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CRISPR Therapeutics Announces the Appointment of Phuong Khanh Morrow, M.D., FACP, as Chief Medical Officer

On May 16, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported the appointment of Phuong Khanh (P.K.) Morrow, M.D., FACP, as Chief Medical Officer, effective May 23, 2022 (Press release, CRISPR Therapeutics, MAY 16, 2022, View Source [SID1234614634]). Dr. Morrow brings more than a decade of leadership experience in global drug development and joins CRISPR Therapeutics to lead the Company’s global clinical development and regulatory operations.

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"P.K.’s leadership experience, deep expertise in oncology drug development, and her track record in bringing novel medicines to patients will be invaluable as we continue to advance our broad portfolio of innovative gene-edited therapies," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "She will play an integral role in shaping our clinical development strategy, and in building and strengthening our organization, and we look forward to her contributions as we continue on our mission to transform medicine."

"I am excited to join CRISPR Therapeutics’ leadership team at this important time in its growth and evolution," said Dr. Phuong Khanh (P.K.) Morrow. "The potential of the Company’s pre-eminent gene editing platform combined with the broad pipeline creates a tremendous opportunity to bring several novel and potentially transformative therapies to patients in need."

During her biopharmaceutical career, Dr. Morrow has demonstrated outstanding leadership in bringing novel medicines through all phases of clinical development and global regulatory approval. Specializing in the therapeutic areas of oncology and hematology, she has been responsible for end-to-end development of numerous drug candidates and for the implementation of strategic partnerships with academic institutions, key opinion leaders and biopharmaceutical co-collaborators to facilitate the successful execution of clinical trials. Dr. Morrow most recently served as Vice President and Global Therapeutic Area Head of Hematology, GI Oncology, GU Oncology, and Bone at Amgen, where she was responsible for guiding and accelerating late development activities addressing marketed hematology programs, Blincyto and Kyprolis, and guiding the late development strategy for programs that focus upon FLT3 and MCL-1. She also led the medical launch activities for Imlygic, Kyprolis, Neulasta Onpro and Blincyto; served as the Global Product General Manager for three early-stage oncology molecules focused upon MCL-1 and KRAS G12C; and led a cross-functional team in the development and registration of Neulasta Onpro and the successful submission of the Neupogen and Neulasta Acute Radiation Syndrome (ARS) sBLAs, leading to the regulatory approval of both products for the ARS indication. In addition, Dr. Morrow was appointed by the U.S. Food and Drug Administration (FDA) to be the industry representative to the Oncology Drug Advisory Committee (ODAC) for a four-year term, ending in 2019.

Previously, Dr. Morrow was Assistant Professor, Department of Breast Medical Oncology at the University of Texas MD Anderson Cancer Center. She co-led the development of the first multidisciplinary breast cancer survivorship clinic at MD Anderson and served as the principal investigator of multiple drug studies. Dr. Morrow received an M.D. from the University of Texas Medical School at Houston, with honors, and completed her Internal Medicine Residency at Baylor College of Medicine and Hematology/Oncology Fellowship at the University of Texas MD Anderson Cancer Center, where she also served as a Chief Fellow. She received a B.S. in Pharmacy from the University of Houston.

UroGen Highlights New Data Presented at AUA that Adds to the Evidence Supporting In-Office Nephrostomy Tube Administration of JELMYTO®, the only Non-Surgical, Kidney-Sparing Treatment for Adults with Low Grade Upper Tract Urothelial Cancer

On May 16, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to creating novel solutions that treat urothelial and specialty cancers, reported that highlights new data on real-world experience utilizing the antegrade approach via nephrostomy tube for administration of JELMYTO (mitomycin) for pyelocalyceal solution (Press release, UroGen Pharma, MAY 16, 2022, View Source [SID1234614633]). This data adds to a growing body of evidence on the safety and efficacy profile of the antegrade method of administration for JELMYTO. These data were presented during a podium presentation at the 2022 American Urological Association (AUA) annual meeting in New Orleans, Louisiana.

"JELMYTO is efficacious as a chemoablative agent in adult patients with low grade upper tract urothelial cancer, and while it’s FDA approved for both antegrade and retrograde administration, prior reports are limited to the retrograde experience," said Kyle Rose, MD, Urologic Oncology Fellow at Moffitt Cancer Center in Tampa, Fla., and study investigator. "These data provide additional evidence that instillation via a nephrostomy tube is an effective instillation method with a safety profile that offers an encouraging option to appropriate patients."

