Portfolio Updates

Bicycle (BCYC) — BCYC has been under pressure recently after the company provided updates on its zelenectide (BT8009) program. The company provided topline Phase I/II Duravelo-1 data of zelenectide+pembro combo in 1L mUC, timing expectations for subsequent Phase II/III Duravelo-2 readouts  (…more)

BIOINVEST BREAKING NEWS – Viking (VKTX) presented excellent updates for both high dose oral and subQ VK2735 at Obesity Week. The data continues to demonstrate best in class potential for the leading 2nd generation oral/subQ GLP-1/GIP program. (…more)

BIOINVEST BREAKING NEWS – MDGL’s Rezdiffra had a very strong 3Q, significantly exceeding very high investor expectations with Rezdiffra revenues of $62 million with a huge beat over the FactSet consensus estimate of $35 million. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) — Celldex reported the eagerly-awaited 52-week data for barzolvolimab (c-KIT antibody) in chronic spontaneous urticaria (CSU) as a late-breaker at EADV occurring in Amsterdam. We believe the data is overwhelming positive, continue to paint the best-in-class compound in urticaria and below address reasons for the stock weakness. (…more)

BIOINVEST BREAKING NEWS – Skye (SKYE) This morning Novo Nordisk announced mixed data for their CB1 receptor inverse agonist, monlunabant with solid weight loss but questionable neuropsychiatric side effects. The data showed that after 16 weeks of treatment, patients with a baseline bodyweight of 110.1 kg lost a mean of 7.1 kg after treatment with 10 mg doses of monlunabant. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) — CLDX released positive topline results from the Company’s 196-patient Phase II clinical trial of barzolvolimab in the most common forms of chronic inducible urticaria (CIndU)—cold urticaria (ColdU) and symptomatic dermographism (SD). (…more)

BIOINVEST BREAKING NEWS – Viking (VKTX) held an excellent Q2:24 call with important positive clinical and regulatory updates for both VK2735 subQ and oral. BUY. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) Based upon the timing of the posting of the Phase III CSU trials, we expect Celldex will announce the first patient treated in the study will happen any day. In addition, the Phased II CindU data is also highly likely to be released this month, then the key 52-week Phase II CSU results will also happen sometime this summer/early fall. (…more)

Special Update: Vaxart (VXRT) – Right at press time VXRT delivered great news after the close when they announced that it received a project award valued at up to $453 million through the Rapid Response Partnership Vehicle (RRPV). Read more…

BIOINVEST BREAKING NEWS – Viking Therapeutics (VKTX) – VKTX is trading down after the release of positive 52 week histology data for VK2809 in NASH. In our view, overall investor concerns around timing and likelihood of partnerships for VKTX in the large Obesity and NASH markets are driving the stock down with today’s move a “sell the news” event. (…more)

BIOINVEST BREAKING NEWS – PGEN delivered excellent data for ‘2012 at ASCO yesterday and is now poised to file for FDA approval. The Company reported positive Phase 1/2 pivotal study results for the investigational PRGN-2012 off-the-shelf (OTS) AdenoVerse gene therapy in patients with recurrent respiratory papillomatosis (RRP) in a Late-Breaking oral presentation at ASCO. (…more)

BIOINVEST BREAKING NEWS – EAACI Data Highlights “Profound” Barzo Angioedema Results – Suggests Every 8 Weeks SC Dosing – Rest of 2024 Is Clinically Busy – BUY (…more)

BIOINVEST BREAKING NEWS – Viking (VKTX) Viking Therapeutics (VKTX) – Oral VK2735 Delivers Excellent Phase I Weight Loss of 5.3% at 28 Days; Highest Dose Not Reached Yet Due to Outstanding Safety. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) On further inspection of the Phase II 12-week CSU data, barzolvolimab’s wide therapeutic window continues to support it Best-In-Class compound profile. The urticaria market is extremely underestimated and we expect barzo forecasts will rise significantly from here. BUY (…more)

BIOINVEST BREAKING NEWS – On Friday, the FDA approved Roche/Genentech’s Xolair as the first treatment to help reduce severe allergic reactions brought on by accidental exposure to certain foods. (…more)

