On February 4, 2019 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") reported that the Medical College of Wisconsin (MCW) has started dosing patients in the second cohort of its novel trial of Actimab-A in combination with CLAG-M in patients with relapsed or refractory AML or Acute Myeloid Leukemia (Press release, Actinium Pharmaceuticals, FEB 4, 2019, View Source [SID1234533052]). This trial is evaluating the impact that the addition of targeted internalized radiation via Actimab-A to the salvage chemotherapy regimen CLAG-M will have on safety and tolerability, response rates, rates of BMT or bone marrow transplant, PFS or progression-free survival, and OS or overall survival.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Dr. Mark Berger, Chief Medical Officer of Actinium, said, "This trial represents an exciting advancement of our CD33 program that is aligned with our clinical strategy to pursue combinations utilizing our ARC drug candidates. We are pleased to be moving ahead with MCW to the second cohort of this trial and we are optimistic that our ARC combination strategy will have a positive impact on patient outcomes by improving response rates, duration of responses and/or increasing the rate of patients receiving a bone marrow transplant. Chemotherapy and external radiation are routinely used in combination in several cancers but despite being radiation sensitive, AML is not treated with external radiation given its diffuse nature. Therefore, the ability to deliver radiation internally in a targeted fashion to AML cells with potency and tolerability gives us great confidence in our ARC approach. In addition, this trial is particularly important as AML patients with relapsed or refractory disease face a poor prognosis with limited treatment options."

In this Phase 1 combination trial patients are administered the salvage chemotherapy regimen CLAG-M, which consists of cladribine, cytarabine, filgrastim, and mitoxantrone, followed by a single dose of Actimab-A. Actimab-A is an ARC or Antibody Radiation-Conjugate that consists or the CD33 targeting monoclonal antibody lintuzumab labelled with the alpha-particle emitting isotope Ac-225 or Actinium-225. In the first dose cohort, patients received 0.25 uCi/kg of Actimab-A. This combination trial is designed as a 3+3 dose escalation study. No dose limiting toxicities (DLTs) were reported in the first patient cohort. As a result, and per the study protocol, the Institutional Review Board (IRB) at MCW has authorized the initiation of the second dosing cohort, in which patients will receive 0.50 uCi/kg of Actimab-A. Assuming no DLTs are observed in the second cohort, three patients will be treated and the study will progress to the third and final cohort will study Actimab-A at a dose of 0.75 uCi/kg.

Sandesh Seth, Actinium’s Chairman and Chief Executive Officer of Actinium, said, "In recent months, Actinium’s R&D and clinical teams have worked together to identify opportunities to further utilize our ARCs in combination with other therapeutic modalities. As a result, we are exploring multiple R&D and clinical initiatives with ARC based combination therapies. With the ARC combination strategy solidified as a corporate focus, we are delighted to see this positive progress from our first Actimab-A combination trial with CLAG-M. We are confident that the use of targeted radiation will prove synergistic with multiple modalities and open several therapeutic opportunities that are not possible with any other technology. We look forward to making continued progress on this front including the planned clinical trials with Actimab-A and venetoclax."

On February 4, 2019 In conjunction with World Cancer Day, the Union for International Cancer Control (UICC) and Pfizer Inc. reported the third round of the Seeding Progress And Resources for the Cancer Community (SPARC): Metastatic Breast Cancer (MBC) Challenge: their pioneering grants initiative designed to support the implementation of projects worldwide that address the specific needs of women with metastatic breast cancer in local communities – many of which extend beyond treatment (Press release, Pfizer, FEB 4, 2019, View Source [SID1234533050]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A total of US$275,000 in funding will be awarded to 10 new organizations, bringing the SPARC awardees network to 50 cancer organizations from all across the globe with a total funding of US$1,565,000. As part of this new round, five grantees from Round 2 of the SPARC MBC Challenge will receive additional funding to continue their projects.

In addition to receiving the seed grant to support their projects, the new awardees will benefit from trainings, best-practice sharing workshops, and mentoring. They will also have the opportunity to participate in key global convening cancer events to network, share knowledge, and showcase their work.

Dr Cary Adams, Chief Executive Officer of the Union for International Cancer Control, said: "We are proud that, together with Pfizer, we will continue to improve the lives of people living with metastatic breast cancer cross the world through this unique programme that combines seed grants, mentoring and peer-to-peer
learning."

