On July 28, 2021 MorphoSys AG (FSE: MOR; NASDAQ: MOR) reported its financial results for the second quarter and the first half year of 2021 (Press release, MorphoSys, JUL 28, 2021, View Source [SID1234585294]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We regained the momentum in Monjuvi sales as we exited the second quarter and are encouraged to see that momentum continuing into Q3," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "We are seeing the positive impact to our business from the vaccination rollout in the U.S., and remain focused on establishing tafasitamab as a standard of care in the treatment of patients with relapsed/refractory DLBCL."

"With the addition of Constellation’s clinical programs to our pipeline, we are in a great position to build a significant presence in hematology-oncology with multiple commercial opportunities."

Tafasitamab Highlights

– Monjuvi(R) (tafasitamab-cxix) U.S. net product sales of € 14.9 million (US$ 18.0 million) for the second quarter of 2021 and € 27.8 million (US$ 33.5 million) for the first half of 2021.

– On April 19, 2021, MorphoSys and Incyte announced that the first patient has been dosed in the placebo-controlled Phase 3 inMIND study evaluating the efficacy and safety of tafasitamab or placebo in combination with lenalidomide and rituximab in patients with relapsed or refractory follicular lymphoma (FL) or marginal zone lymphoma (MZL).

– On May 11, 2021, MorphoSys and Incyte announced that the first patient was dosed in the pivotal Phase 3 frontMIND study evaluating tafasitamab and lenalidomide in addition to rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP) compared to R-CHOP alone as first-line treatment for high-intermediate and high-risk patients with untreated diffuse large B-cell lymphoma (DLBCL).

– From June 4-8, 2021, MorphoSys presented new data from the tafasitamab development program at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Data from the three-year follow-up from the Phase 2 L-MIND study showed a long durability of responses and overall survival in patients with r/r DLBCL.

– On June 25, 2021, MorphoSys and Incyte announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending the conditional marketing authorization for tafasitamab in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplantation (ASCT). The Committee for Orphan Medicinal Products (COMP) has also confirmed the orphan drug designation status in mid-July.

– Together with MorphoSys, Incyte plans to start coreMIND, a pivotal Phase 2 study that will assess tafasitamab in combination with Incyte’s Pi3 kinase delta inhibitor in patients with chronic lymphocytic leukemia (CLL).

– MorphoSys will also initiate MINDway, a study that will look into finding the best treatment schedule for patients with Non-Hodgkin Lymphoma (NHL) who benefit from long-term disease-control from tafasitamab.

Acquisition of Constellation Pharmaceuticals and Strategic Funding Partnership

– On June 2, 2021, MorphoSys entered into a definitive agreement to acquire Constellation Pharmaceuticals (Constellation) for US$ 34.00 per share in cash, which represents a total equity value of US$ 1.7 billion. The transaction has been unanimously approved and subsequently was completed on July 15, 2021.

– MorphoSys gets access to mid- to late-stage product candidates: Pelabresib (CPI 0610) has the potential to be a first-in-class and best-in-class BET inhibitor that is currently being evaluated in a Phase 3 trial for the treatment of myelofibrosis. CPI-0209 is a mid-stage EZH2 inhibitor, which is currently in a Phase 2 clinical trial and has best-in-class potential for treating both hematologic and solid tumors.

– MorphoSys entered into a long-term strategic funding partnership with Royalty Pharma:

– US$ 1.425 billion upfront payment

– Up to US$ 350 million in Development Funding Bonds

– Up to US$ 150 million milestone payments

– Royalty Pharma Investments 2019 ICAV, a subsidiary of Royalty Pharma plc, purchased US$ 100 million in shares (1,337,552 shares) of MorphoSys at a price of € 63.35 per share on July 16, 2021

– MorphoSys also announced today that Jigar Raythatha, President and Chief Executive Officer of Constellation, will resign effective July 31, 2021. Barbara Krebs-Pohl, PhD, Senior Vice President, Global Head of Business Development, Licensing, and Alliance Management at MorphoSys, has been appointed as Site Head of Constellation and Chief Integration Officer.

Tremfya:

– MorphoSys to continue to record Tremfya royalties on its income statement. Royalty Pharma is entitled to receive 100 percent of Tremfya royalties starting with royalties for the second quarter of 2021.

– Tremfya royalties of € 13.7 million for the second quarter of 2021 and € 25.4 million for the first half of 2021.

Financial Results for the Second Quarter of 2021 (IFRS)
Total revenues for the second quarter of 2021 amounted to € 38.2 million (Q2 2020: € 18.4 million). The revenues include success-based payments of € 14.2 million, primarily from Janssen (Q2 2020 success-based payments: € 12.8 million).