BIOINVEST BREAKING NEWS – Viking (VKTX) Viking Therapeutics (VKTX) – LLY’s Phase II MASH Data Puts Pressure on MDGL (March 14 PDUFA) & VKTX (Q1 Phase I Oral Data), Both Still BUYS With Upcoming Catalysts (…more)

BIOINVEST BREAKING NEWS – Viking (VKTX) Viking Therapeutics (VKTX) – Significantly Undervalued After Roche Pays $2.7 Billion For Competitor, Phase I ‘2735 Oral Proof-of-Concept Data in Q1 2024. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) Underwhelming details of NVS remibrutinib (‘REMI’) Phase III trial in chronic spontaneous urticaria (‘CSU’) were released at a major medical meeting last week. Combining that negative event with two more disappointing regulatory/clinical events – the REGN/SNY’s receipt of a CRL of Dupixent and the failure of AMGN/AZN’s tezepelumab – potential competition for Celldex’s barzolvolimab (‘barzo’) is falling by the wayside. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) After the close on Friday, Regeneron and Sanofi announced that the FDA issued a Complete Response Letter (CRL) for Dupixent (“Dupi”) in chronic spontaneous urticaria (CSU). With an expected 2-year delay (or more), this is a major setback for Dupi. And hence, we believe it (…more)

BIOINVEST BREAKING NEWS – PGEN received very good FDA news when the agency recently confirmed that the ongoing Phase I/II single arm trial of PRGN-2012 in RRP will serve as the pivotal study to support accelerated approval and no additional randomized, placebo-controlled trial will be required to support submission of a BLA. (…more)

NEW RECOMMENDATION – We are initiating coverage on Thorne HealthTech (NASDAQ – THRN). For many reasons we believe Thorne is akin to the “Apple” of the nutritional supplement business. (…more)

Madrigal (MDGL) / Viking (VKTX) are both strong BUYS after the overreaction to Lilly’s Phase II, 337 patients, data that showed a 24% weight loss at 48 weeks in the high dose from their new triple drug combo.Madrigal (MDGL) / Viking (VKTX) Strong BUYs After Overreaction to Lilly Phase II Data  (…more)

BIOINVEST BREAKING NEWS – Viking (VKTX) delivered the goods this morning when they announced positive top-line results from its Phase IIb VOYAGER clinical trial of VK2809 in patients with biopsy-confirmed non-alcoholic steatohepatitis (NASH). (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) Celldex shares have been on a downward spiral since the exceptional durability data was presented at the AAAAI meeting in late February; the details are a bit lengthy but we remain steadfast that barzolvolimab (“barzo”) is the best-in-class and exceptionally unique mast cell inhibitor around. (…more)

BIOINVEST SPECIAL UPDATE – Biotechs were under pressure this week as the sudden failure of SVB and drug pricing concerns delivered a double whammy in a continued rising rate environment. In our view, while all biotech stocks are being sold indiscriminately, we believe (…more)

(ESPR) BioInvest News — The full results from the Cholesterol Lowering via Bempedoic acid (BA), an ACL-Inhibiting Regimen (CLEAR) Outcomes trial – were presented at the American College of Cardiology (ACC) Annual meeting on Saturday by Dr. Steve Nissen (the king of CV drug trials) and simultaneously published in the New England Journal of Medicine. (More)

BIOINVEST BREAKING NEWS – Celldex (CLDX) The full Phase 1b trial of barzolimab in chronic spontaneous urticaria (CSU) was presented yesterday and continues to support its potential as the best-in-class treatment for urticaria. (…more)

Bicycle (BCYC) — BCYC announced positive BT8009 Phase I data today in heavily pre-treated cancer patients at ASCO GI. In our view, the strong data point out the potential for BT8009 to become a best in class drug and a significant differentiated treatment option for cancer patients. (…more)

BIOINVEST BREAKING NEWS – Precigen announced the sale of a wholly-owned non-healthcare subsidiary, Trans Ova Genetics, L.C. (“Trans Ova”) for $170 million. The proceeds will go towards retiring roughly $200 million in convertible debt due in 2023. (…more)

Madrigal (MDGL) — Breaking News: MDGL has pitched a “perfect game” with their pristine MAESTRO-NASH Phase III trial hitting both primary, FDA agreed upon endpoints. The FDA had agreed that even hitting one primary endpoint would lead to approval. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) Urticaria Competitor Third Harmonic Discontinues Drug – BUY – After identifying troublesome liver enzyme elevations in patients in its lead program, newly public Third Harmonic (THRD) announced they are discontinuing development of the compound in urticaria. (…more)

