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X-37 Announces $14.5 Million Series A

On November 14, 2019 X-37, LLC, an artificial intelligence-enabled drug discovery company, reported that it has closed a $14.5 million Series A financing (Press release, X-37, NOV 14, 2019, View Source [SID1234551317]). The funding round was led by DCVC Bio and was joined by Alpha Intelligence Capital and Hemi Ventures. The Series A funding will be used to expand the number of drug development programs at X-37 and to advance identified drug leads through laboratory and preclinical testing, with the goal of beginning human clinical trials by 2022. X-37’s development programs encompass novel therapeutics modulating important drug targets to address unmet clinical needs, including ZAP-70 for autoimmune disease, PIM3 and SHP2 for cancer, and Factor XIIa for anticoagulation.

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X-37 was cofounded by Atomwise Inc. and a team of experienced pharmaceutical developers from Velocity Pharmaceutical Development. In addition to the above development programs, the team at X-37 will identify additional high-value drug targets, generate novel drug leads against these targets using Atomwise’s world-class AI platform for structure-based drug design, and develop each of these drug programs to a medically-relevant inflection point, where it can be acquired by or partnered with a major pharmaceutical company to be brought to market.

X-37 makes use of an LLC structure permitting each drug development program to be housed in a separate virtual company under the parent LLC. This structure is tax efficient and flexible, in that it allows X-37 to divest individual drug development programs, while maintaining the parent company and team. X-37 began operation in 2018 and has already generated promising novel hit molecules against several targets of high interest to the pharmaceutical industry.

"We are thrilled with the progress we have made to date and we are looking forward to moving a set of important new drugs into development," said David Collier, M.D., CEO and cofounder of X-37. "With this financing, we have the ability to progress multiple discovery and development programs. Our investors have been a huge help in the genesis of X-37, and we look forward to working closely with them going forward."

"X-37 represents an opportunity to bring new drugs to market very quickly, for challenging targets that have repeatedly stumped pharma and are in dire need of advancement," said Abraham Heifets, CEO and Founder of Atomwise, Inc. "We’re thrilled to work alongside industry veterans who have a track record of efficiently delivering drug after drug for the past 25 years."

"We are very pleased to be investors in X-37," said Kiersten Stead of DCVC Bio. "We have watched with great excitement as Atomwise has refined its deep neural network and chemistry expertise. We identified the team of drug developers at Velocity Pharmaceutical Development as ideal partners for Atomwise because of their experience in target selection and development of drug leads beyond the initial discovery stage. The progress of the combined teams at X-37 has been superb."

Following the financing, Kiersten Stead and Antoine Blondeau of Alpha Intelligence Capital joined the Board of Directors of X-37. The founding leadership team of X-37 is comprised of industry veterans and luminaries, including: David Collier, M.D., Matthew Kerby, Ph.D., PE, James Larrick, M.D., Ph.D., Andrew Perlman, M.D., Ph.D., Anie Roche, J.D., Ph.D., and Ed Schnipper, M.D., who together have brought 17 therapies to the market addressing an even greater number of indications.

About the Investors

DCVC Bio is a Silicon Valley venture capital fund focused on deep technology ventures that lie at the nexus of artificial intelligence (AI) and biotechnology. Alpha Intelligence Capital is a global venture capital fund investing in advanced AI technology-based companies. Hemi Ventures is an early stage venture capital fund investing in the rebels that commercialize nascent technologies to revolutionize industries.

Gossamer Bio Announces Participation in the Guggenheim Healthcare Talks Neuro/Immunology Day

On November 14, 2019 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported that Sheila Gujrathi, M.D., Chief Executive Officer and Co-Founder, will participate in a fireside chat at the Guggenheim Healthcare Talks Neuro/Immunology Day on Monday, November 18 at 1:00 p.m. ET (Press release, Gossamer Bio, NOV 14, 2019, View Source [SID1234551316]).

A live webcast of the presentation will be available on the "Events and Presentations" page in the "Investors" section of the company’s website at View Source A replay of the webcast will be archived on the company’s website for 90 days following the presentation.

Bicycle Therapeutics to Present at the Jefferies 2019 London Healthcare Conference

On November 14, 2019 Bicycle Therapeutics plc (NASDAQ: BCYC), a clinical-stage biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported that management will present at the Jefferies 2019 London Healthcare Conference on Wednesday, November 20, 2019 at 2:40 p.m. GMT (9:40 a.m. ET) in London, England (Press release, Bicycle Therapeutics, NOV 14, 2019, View Source [SID1234551315]).