Cost of Sales: In the second quarter of 2021, cost of sales increased to € 10.1 million (Q2 2020: income of € 7.2 million).

Research and Development (R&D) Expenses: In the second quarter of 2021, research and development expenses were € 40.5 million (Q2 2020: € 30.9 million). Growth over 2020 reflects the increased investment to support the advancement of proprietary programs and consisted primarily of expenses for external laboratory services and personnel expenses.

Selling, General and Administrative (SG&A) Expenses: Selling expenses decreased slightly in the second quarter of 2021 to € 28.5 million (Q2 2020: € 29.3 million) and general and administrative expenses amounted to € 30.5 million (Q2 2020: € 13.8 million). The increase of general and administrative expense in the second quarter was due to the transaction costs related to the Constellation and Royalty Pharma agreements.

Operating Loss: Operating loss amounted to € 71.4 million in the second quarter of 2021 (Q2 2020: operating loss of € 48.4 million).

Consolidated Net Profit / Loss: For the second quarter of 2021, consolidated net profit was € 20.9 million (Q2 2020: consolidated net loss of € 53.1 million).

Financial Results for the First 6 Months of 2021 (IFRS)

Total revenues for the first six months of 2021 amounted to € 85.4 million (H1 2020: € 269.7 million). The revenues include success-based payments of € 43.1 million, primarily from Janssen (H1 2020 success-based payments: € 23.1 million) The year-over-year decline was driven by the upfront payment of the collaboration and license agreement with Incyte in the first quarter 2020 for the out-licensing of tafasitamab outside the U.S.

Cost of Sales: In the first six months of 2021, cost of sales increased to € 15.2 million (H1 2020: income of € 4.0 million).

Research and Development (R&D) Expenses: In the first six months of 2021, research and development expenses were € 73.8 million (H1 2020: € 52.4 million). Growth over 2020 reflects the increased investment to support the advancement of proprietary programs and consisted primarily of expenses for external laboratory services and personnel expenses.

Selling, General and Administrative (SG&A) Expenses: Selling expenses increased in the first six months of 2021 to € 56.6 million (H1 2020: € 42.1 million) and general and administrative expenses amounted to € 40.8 million (H1 2020: € 23.9 million). The year-over-year increase in selling expenses was primarily driven by the full first half year 2021 impact of the expenses for services provided by Incyte as part of the joint U.S. marketing activities for Monjuvi. The year-over-year increase in general and administrative expenses was driven primarily by the transaction costs related to the Constellation and Royalty Pharma agreements.

Operating Loss: Operating loss amounted to € 101.0 million in the first six months of 2021 (H1 2020: operating profit of € 155.1 million).

Consolidated Net Profit / Loss: For the first six months of 2021, consolidated net loss was € 20.7 million (H1 2020: consolidated net profit of € 179.8 million).

Cash and Investments: As of June 30, 2021, the Company had cash and investments of € 1,129.2 million compared to € 1,244.0 million on December 31, 2020. Pro forma cash after the closing of the Constellation and Royalty Pharma transactions, including the sale of ordinary shares, was € 1,168.0 million.

Number of shares: The number of shares issued totaled 32,892,540 at the end of Q2 2021 (year-end 2020: 32,890,046). After the share capital increase on July 16, 2021, to implement the purchase of 1,337,552 new ordinary shares by Royalty Pharma, the number of shares issued totaled 34,227,598.

Adjusted Financial Guidance and Operational Outlook for 2021

in € million Updated Financial Guidance 2021 Prior Financial Guidance (ex-Constellation)

*Group revenues include full year Tremfya royalties and exclude any royalties from potential tafasitamab sales outside of the U.S. as well as any significant milestones from development partners and/or licensing partnerships other than those that were already recorded in the half-year. This revenue guidance is subject to a number of uncertainties including the potential for variability from the first full year of the Monjuvi product launch, the limited visibility that MorphoSys has on the Tremfya royalty stream as well as the ongoing COVID-19 pandemic and the impact on our as well as our partner’s business operations.

**Operating expenses is comprised of R&D and SG&A, inclusive of Incyte’s share of Monjuvi selling costs in the U.S.

MorphoSys expects the following events and activities in 2021:

Tafasitamab:

– Continuation of the phase 1b trial with tafasitamab in previously untreated DLBCL (firstMIND);

– Continuation of the pivotal phase 3 frontMIND trial of tafasitamab in previously untreated DLBCL;

– Continuation of the pivotal phase 3 inMIND trial of tafasitamab in patients with relapsed or refractory follicular lymphoma (r/r FL) or marginal zone lymphoma (MZL);

– Investigation of tafasitamab, plamotamab and lenalidomide in patients with relapsed or refractory DLBCL, first-line DLBCL and relapsed or refractory follicular lymphoma (r/r FL) jointly with Incyte and Xencor (study start expected end of 2021/early 2022);

– Continuation of the L-MIND study of tafasitamab and evaluate the long-term efficacy and safety data;

– Continuation of the phase 3 B-MIND study of tafasitamab in combination with bendamustine for r/r DLBCL;

– Decision of the European Commission on the Marketing Authorization Application (MAA), seeking conditional marketing authorization of tafasitamab in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with r/r DLBCL which is currently under review;

– Support of Incyte in submitting marketing authorization applications in other markets for tafasitamab.