(ESPR) BioInvest News — ESPR has delivered positive top-line data (at least 15% improvement in MACE-4) from their Phase IV Cholesterol Lowering via Bempedoic acid, an ACL-Inhibiting Regimen (CLEAR) Outcomes trial. The trial met its primary endpoint, demonstrating statistically significant risk reduction in MACE-4 in patients treated with 180 mg/day NEXLETOL compared to placebo. (More)

(ESPR) BioInvest News — ESPR held an investor R&D event today and did a very solid job of detailing why the upcoming 14,000 patient global cardiovascular outcomes trial (CVOT), CLEAR, is highly likely to deliver a positive result by reducing major adverse cardio events (MACE) by at least 15%. (More)

BIOINVEST BREAKING NEWS – Last night, MTSL Recommendation MYOV announced that its board has accepted a $2.9 billion (fully-diluted) takeover offer from Sumitovant Biopharma for $27 per share. While we believed that the original $22.50 offer was way too low, the final price does represent (…more)

BIOINVEST BREAKING NEWS – Last night, MTSL Recommendation MYOV announced that its board gracefully declined a $2.4 billion takeover offer from Sumitovant Biopharma, a unit of Japan-based Sumitomo Pharma.  Sumitovant owns 52% of MYOV shares and offered $22.75, a price that we find to be way too low. (…more)

BIOINVEST BREAKING NEWS – TCRT’s stock is down on heavy volume as a blog post from the husband of treated Patient #2 reported that she has stopped therapy due to a mutation. While social media post are not company press releases, (…more)

BIOINVEST SPECIAL UPDATE – Good Medicine for Biotechs: Great Data, Awesome Sales, Monster Deals Yesterday; 100+% Premium Acquisition Today. (…more)

BIOINVEST BREAKING NEWS – Precigen announced the sale of a wholly-owned non-healthcare subsidiary, Trans Ova Genetics, L.C. (“Trans Ova”) for $170 million. The proceeds will go towards retiring roughly $200 million in convertible debt due in 2023. (…more)

Madrigal (MDGL) — MDGL held a two hour conference call after all presentations at EASL to discuss the outstanding resmetirom data presented at yesterday. The Company focused on the 969-patient MAESTRO-NAFLD-1 safety study which showed the compound to have an excellent safety profile along with excellent efficacy. (…more)

Acadia (ACAD) – ACAD received bad news when an FDA AdCom voted 9-3 against recommending pimavanserin for Alzheimer’s disease psychosis (ADP). This is disappointing and significantly lowers the probability of the FDA approving pimavanserin for this new indication on the 8/4/22 PDUFA. Read more…

BIOINVEST BREAKING NEWS – FDA Delays MYOV/Pfizer Approval for Endometriosis on Labeling. MYOV and their partner Pfizer announced today a delay on the Supplemental New Drug Application (sNDA) for MYFEMBREE for the management of moderate to severe pain associated with endometriosis. In our view, this is clearly a delay and not a rejection. (…more)

Bicycle (BCYC) —We Are Buyers On AACR Weakness. BCYC shares are weak today after presenting updated data on BT8009, a lead BTC clinical compound, at the AACR conference over the weekend. Our investment thesis remains intact. (…more)

Madrigal (MDGL) — One of the few broadly positive binary events in biotechnology of late, Madrigal’s best-in-class NASH compound is well on its way with de-risked, late-stage clinical results. With this wholly-owned compound heading to blockbuster status, MDGL remains a Top Pick for 2022. (…more)

Acadia (ACAD) – Yesterday after the close ACAD announced that it plans to resubmit its supplemental New Drug Application (sNDA) to the FDA for pimavanserin for the treatment of hallucinations and delusions associated with dementia. Read more…

(ESPR) BioInvest News — ESPR Raises Cash To Get To CLEAR Outcomes Trial Data in Q12023, Lowering our BUY and our TARGET PRICE. ESPR shares were off 40% after they announced the closing of a $208.8 million of common stock and warrants. (More)

