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XBiotech Announces Preliminary Results of Tender Offer

On February 13, 2020 XBiotech Inc. (NASDAQ: XBIT) ("XBiotech") reported the preliminary results of its "modified Dutch auction" tender offer, which expired at 5:00 p.m., New York City time, on February 12, 2020 (Press release, XBiotech, FEB 13, 2020, View Source [SID1234554303]).

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Based on the preliminary count by American Stock Transfer & Trust Co., LLC, the depositary for the tender offer, a total of 41,164,725 common shares of XBiotech were properly tendered and not properly withdrawn at or below the maximum purchase price of $33.00 per share, including 2,178,233 shares that were tendered by notice of guaranteed delivery.

In accordance with the terms and conditions of the tender offer and based on the preliminary count by the depositary, XBiotech expects to accept for payment, at a purchase price of $30.00, approximately 14,000,000 common shares properly tendered at or below the purchase price and not properly withdrawn before the expiration date, at an aggregate cost of approximately $420 million, excluding fees and expenses relating to the tender offer. The preliminary proration factor for shares that XBiotech expects to purchase pursuant to the tender offer is approximately 30.91 percent, before giving effect to odd lots.

The number of shares to be purchased and the purchase price are preliminary and subject to change. The preliminary information contained in this press release is subject to confirmation by the depositary and is based on the assumption that all shares tendered through notice of guaranteed delivery will be delivered within the two trading day settlement period. The final number of shares to be purchased and the final purchase price will be announced following the expiration of the guaranteed delivery period and completion by the depositary of the confirmation process. Payment for the shares accepted for purchase under the tender offer, and return of all other shares tendered and not purchased, will occur promptly thereafter.

D.F. King & Co., Inc. is the information agent for the Offer. Shareholders with questions may call D.F. King at (212) 269-5550 (banks and brokers) or (866) 856-3065 (all others).

About True Human Therapeutic Antibodies

XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.

Actinium to Highlight Targeted Conditioning Portfolio at 2020 Transplantation & Cellular Therapy Annual Meeting; Phase 3 SIERRA Trial Preliminary Results Selected for Oral Presentation

On February 13, 2020 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported that presentations from its targeted conditioning portfolio have been accepted for presentation at the 2020 Transplantation & Cellular Therapy (TCT) Meetings, which brings together thousands of transplant professionals from over 500 transplant centers worldwide (Press release, Actinium Pharmaceuticals, FEB 13, 2020, View Source [SID1234554302]). TCT is being held February 19-23, 2020 at the Marriott World Center in Orlando, Florida. Notably, data from the pivotal Phase 3 SIERRA trial of Iomab-B have been selected for an oral presentation.

"We are excited that Iomab-B and the SIERRA trial have once again been selected as an oral presentation at TCT," said Dr. Mark Berger, Chief Medical Officer of Actinium. "We look forward to highlighting the potential benefit that Iomab-B can provide to a patient population with active disease who are otherwise ineligible for BMT. We are confident these findings will be received with great enthusiasm. TCT, which assembles leading transplant physicians from top centers in the United States and worldwide, is the ideal venue to showcase the extremely encouraging findings from the SIERRA trial thus far. In addition, our other conference activities are expected to provide significant exposure for this important trial and invaluable interactions with BMT thought leaders. Through the SIERRA trial, we aspire to change the treatment paradigm for older patients with relapsed or refractory AML to make potentially curative BMT via Iomab-B the standard of care for this patient population that continues to have poor outcomes."

Actinium’s TCT Presentations:

Late Breaking Oral Presentation:

Title: Targeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Leads to High Rates of Allogeneic Transplantation and Successful Engraftment in Older Patients with Active, Relapsed or Refractory (rel/ref) AML after Failure of Chemotherapy and Targeted Agents: Preliminary Midpoint Results from the Prospective, Randomized Phase 3 Sierra Trial
Presenter: Boglarka Gyurkocza MD, Memorial Sloan Kettering Cancer Center (and SIERRA Investigator)
Time: Thursday, February 20th, 4:45 PM – 5:00 PM ET
Location: Orlando World Center Marriott – Palms: Sabal
Poster Presentation:

Title: Feasibility of Administering Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] for Targeted Conditioning in Older Patients with Active, Relapsed or Refractory AML without Lead-Lined Rooms: Ongoing Phase 3 Sierra Trial Experience at 6 Study Sites
Presenter: Rajneesh Nath, M.D., Banner MD Anderson Cancer Center (and SIERRA Investigator)
Time: Wednesday, February 19th, 6:30 PM – 8:00 PM ET
Location: Orlando World Center Marriott – Palms: Sabal
About the SIERRA Trial
The SIERRA trial (Study of Iomab-B in Elderly Relapse/Refractory Acute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. Iomab-B is an ARC (Antibody Radiation-Conjugate) comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume. For more information, visit www.sierratrial.com.