The impact of the SPARC MBC Challenge has been significant. Since the initiative launched in 2015, over 8,000 patients from 30 countries have been reached directly through SPARC projects, and more than 125,000 people have benefitted from SPARC advocacy campaigns and information materials. Organizations such as Project Pink Blue and Run for a Cure Africa have reached the next level receiving international awards and additional funds after receiving the SPARC grant. In particular, SPARC contributed to:
Supporting metastatic breast cancer patients through projects such as the creation of an online app to guide patients in finding financial resources to cover some of the expenses of the disease in Canada; a project for young women to better cope with MBC through integrative oncology techniques (occupational therapy, expressive creative techniques, mind body intervention, and acupuncture) in Mexico and training for patients on the importance of treatment adherence in Bulgaria
Strengthening advocates and supporting the development of a positive national policy environment for MBC. SPARC projects have delivered training on advocacy for metastatic breast cancer for individuals from 47 European countries, supported the recent inclusion of medicines for MBC in the National Essential Medicines list in Kyrgyzstan and contributed to the development of supportive national legislation on cancer in Brazil and Nigeria.
Reducing the information gap for patients, with information materials and advocacy campaigns created in 26 local languages. SPARC enabled the creation of the first navigational programs in Nigeria and Trinidad & Tobago.
Building local capacity. Nearly 2,000 health professionals were trained on how to manage the specific needs of MBC patients across all SPARC projects. In Australia, an e-learning training was deployed across the country to help nurses better attend to the needs of MBC patients.
"We’re proud of the far-reaching impact that the SPARC MBC Challenge has had over the past four years on metastatic breast cancer patients around the world, ranging from educational resources to tailored programming," said Andy Schmeltz, Global President, Pfizer Oncology, "With our ongoing partnership with UICC and this year’s renewal, we look forward to continuing to make a difference for breast cancer patients around the globe."

Applications to the new round of the SPARC grants will open at the end of February and new SPARC awardees will be announced in October 2019.

About the Seeding Progress and Resources for the Cancer Community (SPARC): Metastatic Breast Cancer (MBC) Challenge

The SPARC MBC Challenge aims to address critical issues for those at risk of, or living with metastatic breast cancer. The SPARC grants consist of seed-funding, capacity building and convening opportunities to launch and strengthen new projects addressing the needs of MBC patients. Awardees are integrated into the SPARC network where they can share best practices, learn from others and share resources between themselves. The awardees are selected by an independent, external steering committee, consisting of leading experts in the fields of cancer care and chaired by globally renowned oncologist and leading breast cancer advocate, Dr Fatima Cardoso.

On February 4, 2019 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported that management will present company overviews at the following conferences (Press release, Alnylam, FEB 4, 2019, View Source [SID1234533048]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

21st Annual BIO CEO & Investor Conference on Monday, February 11, 2019 at 1:30 pm ET at the Marriott Marquis in New York City

8th Annual SVB Leerink Global Healthcare Conference onWednesday, February 27, 2019 at 10:30 am ET at the Lotte New York Palace Hotel in New York City

A live audio webcast of each presentation will be available on the Investors section of the Company’s website, www.alnylam.com. A replay will be available on the Alnylam website within 48 hours after each event.

On February 4, 2019 Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) reported financial results for the fourth quarter and full year of 2018 (Press release, Alexion, FEB 4, 2019, View Source [SID1234533046]). Total revenues for the full year of 2018 were $4,131.2 million, a 16 percent increase compared to 2017. The positive impact of foreign currency on total revenues year-over-year was less than one percent, or $5.9 million, inclusive of hedging activities. On a GAAP basis, diluted earnings per share (EPS) for the full year of 2018 was $0.35, an 82 percent decrease versus the prior year, inclusive of $1,183.0 million of expense related to the value of the in-process research and development assets acquired in 2018. Non-GAAP diluted EPS for the full year of 2018 was $7.92, a 35 percent increase versus the prior year.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Total revenues in the fourth quarter were $1,128.8 million, a 24 percent increase compared to the same period in 2017. The negative impact of foreign currency on total revenues year-over-year was 1 percent, or $5.5 million, inclusive of hedging activities. On a GAAP basis, diluted EPS in the quarter was $(0.20), a 254 percent decrease versus the prior year, inclusive of $379.3 million of expense related to the value of the in-process research and development asset acquired in connection with our acquisition of Syntimmune. Non-GAAP diluted EPS for the fourth quarter of 2018 was $2.14, a 45 percent increase versus the fourth quarter of 2017.