Dr. Rose presented the abstract Antegrade Administration of Reverse Thermal Mytomycin Gel for Primary Chemoablation of Upper Tract Carcinoma via Percutaneous Nephrostomy Tube: A Multi-Institutional Real-World Experience (Abstract PD58-06) during a podium presentation at the AUA annual meeting on Monday, May 16.

"All 71 patients in the Phase 3 OLYMPUS trial utilized the retrograde approach to administer JELMYTO, therefore we are pleased to see real-world evidence that supports the utilization of the antegrade approach giving physicians and patients more options to administer JELMYTO based on their preference and experience," said Mark Schoenberg, MD, Chief Medical Officer, UroGen.

About This Study

The real-world data from this retrospective analysis was pooled from Moffitt Cancer Center, Tampa, FL; University of Missouri School of Medicine, Columbia, MO; The University of Texas MD Anderson Cancer Center, Houston, TX; and Mayo Clinic, Rochester, MN.

Twenty-six patients received nephrostomy tube administration of JELMYTO, six patients (23%) had solitary kidneys. Nine patients (35%) went on to receive at least one dose of maintenance therapy. Ureteral stenosis occurred in four patients (15%). Other adverse events included fatigue (27%), flank pain (19%), urinary tract infection (12%), sepsis (8%) and hematuria (8%). No patients had impaired renal function during follow-up and no deaths occurred.

Thirteen patients (50%) exhibited a complete response at post-induction ureteroscopy while 12 patients (46%) had a partial response. One patient experienced progression to invasive disease and required a nephroureterectomy. At a median follow-up of seven months (IQR 3-9) post-induction, no patients who experienced a complete response recurred.

The limitations of this study include the retrospective nature, small sample size, and pooled reporting of results. There is a need for larger studies with longer follow-up to study more conclusively any potential advantages of antegrade JELMYTO administration when compared to retrograde instillation.

About the Pivotal OLYMPUS Study

OLYMPUS (Optimized DeLiverY of Mitomycin for Primary UTUC Study) was an open-label, single-arm Phase 3 clinical study of UGN-101 JELMYTO (mitomycin) for pyelocalyceal solution, to evaluate the safety, tolerability and tumor ablative effect of JELMYTO in patients with low-grade Upper Tract Urothelial Cancer UTUC (LG UTUC). Seventy-one patients were treated at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of JELMYTO administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine Complete Response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer and for cause biopsy. Patients who achieved a CR at the PDE timepoint were eligible for the maintenance phase of the trial, during which they could receive monthly maintenance instillations for up to 12 months and were assessed to determine the durability of response with JELMYTO.

In the OLYMPUS study, data was generated for the retrograde administration of JELMYTO. In that study population ureteric obstruction was reported in 58% (n=41) of patients receiving JELMYTO, including 17% (n=12) of patients who experienced Grade 3 obstruction.

About LG UTUC

LG UTUC is a rare disease managed by endoscopic methods and radical nephroureterectomy. Endoscopic resection and laser ablation attempt to preserve the kidney, though there is a high risk of recurrence that may eventually necessitate removal of the kidney. Although kidney removal is the gold standard for treatment of high-grade UTUC, it may be over-treatment in LG UTUC, as kidney removal offers similar five-year survival as kidney-sparing procedures but is associated with significant morbidity. JELMYTO is efficacious as a primary chemoablative therapy in patients with LG UTUC.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for primary chemoablative treatment of LG UTUC in adults. It is recommended for primary treatment of biopsy-proven LG UTUC in patients deemed appropriate candidates for renal-sparing therapy. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

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 U.S. Food and Drug Administration. 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.

Lilly to Participate in UBS Global Healthcare Conference 2022

On May 16, 2022 Eli Lilly and Company (NYSE: LLY) reported that it will attend the UBS Global Healthcare Conference on Tuesday, May 24, 2022 (Press release, Eli Lilly, MAY 16, 2022, View Source [SID1234614632]). Michael Mason, senior vice president, president of Lilly Diabetes, will participate in a fireside chat at 8:30 a.m., Eastern time.

A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

Aprea Therapeutics Announces Acquisition of Atrin Pharmaceuticals Inc. and Reports First Quarter 2022 Financial Results

On May 16, 2022 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics targeting DNA damage response pathways reported that it has acquired Atrin Pharmaceuticals Inc. ("Atrin") and reported financial results for the three months ended March 31, 2022 (Press release, Aprea, MAY 16, 2022, View Source [SID1234614630]).

