BPC September 10 update

Updated September biotech catalysts to watch; Biotech week in review

Weekly watchlist

This week we refresh our list of catalysts slated for September. To start the week all eyes will be on the upcoming ESMO conference with abstracts to be released early Monday morning.

First, let’s review the week that was with notable price-moving events in the healthcare sector. 


Apellis Pharmaceuticals, Inc. (NASDAQ: APLS) reported Thursday divergent top-line results from the Phase 3 DERBY and OAKS studies of intravitreal pegcetacoplan, an investigational targeted C3 therapy, to treat geographic atrophy (GA). The OAKS trial met the primary endpoint and Apellis expects to file a New Drug Application in the first half of 2022. However, the DERBY trial did not meet the primary endpoint.

Iveric Bio, Inc. (NASDAQ: ISEE) shares rallied in response to Apellis’ data release, closing the week up 38% at $14.12. Iveric’s Zimura, a treatment for geographic atrophy, is currently undergoing a Phase 3 trial.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that its Phase 1/2 Phearless study of BMN 307 an AAV5-phenylalanine hydroxylase (PAH) gene therapy to treat phenylketonuria (PKU) has been placed on clinical hold. The FDA’s decision was based on a pre-clinical, non-GLP pharmacology study, and safety concerns that arose from the trial. Shares closed the week down 8% at $77.98.

Mersana Therapeutics (NASDAQ: MRSN) shares fell 41% following its report of interim data from its Phase 1 trial of Upifitamab Rilsodotin (UpRi/ XMT-1536) in patients with ovarian cancer. Only 2 complete responses out of the 75 patients (2.6%) were noted, while the overall response rate came in at 23% among all patients (17/75). 22 patients were not evaluable, predominantly due to deaths (10) and withdrawals (5). Of the 97 patients, 43 (44%) had dose delay, reduction, and/or discontinuation due to Treatment-Related Adverse Events (TRAEs).


Kadmon Holdings, Inc. (NASDAQ: KDMN) announced that they will undergo an acquisition by Sanofi (NASDAQ: SNY). Holders of Kadmon shares will receive $9.50 per share in cash. Sanofi expects to complete the acquisition in the fourth quarter of 2021. Shares closed the week up 68% at $9.09.

ProQR Therapeutics N.V. (NASDAQ: PRQR) closed the week up 19% to $8.20 following news that they will receive a $50 million upfront payment for a license agreement with Eli Lilly to use its Axiomer RNA editing platform. ProQR is eligible to receive up to $1.25 billion in milestone payments.

Adaptimmune Therapeutics PLC(NASDAQ: ADAP) announced that it will enter into a collaboration with Genentech, for the development and commercialization of the cancer-targeted allogeneic T-cell therapies. Adaptimmune receives $150 million upfront, $150 million over the next five years, and an additional $3 billion in milestone payments. Shares closed the week up 26% at $6.37.

Drug Price Stage Catalyst Market Cap

ADAP – Adaptimmune Therapeutics plc
Solid tumors

+0.11  +2%
Phase 1 Phase 1 data released September 13, 2021 - 4% of patients received CR, 28% received PR, 44% received SD, 12% received PD, 3% not evaluable. Additional data due at ESMO September 16-21, 2021.
$842.9 million

ALT – Altimmune Inc.
Non-alcoholic steatohepatitis (NASH) - Healthy Overweight and Obese Volunteers

-1.16  -7%
Phase 1 Phase 1 6-week MAD released June 16, 2021. Weight loss of 5.4% achieved at 6 weeks of treatment with 1.8 mg once weekly dose. 12-week data due September 2021. Phase 2 obesity and NASH trials expected to initiate in Q1 2022.
$605.1 million

ASLN – ASLAN Pharmaceuticals Limited
Atopic dermatitis

+0.19  +6%
Phase 1 Phase 1 top-line data due end of 3Q 2021. Phase 2b trial to be initiated 4Q 2021.
$224.2 million

EDIT – Editas Medicine Inc.
AGN-151587 (EDIT-101) - BRILLIANCE
Leber Congenital Amaurosis type 10 (LCA10).

+2.20  +4%
Phase 1/2 Phase 1/2 initial data due to be presented at the International Symposium on Retinal Degeneration planned for September 29, 2021.
$4.3 billion

EVLO – Evelo Biosciences Inc.
Mild to moderate psoriasis

+1.03  +14%
Phase 2b Phase 2b data due 3Q 2021.
$445.3 million

LIFE – aTyr Pharma Inc.
Pulmonary sarcoidosis

+0.05  +0%
Phase 1/2 Phase 1b/2a trial met primary endpoint September 13, 2021.
$174.4 million

MGNX – MacroGenics Inc.
Solid tumors

-0.51  -2%
Phase 1 Phase 1 data of mCRPC patients showed 25% (4) achieved partial responses (PR) (two confirmed and two unconfirmed). Of NSCLC patients, 25% (4) achieved unconfirmed partial responses (PR). At least one TRAE of any grade was experienced by 78 of 86 patients (91%), with 43 of 86 patients (50%) experiencing a Grade ≥3 TRAE. There were two Grade 5 fatal events: one from an unknown cause and one due to SARS-CoV-2. Noted September 16, 2021.
$1.3 billion

MRTX – Mirati Therapeutics Inc.
Adagrasib (MRTX-849), Erbitux (cetuximab) and KEYTRUDA (pembrolizumab) - KRYSTAL-01
Solid Tumors

+5.03  +3%
Phase 1/2 Phase 1/2 cetuximab data to be orally released at ESMO September 19, 2021.
$9 billion

RDHL – Redhill Biopharma Ltd.
YELIVA (Opaganib)

+0.07  +1%
Phase 2/3 Phase 2/3 preliminary top-line data results did not meet its primary endpoint September 14, 2021.
$228.5 million

SBTX – Silverback Therapeutics Inc.
SBT6050 (monotherapy and pembrolizumab)
HER2 Positive Solid Tumors

+0.05  +0%
Phase 1 Phase 1/1b presented at ESMO September 16, 2021. Among 18 evaluable patients for tumor types of interest, 1 patient had a confirmed partial response (5%). In addition, stable disease was reported in 7 patients (38%).
$439.7 million

SYRS – Syros Pharmaceuticals Inc.
Solid tumors

-0.37  -7%
Phase 1 Phase 1 abstract to be released late September 16, 2021. Phase 1 dose-escalation data to be presented at ESMO September 20, 2021.
$314.6 million

TBPH – Theravance Biopharma Inc.

+0.25  +4%
Phase 3 Phase 3 study did not meet its primary endpoint September 15, 2021.
$522.4 million

TCRR – TCR2 Therapeutics Inc.
Gavo-cel (TC-210)
Mesothelin-positive solid tumors

-5.73  -36%
Phase 1/2 Phase 1/2 data showed 1 Grade 3 and 1 Grade 5 [patient death] reported. 6 patients achieved partial response (PR), and 1 complete response (CR). Overall response rate (ORR) of 31%. By independent review assessment, the ORR was 25% with a DCR Rate of 81%. The median overall survival for patients with mesothelioma is 11.2 months, whereas the median progression free survival (PFS) is 5.9 months, September 17, 2021.
$381.6 million

XENE – Xenon Pharmaceuticals Inc.
XEN1101 (X-TOLE)
Adult Focal Epilepsy

+0.86  +5%
Phase 2 Phase 2b completion of enrollment announced July 7, 2021. Top-line data due late-September to mid-October 2021.
$734.5 million