"2018 was a transformational year for Alexion. I am proud of our many accomplishments, including entering rare neurology and making SOLIRIS for gMG Alexion’s best launch ever, completing the Phase 3 program and launching ULTOMIRIS for PNH in the U.S., delivering groundbreaking Phase 3 data and submitting regulatory filings for SOLIRIS in NMOSD and rebuilding our pipeline through disciplined business development, all while continuing to grow our base business," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "As we look at 2019 and beyond, I am confident that this strong foundation positions us well to achieve our Four Pillars of growth, building durable, blockbuster franchises in PNH/aHUS, metabolics, neurology and FcRn."

Full Year 2018 Financial Highlights

Total net product sales were $4,130.1 million, compared to $3,549.5 million in 2017.
SOLIRIS (eculizumab) net product sales were $3,563.0 million, compared to $3,144.1 million in 2017, representing a 13 percent increase. SOLIRIS volume increased 16 percent year-over-year.
STRENSIQ (asfotase alfa) net product sales were $475.1 million, compared to $339.8 million in 2017, representing a 40 percent increase. STRENSIQ volume increased 47 percent year-over-year.
KANUMA (sebelipase alfa) net product sales were $92.0 million, compared to $65.6 million in 2017, representing a 40 percent increase. KANUMA volume increased 60 percent year-over-year.
GAAP cost of sales was $374.3 million, compared to $454.2 million in 2017. Non-GAAP cost of sales was $352.5 million, compared to $285.8 million in 2017.
GAAP R&D expense was $730.4 million, compared to $878.4 million in 2017. Non-GAAP R&D expense was $646.2 million, compared to $736.3 million in 2017.
GAAP SG&A expense was $1,111.8 million, compared to $1,094.4 million in 2017. Non-GAAP SG&A expense was $953.3 million, compared to $927.8 million in 2017.
GAAP income tax expense was $164.6 million, compared to $104.5 million in 2017. Non-GAAP income tax expense was $310.0 million, compared to $186.7 million in 2017.
GAAP diluted EPS was $0.35, inclusive of $1,183.0 million of expense related to the value of the in-process research and development assets acquired in 2018, compared to $1.97 in 2017. Non-GAAP diluted EPS was $7.92, compared to $5.86 in 2017.
GAAP net income includes total restructuring and related expenses of $50.7 million in 2018, compared to $286.5 million in 2017. GAAP cost of sales includes restructuring related expenses of $5.8 million in 2018, compared to $152.1 million in 2017, GAAP R&D expense includes $0.1 million in 2018, compared to $16.3 million in 2017 and GAAP SG&A expense includes $19.4 million in 2018, compared to $10.9 million in 2017.
Fourth Quarter 2018 Financial Highlights

Total net product sales were $1,128.5 million in the fourth quarter of 2018, compared to $909.4 million in the fourth quarter of 2017.
SOLIRIS net product sales were $976.7 million, compared to $791.9 million in the fourth quarter of 2017, representing a 23 percent increase. SOLIRIS volume increased 28 percent year-over-year.
STRENSIQ net product sales were $126.1 million, compared to $95.6 million in the fourth quarter of 2017, representing a 32 percent increase. STRENSIQ volume increased 43 percent year-over-year.
KANUMA net product sales were $25.7 million, compared to $21.9 million in the fourth quarter of 2017, representing a 17 percent increase. KANUMA volume increased 51 percent year-over-year.
GAAP cost of sales was $96.8 million, compared to $144.6 million in the fourth quarter of 2017. Non-GAAP cost of sales was $93.0 million, compared to $72.5 million in the fourth quarter of 2017.
GAAP R&D expense was $205.6 million, compared to $265.0 million in the fourth quarter of 2017. Non-GAAP R&D expense was $164.0 million, compared to $188.6 million in the fourth quarter of 2017.
GAAP SG&A expense was $318.7 million, compared to $296.4 million in the fourth quarter of 2017. Non-GAAP SG&A expense was $278.0 million, compared to $245.2 million in the fourth quarter of 2017.
GAAP income tax expense was $12.1 million, compared to $59.3 million in the fourth quarter of 2017. Non-GAAP income tax expense was $88.5 million, compared to $46.8 million in the fourth quarter of 2017.
GAAP diluted EPS was $(0.20), inclusive of $379.3 million of expense related to the value of the in-process research and development asset acquired in connection with our acquisition of Syntimmune, compared to $0.13 in the fourth quarter of 2017. Non-GAAP diluted EPS was $2.14, compared to $1.48 in the fourth quarter of 2017.
GAAP net income includes total restructuring and related expenses of $0.5 million in 2018, compared to $95.1 million in 2017. The 2017 statement of operations includes restructuring related expenses of $69.1 million in GAAP cost of sales, $15.3 million in GAAP R&D expense and $4.5 million in GAAP SG&A expense.
Research and Development

