On March 3, 2021 Protagonist Therapeutics, Inc. (Nasdaq: PTGX), reported it will host a conference call and webcast on Wednesday, March 10 at 4:30 pm ET/1:30 pm PT to discuss its fourth quarter and full year 2020 financial results (Press release, Protagonist, MAR 3, 2021, View Source [SID1234576044]).

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Conference Call and Webcast Information

To access the live call, dial 1-844-515-9178 (U.S./Canada) or 1-614-999-9313 (International) and refer to conference ID #: 7756175. A live webcast of the call will also be accessible on the Investors section of the Company’s website at www.protagonist-inc.com. The replay will be available on the Company’s website approximately two hours after the call.

On March 3, 2021 Bio-Techne Corporation (NASDAQ: TECH), a global life sciences company providing innovative tools and bioactive reagents for the research and clinical diagnostic communities, reported it has reached an agreement to acquire Asuragen, Inc. for initial consideration of $215 million in cash plus contingent consideration of up to $105 million upon the achievement of certain future milestones (Press release, Bio-Techne, MAR 3, 2021, View Source [SID1234576105]). The transaction will be financed through a combination of cash on hand and an existing revolving line of credit. Bio-Techne anticipates the acquisition to close in the fourth quarter of its fiscal 2021.

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Founded in 2006, Asuragen is headquartered in Austin, Texas and is a leader in the development, manufacturing and commercialization of genetic carrier screening and oncology testing kits. Asuragen’s products leverage proprietary chemistries which can be used on widely available platforms including, PCR, qPCR, capillary electrophoresis, and next-generation sequencing instruments. This platform agnostic approach enables its clinical laboratory customers to solve complex molecular diagnostic challenges and empowers hospital and regional labs to expand in-house testing capabilities, furthering the decentralization of the molecular diagnostics market, enabling quicker turn-around times and ultimately delivering better patient care. Asuragen’s headquarters includes a scalable, Good Manufacturing Practice (GMP)-compliant 50,000 square foot manufacturing facility as well as a CLIA-certified laboratory. In 2020, Asuragen generated greater than $30 million in revenue globally and its Chief Executive Officer, Matt McManus, will join the Bio-Techne team to continue to lead the legacy Asuragen business as well as the integration process.

Asuragen brings a leading portfolio of best-in-class molecular diagnostic and research products, including its FDA-cleared AmplideX Fragile X Diagnostic and Carrier Screening kit for the screening of prospective parents as potential carriers of Fragile X chromosomal abnormalities as well as its Quantitidex qPCR IS BCR-ABL kit to enable the monitoring of leukemia patients for minimal residual disease. In addition to its existing portfolio of innovative and enabling technologies, Asuragen’s pipeline includes expanded carrier screening panels for various pathologies recognized by The American College of Obstetricians and Gynecologists (ACOG) as areas of concern for prospective new parents. Bio-Techne sees multiple growth synergies following the closing of the transaction, including Asuragen’s capabilities and proven track record in productizing lab-developed tests and commercializing innovative molecular products for broader market adoption.

"Asuragen is very complimentary with Bio-Techne’s existing diagnostics franchise and the addition of this business is expected to drive growth synergies throughout the expanded portfolio," said Chuck Kummeth, President and Chief Executive Officer of Bio-Techne. "We are not only acquiring a financially strong and scalable business, building our diagnostic portfolio and expanding our bandwidth with an additional CLIA-certified and GMP compliant laboratory, but are also adding a team with deep expertise in the intricacies of the global regulatory environment and a proven track record of opening new market channels. This critical mass will be very beneficial to the Genomics and Diagnostics Segment as we commercialize our pipeline of liquid biopsy tests through our Exosome Diagnostics business and also opens the possibility of approaching the market with kitted versions of these products. We anticipate continued traction with Asuragen’s leading portfolio of molecular diagnostic kits for both clinical and research uses and see significant potential in its pipeline of expanded carrier screening panels. Asuragen is a great addition to the Bio-Techne team."

"The Asuragen team is extremely excited to be joining Bio-Techne at this point in our growth trajectory," said Matt McManus, Chief Executive Officer of Asuragen. "Bio-Techne’s global presence, reputation and relationships within the clinical diagnostic and research communities will enable Asuragen to broaden our reach and accelerate penetration into the high-growth molecular diagnostic markets addressed by our portfolio. I am honored to continue to lead the Asuragen team as a part of Bio-Techne. I would like to thank all of the great people of Asuragen for their committed efforts growing our company to this point and am looking forward to the next stage of growth under the Bio-Techne umbrella."

Fredrickson & Byron, P.A. is serving as Bio-Techne’s legal counsel. Perella Weinberg Partners LP is the financial advisor and Vinson & Elkins LLP is serving as legal counsel to Asuragen.

On March 3, 2021 Herantis Pharma Plc ("Herantis"), an innovative clinical stage biotech company pioneering new disease modifying and regenerative biologic and gene therapies, reported its Full Year Report for January – December 2020. It is available on Herantis’ website (Financial information) (Press release, Herantis Pharma, MAR 3, 2021, View Source,c3300578 [SID1234577483]).

