On March 7, 2022 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported the presentation of safety and efficacy results from Cohort 4 of the ZENITH20 clinical trial (Press release, Spectrum Pharmaceuticals, MAR 7, 2022, View Source [SID1234609595]). This data is from 70 first-line patients with non-small lung cancer (NSCLC) with HER2 exon 20 insertion mutations who received 16 mg daily, given as 16 mg once daily (48 patients) or 8 mg twice daily (22 patients) of oral poziotinib. These results showed a confirmed objective response rate (ORR) of 41% (95% CI:30%-54%), as evaluated centrally by an independent image review committee using RECIST 1.1 criteria. The evaluable patient population showed an ORR of 50%. The study met its primary endpoint as the observed lower bound of 30% exceeded the pre-specified lower bound of 20%. The safety profile was consistent with the TKI class. The data is part of an oral presentation at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress 2022 being held virtually March 7-8, 2022.

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"Cohort 4 from our ZENITH 20 study demonstrated positive results for treatment naïve lung cancer patients harboring HER2 Exon 20 insertion mutations. There is currently no approved treatment for NSCLC patients with these mutations," said Francois Lebel, M.D., Chief Medical Officer of Spectrum. "We are encouraged by these findings and look forward to further discussions with the FDA on the regulatory path forward."

The presentation is available on the Spectrum Pharmaceuticals website at: View Source

Safety and Efficacy Data for Cohort 4 of the ZENITH20 Clinical Trial

Cohort 4 enrolled treatment-naïve NSCLC patients with HER2 exon 20 insertion mutations. This cohort investigated the efficacy of poziotinib and included patients dosed either with a QD or BID dosing strategy. Poziotinib 16 mg was administered orally once daily for the first 48 patients followed by an additional 22 patients dosed at 8 mg twice daily. Both dosing regimens allowed dose reductions/interruptions for toxicity. The primary endpoint was ORR evaluated centrally by an independent image review committee using RECIST 1.1 criteria. Secondary endpoints included disease control rate (DCR), duration of response (DoR), progression-free survival (PFS) and safety.

The primary endpoint of ORR was 41% (95% CI:30-54%) in the 70 treated patients including one complete response. The DCR was 73% (95% CI:61-83%). DoR was 5.7 months (range 1.2-19.1+). Median progression free survival (PFS) was 5.6 months (range 0-20.2+). 90% of patients had dose interruptions and 79% had reductions from the 16 mg QD starting dose, while 64% had reductions from the 8mg BID starting dose. The most common treatment related Grade ≥ 3 adverse events were rash (30%), stomatitis (19%), diarrhea (14%), and paronychia (7%). In addition, the incidence of Grade ≥ 3 pneumonitis was low at 3%. The safety profile was consistent with the TKI class and tolerability, dose reductions and interruptions were less frequent with BID dosing.

About the ZENITH20 Clinical Trial

The ZENITH20 study consists of seven cohorts of NSCLC patients. Cohorts 1 (EGFR) and 2 (HER2) in previously treated NSCLC patients with exon 20 mutations, Cohort 3 (EGFR) in first-line patients and Cohort 4 (HER2) in first-line NSCLC patients with exon 20 mutations have completed patient enrollment. Cohorts 1-4 are each independently powered for a pre-specified statistical hypothesis and the primary endpoint is ORR. Cohort 5 includes previously treated or treatment-naïve NSCLC patients with EGFR or HER2 exon 20 insertion mutations. Cohort 6 includes NSCLC patients with classical EGFR mutations who progressed while on treatment with first-line osimertinib and developed an additional EGFR mutation. Cohort 7 includes NSCLC patients with a variety of less common mutations in EGFR or HER2 exons 18-21 or the extracellular or transmembrane domains.

On March 7, 2022 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the fourth quarter and year ended December 31, 2021 and provided pipeline updates (Press release, AnaptysBio, MAR 7, 2022, View Source [SID1234609594]).

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"We advanced our wholly-owned antibody product pipeline and completed a $250 million royalty monetization transaction during 2021," said Hamza Suria, president and chief executive officer of AnaptysBio. "We look forward to multiple clinical data readouts during 2022 and remain focused on developing first-in-class therapeutic antibodies using a capital-efficient business model."

