysis Syndrome (TLS), including fatal outcomes, have occurred. Patients with a high tumor burden should be considered at greater risk for TLS. Adequate hydration is required prior to each dose in Cycle 1, and in subsequent cycles as needed. Consider uric acid lowering drugs in patients at risk for TLS. Monitor for evidence of TLS during treatment and manage promptly, and withhold until resolved.

Pulmonary Toxicity

Acute Respiratory Distress Syndrome (ARDS), acute respiratory failure, and acute diffuse infiltrative pulmonary disease such as pneumonitis and interstitial lung disease have occurred. Some events have been fatal. In the event of drug-induced pulmonary toxicity, discontinue KYPROLIS.

Pulmonary Hypertension

Pulmonary arterial hypertension (PAH) was reported. Evaluate with cardiac imaging and/or other tests as indicated. Withhold KYPROLIS for PAH until resolved or returned to baseline and consider whether to restart based on a benefit/risk assessment.

Dyspnea

Dyspnea was reported in patients treated with KYPROLIS. Evaluate dyspnea to exclude cardiopulmonary conditions including cardiac failure and pulmonary syndromes. Stop KYPROLIS for Grade 3 or 4 dyspnea until resolved or returned to baseline. Consider whether to restart based on a benefit/risk assessment.

Hypertension

Hypertension, including hypertensive crisis and hypertensive emergency, has been observed, some fatal. Control hypertension prior to starting KYPROLIS. Monitor blood pressure regularly in all patients. If hypertension cannot be adequately controlled, withhold KYPROLIS and evaluate. Consider whether to restart based on a benefit/risk assessment.

Venous Thrombosis

Venous thromboembolic events (including deep venous thrombosis and pulmonary embolism) have been observed. Thromboprophylaxis is recommended for patients being treated with the combination of KYPROLIS with dexamethasone or with lenalidomide plus dexamethasone. The thromboprophylaxis regimen should be based on an assessment of the patient’s underlying risks.

Patients using hormonal contraception associated with a risk of thrombosis should consider an alternative method of effective contraception during treatment.

Infusion Reactions

Infusion reactions, including life-threatening reactions, have occurred. Signs and symptoms include fever, chills, arthralgia, myalgia, facial flushing, facial edema, laryngeal edema, vomiting, weakness, shortness of breath, hypotension, syncope, chest tightness, or angina. These reactions can occur immediately following or up to 24 hours after administration. Premedicate with dexamethasone to reduce the incidence and severity of infusion reactions. Inform patients of the risk and of symptoms and seek immediate medical attention if they occur.

Hemorrhage

Fatal or serious cases of hemorrhage have been reported. Hemorrhagic events have included gastrointestinal, pulmonary, and intracranial hemorrhage and epistaxis. Promptly evaluate signs and symptoms of blood loss. Reduce or withhold dose as appropriate.

Thrombocytopenia

KYPROLIS causes thrombocytopenia with recovery to baseline platelet count usually by the start of the next cycle. Monitor platelet counts frequently during treatment. Reduce or withhold dose as appropriate.

Hepatic Toxicity and Hepatic Failure

Cases of hepatic failure, including fatal cases, have occurred. KYPROLIS can cause increased serum transaminases. Monitor liver enzymes regularly regardless of baseline values. Reduce or withhold dose as appropriate.

Thrombotic Microangiopathy

Cases of thrombotic microangiopathy, including thrombotic thrombocytopenic purpura/hemolytic uremic syndrome (TTP/HUS), including fatal outcome, have occurred. Monitor for signs and symptoms of TTP/HUS. Discontinue if diagnosis is suspected. If the diagnosis of TTP/HUS is excluded, KYPROLIS may be restarted. The safety of reinitiating KYPROLIS is not known.

Posterior Reversible Encephalopathy Syndrome (PRES)

Cases of PRES have occurred in patients receiving KYPROLIS. If PRES is suspected, discontinue and evaluate with appropriate imaging. The safety of reinitiating KYPROLIS is not known.

Increased Fatal and Serious Toxicities in Combination with Melphalan and Prednisone in Newly Diagnosed Transplant-ineligible Patients

In a clinical trial of transplant-ineligible patients with newly diagnosed multiple myeloma comparing KYPROLIS, melphalan, and prednisone (KMP) vs bortezomib, melphalan, and prednisone (VMP), a higher incidence of serious and fatal adverse events was observed in patients in the KMP arm. KMP is not indicated for transplant-ineligible patients with newly diagnosed multiple myeloma.

Embryo-fetal Toxicity

KYPROLIS can cause fetal harm when administered to a pregnant woman.

Females of reproductive potential should be advised to avoid becoming pregnant while being treated with KYPROLIS and for 6 months following the final dose. Males of reproductive potential should be advised to avoid fathering a child while being treated with KYPROLIS and for 3 months following the final dose. If this drug is used during pregnancy, or if pregnancy occurs while taking this drug, the patient should be apprised of the potential hazard to the fetus.

Adverse Reactions

The most common adverse reactions in the combination therapy trials: anemia, neutropenia, diarrhea, dyspnea, fatigue, thrombocytopenia, pyrexia, insomnia, muscle spasm, cough, upper respiratory tract infection, hypokalemia.

The most common adverse reactions in monotherapy trials: anemia, fatigue, thrombocytopenia, nausea, pyrexia, dyspnea, diarrhea, headache, cough, edema peripheral.