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"2020 was a fast-paced, purpose-driven, and transitional year for Herantis as we enter the next chapter of development for our fascinating science and innovative disease modifying treatments for patients suffering from debilitating neurological and lymphatic diseases. The year was notable for favorable data in our key programs, a new strategy for our Parkinson’s program, and significant funds raised.

For CDNF, a natural biological molecule, we were very pleased to announce data in August confirming the drug successfully achieved its primary endpoint of safety and tolerability in a First-in-Human Phase I-II study in Parkinson’s disease patients. This was a momentous achievement successfully taking the drug into human subjects. Going forward, we will focus on new routes of administration including nose-to-brain (nasal spray) and skin injection (subcutaneous). This strategy is expected to expand the target population to earlier stage patients, accelerate clinical development, and increase the attractiveness of our CDNF asset to partners.

For xCDNF, an engineered peptide using only the smallest most potent fragments of CDNF, we generated impressive data confirming the potency of the compound plus its ability to cross the blood brain barrier to reach the brain tissue, both of which are critical elements for the success for this therapy. Importantly, as with the new CDNF administration routes above, xCDNF is administered via a simple skin injection without the need for surgery.

It is exciting to have two such compelling assets in our portfolio, and we very much look forward to developing these two programs with their respective merits.

For Lymfactin, our gene therapy product, we were very pleased to announce in November favorable 24-month follow-up review from Phase I Lymfactin trial in Breast Cancer Related Lymphedema (BCRL). Post the FY 2020 financial reporting period, results from Phase II study with Lymfactin in BCRL have been announced separately 2nd March.

We raised a total of approximately EUR 15 million during 2020 with two direct share issues in May and December. 2020 has been a year of considerable progress for Herantis, and we enter 2021 with an optimised foundation, solid business fundamentals, and exciting milestones planned for the year ahead. I am very proud of the accomplishments and the significant advancements Herantis has made this past year to shape its future."

On March 3, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that HOOKIPA’s management team will participate in the upcoming H.C. Wainwright Global Life Sciences Conference held virtually from March 9-10, 2021 (Press release, Hookipa Biotech, MAR 3, 2021, View Source [SID1234575988]):

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Presentation: The conference is being conducted in a virtual format and a webcast of the presentation will become available on Tuesday, March 09, 2021 at 7:00 AM ET

The webcast of the presentation will be available within the Investors & Media section of HOOKIPA’s website at View Source An archived replay will be accessible for 30 days following the event.

On March 3, 2021 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the fourth quarter and full year ended December 31, 2020 and provided a general business update (Press release, Ophthotech, MAR 3, 2021, View Source [SID1234576009]).

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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

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The Company also announced that patient enrollment and retention for GATHER2, its second Phase 3 clinical trial for Zimura (avacincaptad pegol), a novel complement C5 inhibitor, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) are progressing well with enrollment ahead of schedule. The Company is accelerating the timeline for when it expects to complete enrollment in GATHER2 to the third quarter of 2021. If the prespecified 12-month results from GATHER2 are positive, the Company plans to file applications with the results from GATHER1 and GATHER2 to the U.S. Food and Drug Administration and the European Medicines Agency for marketing approval of Zimura for GA.

"We start 2021 with significant momentum as we continue to enroll patients into our Zimura GATHER2 clinical trial and now expect to complete enrollment in the third quarter of this year," stated Glenn P. Sblendorio, Chief Executive Officer and President of Iveric Bio. "We are extremely encouraged by the progress of our clinical trial sites to enroll and retain patients. We continue to work with our investigators to provide a safe environment for patients, which we believe increases the patients’ comfort and confidence to participate in the GATHER2 clinical trial. We are committed to continuing patient enrollment and retention aggressively in the GATHER2 clinical trial while prioritizing patient safety."

Pravin U. Dugel, M.D., Chief Strategy and Business Officer of Iveric Bio added, "Although bringing Zimura to patients suffering from geographic atrophy secondary to age-related macular degeneration remains our top priority, in 2021 and thereafter we will continue to explore the potential development of Zimura in earlier stages of age-related macular degeneration as well as neovascular (wet) macular degeneration. Additionally, in our IC-500 program, we are pursuing HtrA1 inhibition as a target in the treatment of GA and potentially earlier stages of AMD. We are focused on advancing our pipeline of both therapeutic and gene therapy product candidates for treating retinal diseases with the potential to create long-term shareholder value."