Imsidolimab (Anti-IL-36 Receptor) Program

Following an end-of-Phase 2 meeting with the FDA, we initiated our GEMINI-1 Phase 3 trial of imsidolimab in generalized pustular psoriasis (GPP) where the primary endpoint is the proportion of patients achieving a score of clear (0) or almost clear (1) skin on the Generalized Pustular Psoriasis Physician’s Global Assessment (GPPPGA) at week 4 in 45 patients randomized against placebo. These same patients will subsequently be enrolled into GEMINI-2, which will assess 6 months of monthly subcutaneous dosing and safety follow-up.
We anticipate top-line data from the ACORN Phase 2 trial of imsidolimab in moderate-to-severe acne in H1 2022 and from the HARP Phase 2 trial in moderate-to-severe hidradenitis suppurativa in H2 2022.
Rosnilimab (Anti-PD-1 Agonist) Program

We announced positive top-line data from a randomized placebo-controlled healthy volunteer single and multiple ascending dose Phase 1 trial of rosnilimab, our investigational wholly-owned anti-PD-1 agonist therapeutic antibody, previously known as ANB030. Top-line data demonstrated favorable safety, pharmacokinetics and pharmacodynamic results, which supported initiation of our Phase 2 AZURE clinical trial of rosnilimab in alopecia areata.
ANB032 (Anti-BTLA Modulator) Program

We are advancing ANB032, our wholly-owned BTLA modulator antibody, in a healthy volunteer Phase 1 single and multiple ascending dose clinical trial where top-line data is anticipated during the first half of 2022.
GSK Partnered Programs

We completed a royalty monetization agreement with Sagard Healthcare Royalty Partners where AnaptysBio received a $250 million payment in exchange for JEMPERLI royalties due to AnaptysBio on annual commercial sales below $1 billion and certain future milestones starting October 2021. The aggregate JEMPERLI royalties and milestones to be received by Sagard under this Agreement is capped at certain fixed multiples of the upfront payment.
Fourth Quarter Financial Results

Cash, cash equivalents and investments totaled $615.2 million as of December 31, 2021, compared to $411.2 million as of December 31, 2020, for an increase of $204.0 million. The increase relates primarily to cash received from the royalty monetization transaction with Sagard Healthcare Partners offset by cash used for operating activities.
Collaboration revenue was $1.0 million and $63.2 million for the three and twelve months ended December 31, 2021. The $1.0 million earned during the fourth quarter primarily relates to royalty revenue earned for sales of JEMPERLI (dostarlimab) and Zejula by GSK, compared to $60.0 million and $75 million of milestone revenue for the three and twelve months ended December 31, 2020.
Research and development expenses were $26.8 million and $98.5 million for the three and twelve months ended December 31, 2021, compared to $21.6 million and $80.0 million for the three and twelve months ended December 31, 2020. The increase was due primarily to continued advancement of the Company’s clinical programs.
General and administrative expenses were $5.4 million and $21.5 million for the three and twelve months ended December 31, 2021, compared to $5.1 million and $18.9 million for the three and twelve months ended December 31, 2020. The increase was due primarily to personnel-related expenses, including share-based compensation.
Net loss was $32.5 million and $57.8 million for the three and twelve months ended December 31, 2021, or a net loss per share of $1.18, and $2.11, compared to net income of $33.6 million for the three months ended December 31, 2020 or net income per share of $1.23 and a net loss of $19.9 million for the twelve months ended December 31, 2020, or net loss per share of $0.73.

On March 7, 2022 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that it will report fourth quarter and full year 2021 financial results after the U.S. financial markets close on Thursday, March 10, 2022 (Press release, Oncternal Therapeutics, MAR 7, 2022, View Source [SID1234609593]). Oncternal’s management will host a webcast at 2:00 p.m. PT (5:00 p.m. ET) to provide a comprehensive business update and discuss the Company’s financial results.

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The live webcast of the call will be available online at investor.oncternal.com and the call will be archived there for at least 30 days.

On March 7, 2022 Evaxion Biotech A/S (NASDAQ: EVAX), a clinical-stage biotechnology company specializing in the development of AI-driven immunotherapies, reported that now finalized recruitment for Phase 1/2a clinical trial of EVX-02 in adjuvant melanoma patients (Press release, Evaxion Biotech, MAR 7, 2022, View Source [SID1234609592]).