PHASE 3

ULTOMIRIS (ravulizumab-cwvz) – Paroxysmal Nocturnal Hemoglobinuria (PNH): In December 2018, the U.S. Food and Drug Administration (FDA) approved ULTOMIRIS for adults with PNH. Applications for approval in the European Union (EU) and Japan are currently under review. In addition, a Phase 3 study of ULTOMIRIS in children and adolescents with PNH is currently underway.
ULTOMIRIS – Atypical Hemolytic Uremic Syndrome (aHUS): In January 2019, Alexion announced positive topline results from a Phase 3 study of ULTOMIRIS in complement inhibitor naïve patients with aHUS. The study met its primary objective with 53.6 percent of patients demonstrating complete thrombotic microangiopathy (TMA) response. The safety profile of ULTOMIRIS was generally consistent with that seen in Phase 3 studies in PNH. No cases of meningococcal infection were observed. Based on these results, Alexion plans to file for regulatory approval in the U.S. in the first half of 2019 followed by the EU and Japan. In addition, a Phase 3 study of ULTOMIRIS in adolescents and children with aHUS is currently underway.
ULTOMIRIS – Subcutaneous: In late 2018, Alexion initiated a single, PK-based Phase 3 study of ULTOMIRIS delivered subcutaneously once per week to support registration in PNH and aHUS. Data are expected in early 2020.
ULTOMIRIS – Generalized Myasthenia Gravis (gMG): Alexion plans to initiate a Phase 3 study of ULTOMIRIS in gMG in the first quarter of 2019.
ULTOMIRIS – Neuromyelitis Optica Spectrum Disorder (NMOSD): Alexion plans to initiate a Phase 3 study of ULTOMIRIS in NMOSD.
SOLIRIS – NMOSD: Alexion has submitted applications in the U.S. and the EU for the approval of eculizumab for NMOSD. These submissions are based on previously announced results from the Phase 3 PREVENT study, in which 97.9 percent of patients with anti-aquaporin-4 (AQP4) auto antibody-positive NMOSD receiving SOLIRIS on top of stable standard-of-care therapy were free of relapse at 48 weeks compared to 63.2 percent of patients receiving placebo. Alexion also plans to file for regulatory approval in Japan later this year.
ALXN1840 (WTX101) – Wilson Disease: Enrollment is underway in a Phase 3 study of ALXN1840 (WTX101) in Wilson disease, a rare genetic disorder with devastating hepatic and neurological consequences. The study is now powered for superiority versus standard-of-care therapy. ALXN1840 is a first-in-class oral copper-binding agent with a unique mechanism of action to access and bind to serum copper and promote its removal from the liver.
PHASE 1/2