About Transplantation & Cellular Therapy Meetings (TCT)
TCT, formerly known as the BMT Tandem Meetings, are the combined annual meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood & Marrow Transplant Research (CIBMTR). Each year the conference brings together several thousand investigators, clinicians, researchers, nurses and other allied health professionals from over 500 transplant centers from over 50 countries around a full scientific program focused on bone marrow transplant and cellular therapies.

Seattle Genetics Announces FDA Filing Acceptance for Priority Review of Tucatinib New Drug Application (NDA) for Patients with Locally Advanced or Metastatic HER2-Positive Breast Cancer

On February 13, 2020 Seattle Genetics, Inc. (Nasdaq:SGEN) reported that the U.S. Food and Drug Administration (FDA) has accepted for priority review the Company’s New Drug Application (NDA) for the investigational medicine tucatinib (Press release, Seattle Genetics, FEB 13, 2020, View Source [SID1234554301]). This NDA requests FDA approval of tucatinib in combination with trastuzumab and capecitabine for treatment of patients with locally advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received at least three prior HER2-directed agents separately or in combination, in the neoadjuvant, adjuvant or metastatic setting. The filing is based on the results of HER2CLIMB, a randomized pivotal trial comparing tucatinib added to trastuzumab and capecitabine versus trastuzumab and capecitabine alone. HER2CLIMB trial results were recently presented at the 2019 San Antonio Breast Cancer Symposium and published in the New England Journal of Medicine(NEJM). Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of August 20, 2020. Tucatinib is an oral, small molecule tyrosine kinase inhibitor (TKI) that is highly selective for HER2.

"The FDA’s filing of the tucatinib NDA marks an important step forward for patients with locally advanced or metastatic HER2-positive breast cancer, including those with brain metastases," said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. "We are working collaboratively with the FDA throughout the review process to bring this important medicine to patients as quickly as possible."

The NDA for tucatinib was submitted in December 2019 and is being reviewed under the Real-Time Oncology Review (RTOR) Pilot Program. The review of the tucatinib NDA is also being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among participating international partners. Tucatinib was recently granted Breakthrough Therapy designation by the FDA in combination with trastuzumab and capecitabine for the treatment of patients with locally advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have been treated with trastuzumab, pertuzumab, and T-DM1. This designation was based on data from the pivotal HER2CLIMB trial.

About HER2-Positive Breast Cancer

Patients with HER2-positive breast cancer have tumors with high levels of a protein called human epidermal growth factor receptor 2 (HER2), which promotes the growth of cancer cells. An estimated 271,270 new cases of invasive breast cancer will be diagnosed in the U.S. in 2019.1 Between 15 and 20 percent of breast cancer cases worldwide are HER2-positive.2 Historically, HER2-positive breast cancer tends to be more aggressive and more likely to recur than HER2-negative breast cancer.2, 3, 4 In patients with metastatic breast cancer, the most common site of first metastasis is in bone, followed by lung, brain, and liver.5, 6 Up to 50 percent of metastatic HER2-positive breast cancer patients develop brain metastases over time.2, 7 Despite recent treatment advances, there is still a significant need for new therapies that can impact metastatic disease, especially brain metastases. There are currently no approved therapies demonstrating progression-free survival or overall survival benefit for the treatment of patients with HER2-positive metastatic breast cancer after progression on T-DM1.8, 9, 10

About Tucatinib

Tucatinib is an investigational, orally bioavailable, potent tyrosine kinase inhibitor that is highly selective for HER2 without significant inhibition of EGFR. Inhibition of EGFR has been associated with significant toxicities, including skin rash and diarrhea. Tucatinib has shown activity as a single agent and in combination with both chemotherapy and other HER2 targeted agents such as trastuzumab.1,2 Studies of tucatinib in these combinations have shown activity both systemically and in brain metastases. HER2 is a growth factor receptor that is overexpressed in multiple cancers, including breast, colorectal and gastric cancers. HER2 mediates cell growth, differentiation and survival. Tucatinib has been granted orphan drug designation by the FDA for the treatment of breast cancer patients with brain metastases.

In addition to HER2CLIMB, tucatinib is being evaluated in a randomized, double-blind, placebo-controlled, multi-center phase 3 trial of tucatinib called HER2CLIMB-02. This trial is evaluating tucatinib in combination with T-DM1 compared to T-DM1 alone, in patients with unresectable locally advanced or metastatic HER2-positive breast cancer, including those with brain metastases, who have had prior treatment with a taxane and trastuzumab. The primary endpoint is progression-free survival (PFS) per RECIST criteria. Secondary endpoints include overall survival, objective response rate and duration of response. This global trial is expected to enroll approximately 460 patients. More information about the phase 3 trial, including enrolling centers, is available at www.clinicaltrials.gov.