Therapeutics Programs Targeting Age-Related Macular Degeneration

Zimura (avacincaptad pegol): Complement C5 Inhibitor

In April 2020, the U.S. FDA granted Fast Track designation for Zimura for the treatment of GA secondary to dry AMD.
In June 2020, the Company announced positive 18-month results from GATHER1, its first Phase 3 clinical trial for Zimura for the treatment of GA secondary to AMD. The 18-month data supports the previously announced 12-month data from this trial, at which time point Zimura met the pre-specified primary efficacy endpoint with statistical significance. Zimura was generally well tolerated after 18 months of administration.
In late June 2020, the Company announced that the first patient had been dosed in the GATHER2 clinical trial.
In September 2020, the Company announced that the positive 12-month Phase 3 results from its GATHER1 clinical trial with Zimura were published in Ophthalmology, the Journal of the American Academy of Ophthalmology.
In February 2021, Dr. Dugel presented the positive results from GATHER1 at the Angiogenesis, Exudation, Degeneration 2021 – Virtual Edition meeting.
The Company increased the enrollment target in its ongoing Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease. After initially enrolling 95 patients in this trial, the Company plans to enroll approximately 25 additional patients, with the goal of enrolling a total of 120 patients.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

The Company recently revised its development plans for IC-500 to include plans to investigate multiple dosing schedules for this product candidate. Based on current timelines, the Company expects to submit an IND to the FDA for IC-500 in GA secondary to AMD in the second half of 2022.
Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
The Company is preparing an IND for IC-100 and plans to meet with regulatory authorities to discuss its selected doses for a first-in-human clinical trial prior to the submission. The Company plans to file an IND for IC-100 with the FDA and begin enrolling patients in a Phase 1/2 clinical trial for IC-100 in the second half of 2021.
IC-200: BEST1-Related IRDs
The Company is completing IND-enabling activities for IC-200 and plans to file an IND for IC-200 with the FDA and begin enrolling patients in a Phase 1/2 clinical trial for IC-200 in the second half of 2021.
Minigene Programs
The Company, in collaboration with the University of Massachusetts Medical School (UMMS), continues to advance its minigene programs for Leber Congenital Amaurosis Type 10 (LCA10), autosomal recessive Stargardt Disease (ABCA4), and USH2A-related IRDs. The Company expects to select a lead construct for its LCA10 minigene program in the second quarter of 2021. The Company expects to obtain additional results from its Stargardt Disease minigene program in the first half of 2021. The Company expects to obtain preliminary results from its USH2A minigene program in the first half 2021.
Corporate Highlights

During 2020, the Company expanded its Board of Directors and management by adding a number of leading industry experts. Mark S. Blumenkranz, M.D., M.M.S., HJ Smead Professor Emeritus, Department of Ophthalmology, Stanford University joined the Company’s board of directors in July 2020. Pravin U. Dugel, M.D. joined the Company as Chief Strategy and Business Officer in March 2020. Dr. Dugel was previously Managing Partner, Retinal Consultants of Arizona and the Retinal Research Institute; Clinical Professor, USC Eye Institute, Keck School of Medicine, University of Southern California; and Founding Member, Spectra Eye Institute in Sun City, Arizona. Dhaval Desai, PharmD, joined the Company as Chief of Staff in August 2020. Previously, Mr. Desai served as Medical Unit Head at Novartis Eye Care.

In June 2020, the Company raised approximately $150 million in net proceeds in an underwritten public offering of common stock, and pre-funded warrants in lieu of common stock, and a concurrent private placement of common stock.

Fourth Quarter and Year End 2020 Operational Update and 2021 Cash Guidance

As of December 31, 2020, the Company had $210 million in cash, cash equivalents and available for sale securities.
The Company estimates its year-end 2021 cash, cash equivalents and available for sale securities will range between $130 and $140 million. The Company also estimates that its cash, cash equivalents and available for sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses, excluding any potential approval or sales milestones payable to Archemix Corp. or any commercialization expenses for Zimura, into 2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for Zimura, the progression of its IC-100 and IC-200 programs into the clinic, and the advancement of its IC-500 development program. These estimates also assume that the Company will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures related to potentially studying Zimura in other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies or commencement of new sponsored research programs, and any associated development the Company may pursue.
2020 Financial Highlights

R&D Expenses: Research and development expenses were $17.5 million for the quarter ended December 31, 2020, compared to $11.6 million for the same period in 2019. For the year ended December 31, 2020, research and development expenses were $62.8 million compared to $39.6 million for 2019. Research and development expenses increased primarily due to the initiation of GATHER2 and increased manufacturing activities for Zimura, increased manufacturing and preclinical development activities associated with the Company’s IC-100 and IC-200 gene therapy programs and the progression of its IC-500 development program.
G&A Expenses: General and administrative expenses were $8.0 million for the quarter ended December 31, 2020, compared to $6.3 million for the same period in 2019. For the year ended December 31, 2020 general and administrative expenses were $26.0 million compared to $21.6 million for 2019. General and administration expenses increased primarily due to increases in general consulting costs and professional fees.
Income Tax (Benefit): Income tax benefits of $3.7 million and $0.1 million for the years ended December 31, 2020 and 2019, respectively, were recognized to reflect the favorable settlement of local tax audits.
Net Income: The Company reported a net loss for the quarter ended December 31, 2020 of $25.4 million, or ($0.27) per diluted share, compared to a net loss of $17.5 million, or ($0.39) per diluted share, for the same period in 2019. For the year ended December 31, 2020, the Company reported a net loss of $84.5 million or ($1.14) per diluted share, compared to a net loss of $58.9 million or ($1.39) for the same period in 2019.
Conference Call/Web Cast Information

Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for March 3, 2021 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-221-3881 (USA) or 323-794-2590 (International), passcode 9597743. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA), passcode 9597743.