Alkermes (ALKS) — ALKS was blindsided by JNJ after the close yesterday received notices of partial early termination for two license agreements with Janssen Pharmaceutica N.V., a subsidiary of JNJ. These license agreements are for Alkermes’ nanoparticulate formulation technology, known as NanoCrystal® technology. (…more)

BIOINVEST BREAKING NEWS – Precigen held a virtual R&D presentation yesterday, updating data on five clinical trials from the novel Ultra-CAR-T and AdenoVerse platforms. The company has delivered objective responses in late-stage, refractory cancer patients (AML, ovarian, HPV-positive tumors) utilizing their Ultra-CAR-T system plus which offer the fastest (overnight vs. 4-6 weeks) low-cost CAR-T therapies around. (…more)

BioInvest News – SGMO’s PhaseI/II Fabry Disease Data Looks Great – In it’s quarterly update, SGMO delivered news across multiple clinical programs today with the stock reacting positively to the excellent Fabry disease data. (…more)

BIOINVEST BREAKING NEWS – Celldex (CLDX) Delivers Outstanding Phase 1b Data, 19 of 20 Urticarial Patients Demonstrate Complete Response After Single Dose of CDX-0159, Raising BUY to 47 (from 30) and TARGET to 70 (from 40), Best in Class and a Pipeline in a Pill? (…more)

Special Update: Acadia (ACAD) – In another FDA surprise, after the close, the Agency notified ACAD that it has identified deficiencies in the dementia related psychosis (DRP) sNDA for pimavanserin that preclude discussion of labeling and post-marketing requirements. Read more…

Special Update: Five Prime (FPRX) – This morning, FPRX announced that it is being bought by Amgen for $38 per share (~80% premium to yesterday’s closing price), representing an equity value of approximately $1.9 billion.  Read more…

BIOINVEST BREAKING NEWS – This morning NKTR announced that it has signed two deals to advance BEMPEG combos – one with Merck and the other with SFJ.  The first is a Phase II/III trial of BEMPEG in combination with Merck’s KEYTRUDA for first-line treatment of patients with metastatic or un-resectable recurrent squamous cell carcinoma of the head and neck (SCCHN) whose tumors express PD-L1 (Combined Positive Score [CPS] ≥1). (…more)

Special Update: Five Prime (FPRX) – FPRX announced excellent topline results from the global, randomized, double-blind placebo-controlled Phase II FIGHT trial. Read more…

Special Update: Vaxart (VXRT) – Following up on key features and still questions on the exceptional  >90% efficacy in the first Phase II/III trial of their mRNA COVID vaccine from Pfizer/BNTX. BUY VXRT here as Rick Bright is back (see below). Read more…

(ESPR) BioInvest News — Esperion reported financials ($3 million in net sales) after the close yesterday and the stock is under pressure today as the Company continues to struggle with the new drug launch during the COVID-19 crisis.

Special Update: Vaxart (VXRT) – VXRT has delivered two very good pieces of news – the Company has enrolled its first patient in the COVID vaccine study and also released uniformly positive hamster challenge data. Despite this good fundamental news, the stock has been under pressure from short sellers since then based on an SEC filing of a self offering filed on the same day as the clinical/pre-clinical updates. Read more…

BIOINVEST BREAKING NEWS – In late Breaking News, ALKS announced overwhelming and surprising positive votes from the joint meeting of the Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee. The FDA committees met to discuss ALKS 3831 (olanzapine/samidorphan), a novel, once-daily, oral atypical antipsychotic drug candidate for the treatment of adults with both schizophrenia and with bipolar I disorder.

BIOINVEST BREAKING NEWS – MYOV announced results of an additional secondary endpoint from the Phase III HERO study evaluating relugolix in men with advanced prostate cancer. Relugolix did not achieve statistical superiority for castration resistance-free survival compared to leuprolide acetate in men with metastatic disease through 48 weeks. (…more)

BioInvest News – BMRN/SGMO – Surprise Roctavian CRL – Bad For BioMarin, Good For SGMO – This morning, the FDA issued a surprise Complete Response Letter for BioMarin’s Roctavian, its lead hemophilia A gene therapy program. (…more)

BioInvest News – BMRN/SGMO – Surprise Roctavian CRL – Bad For BioMarin, Good For SGMO – This morning, the FDA issued a surprise Complete Response Letter for BioMarin’s Roctavian, its lead hemophilia A gene therapy program. (…more)