Tucatinib is also being evaluated in a multi-center, open-label, single-arm phase 2 clinical trial known as MOUNTAINEER, with tucatinib in combination with trastuzumab in patients with HER2-positive, RAS wildtype metastatic or unresectable colorectal cancer. The primary endpoint of the trial is overall response rate by RECIST criteria. Duration of response, PFS, overall survival and safety and tolerability of the combination regimen are secondary objectives. Results for the first 26 patients were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress. Enrollment of up to 110 patients is ongoing. More information about the MOUNTAINEER trial, including enrolling centers

Synthetic Biologics to Report 2019 Year End Operational Highlights and Financial Results on February 20, 2020

On February 13, 2020 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need, reported operational highlights and financial results for the year ended December 31, 2019 on Thursday, February 20, 2020, and will host a conference call the same day at 4:30 p.m. ET (Press release, Synthetic Biologics, FEB 13, 2020, View Source [SID1234554300]). The dial-in information for the call is as follows:

U.S. (toll free): 1-888-347-5280
International: +1 412-902-4280

Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source." target="_blank" title="View Source." rel="nofollow">View Source An archived replay of the call will be available for approximately ninety (90) days at the same URL, View Source beginning approximately one hour after the call’s conclusion.

Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update

On February 13, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter and full year ended December 31, 2019 and reaffirmed its financial guidance for 2020 (Press release, Ultragenyx Pharmaceutical, FEB 13, 2020, View Source [SID1234554297]).

"In 2019 we created a substantial foundation based on the strong launch of Crysvita and the validation of our gene therapy platform across multiple clinical programs," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In 2020, we anticipate building on this momentum as we look to the potential approvals of UX007 for LC-FAOD and Crysvita for TIO. We also expect to advance our gene therapy programs into pivotal studies and bring two new programs into the clinic while continuing to exercise financial discipline."

Fourth Quarter and Full Year 2019 Financial Results

Net Revenues
For the fourth quarter of 2019, Ultragenyx reported $35.6 million in total revenue. Ultragenyx recognized $29.9 million in total Crysvita revenue. This includes $26.1 million in collaboration revenue in the North American1 profit share territory and $2.2 million in royalty revenue in the European territory from the collaboration and license agreement with Kyowa Kirin Co., Ltd (KKC). Net product sales for Crysvita in other regions were $1.6 million. Mepsevii (vestronidase alfa) product revenue for the fourth quarter of 2019 was $4.4 million, and UX007 named patient revenue was $1.2 million. Ultragenyx recognized $0.1 million in revenue from its research agreement with Bayer.

Net revenue for the year ended December 31, 2019 totaled $103.7 million. In 2019, Ultragenyx recognized $87.3 million in total Crysvita revenue. This includes $74.9 million in collaboration revenue in the North American1 profit share territory and $8.1 million in royalty revenue in the European territory from the collaboration and license agreement with KKC. Net product sales for Crysvita in other regions totaled $4.3 million. Mepsevii product revenue for the year ended December 31, 2019 was $12.6 million, and UX007 named patient revenue was $3.3 million. Ultragenyx recognized $0.5 million in revenue from its research agreement with Bayer in the year ended December 31, 2019.

Operating Expenses
Total operating expenses for the fourth quarter of 2019 were $130.0 million compared with $106.6 million for the same period in 2018, including non-cash stock-based compensation of $19.7 million and $21.1 million in the fourth quarter of 2019 and 2018, respectively. Total operating expenses for the year ended December 31, 2019, were $527.9 million, which includes $15.6 million in expenditures for Arcturus collaboration and license amendment and $20 million for the upfront payment on the GeneTx agreement, compared with operating expenses of $422.9 million for the same period in 2018. The operating expenses include non-cash stock-based compensation of $82.0 million and $80.1 million in the full year of 2019 and 2018, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its pipeline.

For the fourth quarter of 2019, Ultragenyx reported a net loss of $93.8 million, or $1.62 per share, basic and diluted, compared with a net loss for the fourth quarter of 2018 of $87.8 million, or $1.73 per share, basic and diluted. For the year ended December 31, 2019, the net loss was $402.7 million, or $7.12 per share, basic and diluted, compared with a net loss for the same period in 2018 of $197.6 million, or $3.97 per share, basic and diluted. The loss for the fourth quarter of 2019 and for the year ended December 31, 2019 includes unrealized gains of $1.4 million and $13.4 million, respectively, from the fair value adjustments on the investment in Arcturus equity securities. The loss from the full year 2018 was reduced by the gain on the sale of the Mepsevii priority review voucher (PRV) in January 2018 for net proceeds of $130.0 million and a $40.3 million gain from Ultragenyx’s portion of the sales of the PRV received with the Crysvita approval. The net loss for the full year 2019 reflected cash used in operations of $345.4 million compared to $290.6 million for the same period in 2018.