Business Operations Update:

On May 16, 2022 Aprea completed the acquisition of Atrin, a privately held biotechnology company focused on the discovery and development of novel therapeutics targeting proteins in the DNA damage response, or DDR, pathway in oncology through synthetic lethality. The Company believes its cash and cash equivalents as of March 31, 2022 will be sufficient to meet its current projected operating requirements through the second half of 2023.

With the acquisition of the Atrin programs, Aprea intends to shift its primary focus to the ATR inhibitor ATRN-119, which will be studied as both a monotherapy and in combination with standard of care in Phase 1/2a clinical trials in solid tumor malignancies. In parallel with ATRN-119 development, Atrin initiated separate programs for a second-generation ATR inhibitor, ATRN-354, with potentially improved potency and pharmacokinetics, as well as for a potentially highly potent and selective WEE1 inhibitor – ATRN-W1051. These programs are expected to enter IND-enabling studies in 2022 and Aprea anticipates filing Investigational New Drug applications for these programs in 2023. Furthermore, with the acquisition of Atrin, Aprea obtained an active proprietary development platform to drive the identification of new oncology drug targets and biomarkers.

"Aprea has explored a range of strategic alternatives to maximize shareholder value and we believe the acquisition of Atrin represents an opportunity to create substantial value for Aprea shareholders," said Christian Schade, Chairman and Chief Executive Officer of Aprea. "The founders of Atrin are pioneers in the development of novel oncology compounds in the DNA damage pathway. We are excited to work with the Atrin team to begin clinical development of Atrin’s lead ATR program over the coming months, hasten the development of its differentiated WEE1 program, and support a robust platform to build a pipeline of novel inhibitors of the DDR pathway."

"We are excited to join forces with Aprea and expedite the clinical development of our lead programs and drug development platform," said Oren Gilad, Ph.D., the former Chief Executive Officer of Atrin. "Aprea’s knowledge of the p53 pathway in oncology is complimentary to Atrin’s in-house expertise in developing novel targets in the DNA damage response pathway. With supportive resources we look forward to moving our programs into clinical development to better understand the importance of our robust synthetic lethality platform."

Management and Organization

Christian Schade, our current Chairman and Chief Executive Officer will remain with Aprea along with Scott Coiante, our current Senior Vice President and Chief Financial Officer, and Greg Korbel will remain with Aprea, but will transition from his current role as Senior Vice President and Chief Business Officer to Senior Vice President and Chief Operating Officer. Our management team will be expanded with the appointment of Dr. Gilad, the former Chief Executive Officer of Atrin, who will be President of Aprea and Ze’ev Weiss, the former Chief Business Officer of Atrin, who will be the Chief Business Officer of Aprea. After the 2022 annual stockholder meeting of Aprea, Dr. Gilad will assume the role of Chief Executive Officer from Mr. Schade and Mr. Schade will continue as Executive Chairman of the Board of Directors.

In conjunction with the transaction, the Aprea board was expanded to eight members. Immediately following the consummation of the transaction, the Aprea board will consist of, Dr. Gilad, Mike Grissinger, Rif Pamukcu, and Marc Duey, each of whom were members of the Atrin board who will join the Aprea board, and current Aprea Board members Christian Schade, Jack Henneman, Richard Peters and Bernd Seizinger. Christian Schade will remain the Executive Chairman of the Aprea Board for up to six months, and he would remain as Non-Executive Chairman thereafter. Aprea and Atrin have agreed to expand the Aprea board to nine members following Aprea’s 2022 annual stockholder meeting. The Aprea Board plans to fill the remaining vacancy after such meeting.

About the Transactions

The acquisition of Atrin was structured as a stock-for-stock transaction whereby all of Atrin’s outstanding equity interests were exchanged for a combination of shares of Aprea common stock and shares of Series A non-voting convertible preferred stock (the "Series A preferred stock"). Subject to Aprea stockholder approval, each share of Series A preferred stock will, at the option of the holder, convert into 10 shares of common stock, subject to certain beneficial ownership limitations set by each holder. On a pro forma basis and based upon the number of shares of Aprea common stock and preferred stock issued in the acquisition, holders of Aprea equity holders immediately prior to the acquisition will own approximately 41.2% of Aprea on an as-converted basis and former Atrin equity holders will own approximately 58.8% of Aprea on an as-converted basis immediately after these transactions. The acquisition was approved by the Board of Directors of Aprea and the Board of Directors and the requisite equity holders of Atrin. The closing of the transactions was not subject to the approval of Aprea stockholders.