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The Company already conducts the successful EVX-01 program with clinical testing in Australia, Europe and the US, where app. 100 patients suffering from malignant melanoma (metastasized skin cancer) will be treated with EVX-01 therapy in combination with Keytrudaâ from Merck. In the EVX-02 program, Evaxion treats patients with adjuvant melanoma, meaning that their tumors have been successfully removed (surgically), and in the study the focus of the therapy is to avoid relapse.

In the EVX-02 study encouraging preliminary findings around its potential clinical benefit now allows Evaxion to accelerate the development program. Following the finalization of recruitment, Evaxion will advance into a dedicated Phase 2b clinical trial using its patented DNA-based immunotherapy.

For the patients enrolled in the Phase 1/2a clinical trial, Evaxion’s proprietary PIONEERTM AI technology identified a sufficient number of mutations to design a personalized treatment and so far, no serious side effects have been reported.

Lars Wegner, CEO of Evaxion, said:

"Completion of recruitment for the Phase 1/2a clinical trial of EVX-02 is an important milestone, which supports our belief that the Evaxion approach is feasible. EVX-02 appears to be well tolerated and shows encouraging signs as a treatment for adjuvant melanoma patients. The T-cell activation is promising, which allows us to take data and insights from this clinical trial and move into a dedicated Phase 2 clinical trial."

About the EVX-02 Program:

1.Evaxion Biotech has completed recruitment of 16 patients and will now move directly into a dedicated phase 2b clinical trial in adjuvant melanoma.

2.Final data from the current phase 1/2a clinical trial are expected to be announced and reported second quarter of 2023, which is earlier than previously communicated.

3.Expected milestones for the EVX-02 phase 2b study are: Regulatory filing in H1 2022, First patient first visit in H2 2022 and an interim readout in 2023.

On March 7, 2022 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months and year ended December 31, 2021 and provided a corporate update (Press release, DURECT, MAR 7, 2022, View Source [SID1234609591]).

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"We are pleased with the progress made over the past few months in the larsucosterol (DUR-928) AHFIRM trial, including opening international sites and the enrollment rate of patients with severe alcohol-associated hepatitis (AH)," stated James E. Brown, D.V.M., President and CEO of DURECT. "We also signed a U.S. licensing agreement for POSIMIR with Innocoll Pharmaceuticals and look forward to their anticipated launch in the second quarter of 2022."

Fourth Quarter and Recent Business Highlights:

Continued progress in AHFIRM enrollment – DURECT has now dosed over 100 patients in the Phase 2b AHFIRM clinical trial of larsucosterol in severe AH. This includes dosing the first patient in Europe, with 51 global AHFIRM study sites now open, an increase of 15 since our last earnings call. With this progress, our estimated completion of enrollment remains on track for mid-2023.
Successful completion of POSIMIR licensing – DURECT signed an exclusive U.S. licensing agreement for POSIMIR with Innocoll Pharmaceuticals in late 2021. Under the agreement, DURECT will earn low to mid double-digit royalties from net sales of POSIMIR and is eligible to receive up to $136 million in upfronts and milestones, including the $4 million upfront license fee received in January 2022, and a $2 million milestone payment upon the first commercial sale of POSIMIR, which is anticipated in 2Q22.
Increasing awareness of AH market potential – In collaboration with DURECT, Dr. Suthat Liangpunsakul presented a poster at The Liver Meeting 2021 showing increased hospitalizations for AH in the U.S., highlighting both significant comorbidities and high hospitalization costs of over $150,000 per hospitalization for those AH patients who died during their hospital stay.
Important addition to our team – In December 2021, DURECT strengthened its board of directors with the appointment of Pete Garcia, a seasoned financial executive in the biopharmaceutical industry.
Financial highlights for Q4 and full year 2021:

Total revenues were $7.3 million and net loss from continuing operations was $7.0 million for the three months ended December 31, 2021 compared to total revenues of $2.2 million and net loss from continuing operations of $8.8 million for the three months ended December 31, 2020. Total revenues were $14.0 million and net loss from continuing operations was $36.3 million for the year ended December 31, 2021, compared to total revenues of $30.1 million and net loss from continuing operations of $14.3 million for the year ended December 31, 2020.
At December 31, 2021, cash and investments were $70.0 million, compared to cash, cash held in escrow and investments of $56.9 million at December 31, 2020. Debt at December 31, 2021 was $20.6 million, compared to $20.8 million at December 31, 2020. At December 31, 2021, accounts receivable included $5.3 million due from Innocoll as a result of the $4 million upfront fee and a $1.3 million fee primarily to cover manufacturing supplies and excipients and certain equipment transferred to Innocoll; these funds were received in January 2022.
Upcoming Key Milestones:

Complete opening of 60 or more global clinical trial sites across the U.S., E.U., U.K., and Australia and continue acceleration of enrollment in the Phase 2b AHFIRM trial of larsucosterol in severe AH
Provide an update on plans to expand clinical development of larsucosterol in NASH and/or other indications
Commercial launch of POSIMIR by our licensee, Innocoll Pharmaceuticals; expected in Q2 2022
Earnings Conference Call

We will host a conference call today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss fourth quarter 2021 results and provide a corporate update:

Monday, March 7 @ 4:30pm Eastern Time / 1:30 p.m. Pacific Time

Toll Free:

877-869-3847

International:

201-689-8261

Conference ID:

13727525

Webcast:

View Source

A live audio webcast of the presentation will be also available by accessing DURECT’s homepage at www.durect.com and clicking "Investors." If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under "Event Calendar" in the "Investors" section.

About the AHFIRM Trial

Enrollment is ongoing in our Phase 2b randomized, double-blind, placebo-controlled, international, multi-center study in subjects with severe acute alcohol-associated hepatitis (AH) to evaluate saFety and effIcacy of laRsucosterol (DUR-928) treatMent (AHFIRM). The study is comprised of three arms targeting enrollment of 300 total patients, with approximately 100 patients in each arm: (1) Placebo plus standard of care (SOC) which may include the use of methylprednisolone, a corticosteroid, at the discretion of the treating physician; (2) larsucosterol (30 mg); and (3) larsucosterol (90 mg). All patients in the trial receive supportive care. The primary outcome measure is 90-day survival rate for patients treated with larsucosterol compared to those treated with placebo plus SOC. The Company is targeting more than 60 clinical trial sites across the U.S., EU, U.K., and Australia. Reflecting the life-threatening nature of AH and the lack of therapeutic options, the U.S. Food and Drug Administration (FDA) has granted larsucosterol Fast Track Designation for the treatment of AH. We believe demonstration of a robust survival benefit in the AHFIRM trial would support an NDA filing. For more information, refer to ClinicalTrials.gov Identifier: NCT04563026.

About Alcohol-associated Hepatitis (AH)

AH is a life-threatening acute alcohol-associated liver disease (ALD) often caused by chronic heavy alcohol use and a recent period of increased alcohol consumption (i.e., a binge). It is characterized by severe inflammation and destruction of liver tissue (i.e., necrosis), potentially leading to life-threatening complications including liver failure, acute renal injury and multi-organ failure. There are no FDA approved therapies for AH and an analysis of 77 studies published between 1971 and 2016, which included data from a total of 8,184 patients, showed the overall mortality from AH was 26% at 28 days, 29% at 90 days and 44% at 180 days. A subsequent global study published in December 2021, which included 85 tertiary centers in 11 countries across 3 continents, prospectively enrolled 2,581 AH patients with a median MELD score of 23.5, reported mortality at 28 and 90 days of 20% and 31% respectively. Stopping alcohol consumption is not sufficient for recovery in many moderate and severe patients and the use of treatments to reduce liver inflammation, such as corticosteroids, are limited by contraindications and have been shown to provide no survival benefit at 90 days or 1 year. While liver transplantation is becoming more common for alcoholic liver disease patients, including for AH patients, the procedure involves a long waiting period, a burdensome selection process, and costs more than $875,000 on average.

About Larsucosterol (DUR-928)

Larsucosterol is an endogenous sulfated oxysterol and an epigenetic regulator. Epigenetic regulators are compounds that regulate patterns of gene expression without modifying the DNA sequence. DNA hypermethylation, an example of epigenetic dysregulation, results in transcriptomic reprogramming and cellular dysfunction, and has been found to be associated with many acute (e.g., AH) or chronic diseases (e.g., NASH). As an inhibitor of DNA methyltransferases (DNMT1, DNMT3a and 3b), larsucosterol inhibits DNA methylation, which subsequently regulates expression of genes that are involved in cell signaling pathways associated with stress responses, cell death and survival, and lipid biosynthesis. This may ultimately lead to improved cell survival, reduced inflammation, and decreased lipotoxicity. As an epigenetic regulator, the proposed mechanism of action provides further scientific rationale for developing larsucosterol for the treatment of acute organ injury and certain chronic diseases.