ALXN1830 (SYNT001): In the fourth quarter of 2018, Alexion announced the completion of its acquisition of Syntimmune. The acquisition added anti-FcRn antibody ALXN1830 (SYNT001) to the company’s clinical pipeline. ALXN1830 is currently in Phase 1b/2a development in patients with warm autoimmune hemolytic anemia (WAIHA) and in patients with pemphigus vulgaris (PV) or pemphigus foliaceus (PF). In 2019, the company plans to initiate two pivotal trials – one in WAIHA following successful completion of the current Phase 1b/2a study and one in gMG.
ALXN1810 – Subcutaneous: A Phase 1 study of subcutaneous ALXN1210 co-administered with Halozyme’s ENHANZE drug-delivery technology, PH20, is underway. Pending co-formulation data, this next-generation subcutaneous formulation will be called ALXN1810 and has the potential to further extend the dosing interval to once every two weeks or once per month.
ULTOMIRIS – Amyotrophic Lateral Sclerosis (ALS): Alexion plans to initiate a proof-of-concept study for ULTOMIRIS in ALS.
ULTOMIRIS – Primary Progressive Multiple Sclerosis (PPMS): Alexion plans to initiate a proof-of-concept study for ULTOMIRIS in PPMS.
Caelum Biosciences – CAEL101 – Light Chain (AL) Amyloidosis: In January 2019, Alexion entered into a collaboration with Caelum Biosciences to develop CAEL101 for AL amyloidosis, a rare systemic disorder that causes misfolded immunoglobulin light chain protein to build up in and around tissues, resulting in progressive and widespread organ damage. CAEL101 is a first-in-class amyloid fibril targeted therapy designed to improve organ function by reducing or eliminating amyloid deposits in patients with AL amyloidosis. In a Phase 1a/1b study, CAEL101 demonstrated improved organ function, including cardiac and renal function, in patients with relapsed and refractory AL amyloidosis.
PRE-CLINICAL

Dicerna – GalXC: Alexion is collaborating with Dicerna Pharmaceuticals to jointly discover and develop up to four subcutaneously delivered GalXC RNA interference (RNAi) candidates, currently in pre-clinical development, for the treatment of complement-mediated diseases.
Complement Pharma – CP010: Alexion is collaborating with Complement Pharma to co-develop CP010, a pre-clinical C6 inhibitor that has the potential to treat multiple neurological disorders.

2019 financial guidance assumes a GAAP effective tax rate of 13 to 15 percent and non-GAAP effective tax rate of 14 to 16 percent.

Alexion’s financial guidance is based on current foreign exchange rates net of hedging activities and does not include the effect of acquisitions, license and collaboration agreements, intangible asset impairments, litigation charges, changes in fair value of contingent consideration or restructuring and related activity outside of the previously announced activities that may occur after the issuance of this press release.

Conference Call/Webcast Information:

Alexion will host a conference call/audio webcast to discuss the fourth quarter and full year 2018 results today at 8:00 a.m. Eastern Time. To participate in the call, dial 866-762-3111 (USA) or 210-874-7712 (International), conference ID 5972194 shortly before 8:00 a.m. Eastern Time. A replay of the call will be available for a limited period following the call. The audio webcast can be accessed on the Investor page of Alexion’s website at: View Source

On January 28, 2019 Cancer Genetics, Inc. (Nasdaq: CGIX), a leader in enabling precision medicine for immuno-oncology and genomic medicine through molecular markers and diagnostics, reported the pricing of a public offering of 15,217,392 shares of its common stock, offered at a price to the public of $0.23 per share, for gross proceeds of approximately $3.5 million, before deducting placement agent fees and other offering expenses payable by Cancer Genetics (Press release, Cancer Genetics, JAN 28, 2019, View Source [SID1234533044]). The offering is expected to close on or about January 31, 2019, subject to customary closing conditions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering. The offering is being conducted as a "best efforts" offering and the placement agent is not obligated to purchase any securities.

Cancer Genetics intends to use the net proceeds from this offering to pay any amounts required by its lenders, and if any proceeds remain available, to pay certain costs previously incurred in connection with its strategic initiatives and to fund working capital and other general corporate purposes.

A shelf registration statement on Form S-3 relating to the public offering of the shares of common stock described above was filed with the Securities and Exchange Commission ("SEC") and was declared effective on June 5, 2017. A preliminary prospectus supplement describing the terms of the offering has been filed with the SEC. The final terms of the offering will be disclosed in a final prospectus supplement and accompanying prospectus to be filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained, when available, from H.C. Wainwright & Co., LLC, 430 Park Avenue 3rd Floor, New York, NY 10022, or by calling (646) 975-6996 or by emailing [email protected] or at the SEC’s website at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. Any offer, if at all, will be made only by means of the prospectus supplement and accompanying prospectus forming a part of the effective registration statement.