European Commission Approves Seagen’s TUKYSA® (tucatinib) for the Treatment of Patients with Locally Advanced or Metastatic HER2-Positive Breast Cancer

On February 12, 2021 Seagen Inc. (Nasdaq:SGEN) reported that the European Commission (EC) has granted marketing authorization for TUKYSA (tucatinib) in combination with trastuzumab and capecitabine for the treatment of adult patients with HER2-positive locally advanced or metastatic breast cancer who have received at least two prior anti-HER2 treatment regimens (Press release, Seagen, FEB 12, 2021, View Source [SID1234575035]). TUKYSA is an oral, small molecule tyrosine kinase inhibitor (TKI) of HER2, a protein that contributes to cancer cell growth.1,2

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"This approval is a significant advancement for patients in Europe, who will for the first time have an approved medicine demonstrating a survival benefit for HER2-positive metastatic breast cancer after disease progression following two standard anti-HER2 treatment regimens," said Prof. Dr. Med Volkmar Mueller, Deputy Director at the University Medical Center Hamburg-Eppendorf, Hamburg, Germany and investigator for the pivotal trial. "In the HER2CLIMB pivotal trial, the tucatinib combination regimen improved overall and progression-free survival compared to trastuzumab and capecitabine alone, including in patients with active, untreated or progressing brain metastases, a population with significant unmet need."

"The TUKYSA combination is a landmark therapy for patients with HER2-positive metastatic breast cancer with or without brain metastases, extending overall survival in these patients after two prior anti-HER2 treatment regimens," said Clay Siegall, Ph.D., Chief Executive Officer at Seagen. "We are pleased TUKYSA is now approved in Europe, and we look forward to further collaborating with individual countries to ensure it is available to patients."

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion for TUKYSA in December 2020. The approval of TUKYSA is valid in all countries of the European Union, as well as Norway, Liechtenstein, Iceland and Northern Ireland.

HER2CLIMB Efficacy and Safety

Patients who received TUKYSA in combination with trastuzumab and capecitabine in the pivotal trial had a 46 percent reduction in the risk of cancer progression or death (PFS), the primary endpoint, compared to patients who received trastuzumab and capecitabine alone (hazard ratio (HR)=0.54 [95% Confidence Interval (CI): 0.42, 0.71]; p<0.00001) and improved overall survival with a reduction in the risk of death by 34 percent (HR=0.66 [95% CI: 0.50, 0.87]; p=0.0048). The most common adverse reactions occurring in 20 percent or more of patients who received TUKYSA were diarrhea, nausea, vomiting, stomatitis, AST increase, ALT increase, and rash.1

The pivotal trial, HER2CLIMB, is a randomized (2:1), double-blind, placebo-controlled, active comparator, global trial that enrolled 612 patients with HER2-positive unresectable locally advanced or metastatic breast cancer who had previously received, either separately or in combination, trastuzumab, pertuzumab, and trastuzumab emtansine (T-DM1 SmPC).

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. In 2020, more than two million new cases of breast cancer were diagnosed worldwide, including 531,086 in Europe.3 Between 15 and 20 percent of breast cancer cases are HER2-positive.4 HER2-positive breast cancer tends to be more aggressive and more likely to recur than HER2-negative breast cancer.4,5,6 Up to 50 percent of metastatic HER2-positive breast cancer patients develop brain metastases over time.7,8,9

About TUKYSA (tucatinib)

TUKYSA is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. In vitro (in lab studies), TUKYSA inhibited phosphorylation of HER2 and HER3, resulting in inhibition of downstream MAPK and AKT signaling and cell growth (proliferation), and showed anti-tumor activity in HER2-expressing tumor cells. In vivo (in living organisms), TUKYSA inhibited the growth of HER2-expressing tumors. The combination of TUKYSA and the anti-HER2 antibody trastuzumab showed increased anti-tumor activity in vitro and in vivo compared to either medicine alone.

U.S. Important Safety Information

Warnings and Precautions

Diarrhea – TUKYSA can cause severe diarrhea including dehydration, hypotension, acute kidney injury, and death. In HER2CLIMB, 81% of patients who received TUKYSA experienced diarrhea, including 12% with Grade 3 diarrhea and 0.5% with Grade 4 diarrhea. Both patients who developed Grade 4 diarrhea subsequently died, with diarrhea as a contributor to death. The median time to onset of the first episode of diarrhea was 12 days and the median time to resolution was 8 days. Diarrhea led to dose reductions of TUKYSA in 6% of patients and discontinuation of TUKYSA in 1% of patients. Prophylactic use of antidiarrheal treatment was not required on HER2CLIMB.