In connection with acquisition of Atrin, each holder of Aprea common stock as of immediately before the closing of the transaction will be entitled to a non-transferrable contingent value right ("CVR"). Holders of the CVR will be entitled to receive certain payments from proceeds received by Aprea, if any, related to the disposition, if any, of its legacy p53 reactivator assets during the 2-year period following the closing of the transaction.

Wedbush PacGrow is serving as exclusive strategic advisor to Aprea, H.C. Wainwright & Co. is serving as exclusive strategic advisor to Atrin, Sidley Austin LLP is serving as legal counsel to Aprea, and DLA Piper LLP (US) is serving as legal counsel to Atrin.

Immediately after the completion of the acquisition, Aprea is expected to have pro forma cash on hand, before transaction-related expenses, of approximately $48 million (unaudited) and cash runway through the second half of 2023.

Additional details will be available in an updated corporate presentation that can be found in the investor section of the Aprea website at Link.

First Quarter Financial Results

Cash and cash equivalents: As of March 31, 2022, the Company had $47.6 million of cash and cash equivalents compared to $53.1 million of cash and cash equivalents as of December 31, 2021. The Company believes its cash and cash equivalents as of March 31, 2022 will be sufficient to meet its current projected operating requirements through the second half of 2023.
Research and Development (R&D) expenses: R&D expenses were $4.1 million for the quarter ended March 31, 2022, compared to $6.8 million for the comparable period in 2021. The decrease in R&D expenses was primarily due to decreases in clinical trial activity and related costs for (i) our pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for the frontline treatment of TP53 mutant MDS, (ii) our Phase 2 post-transplant MDS/AML clinical trial, (iii) our Phase 1 AML trial, (iv) our Phase 1/2 solid tumor trial and our Phase 1 dose-escalation trial of APR-548.
General and Administrative (G&A) expenses: G&A expenses were $4.0 million for the quarter ended March 31, 2022, compared to $3.4 million for the comparable period in 2021. The increase in G&A expenses was primarily due to increases in legal expense and non-cash stock-based compensation expense.
Net loss: Net loss was $7.9 million, or $0.36 per share for the quarter ended March 31, 2022, compared to a net loss of $9.7 million, or $0.46 per share for the quarter ended March 31, 2021. The Company had 21,974,302 shares of common stock outstanding as of March 31, 2022.
Conference Call and Webcast Details

Aprea will host a webcast on May 16, 2022 at 4:30pm EDT to discuss the acquisition. The webcast can be accessed under the "Events Calendar" in the Investors section of the Aprea website at Link

Proteros Expands Oncology Collaboration and License Agreement with AstraZeneca

On May 16, 2022 Proteros biostructures GmbH ("Proteros"), an expert in integrated structure-based drug discovery, reported an expansion of its collaboration with AstraZeneca (LSE/STO/Nasdaq: AZN) focused on the discovery and development of novel epigenetic drugs (Press release, AstraZeneca, MAY 16, 2022, View Source [SID1234614614]). The new multi-year agreement builds on an ongoing collaboration with AstraZeneca announced in June 2021, to include the development of small molecule inhibitors targeting a second cancer-associated epigenetic protein.

Under the terms of this new agreement, Proteros will receive research funding and will be eligible for success-based research, development, and commercial milestone payments of up to USD 75 million plus tiered royalties on annual net sales. The collaboration combines Proteros’ expertise in the identification and characterization of inhibitors with novel binding mechanisms for technically challenging drug targets with AstraZeneca’s leadership in the discovery and development of oncology medicines.

"The expansion of our agreement with AstraZeneca with an additional drug discovery program reflects our successful ongoing collaboration to identify selective inhibitors for notoriously challenging disease targets," said Dr. Torsten Neuefeind, CEO of Proteros. "This agreement strengthens our collaboration with a global biopharmaceutical leader and we look forward to joining forces again to discover novel inhibitors with the potential to effectively treat cancer patients in the future."

The Proteros platform will ensure high selectivity to a specific target variant within and across multiple protein families through biochemical, biophysical and cellular assays supported by rapid turnaround in the structural determination of drug-target interactions by X-ray crystallography and cryo-Electron Microscopy technologies.