On July 18, 2023 Aptevo Therapeutics Inc. ("Aptevo" or the "Company") (Nasdaq;APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported that its bispecific AML drug candidate APVO436, in combination with emerging standard of care venetoclax and azacitidine, achieved positive duration of remission results in its Phase 1b dose escalation trial (Press release, Aptevo Therapeutics, JUL 18, 2023, View Source [SID1234633292]). The Company is also providing an update of its APVO436 Phase 2 program design that will include both frontline and relapsed/refractory trials beginning later in the second half of 2023.

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Duration of Remission (DOR)
Positive and clinically meaningful DOR results was observed from the company’s bispecific AML drug candidate APVO436 in combination with the emerging standard of care (venetoclax + azacitidine) in venetoclax treatment naïve patients, as follows:

High response rate observed – 82% (9/11) of patients had a favorable response and were eligible for inclusion in the DOR analysis
Multiple patients moved to transplant – 3/11 patients responded sufficiently to move to stem cell transplant – receiving stem cell transplant is the treatment option with the best probability for survival and highest benefit to patients
Sustained complete response – Of the patients with responses, one patient remained on study with sustained complete response for 8 cycles (the maximum allowed per protocol) which translated into at least 8 months of response duration
Median DOR not reached – The median DOR was not reached, which is clinically meaningful because a substantial number of patients either stayed on treatment or moved to transplant and did not experience a relapse event
This DOR data adds to a growing body of clinical evidence (safety, tolerability, efficacy and now durability), that provides strong support for the further development of APVO436 in combination therapy for patients with AML.

Phase 2 Program Update
The Company’s APVO436 Phase 2 program will further evaluate the triplet combination of APVO436 + venetoclax + azacitidine among frontline and relapsed/refractory AML patients who are venetoclax treatment naïve. The Company intends to conduct two trials. The first, among relapsed/refractory patients, will initiate in 2H23. The second, among frontline patients, will initiate in 1H24. Aptevo anticipates that approximately 100 patients will participate between the two trials and that interim results will be available in late 2H24.

APVO436 Dose Escalation Trial Results: Safety and Efficacy Data (previously reported)

Efficacy: APVO436 demonstrated a 91% clinical benefit* in combination with venetoclax + azacitidine in venetoclax treatment naïve patients, more than doubling the response rate in a composite benchmark** across all benefit categories
Safety: APVO436, when given in combination with this regimen, has been shown to be generally safe and well tolerated. Cytokine Release Syndrome (CRS), a common side effect in other trials, was observed in fewer than one quarter of patients within the patient population and in most cases was mild or moderate (grade 1 or 2) and was manageable in the clinic

For more information about previously reported data click HERE.

On July 18, 2023 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that on July 13, 2023, the company approved non-statutory stock options to purchase an aggregate of 66,380 shares of its common stock and 30,685 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 11 new employees (Press release, PTC Therapeutics, JUL 18, 2023, View Source [SID1234633291]). The awards were made pursuant to the NASDAQ inducement grant exception as a component of the new hires’ employment compensation.

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The inducement grants were approved by PTC’s Compensation Committee on July 13, 2023, and are being made as an inducement material to each employee’s acceptance of employment with the company in accordance with NASDAQ Listing Rule 5635(c)(4).

As part of the inducement grants described above and in connection with the hiring of Pierre Gravier as Chief Financial Officer, the company granted Mr. Gravier 26,000 RSUs, in addition to non-statutory stock options to purchase 65,000 shares of its common stock.

All stock option awards have an exercise price of $39.82 per share, the closing price of PTC’s common stock on July 13, 2023, the date of the grant. The stock options each have a 10-year term and vest over four years, with 25% of the original number of shares vesting on the first anniversary of the applicable employee’s new hire date and 6.25% of the original number of shares vesting at the end of each subsequent three-month period thereafter until fully vested, subject to the employee’s continued service with the company through the applicable vesting dates. The RSUs each will vest over four years with 25% of the original number of shares vesting on each annual anniversary of the applicable employee’s new hire date until fully vested, subject to the employee’s continued service with the company through the applicable vesting dates.

On July 18, 2023, Novartis International Pharmaceutical Ltd. ("Novartis") reported that based on a benefit-risk assessment, it is discontinuing its Phase 3 trial (the "Trial") investigating NIS793, an anti-TGFb monoclonal antibody, in first-line metastatic pancreatic ductal adenocarcinoma (mPDAC) (Press release, Xoma, JUL 18, 2023, View Source [SID1234633290]).

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The Trial (NCT04935359) was designed to assess the efficacy and safety of NIS793 in combination with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel and placebo, in mPDAC. Novartis stated it will continue to investigate NIS793 in indications beyond pancreatic ductal adenocarcinoma, including its ongoing Phase 2 study in colorectal cancer.