If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Based on the severity of the diarrhea, interrupt dose, then dose reduce or permanently discontinue TUKYSA.
Hepatotoxicity – TUKYSA can cause severe hepatotoxicity. In HER2CLIMB, 8% of patients who received TUKYSA had an ALT increase >5 × ULN, 6% had an AST increase >5 × ULN, and 1.5% had a bilirubin increase >3 × ULN (Grade ≥3). Hepatotoxicity led to dose reduction of TUKYSA in 8% of patients and discontinuation of TUKYSA in 1.5% of patients.

Monitor ALT, AST, and bilirubin prior to starting TUKYSA, every 3 weeks during treatment, and as clinically indicated. Based on the severity of hepatotoxicity, interrupt dose, then dose reduce or permanently discontinue TUKYSA.
Embryo-Fetal Toxicity – TUKYSA can cause fetal harm. Advise pregnant women and females of reproductive potential risk to a fetus. Advise females of reproductive potential, and male patients with female partners of reproductive potential, to use effective contraception during TUKYSA treatment and for at least 1 week after the last dose.
Adverse Reactions

Serious adverse reactions occurred in 26% of patients who received TUKYSA. Serious adverse reactions in ≥2% of patients who received TUKYSA were diarrhea (4%), vomiting (2.5%), nausea (2%), abdominal pain (2%), and seizure (2%). Fatal adverse reactions occurred in 2% of patients who received TUKYSA including sudden death, sepsis, dehydration, and cardiogenic shock.

Adverse reactions led to treatment discontinuation in 6% of patients who received TUKYSA; those occurring in ≥1% of patients were hepatotoxicity (1.5%) and diarrhea (1%). Adverse reactions led to dose reduction in 21% of patients who received TUKYSA; those occurring in ≥2% of patients were hepatotoxicity (8%) and diarrhea (6%).

The most common adverse reactions in patients who received TUKYSA (≥20%) were diarrhea, palmar-plantar erythrodysesthesia, nausea, fatigue, hepatotoxicity, vomiting, stomatitis, decreased appetite, abdominal pain, headache, anemia, and rash.

Lab Abnormalities

In HER2CLIMB, Grade ≥3 laboratory abnormalities reported in ≥5% of patients who received TUKYSA were: decreased phosphate, increased ALT, decreased potassium, and increased AST. The mean increase in serum creatinine was 32% within the first 21 days of treatment with TUKYSA. The serum creatinine increases persisted throughout treatment and were reversible upon treatment completion. Consider alternative markers of renal function if persistent elevations in serum creatinine are observed.

Drug Interactions

Strong CYP3A or Moderate CYP2C8 Inducers: Concomitant use may decrease TUKYSA activity. Avoid concomitant use of TUKYSA.
Strong or Moderate CYP2C8 Inhibitors: Concomitant use of TUKYSA with a strong CYP2C8 inhibitor may increase the risk of TUKYSA toxicity; avoid concomitant use. Increase monitoring for TUKYSA toxicity with moderate CYP2C8 inhibitors.
CYP3A Substrates: Concomitant use may increase the toxicity associated with a CYP3A substrate. Avoid concomitant use of TUKYSA where minimal concentration changes may lead to serious or life-threatening toxicities. If concomitant use is unavoidable, decrease the CYP3A substrate dosage.
P-gp Substrates: Concomitant use may increase the toxicity associated with a P-gp substrate. Consider reducing the dosage of P-gp substrates where minimal concentration changes may lead to serious or life-threatening toxicity.
Use in Specific Populations

Lactation: Advise women not to breastfeed while taking TUKYSA and for at least 1 week after the last dose.
Renal Impairment: Use of TUKYSA in combination with capecitabine and trastuzumab is not recommended in patients with severe renal impairment (CLcr < 30 mL/min), because capecitabine is contraindicated in patients with severe renal impairment.
Hepatic Impairment: Reduce the dose of TUKYSA for patients with severe (Child-Pugh C) hepatic impairment.

ImmunoGen Reports Recent Progress and 2020 Financial Results

On February 12, 2021 ImmunoGen Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that progress in the business and reported financial results for the quarter and year ended December 31, 2020 (Press release, ImmunoGen, FEB 12, 2021, View Source [SID1234575034]).