Felzartamab:

– Continuation of the M-PLACE and the NewPLACE study in patients with membranous nephropathy;

– Presentation of data from the M-PLACE study at a scientific conference in Q4 2021;

– Start of clinical study in patients with IgA nephropathy (IGNAZ study).

Constellation programs:

– Continuation of the MANIFEST phase 2 study of pelabresib in patients with myelofibrosis;

– Continuation of the MANIFEST-2 phase 3 clinical study with pelabresib in combination with ruxolitinib in patients with primary myelofibrosis;

– Continuation of a Phase 1/2 clinical trial of CPI-0209 in patients with advanced solid and hematological tumors.

MorphoSys will hold its conference call and webcast tomorrow, July 29, 2021, to present the results for the second quarter and first half year of 2021 and the further outlook for 2021.

A live webcast and slides will be made available at the Investors section under "Presentations and Conferences" on MorphoSys’ website at View Source and after the call, a slide-synchronized audio replay of the conference will be available at the same location.

The statement for the second quarter/first 6 months of 2021 (IFRS) is available online:
View Source/Reports

About Tafasitamab
Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi(R) (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi(R) is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

On July 28, 2021 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat specialty cancers and urologic diseases, and Neopharm group ("Neopharm"), reported an exclusive license for Neopharm to market and sell Jelmyto (mitomycin) for pyelocalyceal solution in Israel, subject to regulatory approval (Press release, UroGen Pharma, JUL 28, 2021, View Source [SID1234585293]). Jelmyto is the first and only U.S. Food and Drug Administration approved medicine for adult patients with low-grade upper tract urothelial cancer (LG-UTUC).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Neopharm will lead the regulatory process in Israel, which is supported by the results from the Phase 3 OLYMPUS trial that showed Jelmyto achieved clinically significant disease eradication in adults with LG-UTUC.

"We are excited to work with Neopharm and take the first step in making Jelmyto available to patients in Israel," said Liz Barrett, President and Chief Executive Officer of UroGen. "As we begin the process of geographic expansion for Jelmyto, it was important for us to prioritize Israel as the first opportunity to bring this innovative treatment to patients outside of the United States. Beyond the fact that our company was founded in Israel, and we continue to have a significant presence there, physicians and patients in Israel played a key role in the pivotal study that supported Jelmyto’s approval in the United States."

Jelmyto, which received expedited approval in the United States in April 2020, is an innovative therapy utilizing UroGen’s proprietary sustained release RTGel technology in combination with mitomycin, an established chemotherapy that inhibits DNA synthesis. It has been designed to dwell in the cavity, enabling mitomycin to have longer exposure to and broader coverage of urinary tract tissue, thereby allowing the treatment of tumors by non-surgical means.

"We look forward to working with UroGen to move Jelmyto through the regulatory process in Israel and to make this novel, non-surgical treatment available to appropriate patients as quickly as possible," said Efi Shnaidman, general manager of Neopharm Israel. "We are proud to be the first company outside the US to have started the regulatory approval and commercialization process for Jelmyto, demonstrating Israel’s importance in geographic expansion. Moreover, it is exciting to work with another company who has deep roots in Israel. I am confident that our well-established expertise and heritage of collaboration with innovative biopharmaceutical companies will make Jelmyto a success in Israel."

In addition to Israel, UroGen continues to work with regulators and potential collaborators in key markets to explore opportunities for geographic expansion. UroGen is committed to bringing the promise of Jelmyto to as many patients as possible, as quickly as feasible.

About Jelmyto

Jelmyto (mitomycin) for pyelocalyceal solution, is a drug formulation of mitomycin indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters or nephrostomy tube. The U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC. On April 15, 2020, the FDA approved Jelmyto, making it the first drug approved for the treatment of LG-UTUC in adult patients.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.
Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.