XOMA Corporation (the "Company") licensed NIS793 to Novartis pursuant to that certain License Agreement dated as of September 30, 2015 by and between XOMA (US) LLC, a wholly owned subsidiary of the Company, and Novartis Institutes for Biomedical Research, Inc., previously filed with the Securities and Exchange Commission as Exhibit 10.2 to its Quarterly Report on Form 10-Q for the period ended September 30, 2015.

NIS793 is an investigational compound. Efficacy and safety have not been established. There is no guarantee that NIS793 will become commercially available.

On July 18, 2023 Pieris Pharmaceuticals, Inc. (Nasdaq:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases and cancer, reported a strategic update following recent events that have impacted its inhaled respiratory franchise, including AstraZeneca’s discontinuation of enrollment of the Phase 2a study for elarekibep (Press release, Pieris Pharmaceuticals, JUL 18, 2023, View Source [SID1234633289]). AstraZeneca has now informed the Company of its decision to terminate the parties’ R&D collaboration agreement and hand back elarekibep along with discontinuing the remaining discovery program.

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Pieris’ management and board of directors have assessed several strategic options, which will include focusing on execution of new or expanded partnerships to advance its therapeutic programs, including cinrebafusp alfa (PRS-343), PRS-220 and PRS-400. While it explores potential transactions, Pieris will prioritize capital preservation, with cash, cash equivalents and investments totaling approximately $54.9 million as of June 30, 2023. As part of its cash preservation plan, Pieris initiated a corporate restructuring that will result in a reduction of the Company’s workforce by approximately 70%. These and other cost-saving measures are expected to maximize the opportunity to pursue a range of transactions across both its respiratory and immuno-oncology franchises and its discovery platform. Pieris has retained Stifel, Nicolaus & Company, Inc. as its exclusive financial advisor to evaluate a range of strategic options. These strategic options may include an acquisition, merger, reverse merger, other business combination, sale of assets, financing alternatives, licensing, or other strategic transactions involving the Company. There can be no assurance of a transaction, a successful outcome of these efforts, or the form or timing of any such outcome. The Company does not intend to make any further disclosures regarding the strategic review process unless and until a specific course of action is approved by the Company’s board of directors or until the Company determines that further disclosure is appropriate.

"We are pursuing strategic options across three main areas following the recent developments that have impacted our ability to independently advance our respiratory programs," commented President and CEO Stephen Yoder. "One track is accelerating partnering discussions of PRS-220 and PRS-400. A second focal area is diligently selecting the best possible development partner and deal structure to re-initiate clinical development of cinrebafusp alfa, our former lead immuno-oncology asset, which has shown 100% ORR in five patients in a HER2+ gastric cancer trial that was discontinued for strategic reasons. Third, we will explore whether our balance sheet, position as a public company, and other assets are of strategic value to a range of third parties." Mr. Yoder continued, "While the challenges we recently experienced across our respiratory franchise have forced us to make very difficult personnel decisions, I cannot express enough gratitude to our departing colleagues for their dedication, collaborative spirit and integrity."

On July 18, 2023 Orion Biotechnology Canada Ltd. (Orion), a drug discovery and development company unlocking the therapeutic potential of previously undruggable G Protein-Coupled Receptors (GPCRs), reported that it has successfully completed a discovery campaign against CXCR6, an undrugged small protein GPCR with the potential to provide a new and innovative form of immunotherapy treatment for solid tumor cancers (Press release, Orion Biotechnology, JUL 18, 2023, View Source [SID1234633288]). Completed in only four months, the rapid identification of this first-in-class (FIC) molecule further validates Orion’s PROcisionXTM discovery platform’s industry-leading efficiency.

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CXCR6 is expressed in several subsets of T cells, natural killer T cells, and bone marrow stromal cells, and the main physiological role of the CXCR6-CXCL16 axis is to activate numerous signaling pathways essential to regulating immunity. Boosting signaling through the CXCR6-CXCL16 axis in the tumor microenvironment has the potential to provide a new route to block solid tumor growth by helping to maintain anti-tumor cytotoxic T cells in an aggressive, non-exhausted state.

"We are very excited about the significant progress we continue to make across our portfolio" said Dr. Ian McGowan, the company’s Chief Medical Officer. "Orion’s discovery success will allow for a new and highly differentiated strategy to drug CXCR6 for the treatment of cancer and other inflammatory diseases."

Dr. Oliver Hartley (VP Discovery) commented: "Small protein GPCRs have proven challenging to unlock using established small molecule and antibody-based approaches. This rapid success provides further evidence that Orion’s novel scientific approach can unlock previously undrugged small protein GPCRs and provides a clear illustration of the efficiency of our PROcisionXTM discovery platform."

Orion’s CEO Mark Groper remarked: "Unlocking previously intractable GPCRs represents a tremendous opportunity to develop new treatments across a broad range of therapeutic areas. Our rapid success with a new small protein GPCR marks another key milestone in our mission to deliver medicines that improve the lives of patients. The combination of our highly talented team and our PROcisionXTM platform is allowing our company to rapidly and efficiently build out a multi-product pipeline of high-value GPCR-targeted therapeutics."