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"Despite the challenges of the pandemic, 2020 was a transformative year for ImmunoGen, as we adjusted to new ways of working, accelerated our portfolio, strengthened our management team and balance sheet, and positioned the business for two potential product launches next year," said Mark Enyedy, ImmunoGen’s President and Chief Executive Officer. "Within our portfolio, we advanced accrual in the pivotal SORAYA and confirmatory MIRASOL trials for mirvetuximab soravtansine in patients with ovarian cancer to support our projected timelines for top-line data and regulatory submissions. In addition, we established a second registration program with our CD123-targeting ADC, IMGN632, for which we received Breakthrough Therapy designation and aligned with FDA on a path to full approval in BPDCN. Furthermore, we began dosing patients in the Phase 1 study of IMGC936, our first-in-class ADAM9-targeting ADC for solid tumors, and transitioned IMGN151, our next-generation FRα-targeting ADC, into preclinical development. Finally, through a combination of business development and activity under our ATM facility, we added over $140 million to our balance sheet in the fourth quarter."

Enyedy continued, "With the benefit of our progress in 2020, we enter this year with significant momentum and strong prospects for the business. For mirvetuximab, these include completing enrollment in SORAYA and reporting top-line pivotal data in the third quarter, followed by a planned BLA submission by the end of the year. With IMGN632, we expect top-line pivotal data in BPDCN in 12 to 18 months and anticipate sharing data from our Phase 1b/2 study of IMGN632 in combination with azacitidine and/or venetoclax in AML patients at ASH (Free ASH Whitepaper) in December. We also anticipate completing dose escalation in the clinical study of IMGC936, with initial data late this year or early 2022. Finally, we expect to submit the IND for IMGN151 by year-end. Taken together, our pivotal programs, experienced management team, and strong balance sheet position us well to execute on our strategy and transition ImmunoGen to a fully-integrated oncology company with two products on the market in 2022."

RECENT PROGRESS

Continued patient enrollment in the pivotal SORAYA and confirmatory MIRASOL trials.
Advanced our partnership with Huadong Medicine, having received acceptance of the investigational new drug (IND) application for mirvetuximab in China from the National Medical Products Administration (NMPA).
Aligned with the US Food and Drug Administration (FDA) on a path to full approval for IMGN632, amending our ongoing 801 Phase 1/2 study with a new pivotal cohort of up to 20 frontline blastic plasmacytoid dendritic cell neoplasm (BPDCN) patients.
Presented updated safety and efficacy findings from the Phase 1/2 expansion study of IMGN632 in patients with relapsed/refractory (R/R) BPDCN during an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December. Our collaborators at MD Anderson Cancer Center also presented preclinical data at ASH (Free ASH Whitepaper) in R/R acute myeloid leukemia (AML) that further support the combination of IMGN632 with Vidaza (azacitidine) and Venclexta (venetoclax).
Advanced patient accrual in the Phase 1 dose-escalation study evaluating IMGC936, our novel ADAM9-targeting ADC in co-development with MacroGenics.
UPCOMING EVENTS

Complete patient enrollment in SORAYA, with top-line pivotal data expected in the third quarter of 2021, and submit the biologics license application (BLA) by the end of 2021 to support potential accelerated approval in 2022.
Complete patient enrollment in MIRASOL, with top-line data expected in the first half of 2022.
Present mature data from the Phase 1b FORWARD II cohort evaluating mirvetuximab in combination with Avastin (bevacizumab) in platinum agnostic, recurrent ovarian cancer at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Support initiation of two investigator-sponsored trials of mirvetuximab plus carboplatin, including a randomized Phase 2 study in recurrent platinum-sensitive ovarian cancer and a neo-adjuvant study.
Enroll patients in the pivotal cohort of the 801 Phase 1/2 study of IMGN632 in frontline BPDCN patients and generate top-line data in 12 to 18 months.
Advance the 802 Phase 1b/2 study of IMGN632 in combination with azacitidine and venetoclax in R/R and frontline AML patients and as a monotherapy in minimal residual disease positive (MRD+) AML following frontline induction therapy.
Present updated R/R BPDCN and initial AML combination data for IMGN632 at ASH (Free ASH Whitepaper) 2021.
Complete dose escalation and move to expansion cohorts in the Phase 1 study evaluating IMGC936, with initial data anticipated by the end of 2021 or early 2022.
Submit the investigational new drug (IND) application for IMGN151 by the end of 2021.
FINANCIAL RESULTS

Total revenues in the fourth quarter and year ended December 31, 2020 increased to $85.8 million and $132.3 million, respectively, compared to $44.9 million and $82.3 million for the same periods in 2019. Revenues are comprised of the following components:

License and milestone fees: License and milestone fees of $63.7 million for the year ended 2020, of which $62.4 million was recorded in the fourth quarter, included recognition of $60.5 million of the upfront fee previously received under the Company’s collaboration agreement with Jazz Pharmaceuticals, and $3.2 million in upfront fees previously received from other partners. License and milestone fees of $34.8 million for 2019 included recognition of $22.1 million in upfront fees previously received from partners and $12.7 million in partner milestone payments.
Non-cash royalty revenue: Non-cash royalty revenue in the fourth quarter and year ended December 31, 2020 increased to $23.4 million and $68.5 million, respectively, compared to $15.3 million and $47.4 million for the same periods in 2019 due to rising global sales of Kadcyla during both periods in 2020.
Research and development expenses were $39.6 million for the quarter ended December 31, 2020 compared to $26.1 million for the quarter ended December 31, 2019, and $114.6 million for the year ended December 31, 2020 compared to $114.5 million for the year ended December 31, 2019. The increase in the current quarter was due to greater external manufacturing costs related to the potential commercial launch of mirvetuximab and clinical trials costs driven by advancement of the SORAYA and MIRASOL studies.

General and administrative expenses were flat at $9.7 million and $9.8 million for the quarters ended December 31, 2020 and 2019, respectively, and $38.6 million and $38.5 million for the years ended December 31, 2020 and 2019, respectively.

Restructuring charges of $1.5 million and $21.4 million were recorded in the years ended December 31, 2020 and 2019, respectively, related to the restructuring of the business at the end of the second quarter of 2019, with the current year charge comprised substantially of retention costs.

Net income for the fourth quarter of 2020 was $31.4 million, or $0.16 per diluted share, compared to net income of $4.8 million, or $0.03 per diluted share, for the fourth quarter of 2019. Net loss for the year ended December 31, 2020 was $(44.4) million, or $(0.25) per diluted share, compared to a net loss of $(104.1) million, or $(0.70) per diluted share, for the year ended December 31, 2019.

ImmunoGen had $293.9 million in cash and cash equivalents as of December 31, 2020, compared with $176.2 million as of December 31, 2019, and had $2.1 million of convertible debt outstanding in each period. Cash used in operations was $78.6 million for the year ended December 31, 2020 compared with $88.4 million for the year ended December 31, 2019. Net proceeds from the sale of equipment were $0.5 million for 2020 compared with net capital expenditures of $(0.5) million for 2019.

During the quarter ended December 31, 2020, the Company sold approximately 20 million shares of its common stock through its At-the-Market (ATM) facility, generating gross proceeds to the Company of approximately $100 million. In January 2021, the Company sold an additional 4.5 million shares of its common stock through its ATM facility, generating additional gross proceeds of approximately $35 million.

FINANCIAL GUIDANCE

For 2021, ImmunoGen expects:

revenues between $65 million and $75 million;
operating expenses between $200 million and $210 million; and
cash and cash equivalents at December 31, 2021 to be between $140 million and $150 million.
ImmunoGen expects that its current cash, inclusive of the net proceeds generated from recent sales through its ATM facility, will fund operations into the second half of 2022.

Revenue guidance includes the assumption that a portion of the upfront license fee from Huadong Medicine will be recognized in 2021 beginning with the delivery of the first clinical supply of mirvetuximab to support development in China.

CONFERENCE CALL INFORMATION

ImmunoGen will hold a conference call today at 8:00 a.m. ET to discuss these results. To access the live call by phone, dial (877) 621-5803; the conference ID is 1666147. The call may also be accessed through the Investors and Media section of immunogen.com. Following the call, a replay will be available at the same location.

Enlivex Announces Closing of Previously Announced Bought Deal Offering of Approximately $46.0 Million Ordinary Shares

On February 12, 2021 Enlivex Therapeutics Ltd. (NASDAQ: ENLV), a clinical-stage macrophage reprogramming immunotherapy company targeting diseased macrophages in patients with sepsis, COVID-19 and solid tumors, reported the closing of its previously announced offering of 2,296,107 ordinary shares, par value NIS 0.40 per share, of the Company at a price to the public of $20.00 per ordinary share, less underwriting discounts and commissions (Press release, Enlivex Therapeutics, FEB 12, 2021, View Source [SID1234575033]).

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H.C. Wainwright & Co. acted as the sole book-running manager for the offering.

The Company also has granted to the underwriter a 30-day option to purchase up to 344,416 additional ordinary shares at the public offering price, less underwriting discounts and commissions.

The gross proceeds to Enlivex, before deducting underwriting discounts and commissions and offering expenses, are approximately $45.92 million. The Company intends to use the net proceeds from this offering for (i) clinical, regulatory, manufacturing and research and development activities; (ii) potential acquisitions and in-licensing; and (iii) other general corporate purposes.