On July 28, 2021 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized CAR T platform technology engineered to target patient- and tumor-specific neoantigens, reported the closing of its previously announced private placement with a single healthcare-focused institutional investor for 4,629,630 shares of its common stock (or common stock equivalents) at a purchase price per share of $2.70, priced at-the-market under Nasdaq rules (Press release, Xenetic Biosciences, JUL 28, 2021, View Source [SID1234585292]). The gross proceeds to Xenetic totaled approximately $12.5 million before deducting placement agent fees and other offering expenses.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

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

Additionally, Xenetic issued to the investor warrants to purchase up to 4,629,630 shares of common stock. The warrants to purchase 4,629,630 shares of common stock have an exercise price of $3.30 per share, will be immediately exercisable and will expire three and one half years from the earlier of (a) the six month anniversary of the initial exercise date and (b) the date that the registration statement registering all of the warrant shares underlying the warrants is declared effective. The potential gross proceeds from the exercise of the warrants, if fully exercised on a cash basis, will be approximately $15.3 million. No assurance can be given that any of the warrants will be exercised. Xenetic intends to use the net proceeds from the offering for general working capital purposes.

The securities described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act") and Regulation D promulgated thereunder, and the securities have not been registered under the Act or applicable state securities laws. Accordingly, the securities may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws. Pursuant to a registration rights agreement with the investor, the Company has agreed to file a resale registration statement covering the shares of common stock, common stock equivalents, and shares of common stock underlying the warrants described above within 30 days.

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.

On July 28, 2021 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness a patient’s innate immune system to fight disease, reported that it will host a conference call on Wednesday, August 4, 2021 at 4:30 PM Eastern Time to discuss results for its second quarter ended June 30, 2021 and to provide a corporate update (Press release, INmune Bio, JUL 28, 2021, https://inmunebio.com/index.php/en/news-2/2021/464-muneionctoeporteconduarter2021inanciale20210728 [SID1234585291]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Conference Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call. Please ask for the INmune Bio Second Quarter Conference Call when reaching an operator.

A transcript will follow approximately 24 hours from the scheduled call. A replay will also be available through August 11 by dialing 1-844-512-2921 or 1-412-317-6671 (international) and entering PIN no. 13721921.

On July 28, 2021 Prescient Therapeutics (ASX:PTX) reported its cancer-fighting drug candidate has exhibited an excellent safety in an early clinical trial – and seemed to benefit two patients with aggressive, hard-to-treat lymphoma (Press release, Prescient Therapeutics, JUL 28, 2021, View Source;utm_medium=rss&utm_campaign=very-encouraging-two-cancer-patients-see-partial-remission-and-long-lasting-benefits-after-treatment-with-prescients-ptx-100 [SID1234585290]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Prescient says it is encouraged by a Phase 1b trial evaluating PTX-100 in ten patients: five with solid tumours — pancreatic and colorectal cancer – and five patients with blood cancer, namely multiple myeloma and T cell lymphomas.

The drug was well-tolerated, even at the highest doses, Prescient says. An intravenous infusion, PTX-100 works by blocking an important protein known as GGT-1 that is involved in cancer-causing pathways in cells.

The safety profile of the drug means it might have utility in fragile patients unable to tolerate more toxic therapies, or as a combination agent with other treatments, says Prescient chief executive and managing director Steven Yatomi-Clarke.

And while the primary goal of the study was to evaluate safety, clinical benefit was observed in two patients with aggressive T cell lymphoma.

One patient had tried five different therapies to control their peripheral T cell lymphoma, but none had worked for more than a few months at a time.

Treated with PTX-100, the patient experienced a partial remission in their cancer that has lasted 17 months so far. The patient has undergone 24 cycles of therapy and is still receiving PTX-100.

The other patient also had partial remission, seeing reduced cancerous lesions and relief from their cutaneous T cell lymphoma during 12 months of therapy. This patient had tried three prior treatments that had failed to control their cancer.

Both patients could have expected disease progression within four months using currently available treatments, highlighting the benefits of PTX-100.

"It was surprising to not only see clinical responses in these patients, but for these benefits to endure for a year or longer, together with symptomatic relief," says principal study investor, Professor H. Miles Prince, who called the early results "very encouraging".

A possible quick route to market
Given the success of the Phase 1b study, Prescient plans to progress development of PTX-100 with another study into refractory T cell lymphoma, with a particular focus on peripheral T-cell lymphoma.

There’s a considerable unmet need in treating that aggressive, rare form of non-Hodgkin’s lymphoma with poor survival rates.

The study will evaluate PTX-100 as a monotherapy, meaning the infusion won’t be administered in combination with other drugs.

The company plans to enrol eight to 12 patients in the expansion of the study, which will again be led by Professor Prince at Epworth Hospital in Melbourne.

If that study successfully demonstrates a clinical benefit for PTX-100, the drug could advance directly into another study that could be suitable for registration.

Compared to the usual large Phase 3 studies, a comparatively smaller trial might be all that’s needed to register the drug, Prescient says.

Yatomi-Clarke says the company is pursuing the quickest route to market for PTX-100 by pursuing areas of unmet clinical need.