BIOINVEST NEW RECOMMENDATION – Celldex (CLDX) We are initiating coverage of Celldex Therapeutics (CLDX, $12) with a BUY under $15 and a $30 TARGET PRICE. In our view, CDX-0159, the Company’s anti-C-KIT antibody has blockbuster potential in various mast cell diseases. With a market cap of ~$375 million with ~$200 million in cash, in our view, CLDX is extremely undervalued based on the potential of CDX—0159 alone. (…more)

Special Update: Vaxart (VXRT) – Negating The Egregious NY Times Article – While we rarely rebut a short report or bear raid on one of our recommendations, the The NY Times published one of the more egregious “investigative” reports we have ever seen. Read more…

BIOINVEST BREAKING NEWS – Special Update — NEW RECOMMENDATION: Vaxart (VXRT) – VXRT’s Oral Vaccine for COVID-19 Poised to Enter Human Testing. Read more…

BIOINVEST BREAKING NEWS – Special Update – #ZYNERBA – ZYNE’s stock is down significantly this morning after reporting disappointing top line results from the 14-week pivotal CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) trial. more…

BIOINVEST BREAKING NEWS – In light of the historic, market selloff due to the coronavirus, we thought it was important to update subscribers with a brief summary of the highlights of our recommended stocks during this period of stock corrections unrelated to Company fundamentals. In sum, many of our companies have delivered the positive value creating news that we predicted (…more)

BIOINVEST BREAKING NEWS – After the close, Sangamo announced a worldwide deal with Biogen for gene regulation therapies in neurology.  BIIB is the global leader in developing neuro biologics with the top drugs in multiple sclerosis and an extremely deep Alzheimer’s/ALS R&D pipeline, in our view there is no better partner available than BIIB. (…more)

(ESPR) BioInvest News — FDA approves of NEXLETOL (bempedoic acid)— the first new non-statin once daily LDL-cholesterol lowering medicine in 20 year. With a very seasoned management team that is extremely well-prepared for the imminent launch, on the positive news we reiterate our BUY rating.

BIOINVEST BREAKING NEWS – A very positive long-term follow-up (88% response rate) from the LIBERTY trial was released yesterday and we believe continues to further solidify relugolix in uterine fibroids (UF). Management will file for an NDA for the use of relugolix+ABT in UF in April (plus a separate filing for the treatment of relugoxi in prostate cancer in Q2:20). For relugolix in endometriosis, we expect MYOV to (…more)

BIOINVEST BREAKING NEWS – The FDA rejected NKTR’s Oxycodegol (NKTR-181) by a vote of 27-0 at yesterday’s AdCom meeting. As a result of the negative vote the Company will withdraw the NDA for oxycodegol and make no further investment into the program. We listened to the majority of yesterday’s meeting which was mostly (…more)

BIOINVEST BREAKING NEWS – On January 14-16, there is a Joint Meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and the Drug Safety and Risk Management Advisory Committee. After a close review of the FDA briefing documents, the panel will vote on three options for the approval of NKTR-181 on Tuesday (1/14). The choices are for a narrow approval, a broad approval and a recommendation against approval.  In our view, the fact that NKTR (…more)

BIOINVEST BREAKING NEWS – Nektar (NKTR) and partner BMY announced today that the companies have agreed to a new joint development plan to further the advancement of bempegaldesleukin (bempeg/NKTR-214) plus Opdivo (nivolumab) into two new Phase III/registrational trials, bringing the total to five being tested.  In our view, BMS’ renewed (and public) commitment to the bempeg/Opdivo combo has further de-risked the largely out-of-favor NKTR stock. (…more)

BIOINVEST BREAKING NEWS – Sangamo (SGMO) will have a very busy December, with two clinical data updates coming at the American Society for Hematology (ASH, https://www.hematology.org/Annual-Meeting/). We remain steadfast that SGMO will emerge as the leader in gene therapy for hemophilia A with SB-525. (…more)

BIOINVEST BREAKING NEWS – Medicines Company (MDCO) — From the beginning of the ORION clinical trials program in 2016 until the recent successful three Phase III studies, we have been under the belief that Inclisiran would be much bigger than people think. Today’s announcement tells us that Novartis, too, believe as such – as the consensus suggests that Inclisiran will need to generate at least $2.2 billion annually for the deal to be NPV neutral. (…more)