The securities described above were offered by Enlivex pursuant to a "shelf" registration statement on Form F-3 (File No. 333-232009) previously filed with the Securities and Exchange Commission (the "SEC") on June 7, 2019 and declared effective by the SEC on June 21, 2019 and the Form F-3MEF filed on February 9, 2021 (File No. 333-252926). The offering of the securities described above was made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the securities offered have been filed with the SEC and are available on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (646) 975-6996 or e-mail at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

Entry into a Material Definitive Agreement

On February 12, 2021, CNS Pharmaceuticals, Inc. (the "Company") reported that it entered into a Capital on Demand Sales Agreement (the "Agreement") with JonesTrading Institutional Services LLC and Brookline Capital Markets, a division of Arcadia Securities, LLC (collectively, the "Agents") (Filing, 8-K, CNS Pharmaceuticals, FEB 12, 2021, View Source [SID1234575032]). Pursuant to the terms of the Agreement, the Company may sell from time to time through the Agents shares of the Company’s common stock, par value $0.001 per share ("Common Stock") with an aggregate sales price of up to $20.0 million (the "Shares").

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Any sales of Shares pursuant to the Agreement will be made under the Company’s effective "shelf" registration statement (the "Registration Statement") on Form S-3 (File No. 333-252471), which became effective on February 3, 2021 and the related prospectus supplement and the accompanying prospectus, as filed with the Securities and Exchange Commission (the "SEC") on February 12, 2021.

Under the Agreement, the Company may sell Shares through the Agents by any method that is deemed an "at the market offering" as defined in Rule 415 under the Securities Act of 1933, as amended (the "Securities Act").

Sales of the Shares, if any, may be made at market prices prevailing at the time of sale, subject to such other terms as may be agreed upon at the time of sale, including a minimum sales price that may be stipulated by the Company’s Board of Directors or a duly authorized committee thereof. The Company or the Agents, under certain circumstances and upon notice to the other, may suspend the offering of the Shares under the Agreement. The offering of the Shares pursuant to the Agreement will terminate upon the sale of Shares in an aggregate offering amount equal to $20.0 million, or sooner if either the Company or the Agents terminate the Agreement pursuant to its terms.

The Company will pay a commission to the Agents of 3.0% of the gross proceeds of the sale of the Shares sold under the Agreement and reimburse the Agents for certain expenses. The Company has also provided the Agents with customary indemnification rights. The Company is not obligated to make any sales of Common Stock under the Agreement.

The foregoing description of the Agreement is not complete and is qualified in its entirety by reference to the full text of the Agreement, a copy of which is filed as Exhibit 1.1 to this Current Report on Form 8-K and is incorporated herein by reference. The Agreement is also incorporated by reference into the Registration Statement.

A copy of the opinion of Schiff Hardin LLP relating to the legality of the shares of Common Stock issuable under the Agreement is filed as Exhibit 5.1 to this Current Report on Form 8-K and is also incorporated by reference into the Registration Statement.

The above disclosure shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any offer, solicitation, or sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.

Cassava Sciences Announces $200 Million Registered Direct Offering of Common Stock

On February 12, 2021 Cassava Sciences, Inc. (Nasdaq: SAVA) (the "Company" or "Cassava Sciences"), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported that it has entered into a definitive agreement with several healthcare-focused and other institutional investors for the purchase of 4,081,633 shares of its common stock, at a purchase price per share of $49.00, for gross proceeds of approximately $200.0 million, in a registered direct offering (Press release, Pain Therapeutics, FEB 12, 2021, View Source [SID1234575031]). The closing of the offering is expected to occur on or about February 12, 2021, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

Cassava Sciences intends to use the net proceeds, if any, from this offering for working capital and general corporate purposes, including development of simufilam, the Company’s lead drug candidate for the treatment of Alzheimer’s disease.

The shares of common stock are being offered by Cassava Sciences pursuant to an automatic "shelf" registration statement on Form S-3, which was filed with the U.S. Securities and Exchange Commission (the "SEC") on February 10, 2021 and became effective immediately upon filing, and the base prospectus contained therein. The offering will be made only by means of a prospectus supplement that forms a part of the registration statement.

A prospectus supplement and accompanying base prospectus relating to the shares of common stock being offered will be filed with the SEC. Copies of the final prospectus supplement and accompanying base prospectus may be obtained, when available, on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at 646-975-6996 or e-mail at [email protected].

This press release shall not constitute an offer to sell, or the solicitation of an offer to buy any of the securities described herein, 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.