A live webcast of the presentation can be accessed in the Investors & Media section of Bicycle’s website at www.bicycletherapeutics.com. An archived replay of the webcast will be available for 90 days following the presentation date.

ALX Oncology to Present ALX148 Clinical Data at the 61st American Society of Hematology Annual Meeting (ASH)

On November 14, 2019 ALX Oncology, a clinical-stage immuno-oncology company developing therapies to block the CD47 checkpoint mechanism, reported that ALX148 clinical results have been selected for presentation at the 61st ASH (Free ASH Whitepaper) Annual Meeting & Exposition, December 7 – 10, 2019, Orange County Convention Center, Orlando, FL (Press release, ALX Oncology, NOV 14, 2019, View Source [SID1234551314]).

"This year’s ASH (Free ASH Whitepaper) meeting will be an opportunity to provide important clinical updates on our CD47 program in hematologic malignancies," said Sophia Randolph M.D., Ph.D., Chief Medical Officer of ALX Oncology. "We are excited to share the first clinical efficacy data from the combination of ALX148 with rituximab, showing that ALX148 maximizes clinical activity with a best-in-class safety profile. We are committed to further development of this agent with the potential to transform standards-of-care for patients with cancer."

Abstract Details
The presentation will include updated clinical data from the ongoing phase 1b study of ALX148, administered up to 15 mg/kg once weekly (molar equivalent to 30 mg/kg once weekly of an antibody), in combination with rituximab in patients with relapsed/refractory non-Hodgkin lymphoma.

Title: A Phase 1 Study of ALX148, a CD47 Blocker, in Combination with Rituximab in Patients with Non-Hodgkin Lymphoma
Session Name: 704. Immunotherapies: Poster I
Session Date: Saturday, December 7, 2019
Presentation Time: 5:30pm – 7:30pm
Location: Orange County Convention Center, Hall B
Publication Number: 1953

Aadi Bioscience Breakthrough Therapy nab-Sirolimus (ABI-009) Independent Reviewed Data Released from the AMPECT Registration Trial in Advanced PEComa

On November 14 , 2019 Aadi Bioscience, Inc. (Aadi), a privately held clinical stage biopharmaceutical company, reported its independent radiology reviewed data from the nab-sirolimus (ABI-009) registration trial (AMPECT) for Advanced (metastatic or locally advanced) Malignant PEComa (perivascular epithelioid cell tumor) – a rare form of sarcoma for which there is no currently approved therapy (Press release, Aadi, NOV 14, 2019, View Source [SID1234551313]).

Nanoparticle albumin-bound sirolimus (nab-Sirolimus), an mTOR inhibitor, received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) in Dec 2018, "for the treatment of patients with advanced (metastatic or locally advanced) malignant perivascular epithelioid cell tumor (PEComa)."

The AMPECT trial was conducted at 9 U.S. sites and treated 34 adult patients, with 31 confirmed as PEComa by a central pathology laboratory. PEComa origin sites in these patients included the uterus, pelvis, retroperitoneum, lung, kidney, liver, brain, muscle, ovary, aorta, and small bowel.

Results of the primary analysis were released in an oral presentation (abstract 3206452) at the CTOS 2019 annual meeting in Tokyo, Japan, demonstrating a 39% confirmed independent radiology reviewed objective response rate (ORR) in patients with advanced PEComas originating in various tissues. Responses were rapid and durable with half the responders having an ongoing response duration of 15.3 months or longer and the majority of responders (67%) are still on treatment. AMPECT Investigator and lead author on the oral presentation, Mark Dickson, M.D., from Memorial Sloan Kettering Cancer Center, said "nab-sirolimus delivered highly durable responses in advanced PEComa patients. Most of the responding patients achieved a response by their first assessment at 6 weeks following initiation of therapy and these patients have stayed on therapy for extended periods with a manageable safety profile. We are encouraged by the outcomes in this first-ever prospective clinical trial in advanced PEComa." A second presentation by the principal investigator of the AMPECT trial, Dr Andrew Wagner M.D., Ph.D., from the Dana-Farber Cancer Institute, highlighted the exploratory mutational analysis from the study (abstract 3258096). "Patients having mutations in the TSC2 gene were found to have an 89% confirmed response rate to nab-sirolimus, while patients with TSC1 mutations or no mutations in the TSC1 or TSC2 genes had lower response rates. This is the first prospective study of outcomes vs mutational status of mTOR pathway genes and highlights the relevance of TSC2 mutations in advanced PEComa," Dr. Wagner said.