BIOINVEST BREAKING NEWS – Myovant (MYOV) Legendary blues rockers Little Feat song, ‘Time Loves a Hero,’ says it all for MYOV today as their Phase III prostate cancer data from HERO trial was excellent.  Patient MYOV investors were rewarded this morning as the Phase 3 HERO study of once-daily, oral relugolix (120 mg) met its primary efficacy endpoint and all six key secondary endpoints in men with advanced prostate cancer. (…more)

BIOINVEST BREAKING NEWS – Medicines Company (MDCO) — The oral presentations of the ORION-9 and -10 studies further confirm the revolutionary compound that is Inclisiran. On top of its durable LDL reductions and impeccable safety, the ease of use and high compliance rate, in our view, suggest blockbuster status is likely. With Phase III program successfully complete, we remain steadfast in expecting a much larger market for Inclisiran than the current stock price is forecasting. Raising BUY and TARGET PRICE.

BIOINVEST BREAKING NEWS – We’ve Seen This Before – ZIOP Under Attack By Short Report – REITERATE BUY. ZIOP’s stock has been under pressure the past two days as an error-filled Seeking Alpha (a classic site for anonymous bears) short report was released on the internet. Read more…

BIOINVEST BREAKING NEWS – Ziopharm (ZIOP) – Sleeping Beauty TCR Solid Tumor Trial Posted By NIH Starts Next Week, Oct. 1, REITERATE BUY. The trial discussed below is the accumulation of decades of work by the world’s leading cancer gurus.  While CAR-Ts have shown remarkable successes in blood cancers, treating solid tumors with CAR-Ts has proven to be quite challenging.  (…more)

BIOINVEST BREAKING NEWS – The Medicines Company (MDCO) – Efficacy & Safety “At Least As Favorable As ORION-11” – Positive topline results for the ORION-9 Phase 3 clinical study in patients with Heterozygous Familial Hypercholesterolemia (HeFH). ORION-9 met all primary and secondary endpoints, and … more

BIOINVEST BREAKING NEWS – Special Update – ZYNERBA – ZYNE’s stock has sold off since they announced positive top line results from the open label Phase II BELIEVE 1 (Open Label Study to Assess the Safety and Efficacy of Zygel (ZYN002) Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) clinical trial. Read more…

BIOINVEST BREAKING NEWS – Special Update – ACADIA – ACAD’s Phase III HARMONY trial recently delivered robust statistically superior data (p=0.0033) compared to placebo in time to relapse of dementia-related psychosis (DRP).  In our view, (…more) #acadia #ACAD

BIOINVEST BREAKING NEWS – Special Update – ACAD — In Perfect HARMONY–This morning, ACAD released excellent data from the Phase III HARMONY study, a double-blind, placebo-controlled relapse prevention trial evaluating pimavanserin for the treatment of dementia-related psychosis.  Read more…

The Medicines Company (MDCO) – Inclisiran Indeed!  Following up on the positive top-line results of the ORION 11 trial (released 8/26), the actual data presented at the ESC Congress 2019 were uniformly favorable for both efficacy and safety. In addition, despite the relatively smaller study and not powered for outcomes (n=1607), there was a strong reduction in Cardio Vascular Outcomes (CVO) in the Inclisiran treated arm versus placebo. (… more)

The Medicines Company (MDCO) – With efficacy consistent with the Phase I and II studies and a safety profile at least as favorable as in previous trials (e.g., ORION-1 Phase 2 and ORION-3 open-label extension studies), Inclisiran is well on its way to FDA approval and, in our view, blockbuster status… more

BioMarin (BMRN) – The widely anticipated BioMarin hemophilia A gene therapy delivered good news this morning, albeit with some caveats. BMRN announced both positive Phase I/II and Phase III data for ValRox (valoctocogene roxaparvovec) for adults with severe hemophilia A. 

The Medicines Company (MDCO) – MDCO presented interim results from Group 1 of the ORION-3 Phase II study (n=290) at the NLA (Miami) on Saturday. The long-term 3-year data showed that patients given additional inclisiran doses had LDL cholesterol reduced by >50% over time, consistent with the shorter-term ORION-1 results. (…more)

(ESPR) — FDA Accepts Both BA NDAs with No Need for Advisory Panel – The FDA has accepted both New Drug Applications for bempedoic acid and the bempedoic acid/ezetimibe combination tablet for filing and regulatory review.