Cash, Cash Equivalents and Investments
Cash, cash equivalents, and investments were $760.4 million as of December 31, 2019, including proceeds of $320.0 million received from the sale of the company’s royalty interest in Crysvita in the European territory.

2020 Guidance

Crysvita Guidance in Ultragenyx Territories
In 2020, the company continues to expect Crysvita revenue in the Ultragenyx territories to be between $125 million and $140 million, which excludes the European territory revenue. Ultragenyx territories include the collaboration revenue from the North American profit share territory (US and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). In December 2019, the European territory royalty revenue rights were sold to Royalty Pharma. As a result, the company will no longer receive cash payments from the EU territory royalty revenue rights beginning January 1, 2020, until the respective threshold amount is met; however, the company will continue to record the royalty as "non-cash" revenue.

2020 Expected Net Cash Burn Rate
The company also expects a more than 20 percent reduction in net cash burn (net cash used in operations plus capital expenditures) in 2020 compared to 2019 due to a combination of financial discipline in spending with flattening operating expense growth, combined with the significantly larger growth in revenue.

Recent Updates and Upcoming Milestones

Crysvita for X-linked Hypophosphatemia: Non-dilutive $320 million sale of future European royalties

In December 2019, Ultragenyx sold to Royalty Pharma for $320 million its royalty interest in Crysvita in the European territory, where it is being commercialized by Kyowa Kirin Co., Ltd.
Crysvita for Tumor-Induced Osteomalacia (TIO): Supplemental Biologics License Application (sBLA) submitted

Ultragenyx submitted the sBLA to the U.S. FDA on December 18, 2019 and expects to hear back from FDA on submission acceptance and review designation in February 2020.
UX007 for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): NDA under review by U.S. FDA

The U.S. FDA accepted for review the New Drug Application (NDA) and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application. The review process is on track for a decision by the PDUFA date.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Positive data from higher dose cohort of Phase 1/2 study; data from prophylactic steroid cohort in second half 2020

Recent positive data from Cohort 3 indicated two confirmed responders and a potential third responder out of three total patients, as well as a new responder in Cohort 2. There are currently up to six responders of the nine dosed to date with a more consistent response at higher doses.
Ultragenyx is initiating a fourth cohort (n=3) using prophylactic steroids at the same dose as Cohort 3. Data from the prophylactic steroid cohort are expected in the second half of 2020.
DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Positive data from Phase 1/2 study; enrollment complete in confirmatory cohort and data expected in first half 2020

Enrollment is complete in the confirmatory cohort of three patients at the second dose cohort, with data expected in the first half of 2020. Following the results of the confirmatory cohort, a Phase 3 study could begin in the second half of 2020.
GTX-102 for Angelman Syndrome (AS): Partnered program with GeneTx; IND active and enrollment of first patient expected in first half 2020

An investigational new drug (IND) application is now active and GeneTx has received Institutional Review Board (IRB) approval from the first study site. Enrollment in the Phase 1/2 study of GTX-102 is expected to begin in the first half of 2020.
In February 2020, Ultragenyx paid $25 million after the acceptance of the IND to maintain its option to acquire GeneTx until the earlier of 30 months from the first dosing of a patient in the Phase 1/2 study (subject to extensions) or 90 days after results are available from that study.
UX701 for Wilson Disease: IND expected by the end of the year

An IND application is expected by the end of 2020 for a new gene therapy for Wilson disease, a larger rare metabolic disease. UX701 will be the company’s second clinical program to utilize its HeLa manufacturing system.
DTX201 / BAY 2599023 for Hemophilia A: Partnered with Bayer; Cohort 2 data presented at European Association for Haemophilia and Allied Disorders (EAHAD)

Three cohorts with two patients each, six patients in total, have been dosed with AAVhu37 (DTX201 / BAY 2599023), using material from Ultragenyx’s proprietary HeLa manufacturing platform. Longer term dose cohort 1 and new dose cohort 2 data presented at EAHAD showed three of four patients have achieved clinically meaningful FVIII levels. One patient in Cohort 1 has achieved clinically meaningful FVIII levels and has experienced four bleeds since receiving treatment down from 99 bleeds the prior year. Both patients in dose cohort 2 achieved clinically meaningful FVIII levels out to 24 and 30 weeks. Patient 4 has been off treatment and bleed free for seven months as of the data cut-off date. The second patient in dose cohort 2 had mild ALT/AST elevations that was managed with a short tapering course of corticosteroids.
Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, February 13, 2020, at 2 p.m. PT/ 5 p.m. ET to discuss fourth quarter and full year 2019 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 5068548. The replay of the call will be available for one year.