Neil Desai, Ph.D., Chief Executive Officer of Aadi Bioscience, said "The Aadi Bioscience team is grateful to the patients, families, and clinical trial teams who help expand the boundaries of available care through their participation in clinical trials. These results are an important milestone in the ongoing development of nab-sirolimus across a wide range of diseases and therapeutic indications that are driven by mTOR activation and for which there is a need for new therapies." Dr. Desai added: "Results from the AMPECT study will serve as the basis for the New Drug Application (NDA) for nab-sirolimus, which the company expects to submit to the FDA in the first half of 2020."

Key Data Presented at CTOS

The primary endpoint for the AMPECT study presented at CTOS is independent central radiology reviewed ORR, assessed by RECIST v 1.1. Key secondary endpoints include duration of response (DOR), progression-free survival (PFS), PFS rate at 6 months (PFS6), and safety. The data presented at CTOS represent a May 22, 2019 data cut-off for the primary analysis, with an additional 5.5 months of follow-up (Nov 6, 2019) for the duration of response endpoint.

The data presented are based on 34 patients evaluable for safety and 31 patients evaluable for efficacy per defined criteria in the protocol.

Best Response Assessment

Ninety percent of patients had a best response PR or SD, with disease control (defined as a confirmed response + SD ≥12 weeks) achieved in 71% of patients.

As of Nov 6, 2019, 75% (9/12) of responders had been on therapy for more than 1 year and 42% (5/12) for more than 2 years, with 67% (8/12) still on treatment. Median DOR has not been reached (range [5.6 –33.2+ months]) and 50% of the responders have a response duration that is 15.3 months or longer; the median time to response was 1.4 months (95% CI: 1.3, 2.7).

Median PFS is 8.9 months (95% CI: 5.5, –), PFS rate at 3 months (PFS3) is 79%, PFS6 is 70%, and 26% (9/34) of all patients enrolled remain on treatment. For reference, per a meta-analysis of 10 years of phase 2 trials in advanced soft tissue sarcomas (STS) published by the EORTC STS and Bone Sarcoma Group [2], the PFS3 and PFS6 are widely accepted as a meaningful measure of activity of drugs in STS and may be utilized to determine acceptable criteria of benefit. Drugs yielding a PFS rate of ≥40% at 3 months and ≥14% at 6 months are considered to be ‘potentially active’ in advanced STS [1].

A protocol prespecified exploratory mutational and biomarker analysis was available for 25 patients on the AMPECT trial. Mutational status of the suspect genes TSC1 or TSC2 in the mTOR pathway were analyzed for association with patient response outcomes. Mutation or deletion of TSC1 or TSC2 (no overlap) occurred in 5 (20%) and 9 (36%) patients respectively, while 11 (44%) patients had no alterations in TSC1 or TSC2. Responses occurred in 9/9 (100%, 8 confirmed responses [89%], 1 unconfirmed response [11%]) patients with TSC2 mutations, 1/5 (20%) patients with TSC1 mutations and 1/11 (9%) of patients with no mutations in TSC1 or TSC2.

The safety data presented at CTOS was available for all 34 patients treated on the AMPECT trial. The most common treatment-related hematologic adverse events of any grades included anemia (47%) and thrombocytopenia (32%) and the most common nonhematologic treatment-related adverse events of any grades included mucositis (79%), rash (56%), fatigue (59%), nausea (47%), and diarrhea (38%). Most of these events were grade 1 and 2, were manageable with dose modifications and no grade 4 events were observed. Twelve patients (35%) required dose reductions due to adverse events. Two patients (6%) discontinued nab-sirolimus due to an adverse event. There were no new or unexpected toxicities not previously known for mTOR inhibitor class.

The AMPECT Phase 2 registration trial for Advanced Malignant Perivascular Epithelioid Cell Tumors completed enrollment in late 2018. Aadi previously received agreement from the FDA that this open label study in at least 30 efficacy evaluable patients with a primary endpoint of independently reviewed ORR, could support the submission of an NDA for approval to treat this rare disease assuming a 30% response rate was observed and the lower bound of the 95% confidence interval of the ORR exceeded 14.7%. These data have not been reviewed by any regulatory agencies.