Pacira (PCRX) – On Tuesday (4/30), Heron (HRTX) received a surprise Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for HTX-011 for the management of postoperative pain. The CRL stated that the FDA is unable to approve the NDA in its present form based on the need for additional CMC and non-clinical information.

This morning ALKS released their Q1:19 financial results that showed a big miss for Aristada coming in at $30 million after guiding for $40 million.  While a disappointment, the company did not change overall FY guidance and stated that an inventory work down was the primary cause.

Ionis (IONS) — An update from Spinraza competitor Zolgensma (NVS) and the shift out of health care stocks are driving today’s decline. Shares are down 14% after Novartis presented a positive update to its STRIVE study for Spinal Muscular Atrophy (SMA) yesterday at the MDA 2019 meeting.

BioInvest News – Sangamo (SGMO) has delivered excellent news across their proprietary ZFN drug development platform with promising human data from both the Hemophilia A program with Pfizer and Beta Thal with Sanofi. 

BioInvest News Novavax (NVAX) – This morning, NVAX reported disappointing top-line data from the ResVax Phase III PREPARE study for infant RSV prevention. Unfortunately, the trial failed on the primary endpoint of reducing medically significant RSV lower respiratory tract infection (LRTI).

BioInvest News – NKTR’s stock is under pressure after the early update to NKTR’s Phase 2 PIVOT-02 trial of NKTR-214 in combination with Opdivo which includes 13 additional urothelial cancer (UC) patients vs. the 10 reported at ASCO 2018.   The data remains solid despite the small decrease in ORR which is now 48% vs. 60% prior.

BioInvest News – Sangamo (SGMO) Updated MPS I & II Trials Disappoint But ERT Withdrawals Continue & That’s Key, More Powerful 2nd Generation ZFNs in Clinic by Year-End, Stock Overreaction

(ESPR) — Nets $150 Million in Deal For Europe With Daiichi Sankyo Europe. ESPR entered into a substantial licensing agreement with Daiichi Sankyo Europe (DSE) providing DSE with exclusive rights to commercialize bempedoic acid (BA) and the bempedoic acid/ezetimibe combination pill in the European Economic Area and Switzerland.

BioInvest News – NKTR made a strong presentation at the recent JPM conference with a detailed focus on the company’s I/O pipeline and the upcoming May 29 PDUFA for NKTR-181. NKTR-214 is poised to move into move pivotal studies over the course of 2019 including lung cancer, a major market opportunity.

BioInvest News – Sangamo (SGMO) presented an overview of its clinical and technological gene therapy/editing leadership. With 5 trials underway and data from all five clinical results due this year, to us 2019 will be the year when SGMO gaps ahead of the gene editing crowd.

Ionis (IONS) — Next generation LICA Technology In Focus At JPM. IONS focused on their next generation LICA technology during its recent JPM presentation. IONS’ partner Novartis plans to initiate an outcomes study for AKCEAAPO(a)-LRx following the recent positive end of Phase II meeting with the FDA.

BioInvest BREAKING News (MDGL) – Down Sharply After Positive Update at Liver Meeting, It’s The Unforgiving Market, To Come More In Line With Sentiment & Current Price, Lowering BUY and Target

BioInvest BREAKING News – IONS/AKCA Receive CRL From FDA for Waylivra Despite Positive AdCom. In a somewhat surprising move, ONS/AKCA received a complete response letter (CRL) for Waylivra (volanesorsen) from the FDA. It is also unexpected given the 12-8 Advisory Committee vote in favor of approval on May 10th…

BioInvest BREAKING News – SANGAMO – Q2 CALL BONANZA – Reiterate BUY (And Short CRSP, EDIT, NTLA) First Hemophilia A SB-525 Results Ideal In The ALTA Study-MPSII Patient 6 Treated with SB-913 in the CHAMPIONS Study
-First patient treated in the SB-318 Phase 1/2 EMPOWERS Study for MPS Enrolled the first patient in the Phase 1/2 Thales Study of ST-400 gene-edited cell therapy for beta-thalassemia
Presented further ZFN Safety via the Most Sensitive Assays ..